ff on august 19. stay connected to c-span for an uninterrupted glimpse of democracy get to work. watch the republican and democratic national committee vengeance to the summer on c-span, c-span now, and online at c-span.org. c-span, your unfiltered view of politics powered by cable. announcer: c-span your unfiltered view of government. we are funded by these te comcast. >> you think this is just a community center? it is way more than that. >> comcast is partnering with the 1000 community centers so students from low income families can get the toog. along with these other television providers, giving you a front row seat to democracy. announcer: next medical professionals and public health researchers testify under ways to support people with rare . lawmakers ask about treatment options, pharmaceutical costs

and access to out-of-state care. from the subcommittee on health, this is justhree hours. . >> the subcommittee will come to order and determine will recognize self for five minutes of opening statements. thank you to our witnesses for sitting here today. the legislation before us will take an important step helping to support rare disease. she a rare disease that affects less than 200,000 patients in the united states. according to the national institute of health nih there are over 10000 diseases as many as 30 million americans despite

10% of the u.s. population with a rare disease 95% of these diseases most patients can even begin to think about treatment until they are diagnosed which can be along journey only to discover these options. research and development airbase is challenging especially when it comes to measuring the safety and efficacy of drugs for all patient populations he investment and rare disease therapies risky and unpredictable. numerous steps over the years to better support for approval. research rar diseases and the process by which they seek approval and ensure medicare and medicaid will cover this. congress authorized rare disease priority after program a decade

ago bolster existing the orphan drug act. the doctor reduces financial risk for invaders to obtain resources needed to conduct rare disease research which ultimately patient received therapy more quickly authorizing this for the program and creating is so important. she doctors have been granted and paved the way for groundbreaking therapies. i'd like to thank my colleagues for the bipartisan and ensure access to treatment for rare disease consistency throughout the process. access to care act which will

streamline making it easier to receive necessary care for provider in another state. get the care for rare diseases. harder for patients to access life-saving therapies. in some cases patients travel significant distance lengthy state leaving patients and families responsible for hotels, food and other important expenses. manufacturing paper limited circumstances making it critical and easier for patients. bills buildup this work passing and protecting health care for all patients for those with

disabilities life-saving and provides access for medicaid patients. finally efforts to incre access life-saving therapies we are considering trying patients getting access to affordable treatment to treat rare diseases.

the rare disease. over the last 40 years because the revolution in the development rare disease. fda more than 5000 the american drug act of 1983. last year more than half of all new drugs approved by the fda drug spring hope to millions of americans. there's room for improvement. only 5% of more than 7000 known rare diseases have fda approved

treatment and clinical trials. among the most aggressive our children. you treatments for rare diseases be used. children are not little adults. something congress recognized 20 years ago she requiring this chapter study. as more to ensure children are not left behind. 36% of drugs approved relevant to children since 1999 some oral pediatric data. immigration and drug data i introduced will close the loophole so we have the clinical

data children. the american academy of pediatrics leukemia children's hospital association and the national organization rare disorders, i think all of those organizations. children also benefit from cancer treatment which is why mccall and i introduced the kids act. currently the fda cannot direct critical trials despite combination therapy proving effective in adults rooted legislation has 188 bipartisan endorsed by 50 organizations

research. every member of the committee is part of the fda. finally, i joined representative mccall introducing creating hungry authorization act providing pediatric drug developers pediatric priority and when the drug comes to market or not. 114 authorizations endorsed this bill and last time legislation for reauthorization in the house pass unanimously. no subcommittees considering bills to undermine medicare's

historic new power and beneficiaries are saving money and capped at $35 a month. and 3500 a year. next year's all beneficiaries will have their cost capped. there also unneeded. medicare drug price negotiations should only focus on the top selling high cost drugs without part-time position. unneeded loopholes look forward

to hearing this and i heal back. >> good morning, welcome everyone. we are gathered here today on rare disease day. to promote people with rare diseases and make sure all patients benefit exciting innovation happening. a 12-year-old spinal muscular atrophy type one there's only 10000 rare diseases affecting 30 million americans. fda from treatment.

the technologies and many more innovations researched and developed some of which to find bridges eat treatment but doesn't do a lot more to be done done. just over 40 years ago only 38 drugs were fda approved. degree alone they were approved by the fda and get them intentionally. our job is to be sure it's ready and the market continues. i'm concerned we don't continue to encourage we will be harming the potential for development. many of the bills before us

today provide the necessary to ensure normal therapy continued to be accessible for patients as rapidly as possible as we work to carry out that we were carefully today to ensure doesn't have this effect. we need to make sure constructs are approved, patients can actually access them. concentrated at or limited and it may come down to whether the patient can't afford to travel and life-saving. patient act will make these cost more manageable in the case of a child parent and the care

they need allowing drug manufacturers to support the cost of travel. access to care act with cut red restricts the ability paid in treat kids enrolled in a different program. there are three discounts today and the inflation reduction act. i recognize members of this committee have different opinions to ensure americans have access. i hope you can set aside broader discussions and focus on what's best for rare disease patient because the process that led to the pricing team was so russia, didn't understand how it could have devastating impacts on the rare disease community.

after rejecting a position this committee did not get the opportunity to explore the consequences. i'm hopeful today will see that work and work in a bipartisan way. three bills today or first doing so. a long history of working together to support innovation including things like the 21st centuries act and multiple user feet authorization we must continue to build on this work and that is what we are doing today. look forward to learning more about what we can do from our witnesses finding the committee could move forward with bipartisan legislation should. >> the gentleman from new jersey

jersey. >> eq. i am pleased considering celebrating this care act. it's unfortunate we are also considering a number of proposals will undermine policies to lower prescription drug prices congressional democrats shouliden delivered for the american people as part of the inflation reduction act. ours allowed to leave the reduction ultimately included in the inflation reduction act. medicare is finally going to be able to negotiate prices for american seniors. a few. weeks ago the biden administration first drug price negotiations collective negotiation during the first year.

9 million seniors take drugs negotiation and $3.4 billion greatly and make a difference negotiating lower cost for these drugs. thanks to the official reduction act of those who raised prices should seniors say $618 on average does on 47 drugs despite the fact that it's making it more affordablseniors pharmaceutical industry and congressional republicans are relentlessly attempting to undermine losses and detrimental policy changes will only delay patients. republicans claim we must choose between lower prices and greater innovation. they argue that we want a bit of therapy americand drugs to wipe out life savings more than the

average household in the price of two to three times any other country. i refuse to believe innovation and lower cost for zero-sum game and therefore we know patient seen affordable access to therapies to glory these conditions. i'm just a nuclear discussing this allows potential drugs to be exempt and delays negotiating for the drugs for years. contrary to the sentimental carefully drafted to protect innovation and delivering savings. heart content of the regulation reduction act ensuring the greatest share cost in the program are negotiated to help the american people and crafted exclusion for orphan drugs to treat a disease and was not intended to be for manufacturers to avoid getting the system.

the republican plan laid out today will keep these out of reach for two-minute families. while i cannot support legislation that undermines the inflation reduction actntivize additional development of rare disease treatment in a short regulatory certainty for developers. it's important we consider whether existing programs are providing incentives congress and reauthorized program the drug administration. my conce reports suggest incentivizing research in developing and use public resources to rework development that would have taken place even without the program. look forward to hearing what our witnesses have to say. i understand and appreciate the unique and help innovation and treatment brings especially for

small populations of patients without other therapy options. the hope to reject this which would likely grow billions of dollars in increased prices we should focus on buying census to bring forth locations. >> the gentleman fields back. >> that puts all opening statements. >> now witnesse have five minutes. raise your written testimony you testified before you will see a green light. he will have a minute to correct and move to the next witness. i will introduce all the witnesses and then move to your

statements. >> associate professor public health. alexander -- position and she university of iowa chair and professor department of pediatrics. we have doctor aaron md professor of medicine medical school director program regulation of unix and law and women's hospital. senior fellow ust for public

health policy and economics. as goal public policy. mr. crystal davis connie is. five minutes for your opening statement. >> thank you honorable members of the committee. thank you for the opportunity to testify. the associate research professor professor. i recognize the need bro access affordability which led this testimony is to provide of the current state of innovation

where disease space and rationale for maintaining inflation reduction act. some the following are the key points. improving affordability there's little evidence of house impacted pharmaceutical research and development and we are benefiting from a trail went breakthroughs and rare diseases. we have a drug affordability oblem. one step to address this was the passage of glacial reduction act due to. ira improves drug affordability and price negotiation and reduces out-of-pocket expenditures is to ginny $2000 out-of-pocket max. the biggest impact on suitable

will be price negotiations. price negotiations will reduce medicare spending by 23 billion in the year 2030. that's significant and represent less than 3% of us pharmaceutical 2030 avenue difficult to reconcile innovation with an impact on revenue. ... in 2023, there are 390 orphan drug designations and 90 orphan drug indication approvals in the

u.s. these numbers were merely identical to the five-year average. we've heard mention of irate they impact business decisions. however, we have seen no r&d concept and big pharma. we've seen robust pharma merger and acquisition activity and we even have recently a drug -- encouraged by initial price negotiations. lastly rare diseases remain a focus, therapeutic area for many biopharmaceutical the fa will cause companies to delay seeking approval for rare diseases or drugs with large markup approvals. this is particularly salient in oncology where drugs often receive multiple approvals over their life cycle. companies will certainly look at product launch strategies. however, i suspect they will still seek approval for rare

diseases race on scientific plausibility and market potential. the reason being is these supplemental approvals expand the market for these cancer drugs. even when anticipating negotiated discounts many cancer drugs will still have higher prices in your suggesting a file competition for companies to pursue. we are experiencing a boom in gene therapies thanks in part to the orphan drug act government funding of financial -- hume man genome project and federal risk reward profile. the market is expected to increase fivefold with many current and anticipated gene therapies coming to market with price tags in the hundreds of thousands of dollars. these therapies will treat patients with rare diseases and we need to ensure patients have

access to these truly innovative medicines. i encourage congress to maintain the price negotiation partnership and irate to improve affordability enacted. i also encourage congress to support efforts by cmmi and public affairs to develop innovative payment strategies to maintain a balance of affordability, access and incentives for innovation in rare disease space. thank you. >> thank you for your testimony. the chair will not recognize dr. flotte pick your vitamins for your opening statement expert i.t. chair guthrie ranking member eshoo and members of the subcommittee. i want to thank you for the opportunity to testify today on behalf of the american society of gene cell therapy or asgct took my name is terry flock provost and dean at umass chen medical school and currently the vice president of asgct. our society is a nonprofit professional membership of physicians, scientists patient advocates and other professionals working

together in universities, hospitals and biotechnology companies to expand the discovery and clinical application of gene and cell therapies. my own lifework is rooted in being a pediatric physician scientists working in the rare disease space. there are over 10,000 rare diseases updated% of which are single gene disorders. gene therapy aims to address the underlying causes of disease gene mutations which as you know our mistakes in the basic blueprints of our body. gene therapy can fill in missing parts or correct errors in those blueprints. early in my career doctors like me had few therapeutic options to offer these patients. not being satisfied with this, 1995 i said the first team of researchers to use aav as a vector or carrier in gene therapy trial for cystic

