Pathological Complete Response and Accelerated Drug Approval in Early Breast Cancer
Signé par deux responsables de la Food and Drug Administration, cet article présente les enjeux associés à la prise en compte du critère de réponse pathologique complète dans la procédure accélérée d’autorisation de mise sur le marché des traitements néoadjuvants d'un cancer du sein
New drugs for breast cancer have historically been approved first for patients with metastatic disease who have few remaining options for systemic treatment. Approval for an adjuvant indication occurs years later, after large, randomized trials with prolonged follow-up have been conducted in patients with early-stage disease. Recently, neoadjuvant trials have introduced new drugs preoperatively in patients with localized breast cancer. Such treatment aims to render locally advanced cancers operable, facilitate breast-conserving surgery, and ultimately improve survival. The rate of pathological complete response — absence of residual invasive cancer on pathological evaluation of resected breast specimens and lymph nodes after preoperative therapy — has been used as the primary end point in many neoadjuvant trials...
New England Journal of Medicine , article en libre accès, 2011