Successful market access for gene therapies 2.0

Please find here our updated whitepaper "Successful market access for gene therapies - Strategic challenges and possible solutions" (2023).

To date, the regulatory authorities (EMA and FDA) have approved seven gene therapies in Europe and the US. Although the current research landscape for gene therapy is making strides, there are still some challenges on the long and bumpy road leading to market entry. These obstacles require innovative solutions both in terms of regulatory as well as reimbursement aspects.

In 2018, SKC wrote a comprehensive white paper that examined the strategic challenges and possible solutions for a successful market access of gene therapies. This paper has now been updated by Dr. Esther Kenfack and Prof. Dr. Matthias P. Schönermark and is available in version 2.0. Please feel free to request a free copy below.

In this white paper, a comprehensive analysis of the current regulatory aspects of gene therapies in the US and Europe addresses both existing requirements as well as potential opportunities and risks in market entry. The thorough analysis of precedents provides an overview of the underlying evidence and the current reimbursement scenery for gene therapies already on the market. Since the novelty of gene therapies involves many uncertainties in market entry especially in the reimbursement of on-time administration, the white paper presents possible reimbursement models and makes recommendations for a successful market access.