Gene Therapy

This disclaimer and any questions or matters of any nature arising from or relating to the content or use (or misuse) of this document will be governed by and interpreted in accordance with the laws of the Province of Ontario and the laws of Canada applicable therein, and all proceedings shall be subject to the exclusive jurisdiction of the courts of the Province of Ontario, Canada. [...] Recently approved gene therapies in the US and Europe are offering one-time treatment for severe or terminal conditions that purportedly result in a lifelong benefit.1 Transitioning these new advances from bench to bedside is often challenging, given the lack of long-term evidence related to efficacy and safety, and the costs associated with these therapies.2 Therefore, the regulatory and reimburs [...] The FDA in the US approved the first gene therapy for acute lymphoblastic leukemia (ALL) in children and young adults in August 20174 and the second gene therapy for aggressive lymphoma in adults in October 2017, granting both chimeric antigen receptor (CAR) T-cell technologies Priority Review and Breakthrough Therapy designations.5 In December 2017, the FDA also approved the first non-CAR T-cell, [...] Specifically, it is the Biologics and Genetic Therapies Directorate of Health Canada that oversees the regulation of these technologies.12 ENVIRONMENTAL SCAN Gene Therapy: International Regulatory and Health Technology Assessment (HTA) Activities and Reimbursement Status 4 The FDA in the US provides an interpretation that “gene therapies, including genetically- modified cells, that lead to a durab [...] The classification of gene therapy is challenged by the numerous differences between international regulatory frameworks and the terminology they employ.2 Objective 2: Health Technology Assessment Frameworks for Evaluating Gene Therapy Literature The Environmental Scan did not identify any guidelines specific to gene therapy or frameworks produced by HTA bodies internationally.