When drug trials fail, is it unethical to discard data?

"In our study, we found that over 20,000 patients were involved in 'stalled' drug trials that were never reported. That’s a lot of people whose altruism is not being honored," says Jonathan Kimmelman. (Credit: iStockphoto)

When a drug is proven to be ineffective, unsafe, or both, the information gathered from the trial is often lost, raising questions about waste and ethics.

Because only one in 10 of the drugs that goes into human testing actually gets licensed, most of the information collected in developing new drugs is abandoned, even though the data can be critical for effective care, protecting patients, and discovering better drugs.

“We focused on drug trials in these three areas because they are a major focus for drug development,” says Jonathan Kimmelman, associate professor of biomedical ethics at McGill University.

“We expected to see that many of these trials are never published. What surprised us was the magnitude of the data withholding.”

Published in the British Medical Journal, the study shows that between 2005 and 2009, only about 37 percent of registered trials for “stalled” drug trials were actually published. For the approved drugs developed in the same period, 75 percent of the trials were published.

4 reasons to share information

The researchers cite several ways information sharing—even for drugs that stall in development—can help deliver care or develop drugs more effectively:

  • Allow drug developers to discover what didn’t work, and then adjust the compound or method of delivery so that it might work for other conditions. The drug Viagra failed initially as a drug for treatment of angina, but proved to be an effective drug for erectile dysfunction.
  • Offer information about the safety of other approved drugs. Often, trials of experimental drugs generate valuable evidence about the safety of approved drugs, especially those in the same chemical family.
  • Offer information about the limits of animal models and other experimental techniques. “When a drug works in animal models but not in patients, we have an opportunity to study why our model fell short and to improve it,” says first author Amanda Hakala.
  • Offer safety and efficacy information that might be useful in other parts of the world.  Often, drugs that are considered unsafe and ineffective in one part of the world are approved in another. “Failure to publish these trials deprives patients in those other jurisdictions of state of the art evidence of safety and efficacy,” Kimmelman says.

Many patients who take part in drug trials are given either placebos or ineffective or unsafe drugs.

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“The failure of the drug developers to share their findings runs contrary to the Declaration of Helsinki, which lays out clear ethical guidelines for medical research involving human subjects,” Kimmelman says.

“In our study, we found that over 20,000 patients were involved in ‘stalled’ drug trials that were never reported. That’s a lot of people whose altruism is not being honored.”

Many people are starting to recognize of this lost information, Hakala says.

The European Parliament recently passed legislation that requires sponsors to report on “stalled” trials. And the United States is considering policies that would encourage limited transparency about stalled drug trials.

“Policies like these are no substitute for full publication, but they go a long way to redeeming the sacrifices of the research subjects and advancing the cause of more effective healthcare and better drug development.”

The Canadian Institutes of Health Research funded the study.

Source: McGill University