FDA final guidance on pCR in neoadjuvant breast cancer

FDA published its final guidance on the use of pathologic complete response (pCR) as a surrogate endpoint for accelerated approval of neoadjuvant therapy for high-risk early stage breast cancer. The neoadjuvant setting, which spans the time between diagnosis and removal of the tumor, is thought to be the best opportunity to cure patients.

The guidance is in line with the agency's 2012 draft guidance, which defines pCR as "the absence of any residual invasive cancer on hematoxylin and eosin evaluation of the resected breast specimen and all sampled ipsilateral lymph nodes following completion of neoadjuvant systemic therapy." The indicated population is defined as patients with high risk for mortality and distant disease recurrence despite optimal modern local and systemic therapy, including patients with high-grade tumors lacking estrogen, progesterone and epidermal growth factor receptor 2 ( EGFR2; HER2; ErbB2; neu) receptors. ...