Using Patient Experience Data to Demonstrate the Value of CAR-T Cell Therapies in Clinical Trials of Hematological Cancers

SUMMER 2022, THE EVIDENCE FORUM, WHITE PAPER

Summary

The development of cell and gene therapies, and specifically chimeric antigen receptor T (CAR-T) cell therapy, is having a revolutionary effect on cancers, including the treatment of relapsed and refractory hematological malignancies. Such treatments demonstrate value to patients in terms of clinically meaningful changes in health-related quality of life (HRQL) and are associated with large patient benefits. However, there are also many challenges with CAR-T cell treatment, such as high costs and increasing risks of developing severe adverse events (AEs) after the infusion, as well as potential long-term negative consequences. It is particularly important, therefore, to include patient input and perspectives in the CAR-T drug development process to inform risk-benefit assessments and understand patient burden and treatment acceptability.

There are several mechanisms for capturing patient input (e.g., traditional patient-reported outcomes [PRO] assessments, interviews with clinical trial participants, social media research, patient preference studies) and a range of patient experience data (PED) (e.g., symptoms of the condition and associated impacts on functioning and HRQL, which outcomes are most important to patients, and patient preferences). Such PED is increasingly valued by healthcare decision makers, including regulatory agencies and health technology assessment bodies, to make decisions about approval and access of these treatments.

In this paper, we explore:

• The importance and unique understanding that patient input and patient experience data (i.e., PROs, interview data, patient preferences and any type of data collected directly from the patient) can provide in chimeric antigen receptor T (CAR-T) cell therapy
• Opportunities and barriers in generating PED, including their importance in long-term follow-up
• The unique challenges in capturing patient perspectives for these treatments and possible solutions

Authors

Carla Dias Barbosa, MSc, Senior Research Scientist, Patient-Centered Research
Evidera, a business of PPD, a part of Thermo Fisher Scientific

Fanyang Zeng, MSPH, Research Associate, Patient-Centered Research
Evidera, a business of PPD, a part of Thermo Fisher Scientific