fibrosis. aav vectors one of the basic building blocks of today's gene therapy products. vectors with the genetic information they taken directly target the cause of disease and change the way a cell functions. gene therapies only to be administered one time to have long-lasting potentially curative effect. but of the thousands of rare diseases only handful are being addressed currently with the products that are already licensed and a small subset of the total number are being addressed with drugs in the pipeline. the question now is how can we accelerate the path from gene to therapy and make these challenges f there is a limited medical and scientific capacity? among the rare disease population about half affect children. that is why robust research is needed to help close the knowledge gap for these

children. gene and cell therapy offers a unique opportunity to address those researcher needs. continued investment in basic translational and early clinical research on rare diseases is needed. asgct supports robust funding for the nih to ensure that the u.s. remains a global leader. private sector investments have also played a critical role in the development of gene therapies, maintaining a s in research and private created by the orphan drug act and of the programs is needed to continue growth and success in rare disease research. for example gene therapy products represent more than a quarter of all designations in the pediatric priority review voucher program. this incentive program uses market forces rather than government dollars to reward successful products. this is a win for product taxpayers. we humbly encourage the committee to reauthorize it in a timely manner. cell and gene therapies often

require specialized infrastructure, manufacturing administration facilities. these challenges can be mitigated by innovative trial approaches such as decentralized trials decentralized studies and use of real-world evidence, embracing innovative study designs can benefit patients in need. our goal is to work collaboratively with fda on these and other issues to create a regulatory framework that encourages and supports the development of the available treatments. the society thinks this committee for its role in passing privations in the last fda user fee reauthorization that established the platform technology designation program as well as the advanced manufacturing designation program. in closing i want to express my thanks to the members of the committee for inviting me today to testify on behalf of the asgct. it is our view that since the human gene, human genome progress sorry. human genome project the progress achieved a patient in

cell and gene therapy field is one of sciences greatest achievements. scientists and physicians have an obligation to do everything in their power to help patients and families living with rare genetic diseases. congress we must continue to robustly fund biomedical research and academia, support incentives for private sector investment and ensure the fda as appropriate review processes in place for expediting safe and effective cures for our most vulnerable disease patients. thank you once again for the opportunity to address this important topic. >> thank you dr. flotte. the chair will not recognize doctor forgiven five minutes for an opening seven. >> chair guthrie and ranking member eshoo chairman burr moran rogers, the establishment of the committee think the deputy to participate in the sharing took to discuss h.r. 4758 accelerating kids access to care act. the accelerating kids access to

care act has strong bicameral bipartisan support and as an eye when i am proud that i am a congresswoman miller-meeks along with congresswoman trey hunt of massachusetts on the house cosponsors of the legislation. this legislation will improve children's access to essential health care while intimidating administered a burden for families providers and states. my name is alex, a pediatric neurologist, physician scientist and university of iowa department chair of pediatrics and the physician in chief of our universe or by what stead family children's hospital. our mission at the university of vital and her stead family chilton hospital is to improve the health of children through investigating new cures and treatments, teaching the next generation of pediatricians in pediatric scientists and providing excellent clinical care for children and her family. i'm a member of the association of american medical school pediatric department chairs, the american academy of pediatrics and hospitals membership with the children's hospital association. these organizations along with counts others have been working tirelessly to support the accelerating kids access to care

act. medicaid covers about one half of all children nationally and about one-third of the children cared for at the university of iowa are covered by medicaid. we are taking care of the most vulnerable part of our population, children in poverty. investing in children's health is not just the right thing to do. it also pays off in the long term as we know children receive the services through medicaid have better health as adults. the university violent stead family children hospital is an academic university associate of children's hospital in the state of iowa. the was a violent with over 200 doctors who are solely dedicated to the care of children in many case of university of iowa pediatric care doctors of the only specialist of the kind in the state and often the stranded region. for children with rare diseases and complex medical conditions are university of vital pediatric doctors or usability doctors with any experience with those rare diseases because of this we sometimes have children with rare diseases for whom we're the only place in the state region and the whole country with the knowledge to treat them and we do so with high quality and innovative character design for children

and families to continue to thrive for chilled outside of i will may also be the closest medical center with expertise in a pediatric disease. the bipartisan et cetera and kids access to care act would improve children's access to necessary out-of-state healthcare by streamlining the burdensome and medicaid provider screening and aroma process. today children on medicaid needing care outside their home states often experience delays because some state medicaid programs require out-of-state provided to be screened and rolled into the program even if the provider is already enrolled and in good standing with their home state medicaid program or medicare. as an example dr. ferguson our rheumatology is the world expert for a rare and possibly fatal inflammatory bone condition called chronic recurrent multifocal osteomyelitis or crm oh. children with this disease can have bone inflammation that is painful deform and they can even die from this disease. most doctors don't even see a single case of this rare disease that if you do because it's so

rare they might miss the diagnosis. doctor fergusons work has helped uncover lifesaving treatment for this rare disease. because of her expertise patient come to see dr. ferguson from all over the state of i'm all 50 states and even internationally but as a child is injured by an out of state medicaid program if i wish and picked by dr. ferguson codependent on administrative approval be denied delayed because of other administered burdens. these delays two weeks the musk of which is a cause irrevocable harm to the child's health and future. sadly we know children with this rare disease of already suffered the consequences of this administrative red tape when he could've been up very quickly by dr. ferguson. i've given you an example where a child with rare disease and public medical needs from outside of iowa could be helped by the bill under discussion but, of course, reciprocal is also true. there would be cases where children on iowa medicaid with rare diseases and complex medical needs might be greatly helped by receiving their care in an expedited fashion in

another state. this bill is good for iowa's children and good for children across the country. i urge this committee to act now and passed h.r. 4758, accelerating kids, accelerating kids access to care act. thank you for the opportunity to testify before you today. >> thank you dr. bassuk. the chair will not recognize for five and. >> chairman guthrie, ranking member eshoo chair rodgers ranking member pelota members of the committee. i'm an counsel him and of the the program on regulations or pdx and law at harvard med. thank you for inviting me to this specially a disease day about various proposals covering the care of patients with rare diseases. i want to highlight a few bills being debated and how they do and done i can help such patients. they cure, the orphan cures act and many accident inflation reduction act which created a novel pathway for drug price negotiation by medicare bring in on what all of the goods and services medicare covers. negotiation of prescription drug prices is important because rare disease patients like all patients pay far more for

brand-name drugs in u.s. been comparable countries. the ira's designed to help medicare reach a fair price every flex the drugs clinical values. covers drugs with two wanted none darcy and medicare sales had been on the market already nine to 13 just to the ira has numerous exclusions including drugs for a single rarxemption may have been included to ensure negotiators prioritize drugs that affect broader population but in reality single rare disease revenue. among approved single rare disease drugs the had turned $11 billion in global revenues in its first nine years on the market with medicare spending on this but it's increasing from $3.4 billion in 2012, the $10 billion in 2021. five your net sales of drugs for rent than drugs for more, diseases, thus in these cases developing drugs for rare diseases has been extremely lucrative for manufacturers which is one reason what over half of new drugs approved by the fda chair are rare disease drugs. yet the orphan cures act would

expand rare medicaid negotiation except into drugs approved for multiple rare diseases. then begin multiple rare disease drug in the last decade had $746 million in peak annual medicare expenditures, 35% higher than the median sold rare disease drug. no recent excludes such blockbusters from negotiation process that could help achieve a fair price and increased rare disease patients access to the drugs they need. the often cures act also a delay negotiation on drugs they get a rare disease designation before being approved for more common indication but such a drugs are among the most successful and medicago drugs like -- and not one of them has begun irreverent below $2 billion a year. instead of providing these drugs with a huge undeserved windfall we should be encouraging firms took indications. many act also includes negotiation delay in this case nine to 13 years for so-called advance of drug products, categories defined vaguely which could lead to variable interpretation after enactment.

with the exclusion covered drug like i do captor? approved to treat the specific rare genetic mutation and subset patients with cystic fibrosis? one of its follow-up product for another application listed me to have net value of $33 billion the fuse after their approval. no harm to innovation would from the time the price negotiation of such drugs. another bill up for discussion would renew the rare pediatric disease we knew that you which is when the most ill-conceived drug policy ideas of the last two decades. companies getting rare pediatric disease drug approved can earn the voucher that accelerates fda review of an innovative concentrate by a few months, innovative drugs already get priority review. our controlled studies have found these vouchers do not help start new trials will bring new drugs to market in greenfield, which lets rare pediatric diseases and the fda considers them disruptive to which review process. there are far better ways to promote effective care for rare

disease patients including some of the bills being discussed today. first, the rare act closes a court creator loophole in the orphan drug act and would help ensure drugs for rare diseases face competition in a timely fashion which can help lower prices amateur a stable supply of rare disease drugs. second the als better care act were to direct resources to paying for services for als patients and encourages engagement with clinical trials for new treatment for this important disease. third, the innovation in pediatric drugs act would generate more testing of rare diseases drugs in children which is often waived despite being required before approval and is deferred for years and years after the drug is already approved because there is no incentive or requirements on manufacturers to do these essential studies of these drugs in children. and, therefore children will end up with rare diseases will end up using drugs for years without sufficient guidance because drug companies delay these trials.

bills like these provide useful direct support for clinical care d essential research and will be most meaningful patients with rare diseases. we should proceed with these efforts and avoid drug policy gimmicks like the priority view voucher or bills that undermined efforts to establish their prices on blockbuster rare disease. thank you very much. >> thank you for your testimony. professor chen you're recognized for five minutes for youment. >> thank you chairman guthrie ranking member eshoo and established members of the committee. the opinions offered today are my own and did you not represent views of the university of southern california or the usc schaeffer center. drug innovation has been crucial in addressing unmet patient needs. for rare diseases treatment options are often limited and new drug development can provide help to patients and families facing challenging health conditions. moreover patients highly valued these treatments. our research at usc schaeffer sin has demonstrated that while patients are generally sensitive to out-of-pocket costs they're willing to pay higher prices for

treatments of rare and complex diseases. so to continue developing the types of innovations patients about you manufacturers must expect positive returns under drug development. this undertaking is challenging. on average over 90% of drugs fail and of the small subset that do succeed it takes about 15 ten to 15 years from progress to initial discovery to progress from initial discovery to market approval. this high risk of significant financial loss requires difficult trade-offs. drug manufacturers will typically be forced to pursue drugs with the greatest market potential as measured by the size of the target patient population and potential revenues streams of the specific trait. due to the limited number of patients affected, rare disease at an early present weaker financial incentives for drug innovation. that is why i was a site is created policies like the 1983 orphan drug act to encourage rare disease drug development.

