NCPA - National Center for Policy Analysis


May 5, 2009

As President Barack Obama's new Food and Drug Administration team takes the reins, it must decide what to do with off-label uses of FDA-approved drugs.  Right now these drugs provide immense life-saving opportunities for many sick patients, particularly those threatened by cancer.  The FDA and Congress should protect physicians' and patients' right to off-label uses -- and for the first time allow drug makers to promote off-label uses that prove beneficial, says Richard A. Epstein, a professor of law at the University of Chicago and a senior fellow with the Hoover Institution.

At present, no drug can reach the market without FDA approval, explains Epstein:

  • That approval is explicitly limited to the specific indications that have been subjected to exhaustive clinical tests, which usually take around seven years to complete, typically at the cost of hundreds of millions of dollars.
  • Yet once a drug reaches the market, the FDA has no statutory power to "limit or interfere with the authority of a health-care practitioner to prescribe" how physicians use that drug to treat their patients.

Thus drugs licensed by FDA for one purpose are deployed for a second "off label" use on which clinical trials have not been run or, if run, not completed.  These uses are neither fragmentary nor episodic.  In 2008, Mark Ratner and Trisha Gura reported in Nature that estimates for off-label use of cancer drugs run from 50 percent to 70 percent of total usage, and perhaps higher.

There is ample reason for this extensive off-label use, says Epstein:

  • Cancer patients are often in desperate straits.
  • And when existing treatments fail, patients and physicians alike can rationally conclude that they lose nothing by rolling the dice.

So what should be done, asks Epstein?  No one thinks that unapproved cancer drugs should be freely available to patients in the over-the-counter market.  Yet once a drug passes Phase I clinical trials -- which test for high toxicity -- why not make it available for general distribution through the National Comprehensive Cancer Network (NCCN), a nonprofit alliance of 21 leading cancer centers which gathers and evaluates information on off-label uses, and which has far superior access to specialized medical expertise than the FDA? 

The blunt fact is that no matter how able FDA scientists and physicians are, none of them have the years of experience dealing with particular tumors and particular drugs that specialist scientists and physicians can bring to this project, says Epstein.

Source: Richard A. Epstein, "Cancer Patients Deserve Faster Access to Life-Saving Drugs," Wall Street Journal, May 3, 2009.

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