Discipline: Pharmacology & Therapeutics

Health Technology Assessment of CD19 CAR T-Cell Therapies in the Irish Healthcare Setting

PhD Student: Niamh Carey Supervisor: Dr Laura McCullagh and Prof Michael Barry

Overview of Project: CAR T-cell therapies are innovative treatments for cancer. They are associated with high, upfront costs and a limited evidence base. This creates challenges for key stakeholders, including reimbursement decision-makers. Niamh’s research involved conducting independent Health Technology Assessments (HTAs) of the CD19 CAR T-cell therapies, tisagenlecleucel and axicabtagene ciloleucel. Evidence was attained through primary data collections, systematic literature reviews and clinical evidence synthesis, and the use of advanced decision analytic modelling approaches. This evidence informed the bespoke decision analytic models that were developed to evaluate cost effectiveness and budget impacts of these therapies. Value of information analyses were conducted to estimate the value of simultaneously eliminating all uncertainty of all uncertain parameters in the respective cost-utility models. The parameters, which contributed most to the overall decision uncertainty, were identified. The impact of performance-linked reimbursement agreements on the cost effectiveness and budget impact of these therapies was explored. Long-term impact of project: The outputs of this research can inform reimbursement decision-making of future CAR T-cell therapies globally. It demonstrates how HTA can inform decisions and can identify areas to be prioritised for future research. It illustrates the value of performance-linked reimbursement agreements in sharing financial risk between the payer and manufacturer. Such agreements have not been widely implemented in the Irish and international settings to date.

PhD Candidate Quote : “Of all the skills developed over the course of a PhD, I think soft skills such as resilience, adaptability and collaboration are the most valuable.”