CAR-T Cell Therapy: The State-of-the-Art of Evidence and Barriers in a Real-World Setting
Author(s)
Kumar A1, Bajwa A2, Dowling O3, Sinha S1, Kalra P1
1Novartis Healthcare Pvt. Ltd, Hyderabad, AP, India, 2Novartis Healthcare Pvt. Ltd, Hyderabad, India, 3Novartis Ireland Ltd, Dublin, Ireland
Presentation Documents
OBJECTIVES:
Chimeric antigen receptor (CAR)-T cell therapy involves re-engineering patient’s own T lymphocytes. Although advanced genetic engineering and synthetic biology have enhanced functionality of this novel therapy, there are challenges to adoption in the real-world (RW). The objective of literature review is to ascertain barriers to CAR-T RW adoption and identify evidence gaps informing future RWE generation efforts.METHODS:
Literature review of studies on RW use of approved CAR-T therapies was conducted by keyword search on PubMed. Information on effectiveness, safety, recent developments, gaps in existing knowledge, and barriers to utilization of CAR-T therapies in the RW were extracted.RESULTS:
Majority of the studies focused on therapeutic aspect indicating a paucity of data on RW CAR-T use. Most CAR-Ts approved through abbreviated pathways cause concerns for long-term safety, usually due to neurotoxicity and cytokine release syndrome, which has been addressed through use of registries, surrogate endpoints, external control arms and tokenization. Varied patient responses and relapses have necessitated the need to monitor effectiveness. Whole genome sequencing, next generation sequencing, and liquid biopsies have provided an understanding of genomic instability and other biomarkers relative to patient response. Such techniques complemented with traditional imaging and treatment outcomes can be used to understand RW patient responses to CAR-T. Substantial challenges are posed by person-specific immunology, manufacturing, transportation, cell-banking solutions, and lack of standardization. RWE provides insights on clinical responses in subsets of patient groups and operational logistics enhancing its feasibility. Lack of data on quality of life and patient reported outcomes has inspired investigators to capture these variables. RWE through comparative effectiveness, cost-effectiveness, and drug utilization studies, has provided evidence to influence payers’ perspective bridging the gap between development and use in the RW.CONCLUSIONS:
RWE can provide insights into comparative effectiveness, safety and barriers to adoption of CAR-T cell therapy in RW.Conference/Value in Health Info
2022-11, ISPOR Europe 2022, Vienna, Austria
Value in Health, Volume 25, Issue 12S (December 2022)
Code
EPH137
Topic
Epidemiology & Public Health
Topic Subcategory
Public Health, Safety & Pharmacoepidemiology
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases, STA: Personalized & Precision Medicine