The FDA now requires companies to begin a clinical trial to gather the data needed for a conventional approval before they apply for accelerated approval. It has also begun re-evaluating previous accelerated approvals, a process that has led to high-profile withdrawals. Other safeguards give the FDA more authority to ensure that companies withdraw drugs when required to do so. “FDORA did a number of good things in terms of increasing the FDA’s oversight,” Ramachandran says. But the long-term benefits hinge on its implementation, she adds, particularly of the FDA’s ability “to play more of a role in making clear not just to manufacturers, but also to patients and clinicians, what the level of evidence is at the time of approval”.

Parikh says that the Accelerated Approval Program could be strengthened further by gathering more data during preliminary studies. For instance, investigators could track whether the treatment helps people to survive longer than is typically observed with standard clinical treatments. Comparisons of a drug in a clinical study with real-world data are imperfect, Parikh says, but could still help to guide decision-making. And, Ramachandran says, professional guidelines that more accurately reflect the FDA’s decisions could reduce potential harms. “It’s very low-hanging fruit for the guidelines to explicitly state when a drug has been through the accelerated-approval pathway.”

A spokesperson for the NCCN said that details of the evidence backing the use of a drug are not in the guidelines, but are included in other NCCN resources for clinicians.

Spreading that message internationally could also help regulatory authorities in low- and middle-income countries to weigh up the pros and cons of accelerated-approval drugs when making decisions. Herder says that the uncertainty that accompanies accelerated-approval drugs makes them “by far the riskiest drugs available”. Until that message spreads, he adds, regulators in low- and middle-income countries relying on the FDA’s accelerated-approval decisions might do “more harm than good”.