lynthe sne online at c-span.org. >> three years ago democracy faces the greatest thr today unbroken. [cheering]he annual state of the union address.he country. watch live coverage 8:00 p.m. eastern with our previous program followed by president biden date of the union beach. the republican response and your reaction by taking your phonecalls social media comments. wash the state of the union address eastern oile 30 lab or online at c-span.org. she. >> medical professionals and lookt researers ways to support patients with rare diseases from the house subcommittee o under three hours.

she. >> the subcommittee will come to order and dmi recognize self for five minutes of o statements. thank you to our witnesses for sitting here today. the legislation before us will 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 hea there are over 10000s as many as 30 million amens despite 10% of the u.s. population with a rare disease 95% o diseases most patients can evenosed which can be along journey only

discover these options. airbase is challenging especially when it comes to measuring the safety and efficacy of drugs for patient populations he investment and rare diseaseky and unpredictable. better supportpproval. research rare diseases and the which they seek appr ensure medicare and medicaid will cover this.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 ultimatelyeived therapy more quickly aut this for the program and crea granted

and paved the way for groundbreaking therapies. i'd like to thank my bipartisan and ensure access to treatme disease consistency throughout process. access act which will streamline making it easier to receive necessary[ú for provider in another state. get the care for rareeases. harder for patients to access life-saving therapies. in some casesients travel significant dista lengthy stnd families responsible for hotels, food and other important expenses. limited

circumstances making it for patients. bills buildup this work passing and protecting health care for all patients for those with disabilities life-saving healthcare services access for medicaid patients. finally efforts to increase therapies we are considering patients s to affordable treatment to treat rare diseases.

the rare disease. over the last 40 years bse in the development rare disease. more than 5000 the american ug act of 1983. last year more than half of all new drugs approved by the f drug spring hope to millions of americans. there's room for improvement. than 7000 known rare diseases have fda treatment and clinical trials.

among the most aggressive our children. you treatments for rare diseases be used. children are not little adults. c she chapter study. as more to ensure children are not l drugs approved rel since 1999 some oral pediatric data. introduced will close the loophole so we have the clica data safely treat children. the american a pediatrics leukemialdren's hospital association

national organization rare disorders, i think all of those organizations. children als benefit from cancer treatment which is why mccall and i currently the fda cannot trials proving effective in adults rooted legislation 188 bipartisan endorsed by 50 organizations research. every member of the committee is part of the fda joined representative mccall introducing creating hungry authorization act providing pediatric drug priority

114 authorizations endorsed this bill and l time legislation foron in the house pass u. no considering bills to power and beneficiaries money and capped at $35 a month. and 3500 a year. next y's all beneficiaries their cost capped. there also unneeded. medica d negotiations should only focus on the top

selling high cost drugs withoutrt-time position. ne loopholes look forward hearing this and i heal back. everyone. we areay. to promote people with rare diseases and make sure all patients benefit exciting innovation happening. a 12-year-old spinal muscular only 10000 rare diseases 30 fda from

the technologies and many more innovations researched and which to find bridges eat treatment but done. just over 40 years ago only 38 drugs were fda approved. degree alone they were approved by the fda and get intentionally. our job is to be sure it's ready continues i'm concerned we don't continueq1

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>> and again thank you for your presence on the panel today. it is so important when we talk about a team approach to things, we are facing a situation in this country where we are losing physicians at an absolutely unbelievableate. 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 can lead to the shortages of a previous he mentioned. unfortunately we're see that in

the physicians based we don't do something about it. i know this committee has done some work but we've got a lot still to do. chair guthrie i thank you for the time and w beck. >> the chair recognizes mr. cardin us for fiber. >> thank you chair guthrie and ranking member eshoo for holding this 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 n for hope is ultimately what this is all about. that's why i'm thrilled to see bills that i support up for discussion today including the benefit act which would require the fda to consider relevant patient experienced data in their new drug approval process d 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 pediatrications and it's an incredible legacy to lead to ensure kids with cancer and rare diseases have hope to tre conditions, so thank you to our colleague anna eshoo. my firstor dr. bassuk. in your opinion as a pediatrician, what kin 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 which is a brain death exam because we have no good treatments for brain tumor patients. they are clearly cased to do more than we are doing and we need to do everything that is available to us and you defend that gives us that kind of ability is something we're all going 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 testing where and if at all possible. to be clear this transition is not only an amal 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 tohase out harmful animal tests where possible lease new innovations on t table. i was glad to see the fda modernization act 2.0 yet signed into law last congress. i appreciate efforts to try to implement those policies. most importantly that we must hese efforts are safe and effective for the populations they are intended to your testimony that, quote clearly on regulatory expectations for alternative testing methods isoption of such alternative such must come should be prioritize about how fda can speed effective new alternative methods to be more utilize? >> thank you congressman. complex one of course because when certain new modalities basic platforms of

