RISK-BASED APPROACHES IN GLOBAL REGULATORY SUBMISSIONS FOR RARE DISEASES

Abstract

Rehabilitation of rare disease treatments is fraught as US life-cycle costs for therapies are often determined without regard to international pricing or reimbursement policies, and the patient populations for most of these products are small and sporadic. The objective of this study is to look at risk-based regulatory methodologies such as adaptive trial design, real-world evidence and post-market surveillance that reduce the time to global submission at minimum cost to the patient. The findings point out the benefits of enabling flexible approval pathways to accelerate market entry. However, challenges do include regulatory incontinency and financial risk. The recommendations call for international harmonization, improved data sharing and financial incentives to the pharmaceutical companies. All these strategies can be implemented to support improving regulatory efficiency for the benefit of all patients in rare diseases.