A recent study reveals that 40% of fast-tracked cancer drugs approved by the FDA didn’t show clinical benefit in follow-up trials.
The FDA’s accelerated approvals program, initiated in response to urgent medical needs, has become a common pathway for cancer therapies. However, reliance on surrogate markers rather than direct clinical endpoints poses challenges in assessing true effectiveness. While expedited approvals offer hope for patients, the study underscores the importance of rigorous post-approval evaluations to ensure tangible benefits.
EXAMINING CLINICAL OUTCOMES: A CLOSER LOOK AT THE DATA
Analysis of FDA accelerated approvals for cancer drugs between 2013 and 2017 reveals concerning trends. Nearly 40% of these drugs failed to demonstrate extended patient survival or improved quality of life in subsequent trials. Moreover, delays in confirming efficacy prolong uncertainty for clinicians and patients alike, highlighting the need for streamlined processes and enhanced transparency.
“Although accelerated approval can be useful, some cancer drugs do not end up demonstrating benefit in extending patients’ lives or improving their quality of life,” epidemiologist Ian Liu and colleagues write in their published paper.
Between 2013 and 2023, 59 cancer drugs were given the provisional tick of approval through the FDA’s accelerated approvals pathway, spread across 129 indications or uses.
Among the 46 drug indications approved between 2013 and 2017, results of confirmatory trials were still outstanding for seven medicines by mid-2023. Meanwhile, 10 drugs had been withdrawn.
But 41 percent – or 19 of the 46 cancer drugs granted accelerated approval in that period – didn’t extend patients’ lives or improve their quality of life, the researchers found.
TOWARD REFORM: ADDRESSING SHORTCOMINGS AND IMPLEMENTING CHANGE
Recent legislative reforms aimed at bolstering the accelerated approval pathway signal a step in the right direction. Mandating pre-approval confirmatory trials and regular reporting of study progress offer potential solutions to existing challenges. While the impact of these reforms remains to be seen, they represent a vital effort to enhance the integrity and reliability of cancer drug evaluations.
NAVIGATING THE ROAD AHEAD
As the landscape of cancer drug approvals continues to evolve, it is essential to confront existing shortcomings and embrace meaningful reform. By prioritizing patient outcomes, fostering transparency, and maintaining rigorous evaluation standards, stakeholders can work together to ensure that accelerated approvals fulfill their promise of delivering effective treatments to those in need.
The study has been published in the Journal of the American Medical Association.
