Unintended effects of orphan product designation for rare neurological diseases

Since the introduction of the Orphan Drug Act in 1983, designed to promote development of treatments for rare diseases, at least 378 orphan drugs have been approved. Incentives include financial support, tax credits, and perhaps most importantly, extended market exclusivity. These incentives have encouraged industry interest and accelerated research on rare diseases, allowing patients with orphan diseases access to treatments. However, extended market exclusivity has been associated with unacceptably high drug costs, both for newly developed drugs and for drugs that were previously widely available. We suggest that a paradoxical effect of orphan product exclusivity can be reduced patient access to existing drugs. In addition, the costs of each new drug are arguably unsustainable for patients and for the American health care system. Of all the specialties, neurology has the third highest number of orphan product designations, and neurological diseases account for at least one‐fifth of rare diseases. Citing the use of tetrabenazine for chorea in Huntington disease, adrenocorticotropic hormone for infantile spasms, and enzyme replacement therapy with alglucosidase alpha for Pompe disease, we highlight these paradoxical effects. ANN NEUROL 2012;72:481–490     

Cluster of African trypanosomiasis in travelers to Tanzanian national parks

Game parks in Tanzania have long been considered to be at low risk for African trypanosomiasis; however, nine cases of the disease associated with these parks were recently reported. The outbreak was detected through TropNetEurop, a sentinel surveillance network of clinical sites throughout Europe.     

Screening for type 2 diabetes mellitus in adults: recommendations and rationale

This statement summarizes the current U.S. Preventive Services Task Force (USPSTF) recommendations on screening for type 2 diabetes in adults and updates the 1996 recommendations on this topic. The complete USPSTF recommendation and rationale statement on this topic, which includes a brief review of the supporting evidence, is available through the USPSTF Web site ( http://www.preventiveservices.ahrq.gov ) and the National Guideline Clearinghouse ( http://www.guideline.gov ) and in print through the Agency for Healthcare Research and Quality (AHRQ) Publications Clearinghouse (call 800-358-9295 or e-mail mailto:ahrqpubs@ahrq.gov ). The complete information on which this statement is based, including evidence tables and references, is available in the accompanying article in this issue and in the summary of the evidence and systematic evidence review on this topic on the Web sites already mentioned. The summary of the evidence is also available in print through the AHRQ Publications Clearinghouse.