gene and cell therapy to the fore they require some level of animal testing in order to identify previously unanticipated adverse effects. so that is not a 100% substitution. ..

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

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

>> thank you the drug development especially for oncologyority of research and therapies happen after the initial including earlier stages of cancer and innovation exploring overtime. the cures act is a good first step how can we are incentives for innovation for such things of life saving throughout the lifecycle market?

question. in some cases the conditions 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 now. >> the most positively but would negatively >> 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 thetor 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 eive 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 funding the product safety act

along with my friend and colleague was here joined 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 betterunderstand 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 procuring patients with rare disease? >> there are a variety of products some of which are combined with gene therapy with

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 science advances or to find a better test for unanticipated consequences such as the one talk about. >> we want to see anyone else die, how can we as the safety profile. >> nagano the case in detail to address particular one artregulatory 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 boneft could die from this? >> the purview of the provider as accurately a 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 to address genetic and as you heard more innovation dental treatment down the road.member of the family has told me they don't want to be tested because there's nothing that can be done. that said, do you think what i believe unconstitutionalhe federal government negotiate the way you want what do you think i will

hinder or enhance advancement therapies for people facing raresease? >> 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 t 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 penalties

about medicare medicaid. >> your subject to potentially price negotiations selects a better to not okay. huntington's disease inheriting a disorder he mentioned associated virus targeting pacific cause of disease treatment. are you aware of efforts related to huntington's disease? >> there are there have bee several studies defense.su

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 not h huntington's the new treatment looks promising enough to get or should he go on living his life? always worried about but

couldn't handle the asked at different times n to huntington's. i've learned not to make any guarantees but i will say there's reason to still have hope for cons like this because if we do what we can dot 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 n 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 therapies. we have to do combination therapy. cancer cells are working figure out what's going on with the drug and workaround about why you have to gang up on them and use multiple drugs can look

clinical trials and challenges they faced a quick path? >> it needs to be faster than it is no. there are many ways to do that. right now part of the investment because things take money and time to know will safety and efficacy but very often the window they close while this iso we can do approval and entry into the clinic and we are working with the and if it works in a given cell they will

substitute the gene for patients with a different related condition. >> tc companies embracing studies? >> companies do embrace pediatric studies more t used to having been in the field a long time. i will say there's a lot of risk for companies in this incentives offset the r and remain important. >> ensuring effective treatment is vital but also how innovative treatments and early detection of cancer can save lives. not under consideration today i would like to reiterate support or another bipartisan bill. i like to reiterate support for another bipartisan bill and hr

407 soul medicare cancer screening project to increase access to multi- cancer early detection. can you talk about why help reduce the burden of cancer the most vulnerable childrennd how can increase access help reduce disparities in cancer and equity? >> as it would not detect early horses out of the anything with the patient's. expanding access to clinical trials some changes made in the st that had effects so i what

is a small state 3 million people and the past would notlly hug clinical basis for cancer but now in the last 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 important. there are 30 million fellow americans who suffer from whereand need our help. the vast majority don't have any

cure or effective treatment and that's one reason why we are doing the work we're doing in this committee because we want to address that. it's important we have legislation to bring our healthcare system to the 21st century to help rare diseases get the treatment they need. we introduce the fda modernization 3.0 act. that would establish a program the fda to qualify the group testing methods to reduce or place animal models. it builds on fda modernization act 2.0. as the next step. we are trying to do is really make sure can these models and make sure we are doing

investigational drugs. i know how important it is to make sure improving market and polo a short time after. before not you agree fda author : and non- animal investments when it comes to evaluating safety spoke about in your testimony? >> i mentioned earlier it's a complex question because new platform technologies clearly testing because models don't capitulate every organ of the bodyut adduced there are

