RARE Act

Mr. Speaker, I yield myself such time as I may consume.

Mr. Speaker, I rise today in support of H.R. 3433, the Give Kids a Chance Act, sponsored by Representatives McCaul and Eshoo.

This bill includes several strong bipartisan pieces of legislation, and I am pleased that our Republican colleagues agreed to work with us on advancing important policies.

This legislation will help expand access to care for patients with rare diseases and complex medical needs, increase research and development into rare pediatric diseases, and improve regulatory certainty.

This bill will help in our efforts to bring new treatments to pediatric patients with more advanced forms of cancer. It also includes important sections of H.R. 6664, Representative Eshoo's bill, the Innovation in Pediatric Drugs Act. Specifically, it gives the Food and Drug Administration new enforcement authority to take action against companies that do not conduct required studies in pediatric patients.

The bill also includes critical language introduced by Representative Matsui as part of H.R. 7383, the Retaining Access and Restoring Exclusivity Act, or RARE Act, and this will clarify FDA's long-held treatment of orphan drug exclusivity and bring new treatments to more patients suffering from rare diseases. These provisions all advance our goals of accelerating the development of novel, safe, and effective treatments and improving outcomes for patients.

These bills are going to make a difference, but I remain disappointed that my Republican colleagues have been unwilling to consider necessary changes to require drug manufacturers to study their approved rare disease drugs in pediatric populations.

It is disappointing that my Republican colleagues refuse to hold drug manufacturers' feet to the fire and have decided to move forward with a long-term reauthorization of the priority review voucher program, or PRV, without requiring manufacturers to study rare disease drugs in pediatric patients.

I have been consistent in my belief that we need reforms to the PRV program. Unfortunately, we still have not seen the compelling evidence that this program is functioning how Congress intended it to. That is why I am pleased that the bill includes a requirement for the Government Accountability Office to conduct a new study and report on the effectiveness of the program in attaining our original intent. Previous reports from GAO on the program, and continued research from academics, show that there is little evidence that it incentivized the products Congress intended to help come to market.

I continue to believe that additional guardrails are needed to better target the intent of the program, but despite these concerns, I am pleased that this bill includes important provisions to ensure that our medical product supply chain can be strengthened by working with international partners that believe in both tolerance and respect for every person as well as advancement of science and medicine. I look forward to working with our Republican colleagues to ensure FDA receives the funding necessary to implement this policy and establish an FDA office in Israel.

I am also pleased that this legislation provides the Health Resources and Services Administration the authority to modernize the organ transplant system. This system is lifesaving for many Americans, and I am glad that we can continue to support these much-needed reform efforts.

I believe that we could have achieved more for patients in this legislation. However, since there are strong bipartisan policies included here, I encourage all of my colleagues to vote ``yes'' on this important bill, and I reserve the balance of my time.

Mr. Speaker, I have no additional speakers, and I reserve the balance of my time.

Mr. Speaker, I reserve the balance of my time.

Mr. Speaker, obviously, this bill does a lot to help childhood disorders. It is a very important bill, and I urge that it be passed on a bipartisan basis.

Mr. Speaker, I yield back the balance of my time.

Mr. Speaker, I thank the gentleman from Indiana for yielding.

Mr. Speaker, as a founder of the Childhood Cancer Caucus 15 years ago, I have met countless cancer patients and advocates who have asked me for one thing: to give the kids a chance. They want a chance to receive better and safer treatments that preserve their quality of life, a chance to beat childhood cancer, and a chance to eradicate this disease once and for all.

The Give Kids a Chance Act does just that.

The bill is simple. It directs drug companies researching combination drug therapies for adults to also research them for children, where appropriate. This will allow us to treat children the same as we treat adults.

With 235 cosponsors, this legislation is one of the most bipartisan bills in Congress because there is nothing partisan or political about a child with cancer.

Mr. Speaker, I thank Mr. Bilirakis from Florida and Ms. Anna Eshoo from California, my partner in this. I also

thank Chair Cathy McMorris Rodgers and Ranking Member Frank Pallone for all of their help in making this come together.

Included in this legislation is another one of my bills, the Creating Hope Reauthorization Act. We first passed this legislation in 2012 to create the pediatric priority review voucher program within the FDA because we found that there was no market incentive for pharmaceutical companies to develop cancer treatments for children. This program provides the incentives.

Since its inception in 2012, 53 vouchers have been awarded for treatments of 39 different rare pediatric diseases, 36 of which previously had no FDA-approved treatments. These diseases typically led to death before a child can reach adulthood, but not anymore.

A dear friend of mine, Dr. Allison from MD Anderson in my home State of Texas, was able to use the voucher program to obtain FDA approval for a treatment called CAR-T immunotherapy. Immunotherapy is a breakthrough cancer treatment not just for children but for all adults. He received the Nobel Prize in medicine for it.

This bill would reauthorize that successful program for another 5 years.

We just had our 15-year summit, and these children, I have to say to the ranking member and to the chair, who I hope is listening, how much joy this gave to their lives. These children who have either survived cancer or are going through cancer, some can make it, some may not, but this gives them hope.

I founded the caucus to give them a voice in the Congress because they didn't have one. They didn't have high-powered lobbyists, but they have us. This is the result of their voices.

We wouldn't be standing here today considering this legislation without the help of patient advocates, including my dear childhood cancer friends, Sadie Keller, Sophie Ryan, Brianna Commerford, and Ailani Myers, and so many other brave children who have joined me in advocating tirelessly on this important issue.

By the way, Mr. Speaker, these kids are tougher than any adults I know, and they are my biggest inspiration in life.

Mr. Speaker, I am proud that my colleagues have come together in a bipartisan spirit to do what is right for our children, which will influence them for decades to come.

