I have inserted excerpts and link below from an article about the FDA wanting faster confirmatory trials for drugs approved under the accelerated pathway. Of the 12 oncology accelerated approvals where confirmatory trials are late, note that Keytruda (for one indication) is one of them. There has been a good deal of discussion about Keytruda – that is why I am posting this.
The FDA’s Oncology Center of Excellence on Thursday outlined what more the agency can do to speed up confirmatory trials to ensure drugs approved under the accelerated pathway are clinically beneficial.
Before reviewing two Acrotech Biopharma therapies, FDA officials took questions from an advisory committee of cancer experts on the agency’s wider plans for reforming the accelerated approval process. Acrotech markets two drugs: Folotyn (pralatrexate) and Beleodaq (belinostat), both of which received accelerated approval, are indicated for relapsed or refractory peripheral T cell lymphoma (PTCL) and have lingered for years without completing confirmatory trials.
Gautam Mehta of the FDA’s Office of Oncologic Diseases discussed strategies to help with confirmatory trial delays while noting that the vast majority (85%) of ongoing oncology accelerated approvals are less than five years old. He didn’t explicitly call for time constraints on the trials or AAs, but he noted the FDA’s new authority from Congress allows the agency to require that confirmatory trials be underway and progress reports need to be submitted twice per year. Both changes are expected to help address delays earlier.
Under a slide titled “Confirmatory Trial Well Underway,” Mehta pointed to the positives of having the confirmatory trial “fully enrolled or near full enrollment” at the time of accelerated approval. Doing so helps minimize the risks of delays due to trial enrollment, changes in the treatment landscape and administrative delays in trial initiation or opening sites.
“A failed trial does not mean a failed drug,” FDA’s top cancer expert Richard Pazdur added. “One would also hope if the drug is effective in an earlier line of disease, the drug would be used in the earlier line of disease.”
In its briefing documents, the FDA also published a table of 12 oncology accelerated approvals in which the confirmatory trials are past their original completion date, including ones for Merck’s megablockbuster Keytruda for the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib, as well as Bristol Myers Squibb’s Opdivo for adults and those 12 years and older with microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan.
If anyone would like to see more details about this meeting, you can read the article in Targeted Oncology at this link: