REGULATORY OPERATIONS IN RARE DISEASES: CHALLENGES AND STRATEGIES FOR GLOBAL SUBMISSIONS

Abstract

This research aims to “analyse the challenges associated with regulatory operations in rare diseases and propose strategies for efficient global submissions”. “Rare diseases regulatory operations” require traversing specialised pathways and regulations in efforts to facilitate the advancement and approval of treatments. Both secondary qualitative and quantitative methods were selected to analyse “regulatory operations in rare diseases.” Case study examples, including Novartis, JCR Pharmaceuticals, and Marathon Pharmaceuticals, were included, along with a statistical overview of “rare diseases.” Findings revealed thriving proportions of “rare disease,” and many countries have Orphan Drug designation, which gives such incentives to the medical companies to grow drugs for rare diseases, such as financial benefits, tax breaks, and potentially faster regulatory review.