congressman kevin mccarthy's ter wch lasts until the end of the year. later t hse will consider the spending bill for 2025 funding forilitary construction projects, and the veterans affairs department. the senate also returns for recess on monday at 3:00 p.m. espn. next wk, senate lawmakers will consider several executive minations for the nuclear regulatory commission, and the s. representative to the african union. they're also expeedo take up judicial nominations to the d.c. surior court. watch live coveragof the house on c-span, see the senate on c-span2. also watch all our congressional verage with our free video app, c-span now, or online at cspan.org. >> the the house will be in order. >> this year c-span celebrates 45 years of covering congress like no other. since 1979, we've been your primary source for capitol hill, providing balanced, unfiltered

coverage of government. taking you to where the policies debated and decided, all with the support of america's cable companies. c-span, 45 years and counting, powered by cable. next, medical professionals and public health researchers testifying on ways to support patients with rare diseases. from the house energy and commerce subcommittee on health, this is just under three hours. she■o. >> the subcommittee will come to order and determine will recognize self for five minutes of opening statements. than 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 rare diseases and the process by which they seek approval 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 healthcare services and provides access for medicaid patients. finally efforts to increase access life-saving therapies we are considering trying patients getting access to affordable treatment to treat rare diseases.

>> thank you, mr. chairman, and good morning, colleagues, and all of our guests here in our hearing room. today on rare disease day, we're considering several bills to help the one in 10 americans living with a rare disease. over the last 40 years, there's been a revolution in the development of drugs to treat

rare disease, also known as orphan drugs. f.d.a. has approved more than 5,000 orphan drugs since passage of the orphan drug act of 1983. last year, more than half of all new drugs approved by the f.d.a. were orphan drugs, bringing hope to millions of americans, but there's room for improvement. only 5% of the more than 7,000 known rare diseases have a f.d.a. approved treatment and clinical trials don't reflect the diversity of our nation. among neglected are children. few f.d.a.-approved treatments for rare diseases have been tested to be used in children. children are not little adults. something congress recognized 20 years ago, when it passed my best pharmaceuticals for children act, and my pediatric research equity act to reward

and require pediatric studies. there's more congress can do to ensure that children are not left behind. 36% of drugs approve the rare diseases relevant to children since 1999 lack some or all pediatric data. the innovation and pediatric drug debt, which i introduced with representative mccaul, will close the loopholes so that we have a clinical, so that we have the clinical data we need to safely treat children with new cures. the american academy of pediatrics, the leukemia, a lymphoma society, the children's hospital association, stanford children's health, the alliance for childhood cancer, and the national organization for rare disorders endorsed this bill, and i thank all of those organizations. children should also benefit

from advancements in cancer treatment, which is why representative mccaul and i introduced the give kids a chance act. currently the f.d.a. cannot direct clinical trials to test combinations of drugs in children despite combination therapies proving effective in adults. our legislation gives f.d.a. th authority. legislation has 188 bipartisan cosponsors and hash endorsed by 50 organizations and research. every member of this committee voted for this legislation as part of the f.d.a. user legislation last congress. finally i joined mccaul, burgess, bilirakis in introducing the creating hope reauthorization act, incentivized research by providing pediatric drug developersdevelopers with voucho

speak at reviews of new drug products. the pediatric review voucher program can make the difference between whether a drug comes to market or not. more than 114 organizations endorsed this bill and the last time this legislation was up for reauthorization, it passed the house unanimously by a voice vote. highly instructive to all of us. 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. just over 40 years ago, only 38

drugs were fda approved. degree alone fda and get them intentionally.e 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 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 com■e down to whether the patient can't afford to travel . patient axis 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 tape that restricts the paid in treat kidsdifferent pro.

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 doit whr witnesses finding the committee could move forward with bipartisan legislation should. >> the gentleman from new 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 should biden 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 negotiationse 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 those who raised prices should, seniors say $618 on

average does on 47 drugs despite the fact that it's making it more affordable for nations seniors 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, american family paper high-priced 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 and therefore we know patient seen affordable access to therapies to glory these conditions. i'm just a nuclear discussing this allows potential drugs totg

