2014: A Great Year for Orphan Drugs

By Emily Largent

In America, an orphan disease or condition is defined by law as one that affects fewer than 200,000 people.  Six to seven-thousand different rare diseases have been identified, and new rare diseases are regularly described in the medical literature.  Many of these are conditions for which few or no drugs are available.  The Orphan Drug Act of 1983 sought to counterbalance market forces and spur orphan drug development by offering pharmaceutical companies a variety of incentives, including market exclusivity, tax credits, and research grants.  The act has resulted in many drugs for rare diseases coming to market and many more coming down the research pipeline. 

I have previously written about the ethics of allocating scarce resources, e.g., through insurance plans, to pay for orphan drugs.  Therefore, it is with great interest that I recently noted that 17 of the 41 novel new drugs that FDA approved in 2014 (i.e., 41%) were approved to treat rare diseases.  Examples include Impavido, which treats leishmaniasis, and Sylvant, which treats multicentric Castleman’s Disease, a disorder similar to lymphoma.  A complete list of the 2014 novel new orphan drugs can be found here.

Seventeen is the highest yearly total ever — surpassing the previous high of 13 from 2012.  (By comparison, there were 9 in 2013.)  This is wonderful news for the rare disease community.  It could also end up being good news for some individuals who aren’t affected by rare diseases, as some orphan drugs have ended up being blockbusters — for example, Botox and synthetic erythropoetin (EPO) started as orphan products.  It will be interesting to see what happens in the long-term. 

Despite the growing number of orphan drugs, there continue to be unmet needs for pharmaceuticals.  Last week’s talk by HLS Professor Peter Barton Hutt and Petrie-Flom Fellow Rachel Sachs on barriers to the development of drugs and vaccines for Ebola highlighted one such salient example.  One takeaway from their excellent talk was the need to rethink how we incentivize drug development.  Some discussion was given over to use of prizes and extending market exclusivity beyond the patent term.  It’s a conversation that needs to be continued.  What incentives would you favor?  Why?

Emily Largent

Emily Largent is an Assistant Professor of Medical Ethics and Health Policy at the Perelman School of Medicine. She also teaches at the University of Pennsylvania LawSchool. Her research examines ethical and regulatory issues arising in human subjects research and when integration of clinical research is integrated with clinical care; she has a particular focus on Alzheimer’s disease research. Emily received her PhD in Health Policy (Ethics) from Harvard and her JD from Harvard Law School. Prior to that, she received her BS in Nursing from the University of Pennsylvania School of Nursing and completed a fellowship in the Department of Bioethics at the National Institutes of Health.

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