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By Jennifer Schneider, Sujay V. Kharade, and Nisha Nanaware-Kharade

Jennifer SchneiderSujay KharadeNisha Nanaware-KharadeOrphan drugs are defined as medicines that are approved to treat rare diseases, i.e., diseases affecting fewer than 200,000 individuals in the United States in any given year. Thus, considering such a small patient population, a naïve question that is often proposed especially from a cost-effectiveness point of view is: How could developing orphan drugs be profitable for bio-pharma companies? During a sunrise session at the 2014 AAPS National Biotechnology Conference, this question was addressed by two leading experts in the field: M. Ian Philips, Ph.D., D.Sc., FAHA, Norris Professor of Life Sciences and director of the Center for Rare Disease Therapies at the Keck Graduate Institute of Claremont, Calif., and Marlene E. Haffner, M.D., M.P.H., president and CEO of Haffner Associates, LLC, Rockville, Md., a firm dedicated to drug development with an exclusive emphasis on rare diseases. In addition to answering that question, opportunities and challenges associated with orphan drug development were also discussed.

Philips set the stage for the session by introducing the audience to the niche field of rare diseases and the importance of developing novel orphan drugs. He informed the audience that the Orphan Drug Act passed in 1983 by the Food and Drug Administration (FDA) accelerated the development of orphan drugs resulting in approval of over 400 drugs and biologics. He emphasized how orphan drug development, despite the seemingly small patient population, is a profitable venture and can even reach blockbuster status using Alexion Pharmaceuticals, Inc.’s Soliris (eculizumab) as an example. Soliris is the first agent in its class approved for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, which costs nearly half a million dollars for ongoing yearly treatment.

Haffner expanded further on Philips’ presentation by discussing the opportunities and challenges associated with orphan drug development. Elaborating on future opportunities, she informed the attendees that since many of these drugs are first-in-class treatments with no currently-available treatment options, there is virtually little or no competition for the market. She highlighted the importance of FDA’s orphan drug regulations that provide market-incentives for developing therapies for rare diseases such as favorable regulatory climate for approval, special tax benefits, and seven years protection from competition after approval. On the contrary, she also outlined various challenges such as designing and conducting the clinical trials with limited number of patient and placebo controls. Further, the high prices tags on these drugs often raise questions about insurance coverage, ethical and moral responsibilities, and the sustainability of charging six figures yearly for therapy per patient, especially considering the current healthcare crisis. She also briefly touched on the role of patient advocacy groups in development of orphan drugs.

Overall, the sunrise session provided the attendees a comprehensive overview of the field of orphan drug development, the challenges it presents, and the vast opportunities it offers.

Jennifer Schneider is pursuing a Ph.D. in Pharmaceutical Sciences at the University at Buffalo in Buffalo, N.Y., under the guidance of Sathy V. Balu-Iyer, Ph.D. She served as the student moderator for the Orphan Drugs: Small Markets, Big Opportunities sunrise session at the 2014 AAPS National Biotechnology Conference.
Sujay V. Kharade, M.S. (Pharm.), Ph.D., is a postdoctoral fellow at the Vanderbilt University. He is very active in AAPS and currently serves on various committees. He co-organized the 2014 National Biotechnology Conference session Orphan Drugs: Small Markets, Big Opportunities.
Nisha Nanaware-Kharade, M.S. (Pharm.), Ph.D., is a pharmacokineticist in the Clinical Pharmacology Division of Pharmaceutical Product Development (PPD) Inc., Richmond, Va. She is an active member of AAPS and was the co-organizer and moderator of the 2014 AAPS National Biotechnology Conference sunrise session: Orphan Drugs: Small Markets, Big Opportunities.