Abstract
After years of research and development, gene therapies are now becoming a commercial reality with several products approved by European regulatory authorities [...].
Highlights
After years of research and development, gene therapies are becoming a commercial reality with several products approved by European regulatory authorities
Three gene therapies have been approved by European regulatory authorities, Glybera, Imlygic and Strimvelis, allowing us to perform a first analysis of perceptions by payers
European health technology assessment bodies and public healthcare systems will apply the same criteria and the same scrutiny to the quality of data compared to small molecules or biologics, regardless of the very limited number of eligible patients often found in indications targeted by gene therapies
Summary
After years of research and development, gene therapies are becoming a commercial reality with several products approved by European regulatory authorities. Regulatory approval does not mean that these products are available to patients or are paid for by European health systems. Developers of gene therapies are facing the “fourth hurdle”: convincing health systems that they should invest into highly expensive products with the expectation that long-term patient benefits justify the cost. Three gene therapies have been approved by European regulatory authorities, Glybera, Imlygic and Strimvelis, allowing us to perform a first analysis of perceptions by payers. In the first part of this communication, we will review the outcome of early gene therapy commercialization efforts with a focus on EU5 (Germany, France, UK, Spain and Italy) (Table 1). We will derive initial conclusions and lessons for the developers of gene therapies
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