PBI23 ANALYSIS OF REIMBURSEMENT STATUS OF 8 APPROVED CELL AND GENE THERAPIES IN THE USA, 6 MAJOR EUROPEAN COUNTRIES, AUSTRALIA, AND JAPAN

Abstract

A few advanced therapies such as the cell and gene therapies (CGTs) have been approved during the last couple of years. These one shot CGTs are highly priced and lead to challenges for reimbursement. It is the objective of this research to analyse the current process and decisions of the major HTA bodies worldwide and to provide an overview of the status quo. A targeted research for CGTs was conducted (Embase, Medline, and Google Scholar) to identify the regulatory status and the associated indications. At least one market authorization for one indication of gene therapies or CGTs was considered as inclusion criteria for the research. HTA bodies from UK, France, Italy, Spain, Germany, Sweden, Australia, and Japan were used for the global assessment. For the US, ICER reports, and Blue Cross/Blue Shield was used. Four gene therapies (Voretigene neparvovecrzyl, Alipogene tiparvovec, Nusinersen, GSK-2696273) and four CGTs [CAR-T: Tisagenlecleucel, Axicabtagene ciloleucel, Talimogene laherparepvec, TBI-0301 (HSV-Tk)] were identified. The one-time dose cost of these CGTs varies from 373,000US$ (Axicabtagene ciloleucel) to 850,000US$ (Voretigene neparvovecrzyl). For most of the CGTs, the final decision on reimbursement was pending at the time of the analysis. Different models for reimbursement were used such as a money-back guarantee is given by the manufacturer for Strimvelis. However, a major concern for HTA bodies is the lack of evidence on the durability of the therapy in the long-term. Requests for the set-up of long-term registries have also been made (Strimvelis 15 years follow-up). The lack of evidence on long-term efficacy followed by the high price and impact on the budget are the main reasons for not approving reimbursement of the CGTs. Thus, new reimbursement models are necessary for financing these treatments and make them available for the patients and affordable for the health care systems.