The Durk Pearson & Sandy Shaw®
Life Extension NewsTM
Volume 16 No. 7 • August 2013

The New Economics of Orphan Diseases

Another article1 on hot research areas in the pharmaceutical industry notes that the search for orphan drugs has attracted a lot of interest because of smaller clinical trial size, shorter trial time, and commercial benefits such as fast track FDA approval, tax credits, and fee waivers. Not mentioned in the article, though, is what may prove to be the most important incentive to develop orphan drugs: after getting approval for an orphan drug, off-label uses may be the entryway to much larger markets involving common diseases. Importantly, recent court decisions have provided much support of the First Amendment rights of companies to provide truthful, non-misleading scientific information on off-label uses of drugs approved for other purposes. This is a very effective way to increase the availability of therapies for both rare and common diseases and to improve physician (and public) awareness of these therapies.

“Orphan status is granted to drugs for which the costs of developing and marketing a therapeutic are unlikely to be recovered, and in the U.S., to diseases that affect fewer than 200,000 people …” (The reason why the costs would be unlikely to be recovered is, of course, the huge developmental costs imposed by FDA’s rules and regulations for getting approval.) The article further notes that since the Orphan Drug Act of 1983 was passed, the FDA has approved 350 drugs and biologics for approximately 200 orphan diseases. These treatments are said to account for about 22% of current drug sales, with a total global value of about $50 billion, according to the article.1

We believe that these beneficial court decisions resulted from applying the First Amendment arguments that won our landmark decision in Pearson v. Shalala (U.S. Court of Appeals for the District of Columbia, 1999) (that protects truthful non-misleading speech on the health effects of dietary supplements and foods) to prescription drug marketing.


  1. Dutton. The new economics of orphan diseases. Genetic Engineering & Biotechnology News pp. 12-13 (Jan. 1, 2013).

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