艾昆纬-CAR-T细胞疗法之路

The Path to CAR T–Cell Therapy Uncovering barriers to patient access SASHA LAUKS, MSc, Principal, IQVIA Strategy ConsultingNICHOLAS GOULD, MSE, Associate Principal, IQVIA Strategy ConsultingELIZA BROSGOL, Consultant, IQVIA Strategy ConsultingVALENTINA CEGLIA, PhD, Associate Consultant, IQVIA Strategy ConsultingADAM SOHN, MBA, Vice President, IQVIA Strategy Consulting Table of contentsIntroduction1Methodology2Sample2Overview of survey outputs3Barriers experienced at a referring center4Perceived patient ineligibility4Patient hesitancy5Coordination with ATCs5ATC uncertainties5Barriers experienced at ATCs6Poor patient health6Perceived CAR T-cell therapy risks7Perceived affordability challenges7Logistics7Priority solution areas8Referring oncologists8Treaters and administrators9Patient advocacy groups10Pharmaceutical manufacturers10Conclusion11Appendix12References14About the authors16 Introduction their patient may benefit from CAR T therapy, they mustrefer the patient to an Activated / Authorized TreatmentCenter* (ATC). ATCs are equipped with the necessaryinfrastructure, have trained personnel, and have been CAR T-cell therapies generated intense anticipationahead of their 2017 launch, offering a potentiallytransformative approach to combat cancers thatexperts had previously deemed incurable.1Since initialauthorization, CAR T therapies have expanded rapidlyacross multiple hematologic malignancies, now with Despite the groundbreaking potential of CAR T-celltherapies in improving patient outcomes, access to thesetreatments remains limited. In 2024, IQVIA conductedresearch in a group of community oncologists and CAR TATC treaters to identify the underlying barriers that have In the United States, most patients begin their treatmentjourney at a practice likely located near their home. Theyare often managed by a general oncologist or hematologic respondents were classified as either high or lowreferrers based on the number of LBCL patients they hadreferred for CAR T-cell therapy in the three months priorto completing the survey. A high referrer is defined as Methodology To understand the obstacles throughout the treatmentjourney, IQVIA conducted a double-blind, LBCL-focused,and product-agnostic survey with 166 stakeholders acrossthe provider landscape between April and May of 2024. CAR T treaters and support staff were defined as highif they had managed more than 20 LBCL patients withCAR T-cell therapy in the three months prior to participatingin IQVIA’s survey. A low treater was defined as someonewho had managed at least one and fewer than 20 LBCL Sample The referrers and CAR T treaters included in the sample wereeither hematologists or oncologists. Each stakeholder hadbetween 3-30 years of experience and spent at least 50% Surveyed stakeholders cited a range of barriersacross the CAR-T treatment journey, including disease-related factors, patient-specific concerns, and logisticalchallenges that prevent eligible patients from accessingCAR T-cell therapy.6Referring oncologists often failed Overview of survey outputs IQVIA’s 2024 LBCL research identified and defined eachstage of the CAR T therapy journey, including those thatoccur first in the community setting at a referring center to refer patients because of perceived ineligibility,patient hesitancy, or the complexity of coordinating with At an ATC, there are seven sub-stages, derived fromprevious IQVIA quantitative and qualitative research,beginning with the treating physician conducting an The analysis also investigated where and why patients exitthe treatment journey. As a result of the research findings,IQVIA developed the CAR T therapy waterfall (Figure 2), CAR T-cell therapy physicians and support staffat activated/authorized treatment center (n=99) Barriers experienced there were clear differences in the criteria used byreferrers for determining eligibility that was correlatedto their amount of past experience referring for CAR Ttherapy. The surveyed physicians provided variedresponses when evaluating age, comorbidity (numberand type), and performance status thresholds for In the community setting, there exists an initial set ofchallenges that leads to almost two out of every threepotentially CAR T eligible LBCL patients failing to reach Perceived patient ineligibility Approximately 30% of the drop-off that occurs whilein the referring setting can be attributed to perceivedpatient ineligibility. Referring oncologists perceived While researchers developed the ECOG performancestatus scale to standardize criteria for measuring howcancer impacts a patient’s daily living abilities, surveyedphysicians do not apply limits consistently when usingit to determine patient eligibility for CAR T therapy.10 Coordination with ATCs Referring oncologists also reported that approximately15% of eligible LBCL patients do not proceed with CAR Ttherapy due to challenges coordinating with ATCs.6 ATC uncertainties The uncertainties perceived by the re