FDA TAKES ACTION
Section added by P. Kolchinsky, Editor
The Prescription Drug User Fee Act of 1992 (PDUFA I) allowed companies to pay the FDA to review an NDA faster, assigning more people to the process, perhaps. At first, the FDA promised a response within 12 months of the filing date and eventually pushed this goal down to 10 months. However, a response does not guarantee completion of the review process. By the so-called PDUFA date, the date by which the FDA is expected to give its response, possible verdicts include:
- Approval: The company may proceed with launching its product.
- Non-Approvable: Also known as a Complete Response Letter, a non-approvable letter might require that the company run lengthy additional clinical studies before the FDA will consider giving its approval.
- Approvable: An approvable letter indicates that the FDA agrees that the drug is safe and effective but want more information, which the company must assemble and submit to the FDA. A requirement for a Class I resubmission is considered minor in that it usually involves paperwork, such as a reshuffling of existing clinical data or agreement on how the drug will be labeled, and if any clinical trials are required, these may be done as Phase IV post-marketing studies. Class II resubmission can be more laborious, possibly involving additional clinical studies prior to approval or upgrade and reinspection of manufacturing facilities. If the required information is provided in a timely manner, the FDA will usually review a Class I within 2 months and a Class II within 6 months of resubmission.
If the FDA decides to convene an Advisory Committee (AC) to guide its decision, this meeting will usually take place prior to the PDUFA date. The FDA will usually follow the advice of the AC, but it does not have to.
FDA approval of an NDA should not be confused with Acceptance for Filing of an NDA, which indicates that the application is complete and occurs automatically 60 days after submission of an NDA, unless the FDA raises issues during the 60-day period.
EVEN FASTER
The FDA issues a number of special designations that can speed a much needed drug through development and/or the review process.
Fast Track: At the time of IND review or during subsequent discussion with the FDA, the sponsoring company can request the drug be granted Fast Track status. A Fast Track designation allows for more interaction with the FDA throughout development as well as "rolling" submission to the FDA of the various component of the NDA (i.e. preclinical package, clinical package, CMC) as they are completed.
Accelerated Approval: Drugs that receive Accelerated Approval do not require as much clinical data in their NDA and may be reviewed, for example, on the basis of only Phase II results. With the FDA's consent, such drugs may also be approved on the basis of improvements in surrogate endpoints, which can take less time than showing a clinical benefit.
Priority Review: An NDA that has been assigned Priority Review within 60 days of submission will be acted on within 6 months of submission. All NDAs are considered for Priority Review regardless whether the sponsoring company requests it.
ABOUT THE AUTHOR
Kenneth B. Klein, MD. Endpoint
Dr Klein is a Harvard-trained physician board certified in both internal medicine and gastroenterology. After a career in academic medicine he spent fifteen years in the pharmaceutical industry in the US and Europe.
Nine year ago he founded Endpoint, with offices in Seattle and London. Endpoint provides clients with authoritative advice on the development of medicines, the interpretation of complex preclinical and clinical data, and the creation of novel designs for pivotal clinical studies. Endpoint also has extensive experience with in- and out-licensing strategies, due diligence, coordination of interactions between companies working on a common project, and evaluation of the clinical and commercial potential of drug candidates.
Endpoint's clients include multinational pharmaceutical companies, emerging biomedical ventures, biotechnology investment firms, contract research organizations, regulatory agencies and national health services.