Coalition Letter for Advancing Targeted Therapies for Rare Diseases

As a member of NORD we are proud to have joined forces with 75 other rare disease organizations and signed the “Coalition Letter for Advancing Targeted Therapies for Rare Diseases Act” Click here to view PDF of the Letter. This bill is a result of the efforts of Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association (MDA), Duchenne Alliance, and more within the rare disease patient advocacy community. This legislation clarifies FDA’s existing authority to leverage data previously used in the approval of a targeted drug when approving a new therapy that “incorporates or utilizes the same or similar genetically targeted technology, or the same variant protein targeted technology”. This is particularly important to drug development for patients with diseases that contain various genetic sub-populations. This is also important for diseases that may be genetically related to other diseases, and could potentially benefit from drug development in those other diseases. The bill language for S.2030 can be found here: https://www.congress.gov/bill/114th-congress/senate-bill/2030/text