Capturing the Realities of Hematological Cancer Patients’ Experiences of Novel Immunotherapies: A Call for Alignment and Action to Improve PROM Within CAR-T and Bispecific Antibody Therapies
Author(s)
Eilidh M. Duncan1, Jhulia Salviano, MSc2, Celine Broekhuizen, MSc3, Charalampia Kyriakou, MD4, Samantha Nier, MSc5, Simone Oerlemans, PhD3, Katie Joyner, MSc2, Kate Morgan, BA, MSc2.
1Head of Patient Resarch, Myeloma Patients Europe, Brussels, Belgium, 2Myeloma Patients Europe, Brussels, Belgium, 3IKNL, Utrecht, Netherlands, 4University College London Hospitals NHS Foundation Trust, London, United Kingdom, 5Acute Leukemia Advocates Network, Bern, Switzerland.
1Head of Patient Resarch, Myeloma Patients Europe, Brussels, Belgium, 2Myeloma Patients Europe, Brussels, Belgium, 3IKNL, Utrecht, Netherlands, 4University College London Hospitals NHS Foundation Trust, London, United Kingdom, 5Acute Leukemia Advocates Network, Bern, Switzerland.
OBJECTIVES: Chimeric antigen receptor T-cell therapy (CAR-T) and T-cell engaging bispecific antibodies (BsAbs) are promising treatments for haematological cancers (i.e. lymphoma, leukaemia and myeloma) which are associated with unique toxicities and high symptom burden. This project aimed to identify and promote opportunities to optimise the implementation of patient-reported outcome measures (PROMs) for these treatments for improved alignment, consistency and interpretability of data.
METHODS: This multistakeholder project included patients, family carers and patient organisations from myeloma, lymphoma and leukaemia communities, along with researchers, industry, regulatory and payer representatives. Data was collected through two online workshops and virtual interviews. Thematic analysis was applied using a deductive-inductive approach.
RESULTS: From the 29 workshop participants and 16 interviewees we collected insights on current practice, challenges and limitations of PROM within CAR-T and BsAbs. Reported challenges included PROM selection, alignment of stakeholder expectations, variations in time point measurement. Important aspects of patients experience that impact quality of life, such as emotional burden of treatments, are currently under-captured. Recommendations to optimise PROM use include proposed time points for data capture tailored to CAR-T and BsAbs, alignment on combining PROM/additional items, inclusion of mixed methods capture of patient experiences, development of guidance to facilitate comparisons and shared learnings.
CONCLUSIONS: CAR-T and BsAbs present specific challenges for PROM and require tailored, validated tools that capture treatment-related side effects and burden sufficiently. Current practice of using existing generic and disease-specific PROM risks missing key elements of patients’ experiences. Aligning the use of PROMs, time point measurement and improving reporting requirements would optimise the use of existing PROMs to ensure the evidence base continues to be built whilst treatment-specific modules are currently in development.
METHODS: This multistakeholder project included patients, family carers and patient organisations from myeloma, lymphoma and leukaemia communities, along with researchers, industry, regulatory and payer representatives. Data was collected through two online workshops and virtual interviews. Thematic analysis was applied using a deductive-inductive approach.
RESULTS: From the 29 workshop participants and 16 interviewees we collected insights on current practice, challenges and limitations of PROM within CAR-T and BsAbs. Reported challenges included PROM selection, alignment of stakeholder expectations, variations in time point measurement. Important aspects of patients experience that impact quality of life, such as emotional burden of treatments, are currently under-captured. Recommendations to optimise PROM use include proposed time points for data capture tailored to CAR-T and BsAbs, alignment on combining PROM/additional items, inclusion of mixed methods capture of patient experiences, development of guidance to facilitate comparisons and shared learnings.
CONCLUSIONS: CAR-T and BsAbs present specific challenges for PROM and require tailored, validated tools that capture treatment-related side effects and burden sufficiently. Current practice of using existing generic and disease-specific PROM risks missing key elements of patients’ experiences. Aligning the use of PROMs, time point measurement and improving reporting requirements would optimise the use of existing PROMs to ensure the evidence base continues to be built whilst treatment-specific modules are currently in development.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR37
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology