Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions
Eline van Overbeeke, Sissel Michelsen, Mondher Toumi, Hilde Stevens, Mark Trusheim, Isabelle Huys, Steven Simoens
Abstract
• Challenges blocking market access of GTMPs are highly interrelated. • Developers should seek support and early joint interactions with regulators and payers. • Conditional marketing authorization and reimbursement mechanisms should be explored. • RWE infrastructure and requirements should be developed on an international level. • Efficient innovative pricing and payment models should be implemented. 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number of gene therapy medicinal products (GTMPs) have received marketing authorization (MA), of which some have been withdrawn, and even less have gained reimbursement.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 will 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. This review can inform gene therapy developers on challenges that can be encountered when seeking market access. Moreover, it provides an overview of trends among challenges and potential solutions.