Global Regulatory Operations for Rare Diseases: Overcoming Submission and Approval Barriers

Abstract

The operations of rare diseases have challenges on submission and approval by the
regulators because there are many requirements that need to be met, costly and patients affected
are few. This paper aims to assess the contribution of global regulatory agencies towards the
streamlining of orphan drug approvals. In this paper, with the help of the analysed literature and
identified cases, it is examined that includes the aspects of pricing, risk factors, and shifts in
regulations. It shows that there is a requirement to increase the number of policies to be adaptive
and to have shorter cycling times as well as improving patient access. With these barriers, this
study intends to contribute to the understanding of how rare disease drug approval can be made
efficient across the world.