OBJECTIVES: Advanced therapy medical products (ATMPs) pose specific pricing, reimbursement and market access considerations. The aim of this research is to review ATMP assessments by EU HTA bodies to understand key drivers for securing adoption.

METHODS: HTA websites from France (HAS), Germany (IQWiG/G-BA), Italy (AIFA and regions), Spain (MSSSI and regions) and the UK (NICE, SMC) were searched. Targeted reviews of journal publications, policy documents and grey literature were also performed.

RESULTS: Provenge was withdrawn from the EU in May 2015, ChondroCelect in 2016 and Glybera is due to be withdrawn in October 2017. At HTA-level, in Germany, Glybera, Holoclar, Imlygic and Strimvelis all went through the AMNOG process. Glybera gained an ‘unquantifiable benefit’ outcome as submitted clinical data were not supportive of medical benefit. Reimbursement was later gained through insurers following reclassification to a tissue engineered product (TEP), putting Glybera outside of the AMNOG process. Imlygic obtained an ‘unquantifiable’ outcome as the G-BA considered the choice of comparator (GM-CSF) to be wrong. Strimvelis is still being assessed. ChondroCelect, Glybera and Holoclar were assessed by HAS; ChondroCelect was rejected because of a lack of demonstrated efficacy in clinically recognized trial outcomes. Glybera was awarded an ‘insufficient’ ASMR because of uncertainties around short to medium term safety, and the inability to re-administer after initial treatment. Holoclar achieved an ASMR IV for minor added benefit in a condition with high unmet need.

CONCLUSIONS: Comments from HAS on the lack of recognizable clinical outcomes, and reclassification of Glybera in Germany to a TEP, demonstrates that national HTA process are not yet adapted to the early clinical datasets and novel outcome measures that ATMPs utilise. These products require new approaches to assessing value, and so strong value stories that educate on ATMPs being the solution for burdensome conditions, will be key to future success.