The Unsung Heroes of the FDA

  • by: |
  • 10/23/2012

Good article in the Wall Street Journal about the FDA’s efforts to make not-yet-approved medicines available to patients with life threatening conditions.

If you are a subscriber to the WSJ, the article can be found here.

Kudos to the agency – and well deserved.

Last year, nearly 1,200 patients received treatment with experimental drugs through the compassionate-use program for conditions including hepatitis C, cancer and rare diseases like cystic fibrosis. That is up from about 1,000 patients in 2010, the first year the agency compiled data on the program. The FDA says it has been trying to increase participation, including by helping to set up a Web-based seminar that trains doctors how to make use of the program.

The agency allows manufacturers to make their drugs available, but can't require them to do so, and some companies are reluctant to participate before their products have received marketing approval. The FDA says it has been working to win over more companies. It revised its regulations in 2009 to effectively open the program to a greater number of small drug manufacturers. Most companies that provide drugs for the program do so free of charge, but some seek to recoup their expenses, the FDA says. Health insurers generally don't reimburse patients for the cost of the drugs.

"We get calls from family members and there's this sense these are miracle drugs," says Richard Klein, who heads the FDA's Office of Special Health Issues.

In fact, Mr. Klein says, "you can die faster" if there's an adverse reaction to a drug. And even it if does work, it might only add weeks or months to a patient's life, he says.

OSHI doesn’t get a lot of media coverage, nor do they seek it out. But the work they do is crucial to the mission of the FDA – and to the patients they help.



Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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