Surrogate end points in oncology: aligning drug development incentives and patient needs

Abstract

In a provocative News & Views article (Prasad, V. Surrogate end points in oncology: the speed–uncertainty trade-off from the patients’ perspective. Nat. Rev. Clin. Oncol. 22, 313–314 (2025))¹, Vinay Prasad reviews patient survey data indicating a willingness to wait 16 months to obtain moderate certainty of a drug’s efficacy and 22 months for high certainty. On the basis of these results, he advocates for moving away from surrogate end points, such as progression-free survival (PFS), to place greater emphasis on late-line treatments and incentivize the development of fewer anticancer drugs, in the hope that “truly transformative drugs” would still be developed and only “the most marginal drugs” would be abandoned¹. Although we share the goal of maximizing the development of transformative medicines, the approach Prasad proposes might do more harm than good.

First, point estimates from discrete choice surveys do not fully capture the limited options and highly heterogeneous preferences of real-world patients. Prasad critiques the design of the PERSEUS trial for enrolling all patients with newly diagnosed multiple myeloma instead of only the highest risk subgroup, but the thousands of patients who could benefit from the broader inclusion criteria might disagree — and that is the point. Patients deserve the freedom to choose from a set of treatment options that has not been artificially reduced as a policy preference rather than on the basis of evidence. For some patients, waiting an additional 11–22 months for a new drug to be approved would mean never accessing that treatment within their remaining lifetime.