Estimating the costs of advanced therapy medicinal products: a systematic review

Background Advances in regenerative medicine offer the potential to cure different types of diseases and further improve health outcomes. However, access to Advanced Therapy Medicinal Products (ATMPs) is limited by their often-high costs. Economic evaluation is necessary to assess the cost-effectiveness of these products. This review aims to identify and compare methodologies used in estimating the costs of ATMPs. Methods We conducted a systematic review of studies published in PubMed, EMBASE, and Web of Science that estimated the product costs of ATMPs from inception to February 2026. Titles and abstracts were screened, using in- and exclusion criteria. Relevant publications were subjected to full text screening to assess eligibility and characteristics. Results A total of 7,045 publications were initially identified, of which 136 studies were found eligible, covering 192 cost estimates of ATMPs. Cost estimation strategies included price-based estimates (n=104), cost-based estimates (n=28), and estimates from secondary sources (n=60). Median costs were EUR 292,000 and ranged from as low as EUR 700 for some types of cell-based therapies, to as high as EUR 4,800,000. Price-based estimates are about four times higher than cost-based estimates (p<0.001). Estimated costs demonstrated an annual increase of about 12% over time (p=0.014), but this association was no longer statistically significant when ATMP subclassification and clinical indication were included as covariates. Conclusion This review provides an overview of methodologies used in publications that estimate the product costs of ATMPs. The results indicate a heterogeneity in methods and a wide range of cost estimates. The appropriate cost estimation approach varies with the context and stage of product development, with products in early development having more uncertainty compared to those already on the market.