The BIG-NABCG collaboration developed a road map to improve the design and implementation of future de-escalation trials of systemic adjuvant therapy. Incorporating insights from patients and expert statisticians in the road map, the aim is to obtain robust, practice-changing, and patient-centered results in future de-escalation trials. This opinion paper has just been published in the Journal of Clinical Oncology. The work was funded by the Breast Cancer Research Foundation (BCRF), which also supports the annual meetings between BIG and its counterpart the North American Breast Cancer Group (NABCG).
This important paper is the result of the 2019 BIG-NABCG annual meeting, during which experts focused on treatment de-escalation in clinical trials, tackling various topics including the psychological aspects confronted by patients, the statistical design of such trials, the rationale behind the reduction of loco-regional and other adjuvant chemo or endocrine therapies, and the type of biomarkers that can be used as tools to facilitate de-escalation.
A representative group of patients from Europe and North America was invited to participate in the meeting to discuss their own experiences and express their thoughts about de-escalation clinical trials.
The authors of the paper unanimously observed that, while efforts are being made to de-escalate systemic adjuvant therapy in selected cancer patients, in order to improve quality of life and benefit health economics, only a few of the de-escalation trials conducted in the past have provided clear answers. Some of these trials suffered from poor accrual or had study designs that led to conflicting results. To move research forward, it is essential to understand the lessons from these trials and listen more carefully to the patient voice, which is essential in designing new de-escalation trials.
This consensus paper presents considerations for the development of de-escalation trials with systemic adjuvant treatment including non-inferiority trial design, choice of endpoints and prioritisation of patient’s perspectives. It is a useful tool to help investigators conduct de-escalation trials with robust, practice-changing, and truly patient-centered results.