Patient perspectives on approval speed versus evidentiary certainty in US cancer drug approvals.

Importance Use of the FDA’s Accelerated Approval pathway assumes that patients are willing to accept uncertainty about clinical benefit in exchange for faster access to new cancer drugs. However, little is known about how patients view this trade-off or the circumstances under which they consider it acceptable. Objectives To explore patients’ views on the trade-off between faster approval and evidentiary certainty regarding the clinical benefit of new cancer drugs in the United States. Design, Setting, and Participants Semi-structured interviews were conducted online between January 27 and April 1, 2025 with patients diagnosed with breast cancer. Purposive sampling was used to achieve maximum variation in cancer stage, treatment status, age, and sociodemographic background. Main Outcomes and Measures The primary outcome was patients’ views on the trade-off between faster drug approval and evidentiary certainty. Interview transcripts were thematically analyzed to identify key patterns in understanding, experiences of uncertainty, attitudes toward waiting, treatment priorities, and views on regulatory decision-making. Results Thirty participants from 17 US states were interviewed, ranging in age from 28 to 71 years; 13 (43%) had metastatic disease, 21 (70%) were receiving active treatment, and 27 (90%) had received at least one systemic therapy. Faster approval at the expense of certainty about the clinical benefit of new cancer drugs was considered most acceptable when no alternative treatments existed or when the anticipated benefits were transformative. When clinical benefit was uncertain, participants emphasized the importance of survival and quality of life as priority treatment outcomes, the added risks of adverse effects, and the burden of intensive treatment. Many viewed broader access to clinical trials, rather than faster regulatory approval, as an effective way to address unmet needs while facilitating evidence generation for approval. Conclusion and Relevance In this qualitative study of 30 patients diagnosed with breast cancer, there was a mismatch between when patients considered trading evidentiary certainty for faster approval acceptable, and the conditions under which many new cancer drugs are approved by the FDA. To better align regulatory approvals with patient values, use of Accelerated Approval may be most appropriate for drugs that are likely to address genuine treatment gaps or offer meaningful improvements in clinical outcomes over existing alternatives.