US FDA moots more rigorous trials for accelerated nod of cancer therapy (updated)

TG Therapeutics

24/03/2023 15:58

(Adds details and background)
       March 24 (Reuters) - The U.S. 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  BIIB.O  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  TGTX.O  and Eli Lilly and Co
 LLY.N  were withdrawn in recent years.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    Accelerated approval was withdrawn for use of Merck's
 MRK.N  blockbuster drug Keytruda for advanced gastric cancer
and Bristol Myers Squibb Co's  BMY.N  Opdivo as third-line
treatment of metastatic small cell lung cancer.

 (Reporting by Leroy Leo in Bengaluru; Editing by Maju Samuel
and Sriraj Kalluvila)
 ((Leroy.Dsouza@thomsonreuters.com ; Twitter: https://twitter.com/LeroyLeo7;))