We are excited to announce that U.S. lawmakers have finally reached an agreement on the 21st Century Cures Act, a game-changer for patients with rare diseases. This agreement comes more than one year after the House passed its original version of the bill. The first vote is expected to take place tomorrow.
Late last week, Chairmen Fred Upton (R-MI) and Lamar Alexander (R-TN) of the House Energy and Commerce Committee and the Senate Health, Education, Labor and Pensions Committee (HELP) Committee, respectively, announced a bicameral agreement on an updated 21st Century Cures Act. The House is scheduled to vote on the package tomorrow, and the Senate is expected to vote soon after.
Overall we are quite pleased with the package, and fully support its passage. There are many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients, including:
- Extension of the Rare Pediatric Disease Priority Review Voucher (PRV) Program through September 2020, with an additional two years extension for products that received an application designation.
- Streamlining of FDA review of genetically targeted and protein variant therapies for rare diseases.
- Funding in the form of $4.8 billion over ten years for the NIH to fund the Precision Medicine Initiative, BRAIN Initiative, the Cancer Moonshot, and more.
- Requirement for companies to publicly post their expanded access policies on their website.
- Further expansion of the Patient-Focused Drug Development Initiative and requirements for the FDA to report on how patient experience data was used in regulatory review.
- Creation of the National Neurological Conditions Surveillance System to better quantify the incidence and prevalence of neurological diseases, including rare neurological diseases.
- Expansion of the Humanitarian Use Device program to include devices used by up to 8,000 individuals rather than the current 4,000 individual cap.
While not everything we supported has been included (such as the OPEN Act additional resources for developing natural history registries for rare diseases, and more robust funding for the NIH and FDA), the package still represents a substantial step forward for the development of therapies for rare diseases, the majority of which still have no treatment.
NORD looks forward to continuing to advocate for a strong Rare Pediatric Disease PRV program, appropriate FDA funding to ensure a strong FDA orphan therapy review process, and the replenishment of funds aimed at prevention to address important unmet needs.
Most of all we want to thank our fellow rare disease patient advocates for joining us in this fight. Thank you to the 185 organizations that supported a long-term reauthorization of the Rare Pediatric Disease PRV program, and the more than 1,000 advocates who called their Congressmen to support the 21st Century Cures Act on the unified rare disease day of action earlier this month.
Without you, we would not have gotten this far. But we can’t stop now. The bill has not yet passed, and we still need your help to get it across the finish line. Please consider calling your Congressmen today to add your voice.