Challenges And Opportunities To Commercialize Orphan Drugs For Rare Diseases In The US

January 13, 2019

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.

Spotlight

Dompé

Dompé is a leading Italian biopharmaceutical company and its main focus is developing innovative treatment solutions for rare, often orphan, diseases.

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