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 avoid gettin. the republican plan laid out today will keep these out of reach for two-minute families. while i cannot suprtermines the inflation reduction act we can

incentivize 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 the voucher administration. my concern about 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 t 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 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 witnesses offering statements you will have fivemi. 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 openingst. >> thank you honorable members of the committee. thank you for the opportunity to testify. the associate research professor. i recognize the need broad access, affordability which led innovation. 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 problem. 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 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 my biopharmaceutical companies. 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 theirtainly 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 human 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 drs 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 organization of physicians,, scientists, patient advocates and other professionals working together in universities, hospitals andies to expand t 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 causesne mutations ww 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 ohediseases, only handful ag 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 featured over, , when 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 stable investment 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 thera more tha quarter of all designations in ucher program. this incentive program uses government dollars to reward successful products. th i developers, patient and 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 cane

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 anding

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 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 time-consuming 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 a 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 division director of 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 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 notve and. >> chairman guthrie, ranking member eshoo, chair rodgers, ranking memberel 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 fbrand-nan 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 rare disease. that exemption may have been included to ensure negotiators prioritize drugs that affect broader population but in reality single rare disease drugs generate substantial revenue. among approved single rare di last decade we found and begin drug 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 rare diseases were no different than drugs forus in these casesg 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■h the median sold re disease drug. nox 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 solid clinical development from second 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 drugc 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 fssion 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 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 areo promote effective care for rare

disease patients including some of the bills being 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 waivedpite 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 and 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 your opening statement. >> thank you, chairma 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 affordability and 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 fective 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 thenon-orph. 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 noe 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. ecognizes ms. davis for five minutes for your opening statement. >> chairman guthriean eshoo, chk 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 ames 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. it is crucial with foster research and development of

additional rare disease treatment and that rare disease the treatment upon approval. 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 weord. the stakes are too high. with help of a of the resee 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 hat 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 rare disease community, 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 bn 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 curti 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 ofur relationship, including his partnership in being a caregiver f hunter. although glioblastoma is a rareb 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-6 small patient populatis and pass the orphan drug act in 1983. the oda incentivize development of orphan drugs for rare diseases it unfortunately orphan drugs with more than 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 resurfacing. it's much easier to leverage existing orphan drugs for

additional rare indications than to develop novel drugs however, 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 chen, we're 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 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 >> 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 indiana has come to kentucky for great health goes well. would you talk with your experience? i know you had come to have a 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 community. 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 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 ofja 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 impression that with the federal poverty line 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 anyone with 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 thousands of dollars. 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 fi 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 privilege of representing and the former chief of pediatric o at the natr institute. she said the following about the fda's rare pediatric disease voucher program which my creating hope help of reaun 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 to 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 rare disear clinical programs in gene therapy abandoned. 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 those, 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 innovative treatments 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 y everyone. appreciate you. >> the gir 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 ira. we have heard that true rare disease drugs are not at risk, only big lock buster 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 tha■ktas 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 die indications, , and that would trigger price negotiation. >> thank you. ms. davis, first, , i'm very saddened to hear about your husband, passing of your husband, and thank you for being here today. your strong advocacy. certainly we are hearing from

colleagues across, , my colleags across the 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 s of frontiers of research on both oligonucleotides which a class with a small molecules and on nd 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 fullenges 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 co 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 the alzheimer's care act to improve help for people with alzheimer's disease. 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 would this 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,hysical 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 o 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 ba, 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 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. instead, 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 documents 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 ake 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 found there's been a consistent and continuous increase in capital investment. dr. ballreich, this research similar indicate the ilation reduction act has not, in fact, distance invites investment 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

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 alternatives. 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. thentended to get medicare to lead to a fair price

not a rock-bottom price, a fair price on a drug after it'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 existin products for as long as humanly possible and if they try to direct companies to invest in me 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 guthrd referencing your earlier remarks on the constituents you had 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 evii 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 w 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 we give these therapies, 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' 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 review vouc. seems like there was a recent instance of a rare 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 approvedliabil.

there are ambassadors of provide the essential fun and ringing treatment to market.

really depend on that. the priority review voucher, getting to apply for those and secure those, revise and essential funding mechanism to provide additional funding for the smaller 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 sece approval to have to have funding in place to make sure those the, 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 a 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 f thank you chaird 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 isabout. 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 patient experienced 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 also glad 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 is for 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 a all going to support. >> okay, thank you. also want to discuss importance of using the most humane methods possible to test new innovations and therapeutics. this means we must move away from animal tes 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 p animal tests where possible lease new innovations on the table. i was glad to see the fda dernization 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 such must come should be prioritized, end quote. can you say more about how fda can speed effective new alternative methods to be more utilize? >> thank you, 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 substitution. ..

market treatment as soon as possible especially talking about rare diseases. this committee has all any history working in a bipartisan fashion we have accomplished in this year's past. i believe innovation here rare act. medical breakthroughs have bee made treating rare diseases such as cancer and i fear all the progress accomplished might be in jeopardy for those here's we must together.

we think about small molecules, what comes to mind for treating conditions to manufacture?