research has shown 90% of the innovation stimulated by the orphan drug act would not have occurred in absence of the policy. however, orphan drugs also to be some of the most expensive drugs on the market. so to strike a balance between ensuring nurturing ongoing pharmaceutical innovation prices of new drug treatments need to match the value they deliver. a value-based pricing approach ensures manufacturers are not only encouraged to correct high-value therapies but also discouraged from pursuing less beneficial drugs. three principles should guide pricing policies as relates to rare disease. price should reflect about you which should become pensively assessed ace on the benefits patients and their families prefer. second value as of that should be based on strong evidence, measured not only for rather buys clinical trials but also to the ongoing collection of real-world evidence. third, continued research should be rewarded the aunt a drugs

primary indication. this last point is particularly salient for orphan drugs. under the ira drugs with an orphan designation as their sole fda approved indication are exempt from future drug price negotiations but the ira does not extend this exemption to drugs with multiple rare indications. this diminishes the incentives manufacturers have for exploring alternative uses for existing drugs in treating other rare diseases. follow one indications are critical pathway for more cost effective innovation especially in the realm of rare diseases where the limited sizes of patient market constrain revenues. consider the fact the average id revenues for orphan drugs are 40% lower than they the art for non-orphan drugs. moreover ultra-rare diseases with truly low prevalence black bible r&d incentives without other supporting mechanisms. it is conceivable this publication brought on by the ira can be addressed to policy improvements like the orphan

cares act that could reinforce our societies desire to protect the interests of patients suffering from rare diseases. pharmaceutical prices make it seem like a logical approach and improving accessibility this comes a significant trade-off diminished future drug innovation. price price controls and not the answer because their counter productive from the perspective of improving patient health. instead, ensuring access to cutting-edge treatments among rare diseases necessitates the expiration of innovative financing message such as prescription-based payment models or approaches that amortize the cost insurers pay for a life-saving new treatment based on drug effectiveness. these types of solutions will create a more sustainable system that prioritizes patient access while simultaneously fostering valuable innovation for patients. thank you for the opportunity to testify today and the look for to answering any questions you may have. >> thank you for your testimony.

the chair now recognizes ms. davis for five minutes for your opening statement. >> chairman guthrie ranking member eshoo chairwoman black morris rikers, ranked number lowey, and distinguished members. i thank you for the opportunity to testify on this rare disease day. i am a widow the recently lost her husband to geo blessed over, rare brain cancer. i am a rare disease parent and i'm a rare disease patient advocate. a rare disease committee includes one in ten ten americans affected by more than 10,000 rare diseases. 95% of rare diseases like an approved treatment. at the current pace it will take thousands of years to secure treatment for all rare diseases. meanwhile, a third of june with rare diseases will not survive to their fifth birthday. and development of additional rare disease treatment and that rare disease patients can't access the

treatment upon approval. i joined the rare disease community and 2011 when doctors diagnosed our newborn son hunter. our world forever changed. estimate is like als in babies. and bob speedily to move, swallow and ultimately breathe. at the time was a number one genetic cause of death for infants in the u.s. doctors told us it was the treatment and no hope but we couldn't afford to listen. the stakes are too high. with help of a of the researcher we manufactured a compound in the u.s. and to get to mexico for a trial. eight weeks after his diagnosis hunter was a first sma patient received a disease modifying treatment, and that saved his life. sma has three approved treatments and every state screens newborns for sma. our sma community in the u.s.

has largely achieved what we want for the greater rareommunity presymptomatic diagnosis and treatment. the diagnosis would not be the only devastating rare diagnosis for our family. in june of 2022 my husband curtis was diagnosed with glioblastoma, a a leaf over brain cancer we knew his diagnosis was a death sentence. yet we still held out hope that we could secure another medical miracle. as we had for hunter. sadly, this time science and luck would not be on our side. glioblastoma falls squarely within the 95% of rare conditions lacking a disease modifying treatment. curtis fought fiercely and courageously despite having the porous biomarkers, his tumor invading his motor strip and

being on high-dose steroids, curtis was in the 25% of glioblastoma patients who survived more than one year. this is because curtis had access to innovative treatments that slowed the progression of his glioblastoma for some time. these innovative treatments about us to spend more time and make more cherished memories with curtis. curtis died on june 14 at the age of 54. i was by his side when he died, and i watched him take his final breath. i lost my husband partner in adventure, an amazing father to our five children. he was an exceptional partner in all aspects of our relationship, including his partnership in being a caregiver for although glioblastoma is a rare cancer it is claimed long respected and loved person by those here in our nation's capital including senator ted kennedy, beau biden and senator john mccain.

congress recognize the unmet need for a disease treatments and the additional barriers to orphan drug development born out of the small patient populations and pass the orphan drug act in 1983. the oda incentivize development of orphan drugs for rare diseases it unfortunately orphan drugshan one indications become eligible for price negotiation under the ira. this provision undermines the oda jeopardizes continued research development and funding of orphan drugs that our rare disease community so desperately need. yesterday, arpa-h announced a $48.3 million award to every cure to develop an ai driven platform to revolutionize the future of drug development and resurfacing. it's much easier to leverage existing orphan drugs for additional rare indications than to develop novel drugs the ira sadistically threatens the likelihood that orphan drugs

identified as candidates to treat another indication would reach the rare disease patients they could help. the orphan cares act encourages repurchasing orphan drugs by exempting them from medicare drug price negotiation so long as they only treat rare conditions. this is a fix that our rare disease community needs. thank you. >> thank you for your testimony. thank you for being here and being an advocate. we appreciate you being here today. so that concludes all of her opening statement and we will now move to members questions and i will recognize myself i've been us to begin the question. make it we talked about we heard some testimony on pediatric priority review doctors. i know it's lurid approvals really important for all of us have and its advocates here in the arts today have people come to our office and talk about access to drugs and access to there, can stand bumping against time so it's important with remember that. it may only advance the few months but if you must can be

all the difference in the world. i've had a constituent with a child with muscular dystrophy said if i could can't reverse the damage that the disease does to them but if they could stop it they could stop when you are, so time does matter and so that's important for us. we can't dismiss that in an academic kind of way. so professor talking about key. the term negotiations but it really price control. congress uses the power to tax and audit set up price controls. i know everyone here it has a phd, j. d. or indy and i have neither, but i do have, i did have public school high school economics and price controls always bring timely enough elaborate system to prevent that but always brings the lower quality or shortages. so when you kind of come we talked about europe and what your face and it is frustrating that there are a lot of free writers on the american taxpayer for paying for innovation and the research in drugs but if we don't pay for it we won't get it. what i like to, what i say is you can't get what europe pays

with a getting what europe gets. and so would you talk about the european model for price controls and the limit they have on access to life-saving drugs? >> sure. schaeffer center research my colleagues have shown that if we adopt european price controls in the united states, the outcome is lower life expectancy. the reason for this is because price controls limit incentives for innovation in the u.s. without price controls reset access to new innovation quicker and we see also more new innovation which improves the lives of patients. >> well, thank you. i want to cut a move to dr. bassuk you talked about dr. miller-meeks builder i live in bowling greencontactors of people in my note live in kentucky london dash of world-class center computing the northern kentucky every ten minutes of cincinnati general and wonderful people from -- southern louisville. state lines don't sometimes

become a barrier when your medicaid so that's why that's before figure talked about. also talk to and ms. davis i have built up people pay for expenses also have to travel and so would you can talk about how expenses so have the ability for some manufactures to help assist with lodging travel, meals how they can be a very for people to come to get even if that access to the care? >> happy, happy to answer. we definitely appreciate the goal of that particular bill. anything that supports care coordination and in aspect of care outside of a child state were interested in exploring those policies that will alleviate the burdens for children and families in order for them to devise quality care and it really is a burden some of these really life-saving life altering you heard from ms. davis, spectacularly changing the way kids lives kids who before the kind of

treatment, treatment ms. davis talk about whenever set up, never talk him never eat on their own. these kids cannot walk and talk, and it's really amazing. but for some of these treatments and dr. flotte can discuss is the more, kids have to be from the hospital. they have to be under very close supervision because they can of extreme reactions they need to be taken care of. if they live come just feel like my if they're very far from academic center they're going to need some support. so anything we can do to support that is great. >> were lucky to have access close enough to drive but it gets to the other bill you talked about its across state lines. we have had a crusty. like is that ina kentucky for great health goes well. would you talk with your experience? i know you had come to have a bus in order to travel to make sure you could get to the places you need to go, and just the expense of what you had to go through as a family. >> yeah. our family is very fortunate that we do have the expenses

that we are able to bear that on our own. that's not the case for many families in the rare disease communit they need access to airfare other travel, hotel accommodations. when they are traveling with children you have to remember they are also having parents that are missing work so they can go with the children to these treatments. so there hitting a lot of additional expenses for the families. >> thank you. i appreciate your testimony come all you being here today. my time is expiring so i want you back and recognize the ranking member for five minutes of questions. >> thank you mr. chairman. in listening to each of you i have to say this. you are a blessing to our country. that we have, you know, such experts that have devoted their lives to bring that expertise

for the betterment of humankind. so bravo to each one of you. i'm deeply moved by what you do, and your testimony today. dr. chen i want to take exception to a part of your testimony. i don't have a question, but to leave the impressiont with the federal poverty line for for a family of four in our country at $31,200, that you would reject affordability for drugs in our country, i really just can't stand. i mean, i just rejected out of hand. a child that has a

rare disease would want to bring every last thing from themselves their extended family and friends to help that child. but to reject, you know, affordability when some drugs are hundreds of thousandsllars. i wouldn't be able to afford that and i have a pretty job. and i don't think you would either on your salary. so the affordability of drugs in this country is an essential policy in my view, and we can innovate in we can bring the prices down as well. and anything that stands in the wake of that i have to say i think does not serve the american people well. and it's the congress that has to put that policy in place. it's the congress. and after what, 75 years we finally got to do it.

and bravo to those that voted for it. doctor christa mcauliffe is one of my constituents. she is also a leading cancer research at the stanford university that i the privilege of representing and the former chief of pediatric oncology branch at the national cancer institute. she said the following about the fda's rare pediatric disease voucher program which my creating hope help of reauthorization act would extend for four years quote, before the program i used to go with my hat in hand to big investors to consider a potential drug. now people take a second look and are interested in developing drugs here for example cd 19 car known as -- the first cell-based gene therapy approved in the united states was approved in children first. that would have never happened without the voucher program unquote.

that's quite a compliment. so doctor i mean, this is i know the answer but it wanted for the record. have children benefited from drugs made possible by the rare pediatric disease voucher program, drugs like this? >> in my opinion congressman they certainly have. i just want describe the phenomenon in private sector investment in gene therapy. much of it has been in the high risk biotech sector, later to transition to farmer. many of the investors in that sector are looking at time come looking for a quick relatively quick return. now, you can argue whether they should be doing that are not but the fact is that with, some of the discouraging news in other markets since 2021, over

