Accelerated Approval Prices Are Unrelated to Clinical Value, Study Says

by Peter Johnson

 

The manufacturers of many drugs granted accelerated approval by the FDA do not complete timely confirmatory trials of the drugs’ efficacy, according to a recent study published in the journal Health Affairs — meaning that the pricing for many accelerated approval drugs has nothing to do with their clinical efficacy. The study’s author tells AIS Health that “the market doesn’t work very well” for drugs that have received accelerated approval, and “what it leads us to is overpaying at the beginning and underpaying, potentially, later.”    Most accelerated cancer treatments have not completed confirmatory trials In recent years, many policy experts have criticized the FDA’s confirmatory trial process, observing that many manufacturers don’t meet the deadline for confirmatory trial completion that they have arranged with the agency. At present, there is no standard process or timeline for later review of accelerated approval drugs; while some drugs do lose their accelerated approval status, some policy experts say that the FDA should withdraw more accelerated approvals than it does at present.  Indeed, the Health Affairs study found that between 1992 and 2019, the FDA granted 194 accelerated approvals for cancer indications, with 64 of those drugs relying on a surrogate endpoint that had never been used before. Of those 64 drugs, “more than 75%...used surrogate endpoints with no or a poor proven correlation with overall survival, disease burden, or other relevant metrics of benefits that are meaningful to patients.” The study also notes that many of those drugs “are priced at more than $100,000 per year before the confirmatory trial has been completed.” FDA could step up enforcement There are “two implications” of the paper’s findings, Richard Frank, Ph.D., one of the authors of the study, tells AIS Health. First, they suggest “you shouldn’t be paying full price if you don’t know if it works. So that means lower prices when you don’t know whether it works or not. But for those that work — or work well — if you want the incentive to reward reduced uncertainty and timely completion [of a confirmatory trial], then you ought to pay more when things are resolved positively, in a timely fashion.” Frank, the director of the USC-Brookings Schaeffer Initiative on Health Policy, isn’t sure how to solve this market failure without substantive reforms, but he does argue that the FDA could make progress by using its existing authority to push drugmakers to complete trials.  “Greater enforcement would solve part of the problem, which is drugs either never completing trials or competing them slowly,” he says. “If you enforce the rules and you held FDA’s feet to the fire in terms of having reasonable expectations from the get-go, then I think that would at least get things resolved quicker. In terms of the price, I’m not convinced that will do enough to fix the market to mean that the prices will be more rational.”

 

From Radar on Drug Benefits

 

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