>> thank you the drug development especially for oncology drugs often majority of es happen after the initial approval including earlier stages of cancer and innovation exploring overtime. the cures act is a good first step, how can we ensure there are incentives for innovation for such things of life saving throughout the lifecycle market? >> eq congressman, is a complex question.■

in some cases the conditions based on pacific notations cancer may not meet the rare definition and it is mainly that definition. >> in a variety of ways we work with the fda in any legislation that enhances the investment environment with these types of treatment is essential right now. >> the most posit impacted but would negatively impact? >> discussing in particular position, there are a subset of treatments at our base

emphasized short stretches such as one of the treatments. >> thank you, mr. trip. >> last summer i was contacted by the medical director for the clinic treating a patient. severe first surgical action. after a month in the intensive care unit. recently he seemed manufactured

company with the troubling history. 1870 of these developed infection. in passing, it's discovered her death brings this material produced by the biologic and the patient to receive material from the contaminated lab and as of today the latest outbreak is linked to the death of two people including chandra. i am grateful the chair and testing for tuberculosis act fundg the product safety act as part of days hearing.

along with my friend and colleague was here joined by chandra's sister who also happens to be a step further. thank you honor you today we have been working closely for the food and drug administration and centers for disease control and prevention to better understand reduction in cells and tissues to be maturely improved patient outcomes not harm them. how does the use of human cells to -based products support your practice procuring patients with rare disease? >> there are a variety of products, some of which are

the treatment for sickle cell disease for cellular lp gene alteration and editing.■! what your experiences show if there are a wide range of this elder some of which have the therapies in some not. the revelatory advances or to find a better test for anticipated consequences such as the one talk about. >> we want to see anyone else die, how can we as the safety . >> nagano the case in detail to address particular one art

regulatory frameworks cannot be status with new potential dangers identified. treatment has to be updated. >> do you think the american public understands the risks associated? do think they get a bone graft i could die from this? >> the purview of the provider as accurately as possible sometimes risks and benefits. i don't have any data on the. >> i suspect they are not told also i want to thank you. people on the committee are tired of hearing about this.

no never gave me the risks and that's why these pieces of legislation for important underinsured patients better understand risks associated and improved materials used and i look forward to working with members to ensure we get the bills right. >> mr. griffith, five minutes. >> i come from a little town in southwest virginia go grew up with the family inflicted with intense disease. another family no way of knowing what was going on. that family has learned they

have huntington's disease. he mentioned targeted therapies. >> are a lot therapies developed targeted therapies in particular are there to address genetic and as you heard more innovation dental treatment dow road. >> one member of the family has told me theybe tested because there's nothing that can be done. that said, do you think what i

believe cooperate with the federal government negotiate the way you want what do you think i will hinder or enhance advancement therapies for people f disease? >> a carveout for drugs indication for rare disease. the challenge is drug manufacturers consider the pipeline. it just incentivizes drug manufacturers from entering for the rare disease based. >> let me see if i can translate into the latest terms. if you manufacture medications

for disorder and some of your team says it might help huntington's afraid to try because it's possibly penalties about medicare, medicaid. >> your subject to potentially price negotiations selects a better to not look. okay. huntington's disease inheriting a disorder, he mentioned associated virus targeting pacific cause of disease without treatment. are you aware of efforts related

to huntington's disease? >> there are, there have been several studies defense. two efforts i am aware of and a series of studies for medical school under the leadership one of the people pushing the envelope board for both dbase therapy and nucleotide based therapies or rna either of which is down regulating that chain.

>> your friend who's afraid to find out whether or he has huntington's,looks promising enr should he go on living his life? always worried about but couldn't handle the stress. >> i've been asked at different times nonspecific to huntington's. i've learned not to make any guarantees but i will say there's reason to still have hope for conditions like this because if we do what we can do, industry does what they can do, the foundations do what they can do, we're going to beat most of these diseases. >> appreciate that. my time is up. i yield back.

>> 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 for patients with rare diseases and prioritized innovation to develop new will provide study therapies combinations innovative treatments to help them achieve.

