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Thought Leaders in Europe and US Address Challenges to Making Rare Diseases a Global Public Health Priority
Date:2/28/2011

ortance of harmonization between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) to reduce the cost of clinical trials, especially for makers of therapies used to treat small rare disease populations, was another key issue analyzed by the participants. Clinical trials for rare disease therapies can cost more than 75 million euro ($100 million). As a result, Dr. Hammarstrom said some necessary clinical trials will never be done. "This means that certain types of therapies will be unavailable on both sides of the Atlantic," he said.

In addition, according to Peter Turner, Europe and the US are seldom aligned on clinical trial design. "Quite often we have to design a European trial and then a US trial because of requirements differing across the Atlantic," Mr. Turner explained. Yann Le Cam noted that EURORDIS has been working toward a solution by partnering with the EMA and the FDA on a retrospective analysis of orphan drug designations versus applications to better understand potential common approaches to new therapies. "There were many more commonalities than differences," Mr. Le Cam said. "Ninety-two percent of the decisions were the same."  

AvailabilityThere is a further complication in that the availability of treatments for rare diseases differs from country to country. According to Peter Saltonstall, if a therapy has been used on patients for period of time in Europe and the European safety and efficacy standards have been demonstrated, it is important to find a way to apply that information within the U.S. market.  "There needs to be a way for patients to get the drugs and therapies more quickly," Mr. Saltonstall said.

Yann Le Cam pointed out that globally, 80-85% of people with rare diseases are treated with off-label drugs and therapies. "We should encourage the collection of data during off-label use, both on safety and efficacy. This would be good medical practice that can lead to better
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SOURCE CSL Behring
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