Health-related quality of life data collected in chimeric antigen receptor T-cell (CAR-T) therapy clinical trials. (December 2021)

• Record Type: Journal Article
• Authors: Raymakers, Adam J.N.; Regier, Dean A.; Peacock, Stuart J.; Freeman, Ciara L.
• Is Part Of: Journal of cancer policy. Volume 30(2021)
• Publication Date: 2021-12
• Subjects: Health technology assessment -- Quality of life -- Economic evaluation -- Drug policy -- Clinical trials -- Cost-effectiveness analysis -- Drug reimbursement; Cancer -- Government policy -- Periodicals; Cancer -- Patients -- Services for -- Periodicals; Medical Oncology -- Periodicals; Public Health -- Periodicals; Cancer; Periodicals; 362.196994
• DOI: 10.1016/j.jcpo.2021.100304
• Languages: English
• ISSNs: 2213-5383

Abstract

Highlights: Chimeric antigen receptor T-cell (CAR-T) therapy is a promising approach to the treatment of advanced cancers. Unlike many surrogate outcome measures collect in cancer clinical trials, health-related quality of life (HRQoL) data reflect outcomes that are important to patients. These HRQoL data are also quintessential to assessing the value of these new therapies by decision-makers in order to be provided by health systems. This research highlights the dearth of health-related quality of life data collected alongside clinical trials investigating CAR-T therapies for various cancers. Sponsors of clinical trials should be encouraged to collect these data to reduce decision-uncertainty and improve patient access to valuable therapies. Abstract: Introduction: Chimeric antigen receptor T-cell (CAR-T) therapy represents a novel approach to cancer treatment, particularly advanced cancer. Much of the current evidence for the effectiveness of these therapies is associated with considerable uncertainty. This uncertainty poses a challenge for decision-makers and health systems responsible for granting patients access to these therapies. While the stage of development of the technology is a component of this uncertainty, it can be reduced with relevant data collection alongside clinical trials that is meaningful to patients and decision-makers. The objective of this research was to investigate the frequency with which HRQoL data is collected in currently registered clinical trials investigating CAR-T cancer treatment. Methods: We searched for current CAR-T clinical trials at a registry compiled at United States National Institutes of Health National Library of Medicine (NLM) database. Trials were required to be active, recruiting, or completed. Trials were required to be phase I-IV, listed as 'interventional', and specific to cancer treatment. Results: There were 424 clinical trials that were included in our analysis. The majority of these trials (76 %) were investigating CAR-T therapy in haematological malignancies. Of the included studies, only 29 (6.8 %) included HRQoL as a primary or secondary outcome measure. Only 25 (5.9 %) trials reported collecting data on overall survival. Conclusions: This investigation into clinical trials for CAR-T therapies has shown a failure to collect valuable HRQoL data. Sponsors of clinical trials need to appreciate that clinical trials for novel therapies need to collect relevant data that is paramount to informing decision-making and providing access to patients. Policy statement: The effectiveness of innovative cancer therapies, such as CAR-T, remains associated with considerable uncertainty. This uncertainty can be reduced for decision-makers via the collection of critical HRQoL data. Sponsors of clinical trials should be incentivized to collect these data, particularly where the intention is to use that trial for a reimbursement submission and decision..