Access to Gene Therapies

Access to gene therapies has changed the healthcare landscape. Gene therapies are a field of study for researchers and, at the same time, a never-ending complaint for healthcare payers. For patients, they bring hope and maybe a positive change in life or, above all, even a cure. The first gene therapy approved in the European Union was alipogene tiparvovec with the brand name Glybera. Glybera was also the first gene therapy launched in Germany.

The uncertainty of the available evidence for Glybera constituted a challenge for the mandatory Health Technology Assessment (HTA) in Germany. The HTA decision made by the Federal Joint Committee (G-BA) in 2015 was crucial for the economic success of Glybera. Even Glybera’s orphan designation in the European Union, linked with legal incentives in Germany, did not help: The marketing authorisation holder consequently did not renew the authorisation in 2017.

At the GenoTher Summit, the panel discussion Capital markets and patient access: navigating opportunities and challenges in gene therapy took place on 11 June 2025.  It focused on current challenges of access to gene therapies from the perspectives of Sander van Deventer, Operating Partner at Forbion Venture Capital; Meriem Bouslouk-Marx, Specialist in HTA and Access to Innovative Medicines at MBM Future Health; Frédéric Girard, President at France Biotech; and Salah Ghabri, Senior Scientific Referent at Haute Autorité de la Santé (HAS). In addition, the session’s moderator was Alexandre Lemoalle, President at Ampleia.

During this panel, Meriem Bouslouk-Marx spoke about HTA and reimbursement of gene therapies in Germany. As a former G-BA Officer responsible for Glybera, she then analysed the current trends in access to gene therapies. Ten years after the first HTA decision on a gene therapy, access is still a hot topic.