Early experience with the FDAs regulatory review of novel gene therapies

Introduction Recent advances in gene therapies offer novel therapeutic options for many diseases that are otherwise resistant to pharmacologic treatment. Through gene therapy, scientists now have the opportunity to replace disease-causing genes with healthy copies, inactivate genes functioning improperly and introduce genes into the body to help fight disease.1 As of 31 December 2020, the US Food and Drug Administration (FDA) has approved five gene therapies, and more than 900 are in clinical development.2 Based on an assessment of the current pipeline, the FDA predicts it will approve 10–20 gene therapy products per year by 2025.3 In anticipation of many more approvals in the coming years, the FDA recently released final guidance documents on the clinical development and manufacturing of gene therapy products.2 To facilitate the development of these therapies, the 21st Century Cures Act introduced a new expedited development...
Source: Evidence-Based Medicine - Category: Internal Medicine Authors: Tags: EBM analysis Source Type: research