indications where a small increment on an existing treatment using the same warmth or substitution these models that have these should take this this. >> can you share your observation how it can better peed up the development of these drugs? >> for example described is cystic fibrosis referred to earlier were approved in the earlier predecessors. initially a small range of mutation and the gene, expansion has been done with some form of

the small that has to mutation examples from his patients. in that case it did not require a newic going all the way back to preclinical animal testing. examples that have come about the clinical animal testing and some of those cases animal testing did not predict toxicity. it is a complex regulatory. >> one thing we are trying to do address reforms as it relates transparency in the price of the drug from the

office they are able to garner. if you -- can you describe how the only destroy innovation but also how will make it harder for patients to access medications in the can still prefer higher price drugs to low drugs? >> in addition to space introduced by market revenue the challenge here effects coverage that can limit the patient's ability ton entrance that comes to the market. >> i think all of you for being here americans suffer from rare disease, i think you and look back. >> the chair recognizes the ranking member.

>> focusing on a small subset across the program. includes inclusion for orphan drugs that treat rare disease. 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 dias less auctioning incentives for innovation so it the drug exclusion was expanded to include drugs with rare disease indication and hr 5539, what effect will this have on the negotiation program?

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 d 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

indication would rely on the fair prices out of the negotiations to help improve access to the drugs they need. >> hr five system negotiation or certain subset or molecule drugs so how would delaying negotiations but these drugs impact the medicare program? >> the doesn't seem to be any reason to exclude the drugs from because the kinds of drugs described if t clarity they are extremely profitable drugs and has incentives. >> thank you there's no reason we should further limit drugs medicare to negotiate their prices only undermine the ability to deliver safety for patients particularly those of their condition and continue

reauthorization with a rare disease and other pieces of legislation that will require the emblem mentation. i leave it's important to hear from fda on the other policies under consideration prior to any mmittee action. significant agency resources and important understand some of these policies before moving forward. i want to go back to what i said earlier i think some of you know the drug pricing reform provision including reduction act the intent of drug price negotiation : advances but to reduce dg cost 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.alse premise and panel the reason you can't use it. lack of access -- if you don't have access, what is the. >> is on a broken record thank you i yield back. >> gentlemen yields back. >> thank you very much for holding this hearing to discuss a wide array to improve the lives of not just those with rare disease. i appreciate my colleagues eliminating failed first authorizations for doctors and staff. most important national security preserving the doctor-patientip 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 rat great harm. care and it might as well be called bear first. it shouldn't be acceptable to you. rare diseaseients could face challenges or appropriate treatments and probably most cases medication most effective for treatment are used. these treatments are often dramatically affected. years ago cancer patients a lot

of cancer were labeled first or second line or third line. different order and combinations might have been considered off label. i can'tow often i face but in my practice.congress recognizes by directing pears to look beyond the label and meet the threshold questions. building off those efforts to allow for similar flexibility in the rare disease hr 6094 protect their act would amend this for rare disease medical torture and guidelines in add the fda safety approvals which satisfied medical necessity requirements. i appreciate my colleague for

working with me o important legislation.y should the treatment be used outside what it was originally approved for? small trials and small numbers ofatients near labels. manufacturers could not afford to test all the diseases across these patients in various related diseases. this may lead to medical recommendations and peer-reviewed literature suggests the treatment or

management for rare diseases. the associated costs and therefore improvement. in short access to these treatments supported by peer-reviewed medical literature and i humbly ask my colleagues to join this common sense bill to increase access for lifesaving cures 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 has!0 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 seeing their doctor often requires going to a different state and has a bear.lm fro hampshire

a few weeks old he did surgery to correct an obstruction. her local one could not have the surgery. this partnership and the il 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 a racing against the clock to fin 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 hoping paperwork go through transferred to boston

