Tutorial on FDA Approval Outcomes

J0409042 Every so often, I think it is a good idea to make sure everyone is on the same page for terminology and what it means.  Therefore, today I’m reviewing a few terms and situations.  If you are already knowledgeable about approvals, this won’t interest you. 

And yeah, don’t I wish that were a picture of me….

PDUFA Date or Action Date – This is the date by which the FDA will decide upon a filing for approval.  It may be 6 months (Priority Review) from the date of NDA filing or 10 months from the date of the filing.   Note, that the date of the filing is not publicly revealed by the FDA.  It is a strategic decision by a company whether or not to reveal that date.  Many do, because it is considered by counsel to be material or because they want to juice investors.  In any case, once made public, everyone can start counting on their fingers to know when your "PDUFA" or "Action Date" is going to pass.

Priority Review – This means that the agency is going to review a company’s application in six months rather than the traditional ten months.  This is generally reserved for drugs that offer a significant advancement over current therapies.  The FDA has what are called MAPPs that tell you how these things work.  The Priority Review Mapp can fill you in on details.  Priority Review should not be considered with Accelerated Approval.

Accelerated Approval – Priority Review is when the FDA is going to look at your product more quickly after a company has finished all it’s clinical trials.  Accelerated approval is when the agency allows a company approval of an investigational compound while it is still being studied.  This means it can be used prior to finishing all its clinical trials.  This is done only when the treatment not only offers tremendous promise, but there is little else available in the therapeutic category.  Note that there are marketing restrictions on these drugs.  The FDA Guidance on Accelerated Approval can shed more detailed light. 

Lastly, there are a few approval outcomes.  First, of course, there is the one everyone wants – Approval.  A drug cannot be marketed until it has approval.  Approval is not represented by an advisory committee vote, it is represented by the FDA issuing a letter to the drug sponsor telling them that it has approved the drug.  This occurs days, weeks, or even a few months after an advisory committee meeting, but usually before the PDUFA or Action date noted above. 

A sort of maybe approval is available when a company gets an Approvable Letter.  This means the FDA has told a company that the product could be approved if the company meets certain conditions first.  These conditions could be complex, expensive and cumbersome, such as new clinical trials, or easily addressed, such as a labeling, packaging or small manufacturing issue.  It is noteworthy that the FDA will NOT state the contents of an approvable letter to the media.  That is up to the company.  Some do, some don’t – it is a strategic choice.  I could talk more about this, but I don’t want this posting to be that long….

Next, the FDA can choose to extend a PDUFA date.  Yes, you heard right.  This isn’t done frequently, but if a sponsor submits new data during the period when the drug is being considered for approval, and the FDA deems that this new material is quite substantive, then the PDUFA date can be extended. 

Lastly, there is the outcome no one except competitors like – the non-approval letter.  That means just what you think it does.  That generally means that the effort, like this posting, is over….

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5 Responses to Tutorial on FDA Approval Outcomes

  1. Peter says:

    One point of clarification: the “non-approval letter” actually is a “not approvable letter”, and it is not necessarily the end of the road. A “not approvable letter” explains why your application is deficient at this time, and if you correct these deficiencies and re-submit the application, you may get approval eventually. A prominent recent example is the Plan B OTC switch application, for which Barr Labs received a not approvable letter in 2004 but kept at it.

  2. Mark Senak says:

    Thanks, and you are right, it is not necessarily the end of the line. I have worked with clients before who have come back after a non-approval. I have to admit, I was looking for a glib ending to the posting!

  3. david says:

    It’s worth remembering Orzel, a drug Bristol-Myers Squibb brought forward as an oral formulation of 5-FU. It represented a significant advance for cancer patients’ quality of life (not having to be tethered to an IV pole, for instance). The AdCom voted 8-0 for approval. FDA turned it down flat, citing a lack of studies indicating a survival benefit. AdComs are nice, they aren’t the equal to that letter from FDA, though.

  4. Grtdane says:

    How ofter have you heard of a Pdufa date being missed (Cardiome now 30 days past Pdufa) WITHOUT a reason?
    Published comments from Cardiome say FDA told them they needed more time….and NO additional info/studies requested.
    Totally a rudderless processes, waiting on a black hole at FDA to cough up a response. The drug in question is iv Vernakalant.

  5. Mark Senak says:

    Yes, PDUFA dates do get missed. It isn’t common, but it happens. Also, the communications between the company and the FDA at this time are proprietary, so it could be that there have been discussions that have caused a delay. I have noticed that there have been quite a few more PDUFA extensions as well, where new data exists and the FDA wants to examine it. All of these are rare, but they do happen. And if you go to the FDA Web page, you can see in their annual reports, that they list the percentage of times they were PDUFA compliant, and those where they weren’t.

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