FDA User Fee Act Is Progressing

FDA User Fee Act Is Progressing

Over the last two weeks, the Senate and the House passed their versions of the Food and Drug Administration Safety and Innovation Act. Conference committees will now work together to come up with a compromise, which could potentially be signed by President Obama this summer. The bill is nearly 400 pages long and covers such areas as user fees for novel, generic, and biosimilar medications, device regulation improvements, and drug shortage issues.

One section of this bill that is particularly important for patients addresses the enhancement of accelerated patient access to innovative medicines that treat life-threatening and rare diseases that currently have limited treatment options.

If passed, this Act would enhance the authority of the FDA to expedite the development and approval of these medications that address unmet medical needs. According to the Act, the FDA will need to respond to a new drug sponsor’s request for fast track status within 60 days. If the drug is granted fast track status, the FDA will then take actions to expedite its approval. 

The FDA may also grant “accelerated approval” of a medication for a serious or life-threatening condition if the drug has an effect on a surrogate or clinical endpoint. Oftentimes, these endpoints can be measured earlier than irreversible morbidity or mortality, which will enable faster approval and patient access. The Act also authorizes the FDA to quickly withdraw approval of a medication that received accelerated approval if the sponsor fails to conduct required post-approval studies or if those studies fail to demonstrate clinical benefit.

As the leading provider of specialty program solutions that cater to orphan and ultra orphan products and rare disease patients, we’ll be watching this story closely.