50 rare diseases have had their clinical programs in gene there's an article came out this month in human gene therapy which am happy to share with the committee that describes each one of his programs that was stopped. we are at the mercy and families of patients with rare disease, are at the mercy of investment community to some extent for these highly innovatives that are really going to come out of the small biotech companies. >> i represent many of those small biotech companies. of the more than 7000 rare diseases 70% are rare genetic conditions present from the time a child is born, which i believe ms. davis spoke to. how can we ensure that children are not left out of the new frontier in cell and gene therapy?

i'm over my time. we can take that for the record. >> thank you everyone. appreciate you. >> the gentlelady just back in the chair recognizes chair rodgers for five minutes for questions. >> thank you, mr. chairman. professor chen, i just wanted to give you the opportunity to respond to some of the statement around the affordability of drugs and if you had any insight you would like to add? >> absolutely. let me be clear affordability is very important. this is why we have insurance. insurance was designed to give patients financial protection in the case of rare and catastrophic events. the price of the drug needs to match the value in order to continue to incentivize innovation that is valuable. do we need insurance policies to be able to cover the valuable treatment that patients need. >> thank you. i would also like to ask because last year we had the debate last congress, last congress with the debate around

h.r. three. we had continued hearings on the ir heard that true rare disease drugs are not at risk, onlyr drugs. today we have some witnesses suggesting the evidence is in and we already know the irate instancing typically impacting the development of rare disease treatments. professor chen, how would you respond to those claims? >> i don't think we have enough evidence yet. my own research along with others my colleagues, has shown when you look at the medicare part d act that was introduced in 2003, we didn't see any changes in innovation due to the market size expansion until 2006. 2006. the price negotiations in the ira have not come to fruition yet so we need to wait and see. >> thank you. a few of the bills before us would expand the fda's authority to require drug companies seeking approval for an adult cancer drug, orphan drug come to do studies in pediatric populations as well. if a company were to comply with these mandates and end up getting approval from both the

adult orphan indication and also the pediatric indication, with that trigger price caps? >> some not an expert in the invitation of the ira but as a understand it only drugs with a soul indication for rare disease is exempt. so to the extent those two categories are considered as two different rare disease indications, and that would trigger price negotiation. >> thank you. ms. davis, first i'm very saddened to hear about husband, passing of your husband, and thank you for being here today. your strong advocacy. certainly we are hearing from colleagues across, my colleagues across t aisle their belief in the irate and visions that were put into place. i just want to ask you to give insights srs if you believe there are small things that need to be changed for the rare disease community? >> yes absolutely. when you were a member of this community you see firsthand the struggles of research and development in recruiting patients for clinical trials.

all of these are much more burdensome for the rare disease community and they are much more costly. that's why we have the oda and unfortunately, the ira negatively impacts the efficacy of the oda in driving the innovative research and development that the rare disease community needs to help ensure that we bring treatment for the 95% of rare diseases that lack an approved treatment. >> thank you. dr. flotte excited to see recent advancements in cell and gene therapies for rare disease patients. what are you most excited about over the next five years and are those innovations mostly small molecule or biological products? >> so we, asgct of course represents scientist who are pushing back the boundaries of frontiers of research on both oligonucleotides which a class

with a small molecules and on gene and cell therapies. the thing i most excited about is to see the clinical come to further clinical dissemination of gene pending technology, crispr technology for the treatment of human diseases in semitic cells and affected organs in diseases. >> thank you. in your testimony referenced your experiences working with industry partners on clinical trials. some of which have failed for either scientific reasons for funding challenges. what is your sense of why funding challenges take place? how do you as an investigator respond when disruptions occur and do you sense there's a risk to some exciting innovation that you discussed? >> yeah, i do since there is a risk. as i majored there recently been a number of clinical gene therapy programs that are been discontinued. the advancement of these therapies requires participation from nih from private industry,

from disease foundations from universities as well. so when one of those parts falls out, it is very difficult to continue some of these programs. as the chair said, for the families the clock is ticking for their children if the disease is progressive which most of them are. >> thank you. just let me also say thank you everyone for being here, the witnesses. we appreciate your insights. we are all committed it's an exciting time when you think about innovation but we need to make sure we have access and affordability, and that's the goal today especially as relates to those with rare diseases, so thank you very much. i yield back. >> the chair recognizes mr. sarbanes for five minutes for questions. >> i would like to stop by yielding one minute to ms. schakowsky. >> thank you so much for giving a one minute. i am so grateful that the legislation that i introduced, the alzheimer's care act to

improve help for people with and what i really am happy about is what it does, is that it has multidisciplinary clinics for alzheimer's disease patients. and i just wanted to ask dr. kesselheim why kind of clinic be more helpful than what is available right now? seems to me it would more focused on the 30,000 plus people in the united states that you have alzheimer's disease. >> i mean, i think multidisciplinary care, the kind of talk that would be really important for patients with alzheimer's disease who require care from a number of different disciplines medical doctors respiratory therapist physical

therapist. i think the billiard talk about which would help enhance and coordinate that care would be extremely important in providing the necessary care that patients may need. >> alzheimer's is truly complicated, right? so you need this multidisciplinary group to be there. >> another important part of your bill is it encourages the enrollment in clinical trials which again is the real way that we move science forward in trying to figure out what products actually work for patients with als. >> thank you so much and i yield back and thank you mr. sarbanes for yielding. >> my pleasure. i appreciate today's discussion about how we incentivize research and development for therapies and cures for rare disease populations and the testimony here today has been very powerful. i know we had a discussion about whether inflation reduction act policies to rein in the high cost of prescription drugs may be harming these types of investments but i don't think the data from what i understand

supports that assertion.ead, it tells a different story actually and i like to enter into without objection mr. chairman, into the record a written statement from david mitchell the founder of patients for affordable drugs. >> we have d listed in -- >> thank you. he notes companies have reported increased investment in research and development as this landmark legislation was emerging on the horizon and even in the wake of its passage. for example in 2022, johnson & johnson reported 11.8% increase in r&d spending. merck and 11% increase, moderna cigie 5% increase. with further further increase of projected in 2023 and cbo is found there's been a consistent and continuous increase in capital investment. dr. ballreich, this research similar indicate the inflation

reduction act has not in fact, nvestment in new and innovative therapies and cures and is continue to provide incentives actually for such investment? >> yes. we actually have not seen any tangible evidence that companies have cut their r&d investment. we also look at the venture capital market. overall, total venture capital funds have contracted from their peak but if you look at the biopharmaceutical share of venture capital it's actually expanded since its peak. and there's constant reports of very robust mergers and acquisition activity. happening in the biopharmaceutical space. so there's a lot of doom and gloom rhetoric but we don't see actual dollars since business decisions changing. >> when this was coming together the inflation reduction act was carefully crafted to ensure we are providing the right kinds of incentives, once the spur innovation to truly novel

therapies that can demonstrate high-value versus just the repurposing of older products or similar drugs that already have many alternatives. dr. kesselheim his research for novel therapies they kind of research we should be seeking and incentivizing? i think you would suggest and is the ira in your view drafted in ways they can accomplish this? >> absolutely it is. the ira is intended to get medica to lead to a fair price not a rock-bottom price a fair price on a drug aftert's only been on the market for nine to 13 years. what it will do is what this incentivize companies from the usual strategies to try to extend market exclusivities under existing products for as long as humanly possible and if they try to direct companies to invest in more newer products that will provide meaningful improvements to patient. >> thank you very much. i yield back. >> the time of the gentleman has expired. the chair recognizes dr. burgess for five is for questions. >> thank you chair guthrie and

referencing your earlier remarks on the constituents you had with muscular dystrophy many of these illnesses are by their very nature aggressive in any delay in establishing therapeutic regimen means that there is going to be ground lost that can never be reclaimed. this committee did really important work several years ago on the right to try act back outside to do law and it evil has i mean i hear from constituents particularly in the muscular dystrophy space where that right to try is a significant difference. i just want to remind us all the work we do here on this dais and at the witness table is extremely important for the future. i recently introduced h.r. 7432, sickle cell disease comprehensive care act to allow state medicaid programs to provide comprehensive coordinated care to patients with sickle cell disease through

a home health model. bobby rush was a member of this committee for years was concerned we did the cures for the 21st century we have not really establish anything new for sickle cell disease and he said it's been years since that is happened. and we did have made significant efforts since that time to improve the life of patients were diagnosed with sickle cell disease. the ability to coordinate care i think is going to be so important. i do want to thank congressman danny davis is not part of this committee but has been an advocate for sickle cell and congressman eydie carter who is on this committee for the partnership. i just ask dr. bassuk if you could speak to the role case president can play in helping to ensure that patients the special registers patients get the care they need? >> yes. absolutely. as we've heard before mentioned that we have to correlate the

care take the with these new treatments. you mentioned sickle cell disease here it's been a tricky, it's one of the first or the first genetic disease of renewable we knew the gene and for a long time we didn't have great treatments and now we have two just approved excellent treatments. one is using what dr. flotte referred to earlier this crispr therapy, another one is using a viral vector i believe our antiviral vector to give the right hemoglobin the case having a problem with her hemoglobin. when weherapies for some of them the cellcept removed the back in a patient practice requires literal team who can fill the shrimp for one patient. so we have to correlate the care and that's just one example. thank you. >> and thank you for that answer. ms. davis, i do want to say thank you for being a today and

sharing your very powerful story with you with us. we have the creating hope reauthorization act to extend the fda's authority to issue priority review vouchers. seems like there was a recent instance of a rare small disease company anticipating receiving this voucher and all we learned at 11th i was at the fda did not intend to grant the priority review voucher. could you speak to have this uncertainty adds another layer of complexity in this space that's already complicated enough? >> yes, and i can show where it's working too because the estimate has two approved treatments or three approved treatments and two of those were awarded priority review vouchers. vouchers. so there's an example of showing how it's worke what we need in the rear disease space is predictability and reliability. there are ambassadors of provide the essential fun and ringing treatment to market. really depend on that.

