[PHP24] Funding of gene therapies in europe & the United States

[PHP24] Funding of gene therapies in europe & the United States

2017 Value in Health

Hanna, E. | Remuzat, C. | Jadot, G. | Auquier, P. | Toumi, M. | Volume: 20, Issue: 5, Pages: A18, biomedical technology assessment, female, France, *funding, *gene therapy, Germany, human, investment, *Italy, leasing, male, market, Medline, model, reimbursement, short course therapy, Spain, systematic review, turnover time, United Kingdom, *United States,

OBJECTIVES: Gene therapies (GT) are promising treatments able to potentially cure chronic and disabling diseases after single or short-course administration. Such products deliver long-term benefits after administration. The short-course administration associated to long-term high value lead to high upfront costs that challenge the sustainability of national health insurance systems. As an important number of gene therapies are expected to reach the market, finding a sustainable funding model for GT is needed. The aim of this study is to identify potential funding models for gene therapies in the large 5 EU countries: Germany, United Kingdom, France, Italy, and Spain as well as US. METHODS: A literature review was conducted in PubMed, congress abstracts, Health Technology Assessment bodies’ websites and grey literature. RESULTS: There is no specific path for GT pricing and reimbursement. However, several methodologies have been proposed to set GT price. Four funding models were proposed: “technology leasing reimbursement strategy”, high-cost drug mortgages, high-cost drugs reinsurance, and high-cost drug patient rebates. Some authors suggested that this may jeopardize the future health insurance resources and cannot constitute a generalizable model; they proposed discounts according to the turnover. Other authors proposed constraint optimization models for GT pricing, while others considered those models inapplicable to US as patients change health plan regularly thus disconnecting initial investment and future value. CONCLUSIONS: Current pricing models based on unit price are too one-dimensional for the future needs of the market assuming GT successful arrival to the market. Performance driven managed entry agreements are unlikely to address the short course treatment and long-term value. Many proposed models may be inadequate; they may be too costly on long term or lead to inappropriate return on investment. While GT started reaching the market, no clear research enlightens payers on optimal funding models.