Oncolytics Biotech Inc T.ONC

RE:RE:How does Accelerated Approval work

[Noteable]

This is for the benefit of Quentin30 - who doesn't own any ONCY stock BTW.

" Accelerated approval (AA) targets candidates that are similar to those awarded BTD in terms of qualifying characteristics, such as aiming to treat serious conditions and providing improvements over current therapy. Most importantly, however, AA allows drugs to be approved using surrogate endpoints that are reasonably expected to predict clinical benefits. For example, for some cancer drugs, progression free survival (PFS) may be an effective surrogate endpoint that can be assessed instead of overall survival (OS), which may take much more time to determine, thus delaying the approval of a novel therapy for patients who have the potential to benefit. There is, however, reasonable room for error in extrapolating PFS findings to OS. Consequently, confirmatory trials such as phase 3 double-blind controlled trials are necessary to prove further the efficacy and safety of drugs that have been awarded accelerated approval. These trials must be carried out within a certain time window, after which, the drug may be withdrawn from the market if not determined to be acceptable."

https://www.sciencedirect.com/science/article/pii/S1359644621005729

" .... accelerated approval, which allows the use of surrogate endpoints that are not considered well established but that are determined to be “reasonably likely to predict clinical benefit.” FDA rules define surrogate endpoint as “a laboratory or physical sign that is used in therapeutic trials as a substitute for a clinically meaningful endpoint that is a direct measure of how a patient feels, functions, or survives and that is expected to predict the effect of the therapy” (57 Fed. Reg. 13234 at 13235; see also Fleming, 2005). FDA then requires post-approval studies to develop further evidence about benefits and risks based on clinical outcomes."

https://www.ncbi.nlm.nih.gov/sites/books/NBK56185/

omment by Noteableon Jan 20, 2024 1:31pm

126 Views 

Post# 35837421

RE:RE:RE:How does Accelerated Approval work

January 2024 -  FDA Unveils Groundbreaking Rare Disease Guidance Document

For drugs eligible to follow the Accelerated Approval pathway, a determination of safety and effectiveness may be made based not on measures of direct clinical benefit, but rather on one of two alternative endpoints: (1) a surrogate endpoint that is reasonably likely to predict clinical benefit; or (2) an intermediate clinical endpoint that is reasonably likely to predict clinical benefit. Use of such endpoints may enable the drug to be studied for a shorter treatment duration and receive Accelerated Approval based on these findings. Importantly, however, for products approved under the Accelerated Approval pathway, FDA requires post-approval studies designed to confirm clinical benefit, and, among other things, may withdraw an accelerated approval product from the market for failure to confirm clinical benefit. 

https://www.fda.gov/media/175253/download?attachment

Comment by Noteableon May 12, 2024 1:37pm

106 Views 

Post# 36035948

RE:RE:RE:RE:How does Accelerated Approval work

May 07, 2024 - “The facts clearly show the accelerated approval pathway is providing new hope and meaningful new treatments for patients to fight their disease, and why we shouldn’t be misled by biased studies that distort reality,” concludes BIO.

https://www.bio.org/gooddaybio-archive/setting-record-straight-accelerated-approval

May 08, 2024 - "Using a biomarker to obtain accelerated approval is key to get an initial approval for therapies that address devastating diseases, rather than wait for a clinical endpoint to show efficacy in a longer trial" according to the FDA’s Peter Marks, M.D., Ph.D.

“The wherewithal to do a three-year study or a four-year study without having a revenue stream, it's just beyond many companies that are startups. So having the accelerated approval process is a way to get there,” Marks said during a recent American Society of Gene and Cell Therapy (ASGCT) meeting. 

As long as a biomarker is scientifically validated, Marks said, more products can get to the patients that need them.

https://www.fiercebiotech.com/biotech/preventing-3-billion-visits-fda-year-cbers-peter-marks