Breast cancer patient perspectives on approval speed versus evidentiary certainty in US cancer drug approvals

A qualitative study using semistructured interviews conducted online explored patients’ views on the trade-off between faster approval and evidentiary certainty regarding the clinical benefit of new cancer drugs in the US. Participant were 30 adult female patients with breast cancer (33.3% aged 20-39 years; 40.0% aged 40-59 years; 26.7% aged ≥ 60 years; 43.3% metastatic disease; 70% on active treatment; 90% having received at least one systemic therapy). Faster approval at the expense of certainty about 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 improtance 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 generations for approval. The authors conclude that in this study, there was a mismatch between when patients considered trading evidentiary certainty for faster approval as acceptable and the conditions under which many new cancer drugs are approved by the FDA.