Advanced Therapy Medicinal Products (ATMPs)

The aim of this ongoing qualitative study is to explore the experiences of patients and their family members/caregivers during their CAR-T treatment journey. This understanding will facilitate the identification of specific issues and/or patient and caregiver needs along the treatment pathway that need to be addressed.

As part of the NIHR Blood and Transplant Research Unit in Precision Cellular Therapeutics, the PRO-CAR-T Study is to develop and test the feasibility of a digital system to capture patient-reported symptom and quality of life data from patients who receive CAR-T cell therapies.  

Relevant links:

The PRO-CAR-T study

The use of electronic patient-report outcome (ePRO) systems to capture Patient Reported Outcome (PRO) data in clinical trials is increasing; however, their acceptability, utility, and impacts in clinical trials of advanced therapy medicinal products (ATMPs) are not yet well understood. The PROmics™ system is an electronic data capture system designed to collect and assess PRO data when patients receive an advanced cell or gene therapy. PROmics™ captures PRO data relating to patient-reported adverse events and side effects, global tolerability of the medicinal product, and quality of life and daily functioning, tailored for each disease cohort.The PROmics™ study is part of the Innovate UK/UKRI POLARISE trial, a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with Primary Sclerosing Cholangitis, Rheumatoid Arthritis, Lupus Nephritis and Crohn’s Disease.The aim of the PROmics study is to evaluate the feasibility and acceptability of ePRO data capture using an ePRO system within an advanced therapies trial involving patients with immune-mediated inflammatory disease.

Relevant links:

Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial - Journal of Patient-Reported Outcomes

The aim of this study was to develop an evidence-based resource to facilitate the design of patient-focused educational materials on advanced therapy medicinal products (ATMPs).