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. Id 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[u for provider in another state. Get the care for rareeases. Harder for patients to access lifesaving 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 lifesaving 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. Theres 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 leukemialdrens Hospital AssociationNational 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 ys all beneficiaries their cost capped. There also unneeded. Medica d negotiations should only focus on the top selling high cost drugs withoutrttime 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 12yearold 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 its ready continues im concerned we dont continueq1 t p u i l gr]s,ri alf neai0f mn dih h b pas trroxo community. And again thank you for your presence on the panel today. It is so important when we talk about a Team Approach to things, we are facing a situation in this country where we are losing physicians at an absolutely unbelievableate. We had a hearing last week for the week before on physician burnout. Its 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 were see that in the physicians based we dont do something about it. I know this committee has done some work but weve 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 lifesaving help to people who have had n for hope is ultimately what this is all about. Thats why im 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 its 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 dont 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 thats 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 were 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 years 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 its 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 chandras 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 dont 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 thats 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 huntingtons 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 dont want to be tested because theres 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 huntingtons afraid to try because its penalties about medicare medicaid. Your subject to potentially price negotiations selects a better to not okay. Huntingtons disease inheriting a disorder he mentioned associated virus targeting pacific cause of Disease Treatment. Are you aware of efforts related to huntingtons 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 whos afraid to find out whether or not h huntingtons the new treatment looks promising enough to get or should he go on living his life . Always worried about but couldnt handle the asked at different times n to huntingtons. Ive learned not to make any guarantees but i will say theres 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 were 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. Ive 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 whats 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 theres 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 patients. 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 dont 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. Im 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 dont have any cure or effective treatment and thats one reason why we are doing the work were doing in this committee because we want to address that. Its 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 its a complex question because new platform tec