Gene Therapy For Rare Diseases: Considerations For Both Clinical And Post-Marketing Studies

There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes. The US Food and Drug Administration (FDA) has more than 700 active Investigational New Drug Applications (INDs) for gene and cell therapies and in 2017, the FDA approved two cell-based gene therapies and it is anticipated that gene therapy will become a mainstay treatment for many rare diseases.

In this webinar, Quanticate and QVigilance will examine the challenges of rare diseases studies focusing on gene therapy treatments, including:

• Appropriate study design including Bayesian frameworks
• Statistical analyses, the use of biomarkers, primary endpoint definition and proving drug efficacy right from the start
• The debate to capture as much data as possible due to limited patients, or to not over burden subjects
• Data sources such as historical, real world evidence, clinical trial data
• Pharmacovigilance considerations for the characterization and management of risks including handling and procedural complications and potential for delayed safety and efficacy issues necessitating long term follow-up
• Use of appropriate data capture and data quality oversight methods