Patient Reported Outcomes (PROs) for novel immunotherapies

Project status: ongoing

MPE are leading this pan-haematology project that is working to produce recommendations on what can be done to improve our understanding of blood cancer patients’ experiences of novel immunotherapies.

Novel immunotherapies, such as chimeric antigen receptor T-cell therapy (CAR-T) and T-cell engaging bispecific antibodies, offer innovative ways to treat haematological cancers (i.e., myeloma, leukaemia, and lymphoma). While these novel immunotherapies offer promising results and the potential for disease-free survival, they are not without potentially serious side effects. Capturing the experiences of patients going through these treatments and the impact that they have on their lives is critical.

Patients’ experiences and the impact of treatments on health-related quality of life are captured through Patient Reported Outcome Measures (PROMs). PROMs collected Patient Reported Outcome (PROs) reports directly from patients to provide a systematic way of measuring patients’ views on the impact of disease and treatment on their health and well-being. This data has huge value in healthcare. It can help support shared decision-making about treatment, tailor care to patients’ needs, and identify those most in need of intervention, but can also be used to provide long-term safety and effectiveness data for treatments and inform regulatory and reimbursement approvals processes.

There are currently no PROMs specifically developed to use with haematological cancer patients receiving CAR-T and T-cell engaging bispecific antibodies. As a result, there is inconsistency in which PROMs are being used in different trials and potentially suboptimal PROMs being selected leading to a lack of certainty that patient-relevant side-effects and experiences of treatments are being captured.

This project involves literature review, virtual workshops and interviews with patients, informal care partners, clinicians, researchers, regulators, payers and industry representatives. We are grateful to our collaborators including Acute Leukemia Advocates Network (ALAN), Simone Oerlemans and Celine Broekhuizen from the Netherlands Comprehensive Cancer Organisation (IKNL), and Chara Kyriakou from the University College London Hospitals NHS Trust.