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H D I N S I G H T S HD Insights, Vol. 11 15 Copyright © Huntington Study Group 2015. All rights reserved. H D I N S I G H T S SASINOWSKI: I think that the FDA is being extraordinarily sensitive to the needs of individuals who suffer from rare, chronic, debilitating, life-threatening diseases such as HD. Sponsors should realize that working with the FDA is extremely beneficial. For researchers and patients, it is important to think about ways to deepen an understanding of the natural history of the disease. We need collaboration between patients and the research community to figure out ways in which everyone will measure the same things under the same conditions. Currently, there is tremendous incentive for people to come up with their own ways of doing things; every major research institution wants to have its fingerprint so that it can publish a paper that says, "This is how we do it." That doesn't help, because that means everybody is evaluating everything differently. HD INSIGHTS: When you are not helping companies develop treatments for orphan diseases, how do you spend your time? SASINOWSKI: I recently set up a program called One Wednesday, which hosts community gatherings for 30 minutes of silence every month. Looking at the suffering of patients tells me that we live in a divided world; we divide ourselves between healthy and unhealthy, between economic classes, political factions, religions and even within religions. One Wednesday is one effort to try to bring people to understand a fundamental truth, and that is that we all are one. HD INSIGHTS: Thank you for all your efforts to help us get better treatments for individuals suffering with rare conditions. 1 Sasinowski FJ. Quantum of effectiveness evidence in FDA's approval of orphan drugs: cataloging FDA's flexibility in regulating therapies for persons with rare disorders. Drug Information Journal. 2012;46(2):238-263. 2 Sasinowski FJ, Panico EB, Valentine JE. Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs: Update, July 2010 to June 2014. Therapeutic Innovation & Regulatory Science. 2015. Editor's Desk: The Orphan Drug Act, 1983-2015 Orphan diseases are rare conditions which affect less than 200,000 individuals in the United States. The NIH Office of Rare Diseases website currently lists over 7,000 orphan conditions that affect a total of 25-30 million individuals in the US. Approximately one-fifth of these conditions are neurological. 1 The 1983 Orphan Drug Act encourages pharmaceutical firms to develop new therapies for orphan diseases through several incentive programs, including tax breaks, grant funding, FDA fee waivers, and longer market exclusivity for companies. In the 32 years since the passage of the Orphan Drug Act, the FDA has issued approvals for 463 orphan indications (some compounds are approved for multiple indications), 34 of which are for neurological conditions (see Figure). 2 Only one, tetrabenazine, is for HD. Figure. The total number of FDA-approved drugs for orphan indications has continued to grow since 1983, but drugs for neurological conditions remain only a small fraction of those approved. 0 125 250 375 500 1982 1984 1986 1988 1990 1992 1994 1996 1998 2000 2002 2004 2006 2008 2010 2012 2014 FDA-approved products for non-neurological orphan conditions (includes cancer and HIV/AIDS) FDA-approved products for neurological orphan conditions Figure prepared by Ryan E. Korn, BA and Meredith A. Achey, BM. Source: U.S. Food and Drug Administration Orphan Drug Product designation database. 2015. http://www.accessdata.fda.gov/scripts/opdlisting/oopd/. Search terms: 01/01/1983-05/04/2015, "Only approved products." Accessed May 4, 2015. 1 Murphy SM, Puwanant A, Griggs RC. Unintended effects of orphan product designation for rare neurological diseases. Ann Neurol 2012;72:481-90. 2 U.S. Food and Drug Administration (FDA). Orphan Drug Product designation database. Available from accessdata.fda.gov/scripts/opdlisting/oopd/. Accessed May 4, 2015. Sasinowski, cont…