The US health regulator on Friday proposed cancer drug developers conduct more rigorous trials to seek accelerated approval for their candidates.
The Food and Drug Administration’s move follows criticism for the accelerated approval pathway, as well as an independent federal review into it after the controversial nod for Biogen Inc’s Alzheimer’s treatment Aduhelm.
Accelerated approvals allow quicker nod for therapies that target serious and life-threatening conditions.
The FDA proposed using the gold-standard “randomized controlled trials” (RCT) where patients receive either a therapy or another alternate treatment, compared to conducting a trial testing only the drug without a comparator.
Drug developers usually depend on such “single-arm” studies to gain accelerated approval, and then conduct another trial post-nod to confirm clinical benefits.
The regulator suggested two approaches – one of conducting one RCT each before getting the accelerated nod and then to confirm the benefits, and another of holding just a single trial to seek the nod and then follow-up over the long term.
The single RCT approach will be advantageous as it can also provide a quicker verification of the benefits, the FDA said.
The agency, however, said it would provide recommendation on instances where single-arm trials would be adequate to support accelerated approval.
The proposed changes come after accelerated approvals for cancer drugs by TG Therapeutics and Eli Lilly and Co were withdrawn in recent years.
Withdrawal of accelerated approvals is rare, with 17 drugs being pulled since the pathway was introduced in 1992.
Accelerated approval was withdrawn for use of Merck’s blockbuster drug Keytruda for advanced gastric cancer and Bristol Myers Squibb Co’s Opdivo as third-line treatment of metastatic small cell lung cancer. (Reporting by Leroy Leo in Bengaluru; Editing by Maju Samuall and Sriraj)