the priority review voucher ge and secure those revise and essential funding mechanism to provide additional funding for the smaller biotech companies that so frequently bring our treatments to market. so it's one thing to secure approval for these treatments, but then once they the secure approval to have to have funding in place to make sure those treatments reached the patients, and that is what the priority review vouchers will do and mean for the rare disease community. >> and again thank you for your presence on the panel today. it is so important when we talk about a team approach to things, we are facing a situation in this country where we are losing physicians at an absolutely unbelievable rate. we had a hearing last week for the week before on physician burnout. it's not surprising when you

work people as hard as doctors are worked currently and then turn around with medicare cutting prices. price controls are not always helpful and, in fact, they can lead to the shortages of a previous he mentioned. unfortunately we're see that in the physicians based we don't do something about it. i know this committee has done some work but we've got a lot still to do. chair guthrie i thank you for the time and w beck. >> the chair recognizes mr. cardin us for fiber. >> thank you chair guthrie and ranking member eshoo for holding this important hearing. i also want to thank the witnesses for sharing your expertise and your opinions with us this morning before the american public. providing relief and life-saving help to people who have had little reason for hope is ultimately what this is all about. that's why i'm thrilled to see bills that i support up for discussion today including the benefit act which would require the fda to consider relevant erienced data in their new drug approval process

and the et cetera to get access to care act which would ensure that kids can cross state lines and retain coverage for specialist services to treat rare conditions. i am to see that give kids the chance act unless the bills did as well since it will ultimately improve the potential for treatment options for pediatric cancer. these bills make life altering improvements for those who have had every reason to believe their suffering was inevitable. i also want to thank representative miss an issue for leadership and innovation on pediatric populations and it's an incredible legacy to lead to ensure kids with cancer and rare diseases have hope to treat their conditions, so thank you to our colleague anna eshoo. my first question isor dr. bassuk. in your opinion as a pediatrician, what kind and packed with policy like that

give kids a a chance act which would authorize clinical trials for combinations of drugs to treat pediatric cancer have on families who feel they have exhausted all of their options? >> i don't know the exact details of the bill, but i will say the following. when i was before i was born, if you were born, if your child with leukemia you at 90% chance of dying within five years. by the time i finished medical school year to 90% chance of surviving five years later and that's because of research. last time i was on call i was called to see baby with a brain tumor and had to do something the sometimes pediatric neurologist had to do which is a brain death exam because we have no good treatments for brain tumor patients. they are clearly cases where we need to do more than we are doing and we need to do everything that is available to us and you defend that gives us that kind of ability is something we're all going to

support. ortance of using the most humane possible to test new innovations and therapeutics. this means we must move away from animal testing where and if at all possible. to be clear this transition is not only an animal welfare position but an imperative to advancing human health as well. we know approximately 90% of drugs fail in human test after doing, going through animal trials. in some cases there is reason to believe nonanimal methods are actually more effective and failing to phase out harmful animal tests where possible lease new innovations on the table. i was glad to see the fda modernization act 2.0 yet signed into law last congress. i appreciate efforts to try to implement those policies. most importantly that we must ensure that these efforts are safe and effective for the populations they are intended to treat. dr. flotte you mention in your

testimony that, quote clearly on regulatory expectations for alternative testing methods is crucial, and research development science-based adoption of such alternative such must come should be prioritized, end quote. can you say more about speed effective new alternative methods to be more congressman. the issue is a complex one of course because when certain new modalities basic platforms of gene and cell therapy to the fore they require some level of animal testing in order to identify previously unanticipated adverse effects. so that is not a 100% ..

we see first-hand years in advance what we can do to market those treatments as soon as possible, especially those when we are talking about rare diseases. he has a long-standing history

working in a bipartisan fashion. i'm proud of the work we have accomplished in the years past. i do believe our innovation where can be a risk. . medical breakthroughs have been made treating rare diseases such as cancer and i fear all the progress accomplished might be in jeopardy for those we can and must come together to protect research and rare diseases. i want to thank all of those who have been in the research and development of these to help so many people. we think about small molecules what comes to mind for treating conditions to manufacture?

>> if you're going to put up price control on a high revenue drug and it is a high-value drug all of a sudden intended for the high-value drug has been reduced. it will hinder the ability to receive the drug that is most valuable to our society. >> thank you. is an iterative

process, especially for oncology drugs. the majority of research and cancer therapy after initial approval, including in the earlier stages of cancer and the overall rate of innovation acceleration over time. exploring overtime. the cures act is a good first step how can we ensure there are incentives for innovation things of life throughout the innovation lifecycle continue to come to market. >> thank you. it is question, what it would reiterate many incentives we have talked about the regulatory reviewthe protections the drugs have and in some cases, to define conditions based on specific mutations. as in some type of cancer where it may not meet the red definition.

it may meet that definition. so in a variety of ways, we work collaboratively with the fda. i think any legislation that enhances the investment environment so we can get more private industryunding for these types of treatments is essential right now. >> what diseases might be most impacted by the passage of the act? what would be negatively impacted if it does not pass? >> it is discussed -- doesn't have a particular discussion on the act. there are a subset of treatments in our attachment based on the nucleotides that has synthesized short stretches of dna or rna. such as treatments for sma one and all of the treatment. we feel if they are treating

rail disease, should be treatment and advancement. i was contacted by the medical director of the washington county tuberculosis clinic. receiving a patient for severe postsurgical tuberculosis infection. after a month battling severe tv in the intensive care unit, she died. the position was particularly since she realized she had received a bone graft that would be --

seven of them developed tb infections and eight died. it has been discovered that sandra's death was indeed linked to contaminated biographical material produced again by -- biologic. she was one of 36 patients to receive materials from the contaminated lot. and as of today, the outbreak is linked to the death of two people. including sandra. i'm grateful that the chair included the effective screening and testing for tuberculosis act. and in tissue private safety act as part of today's hearing. i'm leading these bills along with my friend and colleague representative john moolenaar who was here. he's joined by chandra's sister. who also happens to be his

staffer. i want to thank you. we all honor you today and grieve for your sister. so we have been working closely with the fda and cdc to better understand the manufacturing regulations in cells and tissues to make sure they can be used to improve patient outcomes must not harm them. how does the use of human cell tissues and tissue-based products support your practice in caring for patients with rare disease? >> thank you, congressman. there are a variety of different products. some of which are combined with gene therapy as the doctor mentioned with treatments for sickle cell disease require a cellular therapy with gene alteration or editing in order to be used.

what is pointed out is they are a wide range of cell therapies, some of which combined the gene therapies, and some which do not. the regulatory science has to continue to advance in order to find out a better test for these unanticipated consequences such as the one you talked about. we don't want to see anyone else die, how can we maintain the integrity and safety profile of these products? >> i don't know the specific case or the specific details to address that particular one. the point is the regulatory frameworks are testing for quality control cannot be static when new dangers are identified, the treatments -- the quality testing for the treatments has to be updated and improved. >> do you think the american

public understands the risk associated with other donor materials? do you think people who get a bone graft from their dentist think i could die from this? >> i certainly don't have data upon which to base an opinion like that. i would simply state that right now, within the purview of the provider of the physician or the dentist who has accurately as possible summarized the risks and benefits. i don' well-informed patients are. >> i suspect patients are not told at all. people on this committee are tired of hearing about it. but i had multiple bone grafts and no one ever gave me the risk. that is why these pieces of legislation are so important. taken together, they will ensure steps that patients better understand the risk associated with bone graft materials and improve testing and screening of

tuberculosis in donor materials used in medical procedures. i look forward to working with the members of this committee and my dear friend to ensure we get these bills right. i yield back. >> chair recognizes mr. griffith for five minutes of questions. >> i comerom a little town in southwest virginia. but i grew up with a family that was afflicted with huntington's disease. since that time, also another family that through an adoption had no way 40 years ago of knowing what was going on. that family now has learned it also has huntington's disease. in your testimony, you mentioned targeted therapies that directly address the

>> are a lot therapies the targeted therapies in particular are there to address the underlying genetic mechanisms. as we learn more in innovation and move through gene therapy and cell therapy, we have more treatments down the road for us. >> the situation particularly with huntington's disease is so dire, one member of that family has told me he doesn't want to be tested. he's afraid to find out whether he has the disease because there is nothing that can be done for it. do you think some of the revisions of the inflationary reduction act particularly in what i believe have unconstitutional provisions that we don't cooperate with the federal government and negotiate the way they want you to? you think that will hinder or enhanc therapies for people facing these types of rare diseases?

>> already for the sole indication for rare disease. the challenge here is drug manufacturers who enter this space consider the pipeline of indications they might get approval for. . it just incentivizes drug manufacturers from entering , which is a cost effective way of doing r&d for the rare disease space. >> if i can translate into layman's terms. if doing the research and you have one disorder, and some of your think tank team says it might help huntington's disease you are afraid to trap because it would track you into having finds, or penalties put on you.

by medicare, medicaid. is that correct? >> that is right. you are subject to price negotiations. >> you are better off if you don't look? ok. huntington's disease as we have discussed is an inherited disorder. you mentioned the dino associated virus, which targets specific because of the disease and changes the way the cell functions. it treats inherited diseases without treatment. are you aware of any efforts to use this for to research this related to huntington's disease? >> yes congressman. there are. there have been several proof of concept studies using aab to deliver a form of rna interference. huntington's is a disease where you have to silence that gene and not simply add back a good

copy of the were two efforts i'm aware of one was at university of iowa by dr. beverly davis using aab to deliver this technology. the other was a series of studies being done at our institution under the leadership of dr. nila ronan, who is one of the people pushing the envelope forward for both gene-based therapies, and all of the nucleotide based therapies that has sensitized stretches of rna -- synthesized stretchto regulate that offending chain. >> if it is your friend who is afraid to get the test to find out whether or not he's carrying that, do you tell him the new trea test, or should he go on living his life in somewhat -- he's always worried about it, but more blissful because he

thinks he probably could not handle the stress. >> congressman, unfortunately i have been asked this question at different times. not specific to huntington but many of the diseases affecting kids. i have not made any guarantees. but i will say there is reason to still have hope or conditions like this. because if we do what we can do, industry does what they can do, on dacians do what they can do, i'm convinced we are going to beat most of these diseases in the end. >> i appreciate that. my time is up. i yield back. >> the gentleman yields back. >> thank you, mr. chairman. >> thank you mr. chairman. as a physician of treated patients of all ages with various health conditions. i've seen firsthand is a need for patients struggling with rare disease. we need expand access we need to prioritize innovation to develop new and potentially

life-saving treatments. and above all, we must not leave behind the most vulnerable patient's kids. will provide study therapies combinations innovative treatments to help them achieve. >> this this is one part of any individual for cancer they are all going to fail. if you look at adult combination therapies. we have to do combination therapy. cancer cells are working figure out what's going on with the

drug and find a workaround about why you have to gang up on them and use multiple drugs can look clinical trials and challenges they faced a quick path? >> it needs to be faster than it is no. there are many ways to do that. right now part of the investment because things take money and time to know will safety and efficacy but very often the window they close while this is being done so we can do approval and entry into the clinic and we

are working with the and if it works in a given cell they will substitute the gene for patients with a different related condition. >> tc companies embracing studies? >> companies do embrace pediatric studies more than they used to having been in the field a long time. i will say there's a lot of risk for companies in this field so incentives offset the risk and remain important. >> ensuring effective treatment is vital but also how innovative treatments and early detection

of cancer can save lives. not under consideration today i would like to reiterate support or another bipartisan bill. i like to reiterate support for another bipartisan bill and hr 407 soul medicare cancer detection screening project to increase access to multi- cancer early detection. can you talk about why innovative treatment to help reduce the burden of cancer the most vulnerable children and how can increase access help reduce disparities in cancer and equity? >> as discussed it would not detect early horses out of the barn to anything with the

patient's. expanding access to clinical trials some changes made in the past that had effects so i what is a small state 3 million people and the past would not generally hug clinical basis for cancer but now in the last year because of the loss we are talking kids in iowa, they don't have to go ice easter west coast because of legislation. >> i hurt my colleagues to support both hr 433 to help the package work on. >> recorder five minutes.

