CPTA’s Requests to House and Senate
Below is a rundown of requests made by C-Path’s Critical Path for the Ataxias (CPTA) to the U.S. House and Senate in September, 2024.
1. Ask Senators and House Members:
- To pass the FY25 defense appropriations bill with “Hereditary Ataxia” as a topic area in the PRMRP.
- If they would consider submitting a CDMRP/PRMRP appropriations request for the FY26 budget.
- For the contact information for the staffer who handles their appropriations requests.
- Why: Supporting the continued inclusion of Hereditary Ataxia in the CDMRP would provide much needed additional resources to expedite drug development and treat hereditary ataxia.
2. Ask Senators and House Members: To pass a timely FY25 budget with robust funding for the NIH & FDA; no continuing resolutions.
- Why: The NIH and FDA are important federal agencies in developing treatments for Ataxia. They are already underfunded for the work that they do. Delays in passing the FY25 budget directly impact the work of the federal research institutions we depend on. A continuing Resolution (CR) would create confusion regarding long-term funding and delay new research projects that may help hereditary ataxia patients
3. Ask Senators and House Members: To pass the Creating Hope Reauthorization Act so Priority Review Vouchers (PRVs) continue to serve as an important incentive in pediatric rare disease therapy development.
- Why: Developing drugs for rare pediatric diseases is challenging due to the small populations affected, difficulties associated with conducting clinical trials for children, delays in diagnosis and more. 7 out of 7 developers interviewed by the GAO reported that PRVs were a factor in their decisions, and this incentive helps bring about treatments for conditions that wouldn’t otherwise be considered for research and development.
4. Ask Senators and House Members: To pass the Accelerating Access to Kids’ Access to Care Act to allow pediatric providers to enroll more efficiently in multiple state Medicaid programs and improve access to out-of-state care.
- Why: To ensure better, faster healthcare for kids who need it most by providing a streamlined process for providers to enroll in other state Medicaid plans.
5. Ask Senators and House Members: To pass this bill to incentivize companies to continue investing in therapeutics across the disease population.
- Why: This bill would codify how FDA currently interprets the Orphan Drug Act’s exclusivity provisions, limiting the seven-year exclusivity period for orphan products to the approved use of indication, rather than the entire disease or condition as the 11th Circuit held in Catalyst Pharms., Inc vs. Becerra.
6. Ask Senators and House Members: To cosponsor this bill to ensure the patient perspective is considered by FDA reviewers who are evaluating drugs and other medical products.
- Why: So that the patient experience data (patient-reported outcomes, patient testimonials, and natural history data) would be required by the FDA to be included in the risk analysis when reviewing new drug applications.