Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions
2020 Drug Discovery Today
van Overbeeke, Eline | Michelsen, Sissel | Toumi, Mondher | Stevens, Hilde | Trusheim, Mark | Huys, Isabelle | Simoens, Steven |
ATMP, gene therapy, cell therapy, marketing authorization, HTA, coverage, reimbursement,
A limited number of gene therapy medicinal products (GTMPs) have received marketing authorization (MA), of which some have been withdrawn, and even less have gained reimbursement. Many challenges that complicate GTMP market access can occur across multiple jurisdictions and decision-making contexts, but some reimbursement challenges are specific to jurisdictions. The importance of these challenges vary according to the specific therapy being developed, the country where market access is sought, and the efforts made by developers, regulators and payers to implement solutions to overcome these barriers. This review could alert developers to challenges associated with GTMP MA and how to address them.