More drugs being approved for rare diseases in kids

A Harvard School of Public Health (HSPH) health policy expert says the U.S. Food and Drug Administration (FDA) should more closely monitor the increasing number of “orphan” drugs on the market, particularly those designed to treat rare diseases in children.

Orphan drugs target rare diseases traditionally neglected by drug companies. [[Aaron Kesselheim]], research associate in HSPH’s Department of Health Policy and Management, told Reuters Health on February 28, 2012 that government regulators need to increase their oversight of the quality, safety, and effectiveness of these drugs after they are approved for patient use. Kesselheim conducted a study, published in 2011 in the Journal of American Medical Association, which found that, at least with cancer treatments, orphan drugs tend to be approved based on weaker evidence than non-orphan drugs. His research also uncovered increased rates of serious adverse events reported in premarket clinical trials of orphan cancer drugs. “These kinds of drugs could be subject to greater post-market scrutiny,” Kesselheim said.

An FDA study published February 27, 2012 online in Pediatrics found more than three dozen orphan drugs were approved for rare disorders affecting children and teens between 2000 and 2009. In 1983, Congress passed the Orphan Drug Act, which offers companies incentives to develop these drugs, for which profits tend to be slim. An estimated 25 million Americans have 6,000-plus rare diseases. A disease affecting fewer than 200,000 nationwide is considered rare.

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Department of Health Policy and Management