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Gene Therapy: International Regulatory and Health Technology Assessment Activities and Reimbursement Status

Last updated: March 27, 2018
Project Number: ES0325-000
Product Line: Environmental Scans
Result type: Report

Over the last decade, important scientific advances have been made in the field of gene therapy. More recently, the US FDA approved its first two gene therapies for blood cancers, and the National Health Service in the UK announced a decision to fund a gene therapy for an inherited metabolic disorder. In Canada, new drug submissions on gene therapy are anticipated in the near future.

To better understand the current landscape, CADTH is undertaking an Environmental Scan to identify the international regulatory and health technology assessment (HTA) activities on, and reimbursement status of, gene therapy. This Environmental Scan will support the broader initiatives of CADTH’s Gene Therapy Working Group.

The key objectives of this Environmental Scan are:

  • To identify how gene therapy is defined by regulatory and HTA bodies internationally.
  • To identify any guidelines or frameworks for evaluating gene therapy from HTA bodies internationally.
  • To identify any regulatory approvals issued and reimbursement decisions made for gene therapy internationally.