[Free White Paper]

Pharmacovigilance and Risk Management Consideration for Rare Disease Gene Therapies

The development of a pharmacovigilance and risk management system to support a gene therapy for a rare genetic disease requires consideration of the specific challenges and nuances that novel therapies present throughout the lifetime of the product.

Within this White Paper we will explore:

• The extent of rare genetic diseases
• The challenges of pharmacovigilance and risk
  management for rare disease gene therapies
• US (FDA) and EU (EMA) approaches to gene therapy
  pharmacovigilance