Gene therapy is an innovative approach designed to address the genetic root cause of monogenic diseases. The lack of standardized evaluation methodologies for gene therapies provide challenges for medical and health technology assessment and require innovative solutions. This symposium will examine the fundamentals of gene therapy and provide an overview of its clinical application to treat rare diseases, such as spinal muscular atrophy. It will also introduce value-based assessments of gene therapies, including the current and proposed methods of evaluation, assessment of payment models, value-based principles and managing the affordability challenges that could be posed by novel treatments, such as gene therapies.

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