Readout from October 6 Federal Advocacy Drop-In Session

The October 10 Federal Advocacy drop-in session covered three pieces of authorizing legislation and provided an update on possible appropriations for FY2025.  Each of the legislative initiatives have a strong possibility for passage in this Congress, and advocates may be called on to make final pushes between now and the end of the year.

Legislation:

Give Kids a Chance Act has passed the US House and is awaiting approval in the Senate. This bill would help ensure kids with cancer have access to the most modern clinical trials by authorizing the FDA to direct companies to perform a pediatric cancer study for a combination of drugs if the company is seeking approval for a drug to treat an adult cancer and that drug has a molecularly relevant pediatric indication. The Pediatric Voucher Program (PVA, aka the Creating Hope Reauthorization) has been folded into Give Kids a Chance, so it also has House approval.

The PVA was originally passed in 2011 and has been reauthorized twice since then (in 2016 and 2020).  It expands the cost-neutral Food and Drug Administration priority review voucher program, allowing pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases. Two drugs receive priority review for each voucher: the drug winning a voucher for a neglected or rare pediatric disease and the drug using a voucher for another indication. The FDA awarded Priority Review Vouchers (PRV) for four of the six drugs originally approved in the first instance for cancer treatment for children since its passage. Holders of a PRV get a faster FDA drug approval process for a future drug of their choice. The vouchers are transferable (and may be sold or traded) and provide desired incentives for developers of drugs for rare pediatric diseases.

Extension of the PVR program was included in the Continuing Resolution to keep the government operating until December 20 that passed in September.  This means we have to steer Give Kids a Chance through the Senate before the end of the year or the Creating Hope Act will sunset.

H.R. 4758/S. 2372 Accelerating Kids’ Access to Care Act (AKACA). Cancer care for children is not always available close to home, which means many families must travel to other states for treatment.  Accessing care in other states is time consuming and challenging to navigate.  This legislation would improve children’s access to needed out-of-state health care by streamlining the Medicaid provider screening and enrollment process. This bill has also passed in the House, and we have to steer it through the Senate before the end of the year or start again from scratch in the new year. You can read more about the bill here:  AKACA

H.R. 3391/S. 1624 Gabriella Miller Kids First Research Act 2.0: This program helps researchers uncover new insights into the biology of childhood cancer and structural birth defects, including emerging scientific opportunities, rising public health challenges, and knowledge gaps. Since the program was enacted, Congress has provided $125 million to childhood cancer and disease research. Reauthorization legislation will, over the next five year period, enable expansion of the comprehensive shared data resource for scientists researching pediatric cancers and rare disease and support continued development of computational tools to analyze very large, complex genomic, and clinical data sets

Recent Background:  The bill passed in the House March 5, 2024. Sen. Tim Kaine (D-VA), Jerry Moran (R-KS) and 13 other bipartisan Senators reintroduced the Senate version of the bill. If this passes in the Senate and is signed into law, the bill will provide Kids First with a five-year reauthorization of $25M per year.  The bill is currently blocked in the Senate.

The Immediate Ask: It was brought to our attention by Ellyn Miller (Smashing Walnuts) that we need as many constituents as possible from Kentucky to send an email to the office of Sen. Rand Paul (R) to ask for support. If you or someone you know resides in Kentucky, please email Ellyn directly, , and request a template letter to send to his office. She can provide that email address, the letter template that will have the “ask” of Sen. Paul, as well as an area to tell him your story as well as stating that you are a constituent. This should take someone 5 minutes to complete.

Appropriation:

Since 2017, the STAR Act has received annual appropriation of $30 million to the implementation of various components of the Childhood Cancer STAR Act and the Childhood Cancer STAR Reauthorization Act.  This year, both the relevant House and Senate Committees have recommended additional funding of $20 million.  If that recommendation makes it into the final budget, we hope that the agencies will concentrate on the following four areas:

• Enhance childhood cancer survivorship research,
• Broaden the scope of the highly successful Molecular Characterization Initiative,
• Expand the centralized biorepository for the next frontier of research and new treatments, and 
• Fund national pediatric cancer registry infrastructure

We may be called on to actively support this additional funding.