Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of...

Advanced therapy medicinal products (ATMPs) are ground-breaking genetic and cell/tissue-based therapies with the potential to treat and even cure myriad health conditions. However, they have faced multiple challenges in decision making with relatively low success in achieving reimbursement. The aim of this study was to review the published preference-based research on ATMPs to identify value attributes as a starting point in a research workstream aimed at understanding the true value of these innovative therapies. A literature review was used to identify studies across MedLine, Embase, Allied and Complementary Medicine Database, Global Health, ProQuest Central, Social Sciences Premium Collection, Applied Social Sciences Index and Abstracts, Econ Lit, Web of Science and SCOPUS from inception to 3rd March 2024. Search terms for genetic and cell/tissue-based therapies were conducted separately. Studies were included if they were a peer-reviewed primary research study or systematic literature review using stated preference measures to elicit preferences for ATMPs. Thirteen studies were included with 8 pertaining to genetic therapies and 5 to cell/tissue therapies. Attributes of value were similar across the studies and ATMP categories. Attributes were grouped into the following for genetic therapies: clinical benefits, uncertainty, risk, treatment burden, and quality of life; for cell/tissue therapies clinical benefit, uncertainty, risk and treatment burden. There is limited research on the preferences for ATMPs; but these findings suggest that the value attributes of ATMPs are consistent across gene and cell/tissue therapies with clinical benefit, uncertainty, risk, treatment burden, and quality of life being important.