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

children are undergoing treatment. >> absolutely. different medicaid programs so i will takes about 30 from a pretty good but i think so too long the other states require background checks

checks security members and they might still say no. it's can't wait that long. if you are waiting thing we can do on our center you surgeries seizures seizures you will likelyre and more going forward so it comes up unfortunately all too frequently. >> impact to support the exhilarating kids act legislation that would children and their families specialized out-of-state care provider enrollment for these families, i urge my alex to support this bill and with that, i yieldck. >> 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 dis while they help safe and effective treatment options for a few hundred. i'm proud pharmaceutical invaders in my state you like willie on meeting george to develop groundbreaking medicine and it is equally challenging. my good friend doctor joyce. zach would make it critical for the inflation reduction act setting provisions for rare disease treatment for additional obligation good multiple rare

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

negotiations with multiplications whether primarily orphan drugs are not raucous ministers incentive for manufacturers and exploring alternative use for existing drug 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 is not the argument we are saying we are working out transparency and affordability. >> i yield back. >> your recognize for pregnant.

>> thank you for the witnesses being here today it's been an interesting discussion. the pediatric focus today and we have heard this impact children th 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 newborns get in this country. all 55 of these recommended screenings are rare but if you

treat them early it is a normal dietary restrictions and medications and gene therapies we heard about without early detection developmental impacts disability and not like today's discussion. travel costs state lines in regularly for diagnosis and care for rare disease not available in their own state. if the provider is already

enrolled in their own home state. care and treatment and that's support the care act. newborn screening early diagnosis is critical. he mentioned the testimony i hope and to hope, patient fortunate to live within a half hour. one or two patients in a course and the delay specialist. >> we have a case we have a

comprehensive epilepsy study so we have an example is a powerful magnet. fda approval from clinical research including the length of the grain brain you can safely remove our surgeons know how to do this surgery and they sit home and continue to have seizures.

>> the patient's for medical nutrition a medical decision equity act and i look forward to working with the ranking member on this. to quickly address an issue so many times about medicare drug negotiation impacting rar 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 r base. to ensure research and treatments that american patientstc 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 of therapy and diseases. patients and their families help. the orphan drug act signed into law the same year i graduated

from mnessed firsthand innovations and cures the diagnosis of melanoma for the referral into hospice care in short order to ensure patients going to live. cellular therapy was an honest assessment. since the package almost 1200 new medicines approval. i hope everyone regardless of party affiliation want a level of drug innovation to continue to treat diseases and passage of the ira infection 1192 is the acknowledgment that orphan disease treatment are meant to be shielded from the protocol

and it's merely an effort to improve that corporations who ultimately will receive patients patients. in a similar vein, that narrowly targeted therapies. negotiation timelines in for some good drugs to disproportionately impact treat diseases with high unmet medical needs.gj americans.

in 2023 research resulted in 71 novel approval including rare and hard to treat conditions. on the survivor of a rare everyday everyday. from an economic perspective can you speak to the value of research into additional indications and what it means for patients specifically in a rare disease kinetic? >> the postapproval is a more constructive way and the ideal ability to test the drug and other rare disease indications is valuable because it might not

have been there if you had to develop it from the start. >> thank you for sharing the loss of your importance of where conditions for your family and rare disease community? >> we must continue to drive the foundation and access. >> no the patient can only access medications if they first exist. thank you mr. chairman and i yield back. thank you mr. chairman.

>> , healthcare providers assistance to diagnosing a rare disease in five years and it's even longer for patients of color. the majority of rare disease does not have fda approved treatment. i'm glad to see a hearing to address rare disease day. because of how he people are. several of my colleagues in nostalgia program for developing treatments is used to accelerat and otherwise what not been the method.

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

relevant models has to be more productive. based on human cell culture are that they recapitulate the disease in terms of individual patient mutations. so in terms of the diverse applications one has to think about the disease whohe cell models are alternative models reflect the population that the drug is intended for. >> great thank you. dr. chen today marks the end of blackish month will follow the tribes and struggles of black and african americans. sickle cell disease is a genetic blood disorder that affects approximately 100,000 americans.

people 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 whathelp homes are and how they can help address gaps in the care currently received by these patients? >> so medicare excuse me, medicaid health homoss 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 thatare

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 m able to get specialized care they may not that may not be available in their home state. as a pediatric neurologist can you share why it is important to sure children from low-income communities are able to access the services they need? >> yeah. we need to get kids best care available for them where they are. if it's not available where there are we have to bring them to where the care is. that's actually why we and many of the societies would belong to a making two of them, the children's hospital association are absolutely behind this bill. >> thank you. every kid regardless of zip code regardless of income should have the same access to equal care. with that i yield back.

