The Orphan Drug Act (ODA) has incentivized the research and development of therapies for rare diseases. Since its passage in 1983, the ODA has opened new doors and provided alternative and even first-time treatments for individuals affected by rare diseases. The creation of orphan drugs is critical to improving the quality of life for those diagnosed with these conditions.
However, FDA regulations give first-to-market orphan drugs 7 years of market exclusivity pending approval. This incentive leaves other manufacturers facing large roadblocks toward bringing their therapy to market prior to the expiration of the 7-year exclusivity window. Yet, FDA policy does leave workarounds within the legislation for other drug developers to receive second-to-market approval during this timeframe (specific language can be found in 21 CRF 316.31(a)).
The leveraging of this policy stipulation has been used multiple times, including the recent example of the approval for Ruzurgi (amifampridine) by Jacobus Pharmaceuticals. The formal process and application requirements to receiving second-to-market orphan drug approval call for the aid of experienced orphan drug regulatory consultants. Through proper protocols and guidance, overcoming orphan drug exclusivity is possible.