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