Health Technology Assessment for Chimeric Antigen Receptor (CAR) T-cell Therapies for Cancer in Europe, US, and Canada

Author(s)

Stothard C1, Brown A2, Ralston S3, Curry A4, Cork D2, Hollier-Hann G2
1SIRIUS Market Access, Newcastle upon Tyne, NBL, UK, 2SIRIUS Market Access, Newcastle upon Tyne, UK, 3SIRIUS Market Access, London, UK, 4SIRIUS Market Access, Newcastle-Upon-Tyne, UK

OBJECTIVES: Chimeric antigen receptor (CAR) T-cell therapy involves genetically engineering T-cells to produce CARs which, when infused back into the patient, recognise and kill cancer cells expressing the antigen. CAR-T therapies are high-cost, specialised treatments that present many challenges for current HTA methodology. This study aims to identify and review HTAs for CAR-T therapies in Europe, US, and Canada to understand the key HTA challenges and opportunities for these treatments.

METHODS: Searches were conducted to identify HTAs of CAR-T therapies from NICE (England), SMC (Scotland), HAS (France), G-BA (Germany), ICER (US), and CADTH (Canada). HTA decisions and factors affecting decision-making were compared.

RESULTS: HTA organisations assessed tisagenlecleucel for relapsed/ refractory B-cell acute lymphoblastic leukaemia (B-ALL, n=6) or relapsed/ refractory diffuse large B-cell lymphoma (DLBCL, n=5) and axicabtagene ciloleucel for DLBCL (n=5). Assessments highlighted the unmet need and poor patient outcomes associated with the diseases. NICE and SMC recommended all indications via the Cancer Drugs Fund or Patient Access Scheme, respectively, allowing the acceptance of greater economic uncertainty. A key HTA challenge was uncertainty in clinical data, largely sourced from single-arm trials for all indications. This was acknowledged by HAS, which recommended all indications, but requested follow-up data for DLBCL. Likewise, the G-BA highlighted uncertain comparative efficacy and regarded the added benefit to be non-quantifiable for all indications. CADTH assessed tisgenlecleucel and, due to high cost-effectiveness ratios and data immaturity, recommended the therapy under the condition that cost-effectiveness is improved. ICER considered the therapies cost-effective at willingness-to-pay thresholds of $50,000-150,000/QALY, however there was concern regarding the short-term cost impact of axicabtagene ciloleucel.

CONCLUSIONS: A key driver across HTAs was the unmet need and patient benefit associated with CAR-T therapies. However, the uncertainty surrounding immature, non-comparative clinical data and cost-effectiveness were critiqued and positive recommendations relied on organisation-specific schemes and conditions.

Conference/Value in Health Info

2020-11, ISPOR Europe 2020, Milan, Italy

Value in Health, Volume 23, Issue S2 (December 2020)

Code

PCN256

Topic

Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes

Disease

Oncology

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