Recent discussions around the FDA's Accelerated Approval program have been fueled by a JAMA study, which reported that less than 50% of cancer drugs granted accelerated approval demonstrated clinical benefits in confirmatory trials. Critics, including BioCentury's Steve Usdin, argue that these findings are misleading and constitute intellectual malpractice, suggesting that the methodology and endpoints used in the study, such as overall survival, may not be appropriate for evaluating the true effectiveness of these drugs. Furthermore, it was noted that the study created a false impression and that the lead author has potential conflicts of interest, being funded by the same entities that support the Institute for Clinical and Economic Review (ICER).
"In the arena of cancer... where accelerated approval is most frequently used & where FDA has had the most time to work out its procedures & think deeply through the details, a fair analysis of the pathway’s impact would conclude that it has brought important medicines to… https://t.co/BAmm4pgJeR
"These programs could be improved, but the notion implicit in much of the criticism that FDA should always require large, replicated double-blinded placebo-controlled studies with clinical endpoints is nonsense." Read below @biocentury editor's commentary (open access): https://t.co/3v3kjekksp
"For reasons described below, & that @BioCentury has previously chronicled, overall survival is often not an appropriate endpoint for confirming benefit. The paper’s authors [Kesselheim is 1 of them] presented the data in a way that obscured this important point." https://t.co/d6cvE1AlkF
"Academic opponents of accelerated approval [FDA} have cranked out another misleading paper, this time creating the false impression that about 1/2 of the cancer drugs granted accelerated approval turn out to be useless." The lead author is funded by the same ppl who fund ICER https://t.co/3v3kjekksp
Important editorial by my colleague @steveusdin1 . What the numbers really say - and don't say - about Accelerated Approval. (Spoiler alert: it's NOT correct to say half of the drugs approved this way in cancer are ineffective). Via @BioCentury https://t.co/Cn9wShHOWi
Drive-by analysis of accelerated approval is intellectual malpractice. Medical journals and news publications have incorrectly concluded that half of cancer accelerated approvals are useless. BioCentury's @steveusdin1 explains why. https://t.co/aiKjPrvr6X
The FDA's Accelerated Approval program was in the news last week due to a JAMA study that found that <50% of cancer drugs granted accelerated approval show clinical benefits in confirmatory trials. Ask OpenEvidence: How does the FDA's accelerated approval process work?