>> 100% agree. that chair now gentlelady yields back to the chair recognizesr. harshbarger farmers. >> thank you, mr. chairman. thank you to the witnesses for day for being here. i'll start with you dr. chen. today under the pediatric research equity act or pria, sort and orphan drugs are exempt from requirements to conduct pediatric studies. what characteristics do orphan drugs have such they might be exempted from these requirements? with all of the details of the requirements for orphan drugs for pediatrics, but it is important to ensure we have incentives tou tell me, tell me or expand our potential unintended consequences of requiring such studies for orphan drugs on future development decisions? in your testimony you were talking about drug developments. on average 90% of these drugs fail. then you've got ten to 15 years

required to progress them initial discovery to market approval and when you're in that space and i been a pharmacist at 37 years and you know what it takes to get them from clinical trials and up. there's a lot of chance they willhat's absolutely right. we know that without incentives like the orphan drug act 90% of the drugs that we see that it come out of that space to treat specific rare diseases would not have occurred. >> you spoke about the benefits of exploring potential follow-on uses existing orphan drugs to treat other rare diseases. you said follow-on indications represent a critical pathway for faster or cost effective innovation especially in the realm of rare disease treatment. overly narrow constructs of the and -- exemption could significantly discourage future research programs in rare disease and the

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

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

i've only got like 52 seconds left. dr. flotte you talk a little bit about manufacturing technologies decentralized trials in real life stories. tell me how that works to me makes a lot of sense that you would include that. tell me about it bit about that. >> actually one of the real obstacles we have to overcome in clinical trials and gene and cell therapy is having outcomes that reflect in a quantifiable way what were actually seeing in the patient's. standard measures may or may not reflect the benefits that the patient's and families are examples of this. one that happened at the very beginning of a gene therapy a gene therapy was approved by the fda based on the ability to navigate a room in low lights, and this had has never been used as an outcome before but it was

generated from the fact that patients were reporting. and it was what they said was of value to them. of those kinds of outcomes, we need to be flexible about rigid about what will define benefit in each case. >> i totally agree. i know i'm over time so with that i will yield back. >> the chair recognizes ms. trahan for five minutes. >> thank you to the chair and ranking member for holding this important hearing. so grateful for the panel of experts here today and i give a special shout out to dr. flotte from u.s. chan medical school. as a mother of two young daughters it's great to see many of the bills we are focusing on will have a profound impact on the health of our kids. i'm particularly grateful that the accelerating kids act to care act which interviews with congresswoman mary mariannette miller-meeks is included in today's hearing. mr. chairman, i like to request unanimous consent to enter into the record a statement from the children's hospital association and support of this bipartisan legislation. >> we will add that to the

documents list and take action at the end of the meeting. >> thank you, mr. chair. when the necessary clinical care is determined for a child by medical professionals there should be no reason that administrative burdens get in the way. this accelerating kids access to care act is designed to cut through the red tape that kids, and kids get the appropriate care that they urgently need, to express the importance of getting this legislation over the l highlight a story from a patient who was negatively impacted by the burdensome and time-consuming medicaid provider screening and enrollment process. almost three years ago a baby, i will call her lilly was born in a rural state with her esophagus into make separate segments and connected abnormally to her windpipe. because this condition requires particularly conficker procedure to correct it, doctors explained places in the country where it could be done. one of them was boston children's hospital in my home state of massach and repairs are ready to travel to boston where the surgeons were ready to

perform life-saving operation. but instead lily who is a medicaid recipient was waiting for her home state to enroll the eight providers at children's menu to perform this complex surgery. her state confused about the rules concerning out-of-state provider involvement wouldn't authorize the services provided by boston lilies surge was rescheduled more than three times and a laden seven months as a lily waited she lived with a floppy and where they could have collapsed at any moment, a weakened immune system, and routine reading difficulties. so dr. differs from state to state can you please walk us through what it generally takes for provider to enroll in another state medicaid program? >> yet. so as i mentioned previously, it really does differ from state to state, and to the point that it feels like there are 50 do this. i am what i think is sort of on the good side of things here.

we are pretty fast. if we can get someone enroll in 30 days or less, i don't think it's fast enough for kids like lily that you mentioned but there are states because of fingerprinting background, criminal background checks, having to send multiple forms of documentation where we had four four to six months weight. that's more than one case. that's not just one outlier. unfortunately the story that you can do something that we deal with all the time. >> when you see a child in practice or took a long time to get to the right level of expertise to treat the condition, what does that mean that only for their health but for the healthcare costs associated with tha >> yeah, there's no doubt that the healthcare costs are not just u the child and the family but for the whole society. i'll give you i have like us to before i pages of these examples but we had a baby in her neonatal intensive care unit was her for a long time.-of-state medicaid. that original state was approved but we figured out that he had

hearing problem and he needed to get a hearing he's hearing test done and hearing aids put in. the out-of-state medicaid so said he couldn't do it. so that baby actually missed development milestones that he may never make up. that baby may have learning problems the baby n ahat baby could have done. because of this will absolutely unnecessary delays. and that is a huge financial that some expertise but i think that we can probably agree that that's going to have big financial impact. >> and you spoke about this in your testimony beyond the child how does this delay in care impact the well-being of the child's family? >> so actually, and davis mentioned this as well. i mean, there's of course the sufferin getting care and the real financial costs. if you have to move, if you could have gone some really close outside your state which had to go for hours away of your

state and stay at hotel a network. it's financially and emotionally damaging. >> and with that i urge my colleagues to support this bill and i yield back the balance of my time. >> gentlelady's time spac at the chairman recognizes dr. miller-meeks. >> thank you chair guthrie and thank the witnesses for testifying before the subcommittee. i am actually very grateful that we discussing these bills and a please visit the committee considering as you just heard from ms. trahan the aess to cares act which would streamline administrative processes for providers who enroll in another staack of services provided in the patient's home state. i'm also proud to have a fellow iowan and constituent dr. bassuk i too was on the faculty of university of iowa, go hawks here before the committee today. there's currently no standardized federal pathway for this process means providers must enroll any new state medicaid program whenever they see a new patient from that

state and must maintain regular paperwork to remain enrolled in each state medicaid program. and especially in iowa with our four boards with many people from different states who seek care in our home state. when a child from a new state is visiting a doctor as may be the case if a child from another state travels to iowa for care, which is not unusual at all there are often delays in care as a providers sift through paperwork risking the health of children. mr. chairman, ask unanimous consent to insert letters of support from the leukemia and lymphoma society which is undersigned by 215 215 organizations, and from patients rising for so many kids from patients senate in support of speed is if you submit those will add those to the document list and take action at the end of the expert one case that speaks to the need of the filth of also leverage or from another midwest eight who was a rare and safe condition known syndrome in which the aorta narrows leading to multiple series collocation secretary shulkin death if untreated. in this case a referral was made

from another children's hospital to boston children's hospital which operates a leading program to address this condition and a comprehensive care plan was assembled in april of 2022. in the case of the child's home state, all providers at boston children need to be enrolled home state medicaid program before the state would authorize the character by july 2022 the enrollments were still pending three months later which led to a cancellation of care that have been scheduled for the following month august 2022. the home state did not complete its involvement of the boston children's providers until november 2022. in early 2023 boston children's access the child and the coverage of care plan that included additional providers given the child's specific condition was assembled. a authorization was submitted in may 2023 and the care may 2023 and the care was provided in september 2023 nearly a year and half after the initial contact had occurred. and the child could easily have deceased before that time. dr. bassuk, i do know you've answered some of this. in ys a physician and chief at the university of

iowa's stead children somehow some houseplants and administeredburden and delays with providers enrolled in other states medicaid programs that impact pediatric care i can highlight barriers doctor scully face in this process? i think i saw skip that and said does this legislation allow or require other state medicaid ograms t cover services not covered by the patient's home state? >> all right. so i'll answer that second question. no it absolutely doesn't require the home state to cover services that they don't cover. het all. it's about making access easier for kids with medically complex and rare diseases. >> very importantly similarly with this legislation lead to an increase in services provided, or rather the same services ided but in a timely a manner? >> absolutely. it's about getting the right service in in a timely manner, not different services. >> lastly, very important as we looking at cbo scores, do care delays

