Randomized controlled clinical trials and the Human Genome Project

First, relevant disclosures: I work half-time as editor-at-large at MedPage Today in Little Falls, N.J., and I work half-time as editor-in-chief of Cancer Commons from CollabRx in Palo Alto.

Many of you know that I was the editor at JAMA for 17 years and at Medscape for 10 years. You also know that I have been a strong advocate for evidence-based medicine (EBM) for decades and have trumpeted the large randomized controlled clinical trial as the “gold standard” for EBM.

That was in good faith and was the best that we could do for populations with diseases that we believed were reasonably homogeneous. Of course, clinical trial results are reported as medians and ranges, and there are often discordant results amongst clinical trials and thus the need for meta-analyses to work out the differences. And there are always those pesky outliers and what to do with them.

And then came the human genome project, and I thought when we understood the genes, medical science would become a lot simpler.

How wrong I was.

We now know that heterogeneity may be the rule and not the outlier; that it may make no sense to lump patients with diseases that are heterogeneous by genetic mutations and metabolic molecular pathways into groups for large clinical trials.

The great poet Alexander Pope said that “the proper study of mankind is man.” Okay, I’ll admit that is sexist language, man should be “human.” But I say that “the proper study of me is me.” And further, if I were to get a cancer, the proper study of my cancer would be my cancer, a clinical trial with an N of 1, me.

George Lundberg is a MedPage Today Editor-at-Large and former editor of the Journal of the American Medical Association.

Originally published in MedPage Today. Visit MedPageToday.com for more genetics news.

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  • Doc99

    The drive for novel gene-based therapies is running right into the drive for cost-containment. This battle will define the future of medicine for us all.

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