OBJECTIVES: Gene therapies are potentially transformative medicines, but they face significant barriers to timely patient access. In 2022, the Office of Health Economics and Pfizer published recommendations that aim to facilitate improved health technology assessment (HTA) of gene therapies. The recommendations specify changes to HTA methodologies and evidence-generation activities that should be prioritized to enable the full value of gene therapies to be captured in HTA. In 2023, we initiated additional research to explore the extent our recommendations are being achieved in nine European countries, Australia and Canada. We identify examples of best practice and discuss the HTA bodies achieving our recommendations to support countries looking to improve their HTA of gene therapies.

METHODS: We conducted targeted literature searches, including searches of grey literature and HTA agency documentation. We also incorporated input from experts who participated in a roundtable informing the generation of the recommendations, as well as input from local country affiliates. These inputs enabled us to evaluate the extent to which written guidelines are implemented in practice.

RESULTS: Most countries in our sample recognize the lifetime benefits in HTAs and therefore achieve this recommendation. Italy is achieving the recommendation related to the incorporation of outcomes-based payment models. Overall, most work is needed in the recommendation relating to operationalizing additional value elements. This recommendation is being achieved by England, but not by Canada or Switzerland, with the remaining eight countries assessed as only partly achieving this recommendation.

CONCLUSIONS: Although progress is being made towards adjusting HTAs for gene therapies in many countries, there is still much room for progress. Further progress will facilitate patient access and enable the potential transformational benefits to patients of gene therapies, as well as accurately realizing the benefits for health systems and society.