Healthy Skepticism Library item: 475

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Publication type: news

Hensley S.
Drug Firms Address Furor on Tests: New Guidance on Results Aims to Calm Concerns Over Negative Trial Data
The Wall Street Journal 2004 Jun 30

Full text:

The pharmaceutical industry is seeking to calm the storm over alleged suppression of negative clinical-trial results with new standards concerning the public release of data from human tests.

The new guidance to members of the Pharmaceutical Research and Manufacturers of America, the industry’s principal trade group, could become effective as soon as this week, when the organization is expected to post the guidance on its Web site. The group initially issued general principles in October 2002 urging drug companies to make timely public communications of meaningful study results, but didn’t offer specifics. The new guidelines attempt to make the principles more practical to implement.

Under the new plan, the group is urging drug companies to publish trial results, regardless of whether they are positive or negative, in journals, present them at scientific meetings, or post them on the Internet.

While the new standards have been in the works since late last year, they come on the heels of questions raised about unpublished studies that show negative results about antidepressant use in children and adolescents. Some doctors and journals are urging more public disclosure, including requiring drug companies to list their clinical trials in a public registry.

The guts of the current proposal, comprising eight pages of questions and answers, reiterates that companies should publish results on marketed drugs or those destined for market. The benchmark for disclosure is whether a clinical study tested a specific medical hypothesis, such as whether a drug is superior to a placebo, or dummy pill. All late-stage, or so-called Phase III, studies would fall in this category, the PhRMA guidance said. Results from these types of statistically sound studies “allow firm conclusions to be drawn to support product claims,” the guidance says. Some preliminary studies might also meet this criterion, the guidelines said, as could trials of drugs already on the market, such as comparative tests. The principles apply only to trials begun after October 1, 2002.

“We want patients and physicians to understand exactly what we’re doing in clinical trials,” said Peter Corr, who worked on the PhRMA guidance and is a senior vice president for science and technology at Pfizer Inc.

The PhRMA member companies approved them in a recent vote. “I can’t imagine a company would sign on to these policies and not do it,” Dr. Corr says.
“We’re not going to sit on something because it’s a negative trial.”

The standards represent the most specific response by the drug industry to charges that it has played down unfavorable results from human tests of drugs.

“This guidance is much more specific and affirmative on the publication of clinically meaningful study results than anything the trade association has set forth before,” said Mark Barnes, a lawyer specializing in clinical-research issues for law firm Ropes & Gray in New York.

In drawing a line between “hypothesis testing” studies and exploratory ones, the industry introduces a new concept that could lead to a public policy debate on who decides how to classify studies, Mr. Barnes said.

There are exceptions and restrictions that could limit the scope and usefulness of the guidelines. Some studies, both small and large, might be unsuitable for release because their results were found to be invalid, the guidance says. Laboratory malfunctions or unreliable samples could undermine a trial. A study might also be halted early, before a valid conclusion could be reached, after new animal test data became available, for instance.

The majority of “exploratory studies” would be excluded from public dissemination. These generally small studies, designed to gather preliminary information about a drug or disease, could constitute trade secrets because they illuminate the path a company might take in developing new product. But keeping these studies hidden might also deprive doctors of data supporting or refuting use of drugs for unapproved uses.

Dr. Corr says exploratory studies generally aren’t statistically powerful enough to form conclusions that would apply across the board. Occasionally, these pilot studies uncover information that is relevant and sound.
“Sometimes you get a dramatic effect,” he acknowledges. “We would say that should be released if it would alter the treatment of patients.”

The guidelines don’t specify how clinical results should be disseminated.
One reason, the guidance notes, is that some medical journals accept fewer than one in 10 manuscripts for publication. Internet posting of data is another possibility.

Even if trial results become public more often, patients may still have to hunt for them because no central repository for the information exists.

A registry of clinical trials might someday allow for outsiders to track which study results had gone unpublished or weren’t presented at medical meetings. In the meantime, the honor system would prevail, a prospect unlikely to appease critics of the industry for selective disclosure.