The first linked article below relates to recently-passed...

The first linked article below relates to recently-passed legislation that is aimed at assisting the FDA to curb perceived abuse of the Accelerated Approval Program.

The Accelerated Approval Program was introduced to speed up access to new drugs for patients with serious illnesses and limited options. The idea behind it was that a drug maker could apply to have their drug approved more quickly on limited clinical data but the approval was conditional on carrying out a further study to prove the drug’s efficacy and safety. If that confirmatory study failed, the FDA could require the drug’s removal from sale.

This made it possible for a drug approved under this pathway to rake in hundreds of millions of dollars before data confirming efficacy had become available. Which is problematic if you consider that as many as 1 in 5 cancer drugs granted accelerated approval ultimately failed to demonstrate improvements in overall patient survival.

There can be a significant time gap until confirmatory data becomes available, if ever. In an investigation conducted last year, it was found that 42% of outstanding confirmatory trials required after accelerated drug approval either took more than a year to commence or never commenced at all.

One “fix’ to the Accelerated Approval Program contained within the new legislation allows the FDA to require companies to start confirmatory studies before the agency grants accelerated approval to the drug. This requirement remains discretionary rather than mandatory.

The FDA Head of Oncology has stated in recent months that his division wants to see confirmatory trials already underway when companies submit their applications for accelerated approvals. He noted that oncology withdrawals under the accelerated approval pathway took 7.3 years at median 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.

Hence the uncertainty expressed by SYC about the accelerated approval pathway for PTX-100. Clearly, needing to commit to two closely-spaced large trials would significantly impact complexity, cost and feasibility.

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