Editor's Choice

Big trials and human stories

BMJ 2002; 325 doi: https://doi.org/10.1136/bmj.325.7356.0/i (Published 20 July 2002) Cite this as: BMJ 2002;325:i

The publication of a big clinical trial may be a major medical, media, financial, and political event. Results are beamed round the world in moments. Patients may panic. Doctors start debating the implications. Share prices may rocket or tumble. Governments haven't yet fallen because of the result of a trial (making medicine less important than soccer), but they are often obliged to respond.

Last week's big trial, published in JAMA (p 113), was of hormone replacement therapy in postmenopausal women. Part of the study was stopped early because women taking continuous combined oestrogen-progestogen had an increased risk of developing breast cancer. The results show that among 10 000 women taking this treatment there will be each year—compared with women taking no treatment—eight extra cases of invasive breast cancer, seven heart attacks, eight strokes, and eight pulmonary embolisms. But there will also be six fewer bowel cancers and five fewer hip fractures. Overall mortality is not affected. So should a woman take hormone replacement therapy? Researchers can try to unravel the consequences of different treatment regimens. Doctors can offer advice. But ultimately only the woman herself can decide.

Another JAMA trial that is causing continuing controversy is the CLASS trial that compared traditional non-steroidal anti-inflammatory drugs with celecoxib, a COX 2 inhibitor (p 161). The central question is whether the COX 2 inhibitor causes fewer gastrointestinal side effects, as would be expected on theoretical grounds. The JAMA study suggested this was the case. But controversy began when it emerged that more complete information contradicted these results.

We published an editorial on 1 June that extended criticisms of the CLASS trial and how its results were “spun.” The result was a media “firestorm” in the United States and much of Europe but not, interestingly, in Britain. This seemed to happen partly because the suggestion of manipulation of data coincided with anxiety over the manipulation of financial data by Enron and Worldcom. Pharmacia, the manufacturers of celecoxib and the funders of the CLASS trial (who are being acquired by Pfizer (p 123)), were understandably upset. They respond today, but so do the authors of the editorial and others (p 161). BMJ readers—many of whom prescribe these drugs—are probably very confused by the debate over the trials, but the journal will soon be publishing other studies on this important question.

Far away from the hype and complexity of big trials are some compelling human stories. Claire McKenna reviews a play that describes an 18 year old Nigerian pregnant woman being held down and having her clitoris cut away—because it is believed that if the baby's head touches the clitoris when being born either the mother or baby will die (p 169). Rhiannon Tudor Edwards, a health economist, describes beautifully “coming out” as a blind person and getting a dog, Vikki. A blind dog costs £35 000, giving a cost per QALY of £6375—a snip.

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