Providing Medical Information for Orphan Drugs

Medical information departments are responsible for providing information regarding the drugs the company manufactures and the diseases these drugs treat. The medical information departments of orphan drug manufacturers face unique challenges. Although they receive fewer inquiries, those received usually require sophisticated and time-consuming responses, often based on unpublished information and with greater reliance on rare disease experts. Worldwide distribution results in interactions with non-English-speaking individuals and requires familiarity with international legal requirements. Orphan drug medical information staff has greater visibility and is more likely to be contacted regarding adverse events and assistance for investigator-initiated clinical studies. As primary sources of information for patients, they need to be familiar with patient advocacy groups, clinical trial enrollment, and sources of unapproved medications such as expanded access programs. The ideal orphan drug medical information department is staffed with specialized health care professionals with advanced training dedicated to 1 or 2 orphan drug products.