RT Journal Article
T1 Challenges for Economic Evaluations of Advanced Therapy Medicinal Products: A Systematic Review
A1 Olry-de-Labry-Lima, Antonio
A1 Ponce-Polo, Angela
A1 García-Mochón, Leticia
A1 Ortega-Ortega, Marta
A1 Pérez-Troncoso, Daniel
A1 Epstein, David
K1 Advanced therapies
K1 Cost-effectiveness analysis
K1 Health technology assessment
K1 Red Andaluza de Diseño y Traslación de Terapias Avanzadas
AB ObjectivesAdvanced therapy medicinal products (ATMPs) are drugs for human use for the treatment of chronic, degenerative, or life-threatening diseases that are based on genes, tissues, or cells. This article aimed to identify and critically review published economic analyses of ATMPs.MethodsA systematic review of economic analyses of ATMPs was undertaken. Study characteristics, design, sources of data, resources and unit costs, modeling and extrapolation methods, study results, and sensitivity analyses were assessed.ResultsA total of 46 economic analyses of ATMP (from 45 articles) were included; 4 were cell therapy medicinal products, 33 gene therapy medicinal products, and 9 tissue-engineered products. 30 therapies had commercial marketing approval; 39 studies were cost-utility analysis, 5 were cost-effectiveness analysis, and 2 were cost only studies. Four studies predicted that the ATMP offered a step change in the management of the condition and 10 studies estimated that the ATMP would offer a lower mean cost.ConclusionsComparison with historical controls, pooling of data, and use of techniques such as mixture cure fraction models should be used cautiously. Sensitivity analyses should be used across a plausible range of prices. Clinical studies need to be designed to align with health technology assessment requirements, including generic quality of life, and payers should aim for clarity of criteria. Regulators and national payers should aim for compatibility of registers to allow interchange of data. Given the increasing reliance on industry-funded economic analyses, careful critical review is recommended.
PB Blackwell Science
SN 1098-3015
YR 2023
FD 2023-01-06
LK https://hdl.handle.net/10668/23294
UL https://hdl.handle.net/10668/23294
LA en
NO Olry de Labry-Lima A, Ponce-Polo A, García-Mochón L, Ortega-Ortega M, Pérez-Troncoso D, Epstein D. Challenges for Economic Evaluations of Advanced Therapy Medicinal Products: A Systematic Review. Value Health. 2023 Jan;26(1):138-150
NO This work was supported by grant PID2019-105597RA100 from the Spanish Ministry of Science, Innovation and Universities.
DS RISalud
RD Apr 18, 2025