FDA Accelerated Approval PROGRAM, how it works, what the benefits are, etc
A)
In order to get things started, the preliminary events include:
1) a company executes a clinical trial to treat an unmet need for a serious disease
2) upon encouraging surrogate data, a company prepares a presentation for the FDA
3) discussions takes place between the FDA and a company, and a decision is made whether or not pursuing accelerated approval is advisable
If the FDA and a company agree that pursuing an accelerated approval pathway is appropriate, the benefits include:
> utilization of a surrogate endpoint in a p2 trial, rather than a standard endpoint that would take much more time to report on. surrogate endpoints are considered on a case by case basis. ORR as a surrogate is usually granted only when accompanied by DOR, but can be granted on its own.
> potential eventual FDA approval prior to the completion of a p3 trial, rather than after completion of a p3 trial
In order to gain the actual accelerated approval, the requirements include:
1) collection of surrogate endpoint data for full trial cohorts.
2) database lock and data analysis which takes a while.
3) a complete NDA/BLA submission, no different than the NDA/BLA requirement for any other FDA approval, which takes an absolute minimum of 6 months
4) the FDA review period consisting of the 2 month preliminary review and then the full "substantive" review across either 6 months (with priority review, requested upon BLA submission) or 10 months.
5) a confirmatory phase 3 clinical trial being funded and "underway" - which includes an approved protocol, sites being activated and screening having started
> this requirement was codified into federal law in 2023.
C
Once all of this occurs and a drug is approved, it can then be:
marketed / sold
prescribed / prescribed off-label
**************************************************
cytodyn is currently at step A.2
I believe that the accelerated approval pathway is likely valid for cytodyn and mss-crc, pending full ORR data. if cytodyn decides to pursue accelerated approval, then discussions could be concluded by the end of the year. In that case, B.2 (a targeted database lock regarding the surrogate endpoint) could be completed as early as 1st quarter 2027, if the FDA agrees to ORR as the surrogate without DOR. If ORR and also DOR are required, then database lock has to wait until all responsive patients finally progress.
A)
In order to get things started, the preliminary events include:
1) a company executes a clinical trial to treat an unmet need for a serious disease
2) upon encouraging surrogate data, a company prepares a presentation for the FDA
3) discussions takes place between the FDA and a company, and a decision is made whether or not pursuing accelerated approval is advisable
If the FDA and a company agree that pursuing an accelerated approval pathway is appropriate, the benefits include:
> utilization of a surrogate endpoint in a p2 trial, rather than a standard endpoint that would take much more time to report on. surrogate endpoints are considered on a case by case basis. ORR as a surrogate is usually granted only when accompanied by DOR, but can be granted on its own.
> potential eventual FDA approval prior to the completion of a p3 trial, rather than after completion of a p3 trial
In order to gain the actual accelerated approval, the requirements include:
1) collection of surrogate endpoint data for full trial cohorts.
2) database lock and data analysis which takes a while.
3) a complete NDA/BLA submission, no different than the NDA/BLA requirement for any other FDA approval, which takes an absolute minimum of 6 months
4) the FDA review period consisting of the 2 month preliminary review and then the full "substantive" review across either 6 months (with priority review, requested upon BLA submission) or 10 months.
5) a confirmatory phase 3 clinical trial being funded and "underway" - which includes an approved protocol, sites being activated and screening having started
> this requirement was codified into federal law in 2023.
C
Once all of this occurs and a drug is approved, it can then be:
marketed / sold
prescribed / prescribed off-label
**************************************************
cytodyn is currently at step A.2
I believe that the accelerated approval pathway is likely valid for cytodyn and mss-crc, pending full ORR data. if cytodyn decides to pursue accelerated approval, then discussions could be concluded by the end of the year. In that case, B.2 (a targeted database lock regarding the surrogate endpoint) could be completed as early as 1st quarter 2027, if the FDA agrees to ORR as the surrogate without DOR. If ORR and also DOR are required, then database lock has to wait until all responsive patients finally progress.
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