Expedited approval programs are referred to as fast-track programs. The FDA created these programs for new drugs in 1992 under the Prescription Drug User Act (“PDUFA”) with the aim of meeting treatment needs for serious or life-threatening medical conditions more quickly.
What are the options?
- Fast-track designation is granted to a drug for a serious condition for an un-met need that will improve the survival or the day-to-day function of the patient.
- A breakthrough therapy is a drug that treats a serious condition and shows preliminary evidence that it is superior to a drug already on the market with clinically significant endpoints.
- Accelerated approval is granted to a drug that treats a serious condition using surrogate endpoints to show the benefit of the drug. A surrogate endpoint is one that demonstrates it is reasonably likely to predict a real clinical benefit. E.g. shrinking tumors=longer survival
- Priority review is granted to a drug that is highly effective, substantially eliminates adverse reactions that prevent treatment, is safe and effective in a new subpopulation and/or helps patients be more compliant with the drug regimen.
The specific meaning of each of these paths and the distinction among them can be confusing. It is important to determine early in the beginning clinical stages if the drug might fit into either of these pathways. You need an advocate to describe to the FDA in detail how the drug may fit into one of these fast-track programs.
The advantages of being part of a fast-track program for your drug are the FDA accelerates the review of the drug and stays in close contact with the sponsor of the drug. The FDA wants the product to be successful and is part of the review process early in the development. The Agency also has shortened timeframes within which it must respond to the applicant or sponsor. Of course, the economic advantages are obvious!
Is your company missing out on these options?
Jean W. Frydman is a partner in the firm’s Princeton office.