In a recent New England Journal of Medicine Sounding Board, FDA leadership announced that one adequate and well-controlled clinical trial, combined with additional supporting evidence, will become the default basis for drug approval.
The National Breast Cancer Coalition (NBCC) believes that the public is entitled to drugs that are effective and accessible. To achieve that goal, patients deserve high-quality clinical trials that answer meaningful questions. Drug approvals should be based on strong, high-level evidence that a treatment actually benefits patients, not just that it produces a result. Trials should compare new drugs to the best current standard of care and measure outcomes that truly matter to patients such as living longer and living better.
Although the FDA has often approved drugs based on one main trial, this reported change by FDA makes that approach the standard. The agency says it will place greater emphasis on trial quality, including ensuring appropriate comparison groups and clearer outcome measures. NBCC’s concern is what does “emphasis” actually mean in practice. Emphasis alone does not change how trials are designed or conducted unless it translates into concrete requirements applied before studies begin. Pivotal trials should be required to compare new therapies against the best available current treatment, measure outcomes that matter to patients, and demonstrate a magnitude of benefit that justifies widespread adoption and cost. This expectation must also extend to ensuring that trials identify the appropriate dose for patients and reflect real-world clinical care, including access to appropriate treatments after disease progression so that study results accurately reflect whether a new treatment actually improves survival and quality of life outcomes.
From NBCC’s perspective, this is not fundamentally about one versus two trials, but about whether approvals are supported by reliable evidence of meaningful benefit before a drug becomes standard of care. If FDA moves to a one-trial default, it must ensure that confirmatory evidence does not substitute for strong trial design and clinically meaningful results. Post-market studies must be timely, enforceable, and capable of resolving remaining uncertainty.
Equally important, patients and trained patient advocates must be meaningfully involved in decisions about trial design, outcome selection, and magnitudes of benefit that are meaningful to patients before studies begin. Without these safeguards and patient advocate inputs upfront, shifting to a one-trial default standard risks reinforcing existing problems in oncology drug development, outlined in the NEJM article, rather than improving the quality of evidence that patients and clinicians rely on.
