White Paper


Challenges and Opportunities to Commercialize Orphan Drugs for Rare Diseases in the US

Orphan drugs (ODs) for rare diseases (RDs) present pharma companies with the opportunity to address a substantial unmet medical need, with approximately 7,000 RDs, and only about 5% having effective treatments. A large number of patients in the US are affected with these RDs (around 25-30 million people). The majority of RDs affect children. Also, RDs often translate into chronic and deteriorating conditions for patients, the majority being affected and starting in childhood, and often resulting in early death. Significant economic and social burdens exist for patients, caregivers, and the healthcare system to treat RDs.

This white paper explores the unique challenges of ODs for RDs by addressing the following:

a) Characteristics of RDs and ODs.

b) Commercialization Elements for ODs.


There is universal agreement about the success of the Orphan Drug Act in stimulating interest and bringing new drugs for the effective treatment of RDs. Pharma companies have responded with greater focused R&D efforts over time on bringing new ODs to the market given regulatory incentives. However, the model to launch and commercialize successfully ODs is not the same as non-ODs, requiring pharma companies to think and act differently. In many ways, the commercialization of ODs for RDs represents a special case of the industry’s shift to specialty medicines and how pharma companies must differently respond to these new challenges.

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