Orphan drug development: The impact of regulatory and reimbursement frameworks
Journal
Drug discovery today
Journal Volume
27
Journal Issue
6
Pages
1724
Date Issued
2022-06
Author(s)
Abstract
The enactment of orphan drug-specific legislation pioneered by the USA was subsequently followed by many regions, including the European Union (EU), Australia, Japan, and Taiwan. Here, we discuss the associated regulations established and their impacts in the aforementioned regions, which are among the first with frameworks specific for orphan drugs. Varied scopes of rare diseases or orphan drugs, diverse incentives, and heterogeneous types of reimbursement systems imply the prioritization of the agencies concerned. The numbers of designated and approved drugs reflect the impact of the regulatory and reimbursement frameworks. A comparison of the frameworks and their impact in the respective regions could provide valuable information for developing and improving related frameworks for countries worldwide.
Subjects
Orphan drugs; Rare diseases; Regulatory framework; Reimbursement
SDGs
Type
review