OBJECTIVES : Highly innovative gene therapies open up new treatment options for critically ill patients, but could put different burdens on health insurance companies. To assess the potential impact on the German health system, this study quantifies the number of gene therapies in late stages of clinical development. It also provides an overview of the resulting challenges for the statutory health insurance (SHI) system and links the development pipeline with specific challenges for the existing reimbursement system.

METHODS : Long-acting gene therapies were identified through their clinical trials (from phase III) in various clinical trial registries via a systematic search. For all long-acting gene therapies, all diseases in ongoing clinical development from phase II were identified. For all resulting indications, the potential SHI population in Germany was quantified through epidemiologic estimates (incidence and prevalence) for the disease. The results are used to assess the relative importance of different types of challenges for the SHI system resulting from the introduction of long-acting gene therapies.

RESULTS : 45 long-acting gene therapies in late stages of development were identified. These are in development (phase II+) for 42 different diseases. 19 of these diseases are oncological. In terms of epidemiological dimensioning, the 42 diseases are characterized by a high level of heterogeneity. A large part of the identified diseases (17) affect between 1,000 and 10,000 insured persons in Germany (Median 2,675).

CONCLUSIONS : A significant number of long-acting gene therapies will be launched in the coming years. These therapies will provide new therapy options for critically ill patients but will also likely require further development of the German reimbursement and SHI financing system. Besides challenges for the benefit assessment and early patient access, the German allocation system of funds between sickness funds (morbiditätsorientierter Risikostrukturausgleich) is widely affected in various ways by the identified therapies.