Priority Reviews

[georgethenight2]

Has there ever been a rejection of drugs that were applied with a priority review? Asking the board.

[stockwhisperer]

There is never a guarantee with the FDA - for priority review, fast track or otherwise. Would think everyone knows that. It is only about the timing of the FDA’s decision. “Designation of a drug as “Priority” does not alter the scientific/medical standard for approval or the quality of evidence necessary.” www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review

[georgethenight2]

Sept 12, 2021 6:11:44 GMT -5 stockwhisperer said:

There is never a guarantee with the FDA - for priority review, fast track or otherwise. Would think everyone knows that. It is only about the timing of the FDA’s decision. “Designation of a drug as “Priority” does not alter the scientific/medical standard for approval or the quality of evidence necessary.” www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review

Not questioning the FDA standards for approval, but are there actual instances of a fast tracked drug being rejected or given a CSR(correct).

[neil36]

Google the words “FDA priority review rejections”

I didn’t find one particular article to answer your question, but I did find several instances of priority review rejections. But my impression after a few minutes of looking is that approval rates are generally quite high. But like anything to do with the FDA, there are plenty of surprise decisions.

[stockwhisperer]

So true. Not sure that the answer would matter or have any real relevance to the upcoming October response since each FDA approval or rejection is unique to the individual request.

[stockwhisperer]

This is long but very informative - includes some references in the text and footnotes about number of approvals… rarediseases.org/wp-content/uploads/2021/06/NRD-2182-Policy-Report_Accelerated-Approval_FNL.pdf

[akemp3000]

It looks like the approval rate under a PRV is very high with the current typical cost of about $100M. Any company purchasing one should be "almost" certain it will be approved. Saw this...

"There are several risks inherent to using a PRV. The first and most obvious is that FDA is under no obligation to approve a product using a voucher. Both FDASIA and FDAAA stipulate that FDA only needs to come to a decision within six months. As Novartis proved in the first-ever use of a priority review voucher, FDA will not necessarily approve a product just because its sponsor used a voucher.

Priority review will likely accelerate the approval of a good drug to market, but it will not save a bad drug from being rejected. And for companies that are unsuccessful in their use of a priority review voucher, the cost of failure can be especially high due to the cost to procure and use the voucher".

[cretin11]

Thanks for that info. All evidence points to TreT being clearly in the “good drug” category, so that bodes well. UTHR isn’t trying to sneak a “bad drug” through approval. Fingers still crossed but confident we will get the right decision from FDA.