What Constitutes a Valid Surrogate End Point in Cancer Clinical Trials?
Menée auprès de 950 patientes atteintes d'un cancer du sein opérable de stade 2 ou 3 et à haut risque de récidive (âge médian : 49 ans), cette étude évalue, dans le cadre d'un traitement néoadjuvant, l'association entre la réponse pathologique complète et la survie sans événement ainsi que la survie sans récidive distante
Historically, novel postneoadjuvant therapies for patients with early breast cancer have been assessed in multiyear trials. In an effort to address the unmet need of postneoadjuvant therapies in high-risk populations, the US Food and Drug Administration established guidelines for expediting the drug approval process by using pathologic complete response (pCR) as the end point. Previous randomized neoadjuvant trials have suggested that this end point may predict long-term outcome in patients with early-stage breast cancer.In this issue of JAMA Oncology, the I-SPY2 Trial Consortium investigated the association between pCR and survival end points, event-free survival (EFS) and distant recurrence–free survival (DRFS), using data from the I-SPY2 trial, which applied a bayesian adaptive trial design to assess 9 novel neoadjuvant therapeutic combinations for breast cancer. The authors concluded that there is a strong individual-level association between pCR and the survival end points; however, they did not provide the evidence needed to validate pCR as a surrogate outcome for EFS and DRFS.
JAMA Oncology , éditorial, 2019