Abstract
Somatic gene editing (SGE) holds great promise for making genetic therapy possible for many monogenic conditions very soon. Is our current system of European market authorization and reimbursement ready for the expected tsunami of gene therapies? At a recent workshop of the Netherlands ZonMw consortium on ethical, legal, and social implications of personalized medicine, we discussed the current possibilities for bringing new gene therapies to the clinic. In Europe, it is not yet clear whether the route via the European medicines agency as an advanced therapy medicinal product is the most appropriate for evaluation of highly personalized SGE applications, although this may optimally guarantee safety and effectiveness. Compassionate use may ensure faster access than the centralized procedure but does not stimulate the commercial development of products. Prescription to named patients may only provide adequate access for single patients. Temporary authorization of use may allow access to medication half a year before formal market authorization has been granted, but may also have large budget impacts. Magistral compounding under a hospital exemption may be an attractive solution for rare, tailor-made applications at an acceptable price. To approve local experimental use of a therapy on a case-by-case basis may be fast, but does not guarantee optimal safety, effectiveness, and broad implementation. We argue that alternative routes should be considered for products developed for a market of large groups of patients versus unique personalized treatments. A balance between scientific evidence for safety and effectiveness, affordability, and fast access may demand a range of alternative solutions.
Highlights
In the past, monogenic disorders were considered incurable and few treatment options were available
For different parts of the world, market authorization and availability of treatment are governed by different legislative systems, for example, the food and drug administration (FDA) in the USA and the European medicines agency (EMA) in the European Union (EU) [4]
Gene therapies are becoming available at a rapid pace
Summary
Monogenic disorders were considered incurable and few treatment options were available. For different parts of the world, market authorization and availability of treatment are governed by different legislative systems, for example, the food and drug administration (FDA) in the USA and the European medicines agency (EMA) in the European Union (EU) [4] These systems aim to regulate treatments as products, while for genome editing it may be needed to optimize the clinical procedure, both for ex vivo and in vivo editing. A CRISPR-Cas product that is under development for sickle cell disease and beta-thalassemia (CTX001) might serve a relatively large number of patients [6] Would this CRISPR-Cas-based gene therapy become available globally in a fast and affordable manner?. In the opposite case, manufacturers are compensated for every sold product to
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