Oral Presentation Joint 2016 COSA and ANZBCTG Annual Scientific Meeting

Clinical trials in the international neo adjuvant setting (#64)

Laura Esserman 1
  1. UCLA, CA, United States

The implementation of an international platform trial for breast provides a unique opportunity to accelerate the testing of agents that have a high likelihood of success when targeted to the appropriate biomarker subset in phase 3 trials. A platform trial is an extension of clinical practice where the standard for high risk patients is neoadjuvant therapy. Neoadjuvant therapy, in a patient where systemic therapy is indicated, allows the evaluation of response to therapy, and the ability to tailor surgical and adjuvant radiation based on response.  A platform trial augments this approach by adding in standards for data collection, the seamless availability of vetted agents (from phase 2 trials) for the subsets of tumor types for which they are likely to be effective, and a more streamlined approach to generating outcomes, both short and long term. This approach, endorsed by the FDA and the EMA, will enhance global regulatory policy and increase the likelihood that agents demonstrating an improvement in pCR and metastatic free surivival will get to women who could benefit- everywhere in the world. As well, a more efficient, timely, and less costly approach can help to decrease the cost of these agents when they get to market.  The I SPY TRIALs will be used to illustrate this approach. Platform trials that seamlessly integrate into the clinical care process can bring great efficiency and integrate the processes of care and research and eliminate the time consuming duplication of data entry and management of separate studies. As well, platform trials enable a shared control arm, making this approach more cost effective. One of the most important challenges in this new approach is the alignment of credit for the many trial leaders that are critical to the success of a standing international collaborative effort.