PBI35 HEALTH ECONOMICS MODELLING FOR ADVANCED THERAPY MEDICINAL PRODUCTS (ATMP): A TARGETED LITERATURE REVIEW (LR)

Abstract

Characteristics of clinical trials of current ATMPs include small sample sizes, heterogeneous patient populations and uncertainty of long-term efficacy and safety effects. This LR was conducted to understand how health economists have addressed these and other issues when developing economic models for ATMPs. In April 2019, MEDLINE and EMBASE were searched for relevant articles. Original studies reporting economic assessments of gene, cell and tissue engineering therapies were analysed. Additionally, a manual search of documents from key Health Technology Assessment agencies and conferences was performed. The methods and approaches used in the economic models were assessed against a framework adapted from the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. 87 abstracts and articles fulfilled the inclusion criteria for analysis, and several reports were retrieved from the manual search. A notable variability was observed on the chosen time horizon, ranging from one year to lifetime. When long-term time horizons were modelled, various methods for extrapolation were used (not always reported), including assumptions on waning effects or parametric curve averaging to account for the uncertainty surrounding the curve choice. A substantial number of models reported the results from, and argued for, a societal perspective, highlighting the potentially meaningful reduction in indirect costs associated with ATMPs. A considerable variability exists on discounting rates applied to costs and outcomes, and scenario analyses demonstrated that these generally have a major impact on results. The LR showed that innovative payment models were rarely considered in health economic evaluations. The existing methods for the economic evaluation of health technologies seem appropriate for ATMPs. This review, however, has demonstrated that modelling assumptions and approaches adopted in the published literature vary considerably. Given the challenge of assessing the potential value of ATMPs, the scientific community would greatly benefit from a multi-stakeholder consensus on economic modelling for ATMPs.