>> thank you mr. chairman. i'm down here in timeout thank you for being here, this is extremely important and thank you for this hearing all of these bills are extremely important. there are 30 million fellow americans who suffer from where disease and need our help. the vast majority don't have any cure or effective treatment and that's one reason why we are doing the work we're doing in this committee because we want to address that. it's important we have legislation to bring our healthcare system to the 21st century to help rare diseases get the treatment they need. we introduce the fda modernization 3.0 act. that would establish a program the fda to qualify the group testing methods to reduce or place animal models. it builds on fda modernization

act 2.0. as the next step. we are trying to do is really make sure pharmaceutical manufacturers can these models and make sure we are doing investigational drugs. i know how important it is to make sure improving market and polo a short time after. before not you agree fda author : and non- animal investments when it comes to evaluating safety spoke about in your

testimony? >> i mentioned earlier it's a complex question because new platform technologies clearly testing because models don't capitulate every organ of the body but adduced there are indications where a small increment on an existing treatment using the same warmth or substitution these models that have these should take this this. >> can you share your observation how it can better leverage and speed up the development of these drugs? >> for example described is cystic fibrosis referred to

earlier were approved in the earlier predecessors. initially a small range of mutation and the gene, expansion has been done with some form of the small that has to mutation examples from his patients. in that case it did not require a new indication going all the way back to preclinical animal testing. there examples that have come about the clinical animal testing and some of those cases animal testing did not predict

toxicity. it is a complex regulatory. >> one thing we are trying to address reforms as it relates to pricing transparency in the price of the drug from the office they are able to garner. if you -- can you describe how the controls not only destroy innovation but also how will make it harder for patients to access medications in the can still prefer higher price drugs to lower-priced drugs? >> in addition to incentives, venipuncture space here effects coverage that can

limit the patient's ability to access an entrance that comes to the market. >> i think all of you for being here americans suffer from rare disease, i think you and look back. >> the chair recognizes the ranking member. >> focusing on a small subset across the program. make this clear the law includes inclusion for orphan drugs that treat rare disease. exempt from negotiation and that would undermine the benefits he prices high but a series of

questions have to answerickly. he noted in your testimony hr 5539 and hr 5547 make rare disease less affordable while auctioning incentives for innovation so it the drug exclusion was expanded to include drugs with rare disease indication and hr 5539, what effect will this have on the negotiation program?

>> changing the timing of ellen eligibility indication despite the clear sense of law eligibility runs from the initial approval of the drug. would you expect manufacturers would likely attempt to gain provision to avoid negotiation for more than a decade or longer? >> i think it's definitely in the cards and important to recognize if you delay negotiation reduce the number of drugs to qualify for negotiations because they wouldn't qualify for the time actors get around the gains including exclusivity and the indication would rely on the fair prices out of the negotiations to help improve access to the drugs they need. >> hr five system similarly lays negotiation or certain subset or molecule drugs so how would delaying negotiations but these drugs impact the medicare

program? >> the doesn't seem to be any reason to exclude the drugs from negotiation because the kinds of drugs described if there is clarity they are extremely profitable drugs and has incentives. >> thank you there's no reason we should further limit drugs medicare to negotiate their prices only undermine the ability to deliver safety for patients particularly those of their condition and continue reauthorization with a rare disease and other pieces of legislation that will require the emblem mentation. i leave it's important to hear from fda on the other policies under consideration prior to any committee action

significant agency resources and important for the committee to understand some of these policies before moving forward. i want to go back to what i said earlier i think some of you know the drug pricing reform provision including reduction act the intent of drug price negotiation : advances but to reduce drug cost for medicare beneficiaries we can do that and the bills for considering today atonement this act undermine and goes back to saying the public and say over and over you can't negotiate prices and have innovation at the same time. i don't believe that. that's a false premise and panel

the reason you can't use it. lack of access -- if you don't have access, what is the reason. >> is on a broken record but thank you i yield back. >> gentlemen yields back. >> thank you very much for holding this hearing to discuss a wide array to improve the lives of not just those with rare diseaseppreciate my colleagues eliminating failed first authorizations for doctors and staff. most important national security preserving the doctor-patient relationship the driving force behind healthcare priorities since i've been here. it is getting more and more difficult to make sure the right drug gets to the right patient at t great harm.

appropriate care and it might as well be called bear first. it shouldn't be acceptable to you. rare disease patients could face challenges or appropriate treatments and probably most cases medication most effective for treatment are used. these treatments are often dramatically affected. years ago cancer patients a lot of cancer patients were labeled first or second line or third line. different order and combinations might have been considered off

label. i can't tell you how often i face but in my practice. congress recognizes by directing pears to look beyond the label and meet the threshold questions questions. building off those efforts to allow for similar flexibility in the rare disease hr 6094 protect their act would amend this for rare disease based peer-reviewed medical torture and guidelines in addition to the fda safety approvals which satisfied medical necessity requirements. i appreciate my colleague for working with me on this important legi why should the treatment be used

outside what it was originally approved for? small trials and small numbers of patients near labels. manufacturers could not afford to test all the diseases across these patients in various related diseases. this may lead to medical recommendations and peer-reviewed literature suggests the treatment or management for rare diseases. the associated costs and therefore improvement.

in short access to these treatments supported by peer-reviewed medical literature and i humbly ask my colleagues to join this common sense bill to increase access for lifesaving cures and ensure effective treatment of rare disease. i' describe one in my district tallahassee to receive treatment roughly $175,000 in treatment. why didn't they manufacture less effects of drug g? it is cost prohibitive. indications

on the drug already has it simply not cost-effective. flexibility in approach this and i urge everybody to support and protect this act. >> chair recognizes you for five minutes of questioning. >> thank you to the witnesses this hearing as an opportunity to recognize rare disease in the lives of millions of americans across the country including my district new hampshire. a chance for the committee to address b heard questions. many patients with rare diseases seeing their doctor often requires going to a different state and has a bear. a girl from new hampshire a few weeks old he did surgery to

correct an obstruction. her local one could not have the surgery. this partnership and the children's hospital thanks to received the care she needed when needed but other children are not unfortunate. a 1-year-old with a rare genital heart disease is a racing against the clock to find treatment. determined to be too complex and the children's hospital is ready to receive and rover the medicaid program the optimization to provide care. right now she and her family are still waiting hoping paperwork go through transferred to boston to save her life.

have you had patients in your care waiting to access care outside the state could you describe how the way impacted their care? >> we have had that in our state patients of unnecessary hurdles although i want to say we certainly welcome them but one example where somebody was really far from a they were closer to the hospital for many had severe disease and they were left they were told they could go to the other hospital.

. >> absolutely. we have 50 different medicaid programs so i >> in the state of i will and takes 30 days, pretty good but still too long. other states require figure printing, background checks, we have to send social security

numbers and they might still say no. it is a three or four months process and kids cannot wait that long. if you are waiting thing we can do on our center you are having more and more surgeries seizures seizures you will likely have more and more going forward so it comes up unfortunately all too frequently. >> impact to support the exhilarating kids act legislation that would permit as they help children and their families specialized out-of-state care provider enrollment for these families, i urge my alex to support this bill and with that, i yield back back. >> the chair recognizes mr. --

>> get to the witnesses all being here today. we are considering several bills today would increase innovation and improvement and improve patient with red disease. while they help safe and effective treatment options for a few hundred. i'm proud pharmaceutical invaders in my state you like willie on meeting george to develop groundbreaking medicine and it is equally challenging. my good friend doctor joyce. zach would make it critical for the inflation reduction act setting provisions for rare disease treatment for additional obligation good multiple rare

disease population the application of drug coming and discourage investment into care. important we do not love this 20 million americans with red diseases. i was recently informed of constituents recently lost her son just another courageous but with a rare form of disease burden we should incentivize research and develop it and give a chance to healthy and successful lives. in the oral testimony you say

the ira does not extend this exemption from future price negotiations with multiplications whether primarily orphan drugs are not raucous ministers incentive for manufacturers and exploring alternative use for existing drugs and diseases. when the cost of failure is so high how can we better incentivize drug manufacturers and innovative treatments? >> to get evaluate innovation pipeline and in order to incentivize innovation you need to consider the cost from one to

two in two to three and 32 approval. he needed to shake incentivize many factors in the red disease based. >> what you mean by inspected revenue? picking up price negotiations? >> the revenue we need because of it receiving the number of patients. the price of a drug the way we think about it. >> the free-market not the regulators? >> in this competition.

>> thank you. >> i yield the chairman. >> a lot of people have said walkout innovation. economic incentives so stopping everything is not the argument we are saying we are working out transparency and affordability.

>> i yield back. >> your recognize for pregnant. >> thank you for the witnesses being here today it's been an interesting discussion. the pediatric focus today and we have heard this impact children the most. thank you for sharing your story and my condolences. you brought up screening and i want to emphasize the importance of treatment of thesee rar diseases we already have newborn

screening, heal pricks newborns get in this country. all 55 of these recommended screenings are rare but if you treat them early it is a normal life. dietary restrictions and medications and gene therapies we heard about without early detectionacts disability and not like today's discussion.

travel costs state lines in seattle and regularly for diagnosis and care for rare disease not available in their own state. even if the provider is already enrolled in their own home state. care and treatment and that's why i strongly support the care act. newborn screening early diagnosis is critical. he mentioned the testimony i

hope and to hope, patient fortunate to live within a half hour. one or two patients in a course and the delay specialist. >> we have a case we have a comprehensive epilepsy study so we have an example is a powerful magnet. fda approval from clinical research including the length of the grain brain you can safely remove.

our surgeons know how to do this surgery and they sit home and continue to have seizures. >> the patient's for medical nutrition a medical decision equity act and i look forward to working with the ranking member on this. to quickly address an issue so many about medicare drug negotiation impacting rare disease research, this is a red herring. there price negotiations in the pediatric rare disease treatment

not covered by medicare in the first place such. >> i yield back. >> thank you for including two pieces of legislation the orphan cures 537 limiting act. these are critical pieces of legislation and patient treatment and to rare disease base. to ensure research and treatments that american patients walk and deserve are recognized. these are not in any way meant to undermine these mechanisms in

the ira but rather investment into the rare disease space and develop the next generation of therapy and diseases. patients and their families help. the orphan drug act signed into law the same year i graduated from medical and i witnessed firsthand innovations and cures the diagnosis of melanoma for the referral into hospice care in short order to ensure patients going to live. cellular therapy was an honest assessment. since the package almost 1200

new approval. i hope everyone regardless of party affiliation want a level of drug innovation to continue to treat diseases and passage of the ira infection 1192 is the acknowledgment that orphan disease treatment are meant to be shielded from the protocol and it's merely an effort to improve that corporations who ultimately will receive patients patients. in a similar vein, that narrowly targeted therapies.

negotiation timelines in for some good drugs to disproportionately impact treat diseases with high unmet medical needs. 30 million americans. in 2023 research resulted invel approval including rare and hard to treat conditions. on the survivor of a rare cancer

and it still impacts me everyday everyday. from an economic perspective can you speak to the value of research into additional indications and what it means for patients specifically in a rare disease kinetic? >> the postapproval is a more constructive way and the ideal ability to test the drug and other rare disease indications is valuable because it might not have been there if you had to develop it from the start. >> thank you for sharing the loss of your husband. can you speak to the importance of where conditions for your family and rare disease

community? >> we must continue to drive the foundation and access treatment. >> no the patient can only access medications if they first exist. thank you mr. chairman and i yield back. >> five minutes of questioning. thank you mr. chairman. >> , healthcare providers assistance to diagnosing a rare disease in five years and it's even longer for patients of color. the majority of rare disease does not have fda approved

treatment. i'm glad to see address rare disease day. because of how he people are. several of my colleagues in nostalgia program for developing treatments is used to accelerate rare disease and otherwise what not been the method. >> tech sector and eligibility for prb as an incentive to risks.

