Editorial: Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders (The need for a biomarker exploration phase in clinical trials - Phase 2m)
David Q. Beversdorf, Evdokia Anagnostou, Antonio Y. Hardan, Paul P. Wang, Craig A. Erickson, Thomas Frazier, Jeremy Veenstra‐VanderWeele
Abstract
Editorial on the Research Topic Precision medicine approaches for heterogeneous conditions such as autism spectrum disorders (The need for a biomarker exploration phase in clinical trials -Phase m) Significant progress has been made in understanding the biology of autism spectrum disorder (ASD), providing rational hypotheses for interventions to address the core symptoms.However, clinical trials of these interventions have failed to yield positive results to date.In many of these studies, a subset of participants appear to respond well, but a significant benefit is not found in the overall intent-to-treat group.Due to the etiological heterogeneity of ASD, we anticipate that this will continue to be a challenge in future clinical trials.It will be critical to identify the patients that are most likely to respond to a treatment and to target those subjects in later phase trials.We Frontiers in Psychiatry frontiersin.orgBeversdorf et al. ./fpsyt. .therefore propose the explicit inclusion of "Phase 2m" as part of the pathway of clinical drug development, specifically for the development of a biomarker profile that can be incorporated into later phase 2 and 3 clinical trials.Such a precision medicine approach has the potential to optimize the likelihood of success in future clinical trials to benefit patients.