>> this is one part of any individual for cancer, they are all going to fail. if you look at adult combinatio. 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 auick 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 cpa 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 riso

centives offset the risk and rema 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 whyinnovativp

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 las year because of the loss we are talking about 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 ra a have these should take this. >> can you share your important. there are 30 million fellow observation how it can better americanwho suffer from where leverage and speed up the disease and need our help. development of these drugs? the vast majority don't have any >> for example described is cure or effective treatment and that's one reason why we are cystic fibrosis referred to doing the workf, we're doing in this committee because we want to address that. earlier were approved in the it's important we have

legislation to bring our healthcare system to the 21st healthcare system to the 21s ras get the treatment they need. initially a small range of we introduce the fda modernization 3.0 act. mutation and the gene, expansion that would establish a program the fda to qualify the group has been done with some form of testing methods to reduce or an. the small that has to mutation it builds on fda modernization examples from his patients. act 2.0. as the next step. in that case it did not require we are trying to do is really a new indication going all the way back to preclinical animal make sure pharmaceutical testing. there examples that have come manufacturers can these models about the clinical animal and make sure we are doing testing and some of those cases investigational drugs. animal testing did not predict i know how important it is to make sure improving market and toxicity. it is a complex regulatory. polo a short time after.

>> one thing we are trying to do before not you agree fda author address reforms as it relates to pricing transparency in the price of the drug from the : and non- animal investments office they are able to garner. when it comes to evaluating if you -- can you■5mz describe w safety spoke about in■k your the controls not only destroy testimony? >> i mentioned earlier, it's a innovation but also how will make it harder for patients to access medications in the can complex question because new platform technologies clearly still prefer higher drugs testing because models don't to lower-priced drugs? capitulate every organ of the >> in addition to incentives, body but adduced there are venipuncture introduced by market revenue, the challenge en indications where a small increment on an existing same warmth

limit the patient's ability to or substitution these models access an entrance that comes to th market. >> i think all of you for being that have these should take here, americans suffer from rare >> can you share your observation how it can better disease, i think you and look back. >> the chair recog ranking member. >> focusing on a small subset across the program. make this clear, the law includes ilusion 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 answer

quickly. 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 a 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 fe similarly lays negotiation or certain subset or molecule drugs so how would hese drugs impact the medicare

>> 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 fr 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 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 reasont thank you, i yield b■wack. >> 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 disease. i appreciate 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 the right time and causes great harm.

appropriate 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 ■jjfor treatment are used. these treatments are often dramatically affected.■$ years ago cancer87 patients, a t of cancer patients were labeled first or second line or third line. different order and combinations mit been considered off

label. i can't tell you how often i face but i my practice. congress recognizes by directing pears to look beyond the label and meet the threshold o 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 legislation.

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 literaturj and i humbly ask my colleagues to join this common sense bill to increase access ensure effective treatment of rare disease. i'd like to describe one in my district, tallahassee to receive treatment roughly $175,000 in treatment. why didn't they manufacture less effects of drug go?

it is cost prohibitive. label indications on the drug already hasy 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 barriers as you have 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.r local e surgery. this partnership and the children's hospital thanks to the collaboration she received the care she needed when needed but other children are not unfortunate. a 1-year-old with a rare genital heart disease is 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 waympacted 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 closer to the hospital for many had severe disease and they were left, they were told they could go to the other hospital. ■i

children are undergoing treatment. >> absolutely. we have 50 different medicaid programs so i will takes about 30 days from a pretty g but i

think so too long the other states require background■ checks, security members and they might still say no.■á it's can't wait that long. if you are waiting thing we can do on our center you are having more and more surgeries 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 theirciad out-of-state care provider enrollment for these families, s bill and with that, i yield

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 shoe 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 nothe saying we at

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 these rare diseases we already have newborn screening, heal pricks newborns get in this country. all 55 of these recommendedscreu treat them early, it is a normal life. dietary restrictions and medications and gene therapies detection developmental impacts,

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 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 safelyr. 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 l■s■.ook forwao working with the ranking member on this. to quickly address an issue so many times 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 in, 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 medicines received fda 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. in a similar vein, that narrowly targeted therapies. negotiation timelines in for some good drugsionately impact t diseases with high unmet medical needs. 30 million americans.■o

in 2023 research resulted■ in 71 novel approval including rare and hard to treat conditions. on the survivor of a rare cancer and it still impacts me everyday. from an economic perspective, can you speak to 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 pro 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 a hearing to address rare disease day. because of how he people/w 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 offset risks. >> i want to thank the foundation and stakeholders who have worked to support this 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 patientations. so in terms of the diverse applications one has to think about the disease, who at the facts and have the cell models arect 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 african dissent affected at 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. 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 ce. 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 >> 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