The United States Orphan Drug Act (US ODA) was passed in 1983, as way to stimulate the development of new therapies for rare diseases. According to the US ODA, orphan drugs are promising therapies intended to treat diseases affecting fewer than 200,000 people in the U.S. Products that fall into this category are eligible to receive financial incentives, including federally-funded grants and contracts for clinical trials, a 50% tax credit on trial costs, and most importantly seven years of market exclusivity starting from the date of drug approval. To date, the research done on rare diseases has led to a wealth of medical knowledge in different fields of medical research. An estimated 357 drugs have been approved, and more than 2,100 additional products are now in the pipeline. Some of the drugs developed under the US ODA framework have become blockbusters. For example Botox, which was initially approved to treat rare conditions involving involuntary muscle contractions, has a very popular cosmetic application.
Although the US ODA has been instrumental in the development of new products, a new impetus is needed to change the perception that it’s not worthwhile to develop drugs for rare diseases. That’s why the Food and Drug Administration’s Office of Orphan Products Designation, (OPPD) was recently charged with reevaluating the review process for orphan drugs in order to further expedite and simplify the approval of new products. In addition to this, the recent launch of a series of workshops, organized by the OPPD and targeted to companies developing orphan drugs, will provide practical guidance on financial incentives and application procedures.
These measures are aimed at easing the development of new products, while improving the availability of rare diseases treatments and reducing the financial risks for the manufacturer. A faster and simplified approval process will shorten the development times and provide faster access to effective treatments for to the rare disease community.
Sources: Braun MM, Farag-El-Massah S, Xu K, Coté TR. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nature Reviews, Drug Discovery 2010 June; Wapner J. Adoption Agents: Keeping Interest in Orphan Drugs Alive Scientific American Magazine June 2010
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Hi, my name is Leisa Greathouse, the mother of Samuel who died at age 2 from a rare blood disease known as Langerhans Cell Histiocytosis (LCH). I volunteer for the Histiocytosis Association (HA) and am a previous contributor to R.A.R.E. Blog. I’m submitting a comment here to ask for permission to reprint this article for the HA eNewsletter, which is scheduled to be emailed to its members on Wednesday, Oct. 20. Please email me at lmbrown357@aol.com to let me know whether or not I can submit this article in its entirety to HA for republication in their eNewsletter. Thank you.