A Review of Methodological Considerations for Economic Evaluations of Gene Therapies and Their Application in Literature

Value in Health
September 2020 Volume 23, Issue 9, p1119-1280
https://www.valueinhealthjournal.com/issue/S1098-3015(20)X0011-8

 

SYSTEMATIC LITERATURE REVIEW
A Review of Methodological Considerations for Economic Evaluations of Gene Therapies and Their Application in Literature
Renske M.T. ten Ham, Olaf H. Klungel, Hubert G.M. Leufkens, Geert W.J. Frederix
p1268–1280
Published online: August 18, 2020
Results
The first literature search yielded 13 articles discussing methodological considerations. The second search provided 12 EEs. Considerations identified were payment models, definition of perspectives, addressing uncertainty, data extrapolation, discount rates, novel value elements, and use of indirect and surrogate endpoints. All EEs scored satisfactory to good according to Consolidated Health Economic Evaluation Reporting Standards. Regarding methodological application, we found 1 methodological element (payment models) was applied in 2 base cases. Scenarios explored alternative perspectives, survival assumptions, and extrapolation methods in 10 EEs.
Conclusions
Although EE quality of reporting was considered good, their informativeness for health technology assessment and decision makers seemed limited owing to many uncertainties. We suggest accepted EE methods can broadly be applied to GTs, but few elements may need adjustment. Further research and multi-stakeholder consensus is needed to determine appropriateness and application of individual methodological considerations. For now, we recommend including scenario analyses to explore impact of methodological choices and (clinical) uncertainties. This study contributes to better understanding of perceived appropriate evaluation of GTs and informs best modeling practices.