Exploring the Value and Impact of Patient-Reported Outcomes in Patient-Centric CAR-T Clinical Trials

Such remarkable advances in clinical outcomes make the evaluation of quality of life and patient-reported outcomes crucial to the complete therapeutic appraisal. As ongoing clinical trials on CAR-T cells provide additional insights into the common toxicities, rate of response, durability of remission and potential expansion into new indications of multiple hematologic and solid malignancies, it becomes vital to consider the patients’ perspective, symptomatic burden and overall quality of life impacted as a result of treatment. Despite the latest U.S. Food and Drug Administration (FDA) CAR-T approvals for use in relapsed or refractory leukemia and lymphomas, and the associated inspiring response rates, the industry as a whole has a deficit of data related to patient-reported toxicity and the impact various CAR-T constructs may have on a patient’s health-related quality of life.

An essential component of optimal patient care for those with chronic disorders, including patients with cancer and hematological diseases, is patient-centricity. In recent decades, the pharmaceutical industry and its regulators have placed increasing emphasis on placing patients at the center of healthcare research and drug development, seeking to improve their experience and ensure maximum value for the use of medicines, therapies and health services. Key aspects of patient-centric care embrace patients’ active and central role in managing their disease. Moreover, understanding the patients’ concerns, treatment impacts and their expectations of benefit from new medicines is a critical step in the development of products that can change the course of a disease and patient lives.