Abstract
The standardization and implementation of new endpoints are crucial for the development of novel therapeutic agents. These endpoints help streamline clinical development while maintaining high standards of care for innovative treatments, ultimately leading to improved patient outcomes. The Measurable Residual Disease Partnership and Alliance in Acute Myeloid Leukemia Clinical Treatment (MPAACT) consortium has consulted key opinion leaders (KOL), payers, and a patient advocate to discuss required actions to facilitate the use of measurable residual disease (MRD) as a surrogate endpoint for overall survival (OS) in clinical trials. Methods: Policy-oriented perspectives were captured with ten semi-structured interviews with Acute Myeloid Leukemia (AML) experts from the United States of America (USA) and Europe. Two advisory boards with eleven experts followed: the first shared perspectives; the second informed recommendations for MRD acceptance in AML. Results: Critical gaps for MRD adoption in AML include a lack of mature OS data in AML trials, inconsistent MRD definitions, and insufficient data correlating MRD with OS. Conclusions: This paper calls on healthcare professionals, payers, and patient advocates to collaborate on: (1) establishing aligned MRD guidelines and definitions in AML trials, (2) recognizing MRD as a promising surrogate endpoint, and (3) contributing to robust evidence supporting the MRD-OS correlation.
Keywords
Measurable residual disease (MRD), Acute myeloid leukemia (AML), Surrogate endpoints, Overall survival (OS), Patient access, Health technology assessment (HTA)