Mikaela Naylon Give Kids A Chance Act

Floor Speech

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Mr. PALLONE. Mr. Speaker, I thank the sponsor of this legislation on the Democratic side, the gentlewoman from Michigan (Mrs. Dingell), for yielding.

I rise today in support of this legislation, the Mikaela Naylon Give Kids a Chance Act, sponsored by Representatives Dingell and McCaul.

This bill includes several strong, bipartisan pieces of legislation to help expand access to care for patients with rare diseases and complex medical needs. It increases research and development into rare pediatric diseases, improves regulatory certainty, strengthens the medical product supply chain, and enhances patient access to lower-cost medicines.

This bill will help in our efforts to bring new treatments to pediatric patients with more advanced forms of cancer. It also includes important enforcement authority for the Food and Drug Administration to take action against companies that do not conduct required studies for pediatric patients.

The bill also includes critical language to clarify the FDA's long- held treatment of orphan drug exclusivity and bring new treatments to more patients suffering from rare diseases. It will also allow for disclosure of certain information related to drug formulations to generic drug manufacturers to bring lower-cost medications to patients more quickly.

These provisions all advance our goal of accelerating the development of novel, safe, effective, and affordable treatments while improving outcomes for patients.

I am also pleased that the legislation provides the Health Resources and Services Administration, or HRSA, with the authority to modernize the organ transplant system. This language will allow HRSA to collect registration fees and distribute them among the multiple contractors that will be supporting the OPTN. The system is lifesaving for Americans, and I am glad that we can continue to support these much- needed reform efforts.

Mr. Speaker, this bill also 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.

As a whole, this bill is going to make a difference, but I continue to believe that additional guardrails are needed for the PRV program to function the way Congress intended it to.

Previous reports from the Government Accountability Office on the program and continued research from academics show that there is little evidence that it incentivizes the products that Congress intended to help come to market.

This is especially concerning as the FDA has introduced a new priority review program with potentially massive financial benefits for pharmaceutical companies without any transparency or congressional authorization. That is why I am pleased that this bill includes a requirement for the GAO to conduct a new study and report on the effectiveness of the PRV program in achieving our original intent.

Let us not overstate the impact of this bill, however. While we are working together to advance the bill forward on incremental but important bipartisan legislation to advance both drug discovery and drug affordability, the Trump administration continues its devastating attacks on public health, including in cancer research.

The Trump administration's substantial cuts to research funding for the biomedical community is chilling the innovation that we are trying to promote through this bill.

The Trump administration is decimating research and endangering the pipeline of new cancer therapies that could come to FDA for approval. Their actions are putting patients at risk and leaving pediatric patients without the treatments that we are trying to ensure can be tested and brought to them.

Meanwhile, the Trump administration is using the FDA as a tool to provide lucrative gifts to drugmakers and allies of President Trump, undermining the Agency's science-based review process, and endangering the health of the American public with potentially unsafe and rushed drug reviews.

The Trump administration is also decimating our vaccine infrastructure, from the programs that cover our vaccines and ensure that patients can afford them to destroying the pipeline to future vaccines through their ideological, pseudoscience agenda.

Mr. Speaker, one day, I hope that my Republican colleagues will join us in calling on the Trump administration to reverse these terrible policies that are only harming the public health.

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