After hundreds of Hill meetings, emails and calls to Congress, we are seeing the true impact of Rare Disease Patient Advocacy on the 21st Century Cures Legislation. The latest draft released this week includes many priorities for the rare disease community – including 3 out of the 4 pieces of legislation advocates lobbied for during Rare Disease Week 2015:
- HR 971, Orphan Product Extensions Now Accelerating Cures and Treatments Act(OPEN ACT)
- HR 292, Advancing Research for Neurological Diseases Act
- HR 909, Compassionate use Reform & Enhancement (CURE) Act
Other important provisions benefiting the rare disease community include, making the Pediatric Priority Review voucher program permanent, the NIH Innovation Fund, language on Precision Medicine for Orphan diseases, and patient focused drug development.
We wish to thank all the Patient Advocates who have taken action to support rare disease legislation and to the Energy & Commerce Committee Members and their Staff for making rare disease patients a priority.
We are so excited about the impact we are making together! However, the battle is hardly over. The 21st Century Cures Legislation faces some tough challenges in the upcoming weeks and months. We need rare disease advocates to keep up the pressure on Congress to ensure the rare disease provisions are included in the final legislation and to ensure Congress passes the legislation. Please click here and take 2 minutes today to thank Members of Congress for making rare disease patients a priority! Remember legislation will only happen if YOU take action.