There’s an excellent poster (@hottod) who posts in another HC page (another immunotherapy stock that I own) and she brought up the issue about the recent changes to FDA’s Accelerated Approval Program (AAP) “to treat serious conditions”. I’ve pasted her contribution below.
That got me thinking about how this could apply to IMM. HNSCC is obviously essentially in a registrational trial (albeit in a phase 2b study, and it depends on how strong the results are).
But under the AAP there is nothing stopping IMM applying for approval for MBC/TNBC and NSCLC once the phase 2/3 studies commence. Even with the recent changes the FDA only needs evidence that the confirmatory studies are already underway if there is already solid evidence that the drug works.
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The first linked article below relates to recently-passedlegislation that is aimed at assisting the FDA to curb perceived abuse of theAccelerated Approval Program.
The Accelerated Approval Program was introduced tospeed up access to new drugs for patients with serious illnesses and limitedoptions. The idea behind it was that a drug maker could apply to have theirdrug approved more quickly on limited clinical data but the approval wasconditional on carrying out a further study to prove the drug’s efficacy andsafety. If that confirmatory study failed, the FDA could require the drug’sremoval from sale.
This made it possible for a drug approved underthis pathway to rake in hundreds of millions of dollars before data confirmingefficacy had become available. Which is problematic if you consider that asmany as 1 in 5 cancer drugs granted accelerated approval ultimately failed todemonstrate improvements in overall patient survival.
There can be a significant time gap untilconfirmatory data becomes available, if ever. In an investigation conductedlast year, it was found that 42% of outstanding confirmatory trials requiredafter accelerated drug approval either took more than a year to commence ornever commenced at all.
One “fix’ to the Accelerated Approval Programcontained within the new legislation allows the FDA to require companies tostart confirmatory studies before the agency grants accelerated approval to thedrug. This requirement remains discretionary rather than mandatory.
The FDA Head of Oncology has stated in recentmonths that his division wants to see confirmatory trials already underway whencompanies submit their applications for accelerated approvals. He noted thatoncology withdrawals under the accelerated approval pathway took 7.3 years atmedian if the confirmatory trial wasn’t ongoing at the time of the green light.That compared with 3.8 years if the confirmatory study was already underway.
https://www.npr.org/sections/health...forcement-drug-approval-manufacturer-promises
https://www.npr.org/sections/health...flawed-approach-to-accelerated-drug-approvals
https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program
https://www.fiercebiotech.com/biote...ry-trial-underway-accelerated-approval-expect
(20min delay)