Mr. Speaker, I move to suspend the rules and pass the bill (H.R. 3433) to amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes, as amended.

Mr. Speaker, I ask unanimous consent that all Members may have 5 legislative days in which to revise and extend their remarks and include extraneous material in the Record on the bill.

Mr. Speaker, I yield myself such time as I may consume.

Mr. Speaker, I rise in support of H.R. 3433, the Give Kids a Chance Act of 2024, and thank Representatives McCaul, Eshoo, and Bilirakis for their hard work on this important piece of legislation.

Mr. Speaker, I am excited that we have reached broad bipartisan agreement on this comprehensive bill, which will provide hope to approximately 30 million Americans affected by rare diseases.

Currently, only about 500 of these diseases have FDA-approved treatments. This legislation will foster the development of more pediatric rare disease treatments by strengthening the orphan drug pathway, ensuring drugs are studied for children battling cancer, and reauthorizing the rare pediatric disease priority review voucher program, which is set to expire on September 30.

The PRV program has been a lifeline for patients who require specialized treatments that would not be developed without the incentive this program provides.

I am also supportive of the other provisions in this bill, which would increase organ transplant rates by strengthening OPTN operations and require the FDA to establish an office in a country that has signed the Abraham Accords.

I am grateful to Representatives Harshbarger and Vargas for their leadership on this effort to facilitate operations between the FDA and regulatory authorities and innovators in Abraham Accords countries.

There has been a lot of discussion about moving supply chains for medical products back to the U.S. and our allies, and this office is a good step to help do just that.

Mr. Speaker, I encourage my colleagues to support this bill, and I reserve the balance of my time.

Mr. Speaker, I yield 5 minutes to the gentleman from Texas (Mr. McCaul).

Mr. Speaker, I yield 3 minutes to the gentleman from Florida (Mr. Bilirakis).

Mr. Speaker, I yield 5 minutes to the gentlewoman from Tennessee (Mrs. Harshbarger).

Mr. Speaker, in closing, I encourage a ``yes'' vote on this bill, and I yield back the balance of my time.

Mr. Speaker, I thank Dr. Bucshon, a great friend of mine who does an outstanding job on our committee, for yielding.

Mr. Speaker, I rise in strong support of H.R. 3433, the Give Kids a Chance Act, by Representative McCaul. His legislation strengthens pathways for treatments and cures for children across the Nation.

The Give Kids a Chance Act would require that new studies of pediatric cancer trials focus on combination therapies rather than solely single-use drugs. This bill requires these pathways for children with cancer in cases where both the drugs are FDA approved and owned by the same company.

These combination products may help unlock new treatment options for pediatric cancers and help extend the lives of children battling a rare cancer.

I am proud to cosponsor this legislation. Furthermore, I am particularly glad to see that this comprehensive package contains provisions of two bills I co-lead, the Creating Hope Reauthorization Act and the RARE Act. These bills will support innovation for pediatric rare disease treatments and cures.

The Creating Hope Reauthorization Act will extend the FDA's rare pediatric priority review voucher program for another 5 years, through fiscal year 2029. This voucher serves as a critical and necessary incentive for innovators to invest in research and development of pediatric rare diseases.

Due to their extremely small patient populations, these diseases too often go unnoticed, unfortunately. However, the voucher program allows innovative rare disease companies to receive FDA priority review if they get an approval for a pediatric rare disease. These vouchers can then be used to expedite consideration of new treatments in the pipeline, or transferred or sold to another company to recoup investment losses.

This successful FDA incentive does not cost any taxpayer dollars and must be extended to continue the pipeline of treatments for children with rare diseases.

I was proud to lead the effort in the Energy and Commerce Committee with my good friend and colleague, Anna Eshoo. We are going to miss her. She is retiring, but hopefully, she will be around to give us some advice in the future.

Again, we have bipartisan agreement on this. It is so very important for our children.

Mr. Speaker, I ask my colleagues to join me in supporting H.R. 3433 off the floor today.

Mr. Speaker, today, the House will vote on my legislation, H.R. 3433, the Give Kids a Chance Act.

My thanks to Reps. Michael McCaul (R-TX) and Gus Bilirakis (R-FL), the chairwoman of the Energy and Commerce Committee Cathy McMorris Rodgers (R-WA) and Ranking Member Frank Pallone (D-NJ) for their very hard work on this important effort.

H.R. 3433 is the most comprehensive legislation to improve and address childhood cancer and rare disease in over a decade.

As medicines become more sophisticated and we improve our understanding of the

deadliest diseases facing children in our country, our laws must keep up.

This legislation will ensure children have access to the most innovative treatments and therapies for cancer and rare disease, and it does that by doing the following.

It reauthorizes the FDA's pediatric priority voucher program for five years. The program expires September 23, 2024 and we cannot let critical tools to encourage the development of drugs for children to lapse.

It directs companies to conduct pediatric trials with combinations of drugs. More than 40 combination therapies are approved for adults, but only 2 are approved for children. This legislation fixes that inequity. Every member of the Energy and Commerce Committee and 392 Members of the House voted for this provision as part of the user fees package last Congress.

It brings the FDA's enforcement capabilities for children on par with that of adults, giving the FDA new options to ensure pediatric studies are completed on time. Today, the FDA can only remove a drug from the market if pediatric studies are not completed. This bill gives the FDA more flexibility to ensure companies follow the law.

The bill dedicates existing funds for pediatric research through the NIH's Best Pharmaceuticals for Children Act Program over the next three years, the program's first funding update since it was authorized in 2002. Zero new funding will be used.

This bill was advanced by the House Energy and Commerce Committee unanimously last week.

Children are not little adults. They deserve effective medicines, just as adults do. The Give Kids a Chance Act will get children what they deserve and save lives.

I urge my colleagues to vote for this critical legislation.