The U.S. Food and Drug Administration
When an experimental enzyme-replacement drug for Hunter syndrome came along a decade ago, parents of children with the disorder were understandably desperate to get their kids the new medicine, called Elaprase.
Many families traveled hundreds of miles so that their children could take part in the drug’s key clinical trial. They may have expected that the trial would be an example of effective regulation hastening the timely arrival of a safe, new treatment. Instead, what these families experienced exemplified a broken and dysfunctional approach to drug trials, driven by an FDA culture poorly suited to serving the needs of the sickest patients.
The story of the Elaprase trial is important not because it stands out as an exception, but rather because it is increasingly characteristic of the FDA’s drug-review culture. That culture is the product of a poorly understood, but now well-established, attitude within the agency: an excessive desire for certainty.
This desire is primarily driven not by fear of unforeseen dangerous side eff ects caused by drugs under review, but rather by a deepening mistrust of the doctors who eventually prescribe such medicines and the companies that market them. And that mistrust, in turn, is impeding the availability of safe, effective drugs that could today be helping real patients.
Fortunately, however, this harmful culture can be readily improved — by implementing a few straightforward reforms of the FDA’s responsibilities and structure.
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