艾昆纬-CAR-T细胞疗法之路（英）

White PaperThe Path to CAR T–Cell Therapy Uncovering barriers to patient access SASHA LAUKS, MSc, Principal, IQVIA Strategy Consulting NICHOLAS GOULD, MSE, Associate Principal, IQVIA Strategy Consulting ELIZA BROSGOL, Consultant, IQVIA Strategy Consulting VALENTINA CEGLIA, PhD, Associate Consultant, IQVIA Strategy Consulting ADAM SOHN, MBA, Vice President, IQVIA Strategy ConsultingTable of contentsIntroduction 1Methodology 2Sample 2Overview of survey outputs 3Barriers experienced at a referring center 4Perceived patient ineligibility 4Patient hesitancy 5Coordination with ATCs 5ATC uncertainties 5Barriers experienced at ATCs 6Poor patient health 6Perceived CAR T-cell therapy risks 7Perceived affordability challenges 7Logistics 7Priority solution areas 8Referring oncologists 8Treaters and administrators 9Patient advocacy groups 10Pharmaceutical manufacturers 10Conclusion 11Appendix 12References 14About the authors 16 iqvia.com | 1CAR T-cell therapies generated intense anticipation ahead of their 2017 launch, offering a potentially transformative approach to combat cancers that experts had previously deemed incurable.1 Since initial authorization, CAR T therapies have expanded rapidly across multiple hematologic malignancies, now with six FDA-approved products addressing a broad range of relapsed or refractory B-cell lymphomas, leukemias, and multiple myeloma (Appendix, Table 1).2,3,4 These CAR T therapies have demonstrated superior efficacy compared to standard-of-care treatments and typically acceptable risk-benefit profiles, prompting label updates that approved CAR T usage in earlier lines of therapy, and eliminated the FDA’s Risk Evaluation and Mitigation Strategy (REMS) program.4,5In the United States, most patients begin their treatment journey at a practice likely located near their home. They are often managed by a general oncologist or hematologic oncologist at an institution without advanced CAR T-cell treatment capabilities. When these oncologists believe their patient may benefit from CAR T therapy, they must refer the patient to an Activated / Authorized Treatment Center* (ATC). ATCs are equipped with the necessary infrastructure, have trained personnel, and have been onboarded by manufacturers to administer CAR T-cell therapy. At an ATC, a new healthcare provider (treater) is added to the patient’s care team. Despite the groundbreaking potential of CAR T-cell therapies in improving patient outcomes, access to these treatments remains limited. In 2024, IQVIA conducted research in a group of community oncologists and CAR T ATC treaters to identify the underlying barriers that have hindered patients’ access to CAR T for the treatment of Large B-Cell Lymphoma (LBCL). This research uncovered a substantial disparity between the number of patients who may be eligible for the treatment and those who ultimately receive it. Addressing these obstacles is essential to bridge the divide between the transformative potential of CA