>> i want to thank the foundation and stakeholders who have worked to support this bill bill. fda modernization act 2.0 was passed into law in 2022. to support the use of alternative messages for the animals nonclinical test. fourteen months later the fda has not lamented the law. animal wallace act provided additional clarity by the fda and utilized drug developers.minimal biological models of the fda drug testing did not resemble human biology but human relevant models has to be more productive. ...

based on human cell culture are that they recapitulate the disease in terms of individual patient mutations. so in terms of the diverse applications one has to think about the disease who at the facts and have the cell models are alternative models reflect the population that the drug is intended for. >> great thank you. dr. chen today marks the end of blackish month will follow the tribes and struggles of black and african americans. sickle cell disease is a genetic blood disorder that affects approximately 100,000 americans. people afr much higher rates compared to other groups. despite the sickle cell disease has been historically underfunded. sickle cell disease comprehensive care act would allow state medicaid health home programs provide care to people with sickle cell disease. can you describe what medicaid

help homes are and how they can help address gaps in the care currently received by these patients? >> so medicare excuse me, medicaid health homes are very durable across each state state and political programs. the care record nation essentially medicare health homes offer tries to ensure patients have access to the primary care specialist, specialist care that they need. in her own research looking at care record nation models such as the accountable care organizations we found care record nation is important in reducing healthcare expenditures. >> so will this help families able to provide care for the loved one? >> to the extent that care record nation can help you access the medical care you need absolutely. >> thank you. dr. bassuk, the district has a significant medicaid population which is why i'm a proud cosponsor the children kids

access to care act. this will clarify medicaid and chip process so families are able to get specialized care they may not that may not be available in their home state. as a pediatric neurologist can you share why it is important to sure children from low-income communities are able to access the services they need? >> yeah. we need to get kids best care available for them where they are. if it's not available where there are we have to bring them to where the care is. that's actually why we and many of the societies would belong to a making two of them, the children's hospital association are absolutely behind this bill. >> thank you. every kid regardless of zip code regardless of income should have the same access to equal care. with that i yield back. >> 100% agree. that chair now gentlelady yields back to the chair recognizes mr. harshbarger for farmers. >> thank you, mr. chairman. thank you to the witnesses for day for being here.

i'll start with you dr. chen. today under the pediatric research equity act or pria, sort and orphan drugs are exempt from requirements to conduct pediatric studies. what characteristics do orphan drugs have such they might be exempted from these requirements? >> i'm not quite familiar with all of the details of the requirements for orphan drugs for pediatrics, but it is important to ensure we have incentives to innovate in that specific area. >> okay. could you tell me, tell me or expand our potential unintended consequences of requiring such studies for orphan drugs on future development decisions? in your testimony you were talking about drug development investments. on average 90% of these drugs fail. then you've got ten to 15 years required to progress them initial discovery to market approval and when you're in

that space and i been a pharmacist at 37 years and you know what it takes to get them from clinical trials and up. there's a lot of chance they will never make it to market. >> that's absolutely right. we know that without incentives like the orphan drug act 90% of the drugs that we see that it come out of that space to treat specific rare diseases would not have occurred. >> you spoke about the benefits of exploring potential follow-on uses existing orphan drugs to treat other rare diseases. you said follow-on indications represent a critical pathway for faster or cost effective innovation especially in the realm of rare disease treatment. overly narrow constructs of the and -- exemption could significantly discourage future research programs in rare disease and the orphan cares act would correct this by allowing medicines they

treat one or more rare diseases and only rare diseases to remain exempt from medicare drug pricing negotiations. so my question is, do you believe this solution would reduce the burden on innovative to pursue research and development for the millions of rare disease patients without treatment? >> it's precisely because follow-on indications represent a cost-effective way to do r&d and explore in this space is very important. it's important to note that as with her today only i've hundred of the rare diseases have the cure and some of these are very low prevalence populations. because of that the incentives for innovating and that space is particularly low. >> some of these rare or ultra rare disease may have 50 patients. i mean, very low number of people. the bottom line is, if you're wanting to invest in these companies you have to have an incentive to do that. it has to be profitable. how many times have we use drugs

off-label, physicians in the audience? we have used the multiple times for different diseases. they may come up with something that it cures later on down the road you have to go back and go through the whole process again. it's not conducive. i guess the last thing dr. chen, as an expert in health policy as an economist can you explain the challenges posed by the current policy environment to where disease drug development and what's future look like if it's left unchanged? >> the challenge here is designing a policy where we ensure we get the drugs patients really value and we want to create incentives for manufacturers to innovate for those high-value drugs. in particular for the rare disease space we know patients really value those treatments and we should take into account that just the clinical benefit of this drugs but also the patient preferences for those drugs. >> it may not have stopped

innovation but it certainly is stifled it. that's my opinion. i've only got like 52 seconds left. dr. flotte you talk a little bit about manufacturing technologies decentralized trials in real life stories. tell me how that works to me makes a lot of sense that you would include that. tell me about it bit about that. >> actually one of the real obstacles we have to overcome in clinical trials and gene and cell therapy is having outcomes that reflect in a quantifiable way what were actually seeing in the patient's. standard measures may or may not reflect the benefits that the patient's and families are perceiving and are a few examples of this. one that happened at the very beginning of a gene therapy a gene therapy was approved by the fda based on the ability to navigate a room in low lights,

and this had has never been used as an outcome before but it was generated from the fact that this is what the patients were reporting. and it was what they said was of value to them. of those kinds of outcomes, we those and not rigid about what will define benefit in each case. >> i totally agree. i know i'm over time so with that i will yield back. >> the chair recognizes ms. trahan for five minutes. >> thank you to the chair and ranking member for holding this important hearing. so grateful for the panel of experts here today and i give a special shout out to dr. flotte from u.s. chan medical school. as a mother of two young daughters it's great to see many of the bills we are focusing on will have a profound impact on the health of our kids. i'm particularly grateful that the accelerating kids act to care act which interviews with congresswoman mary mariannette miller-meeks is included in today's hearing. mr. chairman, i like to request

unanimous consent to enter into the record a statement from the children's hospital association and support of this bipartisan legislation. >> we will add that to the documents list and take action at the end of the meeting. >> thank you, mr. chair. when the necessary clinical care is determined for a child by medical professionals there should be no reason that administrative burdens get in the way. this accelerating kids access to care act is designed to cut through the red tape that kids, and kids get the appropriate care that they urgently need, to express the importance of getting this legislation over the finish line i would like to highlight a story from a patient who was negatively impacted by the burdensome and time-consuming medicaid provider screening and enrollment process. almost three years ago a baby, i will call her lilly was born in a rural state with her esophagus into make separate segments and connected abnormally to her windpipe. because this condition requires particularly conficker procedure to correct it, doctors explained

to the family there are only two places in the country where one of them was boston children's hospital in my home state of massausetts. immediately lilly and repairs are ready to travel to boston where the surgeons were ready to perform life-saving operation. but instead lily who is a medicaid recipient was waiting for her home state to enroll the eight providers at boston children's menu to perform this complex surgery. her state confused about the rules concerning out-of-state provider involvement wouldn't children. lilies surge was rescheduled more than three times and a laden over seven months as a lily waited she lived with a floppy and where they could have collapsed at any moment,g( a weakened immune system, and routine reading difficulties. so dr. bassuk, while the process differs from state to state can you please walk us through what it generally takes for a provider to enroll in another state medicaid program? >> yet. so as i mentioned previously, it

really does differ from state to state, and to the point that it feels like there are 50 different ways to do this. i am what i think is sort of on the good side of things here. we are pretty fast. if we can get someone enroll in 30 days or less, i don't think it's fast enough for kids like lily that you mentioned but there are states because of fingerprinting background, criminal background checks, having to send multiple forms of documentation where we had four four to six months weight. that's more than one case. that's not just one outlier. unfortunately the story that you can do something that we deal with all the time. >> when you see a child in your practice or took a long time to get to the right level of expertise to treat the condition, what does that mean that only for their health but for the healthcare costs associated with that child? >> yeah, there's no doubt that the healthcare costs are not

just up for the child and the family but for the whole society. i'll give you i have like us to before i pages of these examples but we had a baby in her neonatal intensive care unit was her for a long time. these oaths out-of-state medicaid. that original state was approved but we figured out that he had hearing problem and he needed to get a hearing he's hearing test done and hearing aids put in. the out-of-state medicaid so said he couldn't do it. so that baby actually missed development milestones that he may never make up. that baby may have learning problems the baby not be able to work the way that baby could have done. because of this will absolutelynecessary delays. and that is a huge financial that some expertise but i think that we can probably agree that that's going to have big financial impact. >> and you spoke about this in your testimony beyond the child how does this delay in care impact the well-being of the child's family?