increase costs to the health care system? >> you know, in pediatrics we say pay me now or pay me more later. so yes. >> thank you. pay me now or pay me more later. dr. chen, the orphan cares act which is again t same moniker i'm hanging out emulator, which exemption for medicare drug after drug price negotiation to include treatments effective against more than one rare disease. in contrast to my colleagues, whatever research and innovation goes on regards of age group can be transferred to another age group. all of that foundation of knowledge and and innovation is transferable which also means research is done to develop one drug to treat one disease may in fact, find out it treats another disease or a side effect that we didn't even realize. why is this legislation important for ensuring we continue to make progress in developing and cures for more than 90% of rare disease patients that do not have fda approved therapies?

>> so you want to take into account all of the potential drug has a viability for. some of those diseases some of those might not be in the bottom line is the entire pipeline matters. >> ms. davis i had a question for you but my time has run out. it's on small molecule medicines, thank you so much for beingn research and development. i would like to is that the collation to be answered -- >> absolut thank you. >> i yield back. >> a gentlelady yields back and the chair recognizes you for five minutes. >> thank you very much to the chair and two are witnesses. i think my microphone is malfunctioning here. all right. technical difficulties resolved. i would like to thank the chair and the committee for including my bill h.r. 6020, the honoring our living donors act in this

hearing. i . i introduced this along with my cosponsor congresswoman delbene in to solve a really important problem with the living donor reimbursement program. i think the people in israel are probably not surprised to hear that we have currently over 92,000 americans waiting for a kidney transplant, and yet the number of living donors was only about 6000 last year, and it is declining rather than increasing. is a really serious problem. so anything that we can do to incentivize and increase a number of living donors i think is going to be something that not only saves lives but is of service to a lot of the people that are waiting on that list. it's an incredible sacrifice to be an organ donor and the impediments to being one are severe. that's why congress of years ago

authorized this living owner of doery modest way reimburses costs like travel expenses dependent care, things of that nature for living donors. and by modest i mean the program out at $6000 per donor. it is modest. it's also means a donor or the recipient of the oregon cannot have income over 350% of the poverty line. this is a probably guess it makes no sense to include the income of the recipient in this calculation. that is nothing to do with whether or not the org is going to be able to make ends meet in donating the oregon. that's what this bill does. it makes that 350% means test apply only to the income of the donor, not the income of the recipient. another thing the bill does is commission a a study on the program to seek ways that we can be more effective in both getting these donations funded

and into communicating existence of the program to the recipients. want to think very much, mr. chairman for including that bill industry. i certainly hopean get speedy passage for that to fix this problem. dr. bassuk, your testimony really interested me and and i wanted to talk about it, with respect to organ donations. recent studies have indicated that in addition to frequently needing organ donations rare disease patients can't themselves be living donors. it's a misperception to think they can't be. they can be, and they have been,alk about the importance of breaking down the barriers for rare disease patients to get care, including organ donation? >> yeah. i think i mean, i'll just reiterate what you said. patients with a rare disease kids with rare disease need all the treatment anybody else gets, and the special treatments that they need as well, and they

deathly should not be excluded from standard therapy. >> right. thank you. ms. davis,hi i tast question are here it's a think it's appropriate to give you the last word. after considering everything that's in said in the searing if you could tell people wanting to take away from hearing today what would it be? >> keep in mind that it take thousands of years at the current pace to reach treatments for all patients with rare conditions. th unacceptable, unacceptably and hope that that is unacceptable to all of you as well. we know that the orphan drug act is working. we seem the great insinuations that is provided in bringing novel therapy to patients in need. i urge all to turn to those incentives extend those and protect our orphan drugs from restrict innovation and access

to the treatments that can help our patients, help our patients live better lives help our patients live longer lives and help us have more days to spend and love our loved ones with rare conditions. thank you. >> thank you for your testimony of thank you to all other all of our witnesses for your valuable testimony today. i yield back. >> that concludes the members of the committee testimony. we do have 1% that is asked to wait on from the full committee. i will now recognize ms. matsui from california for five minutes and that will be the final witness. >> thank you very much, mr. chairman. i want to thank you and the ranking member for holding this very important hearing. rare disease day is the time to race the winds come celebrate our advances and commit to building our progress. ms. davis, i've been moved by your testimony. as cochair of the congressional rare disease caucus, this is especially iortant to me. i'm grateful to my cochair for

