Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries.

ABSTRACT:

Objective

To review existing regulations and policies utilised by countries to enable patient access to orphan drugs.

Methods

A review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country.

Results

Fifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country's legislation and regulations including national orphan drug policies, orp

SUBMITTER: Gammie T 

PROVIDER: S-EPMC4599885 | biostudies-literature | 2015

REPOSITORIES: biostudies-literature