>> so actually, and ms. davis mentioned this as well. i mean, there's of course the suffering of senior child suffer the anguish of the not getting care and the real financial costs. if you have to move, if you could have gone some really close outside your state which had to go for hours away of your state and stay at hotel a network. it's financially and emotionally damaging. >> and with that i urge my colleagues to support this bill and i yield back the balance of my time. >> gentlelady's time spac at the chairman recognizes dr. miller-meeks. >> thank you chair guthrie and thank the witnesses for testifying before the subcommittee. i am actually very grateful that we discussing these bills and a please visit the committee considering as you just heard from ms. trahan the accelerating kids access to cares act which would streamline administrative processes for providers who enroll in another state medicaid

program due to a lack of services provided in the patient's home state. i'm also proud to have a fellow iowan and constituent dr. bassuk i too was on the faculty of university of iowa, go hawkse before the committee today. there's currently no standardized federal pathway for this process means that providers must enroll any new state medicaid program whenever they see a new patient from that state and must maintain regular paperwork to remain in each state medicaid program. and especially in iowa with our four boards with many people from different states who seek care in our home state. when a child from a new state is visiting a doctor as may be the case if a child from another state travels to iowa for care, which is not unusual at all there are often delays in care as a providers sift through paperwork risking the health of children. mr. chairman, ask unanimous consent to insert letters of support from the leukemia and lymphoma society which is undersigned by 215 215 organizations, and from patients

rising for so many kids access to care and from patients senate in support of speed is if you submit those will add those to the document list and take action at the end of the expert one case that speaks to the need of the filth of also leverage or from another midwest eight who was a rare and safe condition known as mid-aortic syndrome narrows leading to multiple series collocation secretary shulkin death if untreated. in this case a referral was made from another children's hospital to boston children's hospital which operates a leading program to address this condition and a comprehensive care plan was assembled in april of 2022. in the case of the child's home state, all providers at boston children need to be enrolled in home state medicaid program before the state would authorize the character b july 2022 the enrollments were still pending three months later which led to a cancellation of care that have been scheduled for the following month august 2022. the home state did not complete its involvement of the boston children's providers until november 2022.

in early 2023 boston children's access the child and the coverage of care plan that included additional providers given the child's specific condition was assembled. a request for authorization was submitted in may 2023 and the care may 2023 and the care was provided in september 2023 nearly a year and half after the initial contact had occurred. and the child could easily have deceased before that time. dr. bassuk, i do know you've answered some of this. in your capacity as a physician and chief at the university of iowa's stead children somehow some houseplants and administered a burden and delays with providers enrolled in other states medicaid programs that impact pediatric care i can highlight barriers doctor scully face in this process? i think i saw skip over that and said does this legislation allow or require other state medicaid programs to cover services not covered by the patient's home state? >> all right. so i'll answer that second question. no it absolutely doesn't

require the home state to cover services that they don't cover. that's not what the bill is about at all. it's about making access easier for kids with medically complex and rare diseases. >> very importantly similarly with this legislation lead to an increase in services provided, or rather the same services provided but in a timely a manner? >> absolutely. it's about getting the right service in in a timely manner, not different services. >> lastly, very important as we looking at cbo scores, do care delays increase costs to the health care system? >> you know, in pediatrics we say pay me now or pay me more later. so yes. >> thank you. pay me now or pay me more later. dr. chen, the orphan cares act which is again the same moniker i'm hanging out emulator, which i probably cosponsor fixes the exemption for medicare drug after drug price negotiation to include treatments effective

against more than one rare disease. in contrast to my colleagues, whatever research and innovation goes on regards of age group can be transferred to another age group. all of that foundation of knowledge and science and innovation is transferable which also means research is done to develop one drug to treat one disease may in fact, find out it treats another disease or a side effect that we didn't even realize. why is this legislation important for ensuring we continue to make progress in developing treatments and cures for more than 90% of rare disease patients that do not have fda approved therapies? >> so you want to take into account all of the potential indications a drug has a viability for. some of those may be rare diseases some of those might not be in the bottom line is the entire pipeline matters. >> ms. davis i had a question for you but my time has run out. it's on small molecule medicines, thank you so much for being here. and the impact of the ira on

research and development. i would like to is that the collation to be answered -- >> absolute, thank you. >> i yield back. >> a gentlelady yields back and the chair recognizes you for five minutes. >> thank you very much to the chair and two are witnesses. i think my microphone is malfunctioning here. all right. technical difficulties resolved. i would like to thank the chair and the committee for including my bill h.r. 6020, the honoring our living donors act in this hearing. i . i introduced this along with my cosponsor congresswoman delbene in to solve a really important problem with the living donor reimbursement program. i think the people in israel are probably not surprised to hear that we have currently over 92,000 americans waiting for a kidney transplant, and yet the

number of living donors was only about 6000 last year, and it is declining rather than increasing. that is a really serious problem. so anything that we can do to incentivize and increase a number of living donors i think is going to be something that not only saves lives but is of service to a lot of the people that are waiting on that list. it's an incredible sacrifice to be an organ donor and the impediments to being one are severe. that's why congress of years ago authorized this living owner of donor reimbursement program whh in a very modest way reimburses costs like travel expenses dependent care, things of that nature for living donors. and by modest i mean the program is camped out at $6000 per donor. it is modest.

it's also means tested. a donor or the recipient of the oregon cannot have income over 350% of the poverty line. this is a probably guess it makes no sense to include the income of the recipient in this calculation. that is nothing to do with whether or not the organ donor is going to be able to make ends meet in donating the oregon. that's what this bill does. it makes that 350% means test apply only to the income of the donor, not the income of thel does is commission a a study on the program to seek ways that we can be more effective in both getting these donations funded and intoommunicating existence of the program to the recipients. i want to think very much, mr. chairman for including that bill industry. i certainly hope that we can get speedy passage for that to fix this problem. dr. bassuk, your testimony

really interested me and and i wanted to talk about it, with respect to organ donations. recent studies have indicated that in addition to frequently needing organ donations rare disease patients can't themselves be living donors. it's a misperception to think they can't be. they can be, and they have been, very effectively. can you talk about the importance of breaking down the barriers for rare disease patients to get care, including organ donation? >> yeah. i think i mean, i'll just reiterate what you said. patients with a rare disease kids with rare disease need all the treatment anybody else gets, and the special treatments that they need as well, and they deathly should not be excluded from standard therapy. >> right. thank you. ms. davis, i think i'm probably the last question are here it's a think it's appropriate to give you the last word.

after considering everything that's in said in the searing if you could tell people wanting to take away from hearing today what would it be? >> keep in mind that it will take thousands of years at the current pace to reach treatments for all patients with rare conditions. that's unacceptable, unacceptably and hope that that is unacceptable to all of you as well. we know that the orphan drug act is working. we seem the great insinuations that is provided in bringing novel therapy to patients in need. i urge all to turn to those incentives extend those protect our orphan drugs from harmful policies that will restrict innovation and access to the treatments that can help our patients, help our patients live better lives help our patients live longer lives and help us have more days to spend

and love our loved ones with rare conditions. thank you. >> thank you for your testimony of thank you to all other all of our witnses for your valuable testimony today. i yield back. >> that concludes the members of the committee testimony. we do have 1% that is asked to wait on from the full committee. i will now recognize ms. matsui from california for five minutes and that will be the final witness. >> thank you very much, mr. chairman. i want to thank you and the ranking member for holding this very important hearing. rare disease day is the time to race the winds come celebrate our advances and commit to building our progress. ms. davis, i've been moved by your testimony. al rare disease caucus, this is especially important to me. i'm grateful to my cochair for all of our bipartisan work on rare disease issues, and i'm thrilled to see so many bipartisan bills notice on today's agenda, including three bills i introduced, the benefit act, the rare act and protect

rare act. however, disappointed this been hijacked by bills to dismantle the ira. the ira was a major step forward in assuring americans including those with rare diseases don't have to choose between paying for their prescriptions and basic necessities. this is especially frustrating when you are so many commonsense proposals to ensure rare disease patients have access to effective and affordable therapies. i hope this committee can work together on these solutions rather than undermining the successful policies in the ira. i would like to turn to one such solution a benefit act. in recent years congresses work with the fda and patients has spurred the development of meaningful patient experience of data being submitted to the fda including as part of new drug applications. one way of continuing this momentum is to assure there is clarity around whether and how the fda uses the patient experience data. to address this gap i introduced

a benefit act to require fda to describe how patient experience data was incorporated in the review process. dr. flotte can you comment on the importance of incorporating patient experience of david into drug reviews? >> thank you congressman. it turns out to be as an obstacle that we're facing right now in the development of several gene therapies diseases. we can compare patients who have disease with those who do not but that doesn't always give us a clear-cut in point when racing benefits from a treatment. and what could be more important than how to benefit his experience by the patient and their families, particularly parents. the ability to get through to get more drugs through the pipeline is what families want. and if these treatments are actually producing benefits that

are affecting patient experience, there has to be real validity to that. >> right thank you. next i like to turn to the rare act. i introduced the rare act with -- to codify long-standing fda policy on orphan exclusivity and ensure companies are not gaming the system to get new market. dr. kesselheim how would the rare act ensure continued development of additional therapies for rare diseases. >> thank you. i think the rare act as an important bill because under the 11th circuit decision that created this loophole of the orphan drug act a manufacturer now can get a rare disease designation for a particular disease and as that time is ending get into designation for a slightly different version or slightly expanded population, and to that additional seven years prevent other competitors

from coming on the market for the initial indication. and in doing so create almost perpetual state of market exclusivity that would prevent the competition that would both lower prices for patients and help incentivize new discoveries of the future. so i think a rare act is incredibly important to a close that loophole and come back to the fda's long-standing original interpretation of the orphan drug act. >> thank you. and now want to turn to another issue for rare disease patients, affording the necessary medications. over 90% of patients lacked an fda approved treatment for the rare disease. as i introduced introduced to protect rare act with represent done to ensure patients have access medically necessary off-label therapies. to show a critical this can be i would like to share a story from what my constituents. janet was in her 30s raising a six year old daughter when she was diagnosed with a rare and fatal disease called -- despite

her doctors prescribe medications at the standard of care for her condition her insurance would not cover the stream is because they were off-label. rather than focusing on healing and spending time with her family janet was forced to fight for care while her symptoms and pain worsened and her finances suffered. i know i don't have much time here. i want to ask the question. dr. kesselheim you know from your focus groups with prejudice patients that they are struggling to afford necessary care is a common thing. how with the policies on ira ease the financial burdens of rare disease patients and families? asked the question to realize i've got over so i've would speak to give a brief answer. we'll give would give you a few seconds. i think the ira is actually important to rare disease patient and it comes because it helps promote fair prices for these products after the bodies have been on the market, and fair prices help translate to better access to patients, to the drugs that they need. >> thank you very much. thank you mr. chairman.

>> that concludes all members questions and i do say there are a lot of people in the room that represent families or people that have rare diseases or have children or family members with rare diseases. ms. davis i know you are speaking for them today and we really appreciate your willingness to do so. that's how our loved ones live on. we see it all the time in congress and makes a difference. paleness bills the past, given things and move forward because of advocacy, so thank you all so much. we were taught by how wonderful the panel is and how good you guys are in representing what you do. we really appreciate that. so i do have staff documents for the record list and it includes what mr. sarbanes, doctor shrine dr. miller-meeks brought forward. i ask unanimous consent to insert in the wreck the documents included on the list. without objection so ordered. and i will remind members decked in business days to submit questions for the record and os the witnesses if they respond promptly. members should submit their questions at the close of

business on march 14. without objection -- did you -- >> mr. chairman, i'd like to respond to what you said earlier about cbo and cheers if i might. there is a from cbo to ms. errington and dr. burgess from our committee mr. harrington, the chairman of the house budget committee. the letter was from cbo said that, quote the share of venture capital reaching pharmaceutical companies has been trending upward, end of quote, since the drug negotiation program was enacted. so i think that that's important important, when i'm presenting i think it's important to be part

of the record today because they are, i think this stands contrary to what testimony has been given. so i thank you for give me the time to do so. >> window investments company and we all know where disease are included in that. so thank you so much for being part of this subcommittee hearing. as i said, members submit a question by the close of visit march 14. without objection the subcommittee is adjourned. writes a lot. thanks everybody for being here. thank you. [inaudible conversations] [inaudible conversations] [inaudible conversations]

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