all of our bipartisan work on rare disease issues, and i'm thrilled to see so many bipartisan bile on today's agenda, including three bills i introduced, the benefit act, the rare act and protect rare act. however, disappointed this hearing has been hijacked by bills to dismantle the ira. the ira was a major step forward in assuring aeases don't have to choose between paying for their prescriptions and basic necessities. this is especially frustrating when you are so many commonsense proposals to ensure rare disease patients have access to safe, effective and affordable therapies. i hope this committee can work together on these solutions rather than undermining the ccessful policies in the ira. i would like to turn to one such solution a benefit act. in recent years congresses work with the fda and patients has spurred the development of meaningful patient experience of data being submitted to the fda including as part of new drugay of

continuing this momentum is to assure there is clarity around whether and how the fda uses the patient experience data. to address this a benefit act to require fda to describe how patient experience data was incorporated in the review process. dr. flotte can you comment on the importance of incorporating patient experience of david into drug reviews? >> thank you congressman. it turns out an obstacle that we're facing right now in the development of several gene therapies for rare diseases. we can compare patients who have disease with those who do not but that doesn't always give us a clear-cut in point when racing benefits from a. and what could be more important than how to benefit his experience by the patient and their families, particularlybility

to get through to get more drugs through the pipeline is what families want.hese treatments are actually producing benefits that are affecting patient experience, there has to be real validity to that. >> right thank you. next i like to turn to the rare act. i introduced the rare act with -- to codify long-standing fda policy on orphan exclusivity and ensure companies are not gaming the system to get new innovative therapies off the market. dr. kesselheim how would the rare act ensure continued development of additional therapies for rare diseases. >> thank you. i think the rare act as an important bill because under the 11th circuit decision that created this loophole of the orphan drug act a manufacturer now can get a rare disease designation for a particular disease and as that time is

ending get into designation for a slightly different version or slightly expanded population, and to that additional seven years prevent other competitors from coming on the market for the initial indication. and in doing so create almost perpetual state of market exclusivity that would prevent the competition that would both lower prices for patients and help incentivize newiscoveries of the future. so i think a rare act is incredibly important to a close that loophole and come back to the fda's long-standing original interpretation of the orphan drug act. >> thank you. and now want to turn to another issue for rare disease patients, affording the necessary medications. over 90% of patients lacked an fda approved treatment for the rare disease. as i introduced introduced to protect rare act with represent done to ensure patients have access medically necessary off-label therapies. to show a critical this can be i would like to share a story from what my constituents.

janet was in her 30s raising a six year old daughter when she was diagnosed with a rare and fatal disease called -- despite her doctors prescribe medications at the standard of care for insurance would not cover the stream is because they were off-label. rather than focusing on healing and spending time with her family janet was forced to fight for care while her symptoms and pain worsened and her finances suffered. i know i don't have much time here.i want to ask the question. dr. kesselheim you know from your focus groups with prejudice patients that they are struggling to afford necessary care is a common thing. how with the policies on ira ease the financial burdens of rare disease patients and families? as to realize i've got over so i've would speak to give a brief answer. we'll give would give you a few seconds. i think the ira is actually important to rare disease patient and it comes because it helps promote fair prices for these products after the bodies have been on the market, and fair prices help translate to better access to patients, to

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

promptly. members should submit their questions at the close of business on march 14. without objection -- did you -- >> mr. chairman, i'd like to respond to what you said earlier about cbo and cheers if i might. there is a december 2023 letter cbo to ms. errington and dr. burgess from our committee mr. harrington, the chairman of the house budget committee. the letter was from cbo and it said that, quote the venture capital reaching pharmaceutical companies has been trending upward, end of quote, since the drug negotiation program was enacted. so i think that that's important important, when i'm presenting i

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

[inaudible conversations] [iud conversations] >> the u.s. supreme court ruled in favor of donald trump in the case challenging his eligibility to be on the ballot in 2024. the court overturned the colorado court ruling the said former p eligible to run for office again because of his actions leading up to the generous six capitol. in the unanimous decision the court said the colorado supreme court had wrongly assumed state

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