Room for improvement in breast cancer clinical trial design: taking advantage of the preoperative setting

Abstract

Achieving approval of new oncologic drugs in breast cancer (BC) is lengthy and costly. After approval in a randomized Phase III trial in metastatic BC, an agent is then evaluated in stage I-III BC. It can take a decade for drug approval in early-stage BC, given the large sample size and long follow-up to detect improvements in disease-free or overall survival. One way to reduce this time period is conducting preoperative trials. In neoadjuvant BC trials, improvements in pathologic complete response in randomized trials of chemotherapy with and without a new agent can lead to accelerated approval. In exploratory investigational new drug trials, such as Phase 0 trials, new drugs can be evaluated for a limited time prior to traditional dose escalation trials. The US FDA has released guidelines for utilization of preoperative trials. The goal is to administer a new agent to the right subset of BC patients quicker and more effectively.