OBJECTIVES: The number of available advanced therapy medicinal products (ATMPs) has grown considerably in recent years. These treatments are likely to present promising results for a wide range of diseases, but also high prices. Robust methodologies are needed to evaluate such therapies and ensure value for money for payers and health systems. The objective of this work is to compile the methodological aspects of conducting economic evaluations of ATMPs.

Methodology: A systematic review was carried out and the following databases were consulted (11 September 2020): PubMed, Embase, Web of Science (WOS) and The Cochrane Library, complemented by exploratory search in Google Scholar. Two systematic reviews were located that served to identify further publications through the reference list. The search strategy was constructed with controlled and free terms, including the commercial names of ATMPs. Inclusion and exclusion criteria: all articles that carried out a cost analysis or economic evaluation of ATMPs were included. Those articles that evaluated the production process were excluded; the search was limited to the previous 15 years.

Results: A total of 1522 papers were assessed for eligibility, and 38 were chosen for the review. 3 papers (8%) reviewed cell therapies, 26 (68%) papers reviewed gene therapies, and 9 (24%) papers reviewed tissue-engineered products. 32 studies were Cost-Utility Analysis (CUA), 2 Cost-Effectiveness Analysis (CEA) and 4 were cost only studies. This review includes therapies which are represented in three of the four “quadrants” of the cost-effectiveness plane. In addition, some broad themes emerge as common issues in the economic evaluations of ATMPs: size and design of clinical trials, clinical evidence, long term, risk of bias, among others.

Conclusion: This work makes it possible to identify the gaps in the existing literature, reporting the main methodological approaches and offering some key recommendations for researchers and policy-maker.