Theralase Technologies Inc. V.TLT

AA comes with BTD ? More trials ahead for 1433 it appears ?

[Oden6570]

Yes, a confirmatory trial is required to transition an accelerated FDA approval into a full approval: 

• Purpose

  Confirmatory trials are the final step in the process of transitioning a drug from accelerated approval to full approval. They are designed to confirm that a drug's early benefits are sustained in longer-term studies. 
• Process

  If the confirmatory trial shows that the drug provides clinical benefit, the FDA grants traditional approval. If the trial does not show clinical benefit, the FDA can remove the drug from the market. 
• Accelerated approval

  Accelerated approval is based on surrogate endpoints, such as a laboratory measurement or radiographic image, that are reasonably likely to predict clinical benefit. 
• Revocation

  The FDA can revoke accelerated approval if the confirmatory trial is not conducted or if the trial shows that the drug's risks outweigh its benefits. 

A key feature of the accelerated approval pathway is that the FDA requires manufacturers to conduct postapproval studies to confirm a drug’s clinical benefit and risk profile. Accelerated approval can be revoked if the confirmatory (postapproval) trial is never done or if the trial demonstrates that the risks associated with a drug outweigh its benefits. The most widely discussed example of this occurred with bevacizumab, which was granted accelerated approval for the treatment of metastatic breast cancer in 2007 based on improvements in PFS reported in an open-label randomized clinical trial (RCT) of 722 patients.⁸ When subsequent confirmatory trials failed to demonstrate a benefit in OS but did demonstrate an increase in toxic effects (potentially deadly thromboembolic disease), the FDA revoked approval for this indication in 2011.⁸

The FDA recently published an article on the 25-year experience with the accelerated approval pathway that examined the fate of 93 oncology indications granted accelerated approval from December 11, 1992, through May 31, 2017

Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval - PMC