Rare Disease Legislative Recommendations
The following policy overview reflects the values and current focus of MLD Foundation.
Rare Disease Policy Leaders
We encourage all rare disease organizations to join and follow the policy efforts at:
- The EveryLife Foundation/RDLA
- NORD … broad policy engagement
- Haystack Project … access & reimbursement issues
- Genetic Alliance
- EPICrd … a patient driven effort … Rare Disease Medicaid policy
- ALD Alliance … newborn screening initiatives
- MLD Foundation … newborn screening, access, reimbursement, Medicaid
- RAAP – Rare Access Action Project
- CAHC – Council for Affordable Health Care
- PIPC – Partnership to Improve Patient Care … QALYs, 340B rule
- Global Genes
Rare Disease Legislation
The following policy overview reflects the values and current focus of MLD Foundation. We are always happy to mentor and help direct your efforts to maximize your impact for rare disease and your specific community.
Please follow the links to the primary sources of information and sponsoring organizations for various policy initiatives.
As of February 2025
- Ensure steady and robust leadership, federal biomedical research funding, and public health agency support
- Reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program (118th HR 7384 and S. 4583, the Creating Hope Act)
- Pass Accelerating Kids’ Access to Care Act (118th S.2372 HR 4758)
- • Join the Rare Disease Congressional Caucus (Everyone must join again for the 119th Congress)
- PROTECT Rare Act – assuring disease parity in accessing medically necessary care (118th HR 6094 … Reps. Matsui, Dunn, Kelly and Thompson)
- Oppose co-Pay Accumulators
- Oppose Step Therapies – Safe Step Act
- EPICrd Act – Medicaid access (coming soon – patient advocacy driven – MLD Foundation is a prime creator of this legislation)
- MVP Act – Gene Therapy (and other expensive therapies) Medicaid Value Based Payments for Patients Act (118th HR 2666 … Reps. Eshoo, Joyce, Auchincloss, Miller-Meeks)
- Support Federal Funding to help states launch RUSP-recommended Newborn Screens (NBSSL – Newborn Screening Saves Lives Act & others)
- Require the FDA resume self-certification of LDTs (Lab Developed & certified Tests) for Newborn Screening
- Support PBM reform
- Oppose most PDABs – Prescription Drug Affordability Boards (Most of today’s PDABs will restrict rare disease drug access)
- Oppose use of QALYs (discriminatory Quality Adjusted Life Years) for value assessment