Accelerated cancer drug approvals deliver limited survival gains at high cost

Harvard Pilgrim Health Care Institute

Early access to new cancer drugs, granted accelerated approval by the U.S. Food and Drug Administration (FDA), has provided mixed benefits for patients while costing Medicare billions of dollars, reveals new research published in the journal BMJ Medicine.

Researchers found that between 2012 and 2020, 178,000 Medicare beneficiaries received early access to cancer drugs through the FDA’s accelerated approval pathway. While the pathway aims to give patients faster access to promising treatments, fewer than half of these drugs were later shown to help people live longer.

Key Findings:

• Limited Survival Gains: Only 45% of Medicare beneficiaries who received accelerated approval drugs were treated with drugs that ultimately improved survival.
• Life-Years Added: Early access to accelerated approval drugs delivered an estimated 76,000 extra years of life for Medicare beneficiaries.
• Concentrated Benefits: Just three drugs—for melanoma and lung cancer—accounted for over two-thirds of all extra life-years gained.
• High Cost: Medicare spent over $20 billion more on early access to cancer drugs than on alternative treatments, averaging $263,000 per additional year of life. Costs ranged from $26,000 per life-year for melanoma drugs to $4.5 million per life-year for some breast cancer drugs.

Huseyin Naci, Associate Professor of Health Policy at the London School of Economics, and lead author of the study, said: "Early access to new cancer drugs through FDA’s accelerated approval pathway can save lives, but our findings show that most offered little or no survival benefit while costing Medicare substantial sums. This raises important questions about how to balance rapid access to novel treatments with solid evidence of effectiveness."

FDA’s accelerated pathway allows new drugs to enter the market based on interim measures rather than waiting for long-term results like overall survival. Pharmaceutical companies are required to run follow-up studies after initial approval to confirm the benefits, but these studies are not always completed. From 1992 to 2020, nearly half of the drugs approved through this pathway were still missing these follow-up studies.

Joseph Ross, Professor of Medicine and Public Health at Yale University and co-author of the study, said: “Treatments which have been approved for use must be followed up with trials measuring overall survival and then appropriate regulatory actions put in place. This will help direct Medicare funds towards treatments with proven clinical benefits.”

Anita Wagner, Associate Professor of Population Medicine at Harvard Medical School, and senior author of the study, added “Importantly, FDA should also clearly communicate the degree of uncertainty about drugs which have had accelerated approvals. This will help clinicians and patients be more informed as they make important health decisions.”

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Journal

BMJ Medicine

Article Title

Costs and benefits of early access to new cancer drugs through the US Food and Drug Administration’s accelerated approval pathway: retrospective observational study and economic evaluation

Article Publication Date

17-Dec-2025