[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