Authors: Ben Barron-Millar, PhD; Malcolm Boyd, MSc. Kintiga, Cambridge, United Kingdom.
Abstract
OBJECTIVES
Advances therapy medicinal products (ATMPs) present significant challenges for health technology assessment (HTA), driven by high upfront costs, immature evidence and uncertainty around long-term outcomes. England has implemented flexible appraisal pathways and managed access mechanisms, to enable access while mitigating considerable uncertainty. This study describes National Institute for Health and Care Excellence (NICE) reimbursement outcomes for ATMPs in England with a focus on reimbursed indication alignment with marketing authorisation (MA) versus optimisation, and the rationale for managed access.
METHODS
RESULTS
To date, 14 ATMP treatments have been evaluated by NICE, generating 18 reimbursement recommendations due to multiple indications per agent. In terms of HTA routing, five highly specialised technology (HST) appraisals and 13 standard technology appraisals (STAs) were reported, with 50% (n=9) being optimised for reimbursed indication versus original MA. This definition of target subpopulations allowed the HTA to highlight patient populations with the highest unmet need and most plausible cost-effectiveness.
The most common indications were oncology and/or haematology related (n=10), driven largely by CAR-T therapies for aggressive B-cell lymphomas. Of the appraised ATMP indications, routine commissioning was recommended (n=9) as well as optimised/restricted outcomes (n=9). Overall, 9 ATMP indications have entered into managed access arrangements, primarily to address uncertainty in long-term durability, immature survival data, and reliance on single-arm studies.
CONCLUSIONS


