Congress is grappling with a busy and complex agenda before adjourning on September 27 through the election. Congress is on track to meet its deadline of October 1 to pass a government funding measure and there are several other important issues that the life sciences community is advocating for Congress to prioritize by the end of the year. Two of the key issues CBSA is keeping a close eye on include the reauthorization of the rare pediatric disease priority review voucher (PPRV) program (S. 4583 and H.R. 7384) and the Ensuring Patient Access to Critical Breakthrough Products Act of 2023 (H.R. 1691).  

• Reauthorization of the Rare Pediatric Disease Priority Review Voucher (PPRV) Program: The PPRV program is set to expire on September 30, 2024, but bipartisan and bicameral legislation, S. 4583 and H.R. 7384, would reauthorize the program, allowing for a continued path forward to additional innovative therapies for pediatric rare disease patients.
  • On September 18, the House Energy and Commerce Committee voted unanimously (43-0) to pass a pediatric package favorably out of committee. This package included H.R. 7384, the Creating Hope Reauthorization Act of 2024, which would extend the PPRV program for another five years.
  • On September 23, the U.S. House of Representatives passed the pediatric package, including the five-year reauthorization of the PPRV program, by voice vote.  
  • As of September 23, it looks likely that the continuing resolution (CR) Congress is expected to pass in the coming days to extend funding for the federal government will also include an extension of the PPRV program through the duration of the CR—December 20 (so a full reauthorization of the program would still need to be included in the year-end package).
  • The Senate plans to mark up S. 4583 on September 26. CBSA and BIO are hopeful that this progress will pave the way for inclusion of the Creating Hope Reauthorization Act in an end-of-year package when Congress returns after the election. 
  • CBSA still urges members to reach out to Senator Hickenlooper, as he is an important voice on the Senate Health, Education, Labor, and Pensions (HELP) Committee, and other elected officials from Colorado to encourage them to support and co-sponsor the Creating Hope Reauthorization Act.
    • Read this CBSA policy blog from June 25: Urge Congress to Reauthorize the Rare Pediatric Disease Priority Review Voucher (PPRV) Program.
    • See this action alert from the National Organization for Rare Disorders (NORD) and the EveryLife Foundation. Also see NORD’s PPRV one-pager, white paper, and impact report.
  • CBSA and 47 other organizations signed onto this Council of State Bioscience Associations (CSBA) letter expressing support for the program submitted to Congressional leaders on September 9. 

• The Ensuring Patient Access to Critical Breakthrough Products Act of 2023 (H.R. 1691)
  • H.R. 1691, the “Breakthrough Act,” would create a timely, streamlined pathway for Medicare patients to access innovative medical technologies.
  • As CBSA described in its August 26 policy blog, Transitional Coverage for Emerging Technologies (TCET) Final Notice and the “Breakthrough Act,” the Centers for Medicare & Medicaid Services (CMS) released the long-awaited TCET Pathway Final Notice in August. Although this rule is a step in the right direction, CBSA continues to urge Congress to pass the bipartisan “Breakthrough Act” (H.R. 1691) to more fully address seniors’ need for access to breakthrough diagnostics and treatments soon after they are approved by the Food & Drug Administration (FDA).
  • CBSA recently signed on to an H.R. 1691 support letter from AdvaMed. 

Other topics CBSA is monitoring include the Pioneering Antimicrobial Subscriptions to End Upsurging Resistance (PASTEUR) Act, the Verifying Accurate Leading-edge IVCT Development (VALID) Act, intellectual property protections, IRA fixes like the Optimizing Research Progress Hope and New (ORPHAN) Cures Act and the Ensuring Pathways to Innovative Cures (EPIC) Act, the Multi-Cancer Early Detection (MCED) Screening Coverage Act, and PBM reform. CBSA will continue to advocate for policies that promote innovation and improve patient access to life-saving therapies and technologies. If you are interested in contributing to CBSA’s advocacy efforts, please reach out to CBSA’s Vice President and Counsel for Policy + Advocacy, Amy Goodman.