Defining Patient-Centered Outcomes in CAR-T-cell Therapy: Is There a Need for a Patient-Informed CAR-T PROM?
Author(s)
Lorna M. Richards, PhD1, Jane Bentley, PhD2, Puneet Kumar, Masters of Pharmacy3, Julie Prock, MPH4, Geof Gray5.
1Insights & Evidence Generation, Syneos Health, Bridgewater, NJ, USA, 2Strategy and Delivery, Syneos Health, London, United Kingdom, 3Consulting, Syneos Health, London, United Kingdom, 4Insights and Evidence Generation, Syneos Health, Bridgewater, NJ, USA, 5Ilam, Christchurch, New Zealand.
1Insights & Evidence Generation, Syneos Health, Bridgewater, NJ, USA, 2Strategy and Delivery, Syneos Health, London, United Kingdom, 3Consulting, Syneos Health, London, United Kingdom, 4Insights and Evidence Generation, Syneos Health, Bridgewater, NJ, USA, 5Ilam, Christchurch, New Zealand.
OBJECTIVES: CAR-T cell therapy offers transformative potential and presents unique burdens that generic patient-reported outcome measures (PROMs) may fail to capture. Centering patient perspectives ensures value-driven, equitable care and informs meaningful outcome measurement.
To characterize the treatment experience and health-related quality of life (HRQoL) among individuals undergoing CAR-T cell therapy and to support the development of a patient-informed, CAR-T specific PROM.
METHODS: Three sequential HIPAA-compliant, web-based surveys (30-40 minutes each) were completed by individuals who had received (Group 1) or seriously considering, (Group 2) CAR-T cell therapy. (Group 2). Survey 1 assessed the relevance of PROMIS-29 v2.0. Survey 2 explored PROMIS-29 content gaps, relevance of altered or new questions, and identified domains important to patients. Survey 3 evaluated the draft CAR-T specific PROM for clarity, feasibility, and perceived value. Data were analyzed using mixed methods, combining Likert scale responses and qualitative thematic analysis.
RESULTS: In Survey 1 (n=22), participants found PROMIS-29 generally relevant and reported limitations in capturing social functioning and physical decline. 30.8% of infused and 44.4% of considering participants reported likely difficulty completing PROMs during the first post-treatment week. Survey 2 (n=21) identified content gaps including disease-related anxiety, immunosuppression-driven isolation, exercise, ability to complete ADLs, memory, sleep, and unmet expectations around treatment outcomes. Survey 3 (n=19) showed high feasibility of the draft CAR-T specific PROM. 89.5% were able to complete the tool independently; 63.2% found it useful even without clinician input. Participants noted the value of integrating it into patient-provider communication and long-term care planning.
CONCLUSIONS: Patient feedback was essential in creating a relevant and practical CAR-T specific PROM. These results highlight the importance of patient-centered collaboration in developing tools that enhance care quality, facilitate shared decision-making, and better align clinical innovation with patient experiences.
To characterize the treatment experience and health-related quality of life (HRQoL) among individuals undergoing CAR-T cell therapy and to support the development of a patient-informed, CAR-T specific PROM.
METHODS: Three sequential HIPAA-compliant, web-based surveys (30-40 minutes each) were completed by individuals who had received (Group 1) or seriously considering, (Group 2) CAR-T cell therapy. (Group 2). Survey 1 assessed the relevance of PROMIS-29 v2.0. Survey 2 explored PROMIS-29 content gaps, relevance of altered or new questions, and identified domains important to patients. Survey 3 evaluated the draft CAR-T specific PROM for clarity, feasibility, and perceived value. Data were analyzed using mixed methods, combining Likert scale responses and qualitative thematic analysis.
RESULTS: In Survey 1 (n=22), participants found PROMIS-29 generally relevant and reported limitations in capturing social functioning and physical decline. 30.8% of infused and 44.4% of considering participants reported likely difficulty completing PROMs during the first post-treatment week. Survey 2 (n=21) identified content gaps including disease-related anxiety, immunosuppression-driven isolation, exercise, ability to complete ADLs, memory, sleep, and unmet expectations around treatment outcomes. Survey 3 (n=19) showed high feasibility of the draft CAR-T specific PROM. 89.5% were able to complete the tool independently; 63.2% found it useful even without clinician input. Participants noted the value of integrating it into patient-provider communication and long-term care planning.
CONCLUSIONS: Patient feedback was essential in creating a relevant and practical CAR-T specific PROM. These results highlight the importance of patient-centered collaboration in developing tools that enhance care quality, facilitate shared decision-making, and better align clinical innovation with patient experiences.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
MSR67
Topic
Methodological & Statistical Research, Patient-Centered Research, Study Approaches
Topic Subcategory
PRO & Related Methods
Disease
Oncology, Personalized & Precision Medicine