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JOURNAL ARTICLE: LACK OF RARE-DISEASE DRUG APPROVALS

March 8, 2006

Despite the fact that more than 5,000 rare conditions currently exist in Europe, only 7 percent of drug applications for treating these conditions were approved between 2000 and 2004, according to an article published in the British Journal of Clinical Pharmacology. During that same time period, notes the article, more than 79 percent of other drug applications submitted to the European Agency for the Evaluation of Medical Products (EMEA) were approved. The journal cites a lack of incentives, financial and otherwise, as the primary source of this problem. In the U.S., by contrast, where tax incentives and research aids simplify rare-disease drug authorization procedures, more than 1,000 "orphan drug" applications were approved between 1983 and 2002.

"It's difficult to find a balance between the urgent need for drugs for patients with rare diseases and guaranteeing their quality, efficacy, safety and, where necessary, making comparisons with existing drugs," said one of the study's authors, Silvio Garattini, a professor at Milan's Mario Negri Institute for Pharmacological Research. "The lack of reliable methods for evaluating 'orphan drugs' in a small number of people probably explains the poor quality of the applications."

Between August 2000 and December 2004, the EMEA's Committee on Orphan Medical Products, which specializes in rare-disease drugs, reviewed 255 applications for such medications. Only 18 of these were approved, in contrast to the193 marketing authorization applications approved for non-rare conditions.

"We need more incentives in Europe to develop orphan drugs and to develop them cost-effectively, so as not to compromise our ability to manage other diseases," said Jeffrey Aronson, chair of the journal's editorial board and a reader in Clinical Pharmacology at Oxford University. "The tension between equity and affordability is unbearable and pulls in both directions -- those with rare diseases deserve to be treated but those with common diseases should not be expected to subsidise them."