Providing Medical Information for Orphan Drugs
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Providing Medical Information for Orphan Drugs Sean Turbeville, PhD1, David A. Wells, PhD, RPh2, and Carl S. Hornfeldt, PhD, RPh3
Abstract 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they receive fewer inquiries, those received usually require sophisticated and time-consuming responses, often based on unpublished information and with greater reliance on rare disease experts.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. Keywords orphan drugs, rare disease, drug information, medical information, orphan drug manufacturer
Introduction In the United States, rare diseases are defined as disorders affecting 200,000 people.1 The European Union (EU) defines rare diseases as disorders with a prevalence of 5 per 10,000 people,2 and other countries use similar definitions.3 Although each rare disease affects a relatively small number of individuals, it is estimated that the world population is affected by more than 7000 rare diseases, believed to collectively affect 30 to 40 million people worldwide.4 For many years, there was little interest in developing drug treatments for these patients because of the small market size relative to the incurred drug development costs, the lack of patent protection for existing or natural drug entities, and product liability.1,5 To increase the availability of drug therapies for treating these rare disorders, the US Congress passed the Orphan Drug Act in 1983, which encouraged the pharmaceutical industry to develop therapies for these diseases by providing several financial incentives. Provision of the act included an expedited review process, protocol assistance, grant funding, tax credits for research expenses, and a 7-year period of marketing exclusivity.1,6,7 To promote orphan drug development, the Office of Orphan Products Development was created
within the Food and Drug Administration (FDA). Similar legislation was enacted in the EU to stimulate orphan drug development with oversight provided by the European Medicines Agency’s Committee on Orphan Medicinal Products.2 The US Orphan Drug Act has been highly successful in bringing effective therapies to previously underserved patient populations. During the 10-year period prior to pa
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