Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis

With three hundred million people living with rare diseases worldwide, this latest article via Value in Health (ISPOR) provides an overview of global policies that optimize development, licensing, pricing, and reimbursement of orphan drugs.

The Orphan Drug Act, enacted by the US FDA in 1983, was the first legislation to regulate orphan drugs and offered financial and regulatory incentives to drug manufacturers. Since then, authorities worldwide have set up orphan drug legislation, including the European Union, Singapore, Japan, Australia, South Korea, and Taiwan. In recent years, increasing numbers of non-high-income countries have included orphan drug policy (ODP) development in their national health policy agenda. 

Legislation and policies related to access and regulation of orphan drugs were examined from 194 World Health Organization member countries plus six areas. Ninety-two countries/areas were identified as having an ODP. In particular, the world’s top 2 populous countries, China and India, recently adopted orphan drug policies. 

While observing the global growth of ODP establishment, the researchers conclude drug authorities should be prepared to develop or refine current policies to optimize patient access to orphan drugs. In particular, policy improvements in the areas of price regulation and market incentives to ensure affordability for payers with sufficient returns for manufacturers.