Beyond maximum grade: using patient-generated data to inform tolerability of treatments for haematological malignancies.

Abstract

Incorporating patient-generated data into drug development is crucial for assessing the tolerability of treatments, particularly in patients with haematological malignancies, some of whom receive high-intensity, short-duration treatments and others who endure chronic therapies for months to years at a time. With increasing use of novel therapies such as oral targeted agents and immunotherapy, including chimeric antigen receptor T-cell therapy and bispecific antibodies across different haematological malignancies, new types of toxicity assessment techniques that harness patient-generated data, including patient-reported outcomes (PROs) are required to fully evaluate short-term and long-term side-effects. In this second paper in this Series, we describe progress in PRO implementation in clinical trials and outline future directions for use of patient-generated data, including PRO implementation in early-phase trials, novel PRO-based endpoints in haematology trials, and updated PRO measures that reflect treatment advances across haematological malignancies.