A gold standard, made better

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Candace Stuart, Editor

Randomized controlled clinical trials remain the gold standard for providing evidence to guide clinical care, but the growing might of computational modeling is likely to make tomorrow’s trials more efficient.   

Take two unrelated findings reported this week. In today’s New England Journal of Medicine, Sam Schulman, MD, PhD, of the Thrombosis and Atherosclerosis Research Institute in Hamilton, Ontario, and colleagues reported results from the RE-MEDY and RE-SONATE trials. The first study, a noninferiority trial, compared extended use of dabigatran (Pradaxa, Boehringer Ingelheim) with warfarin in the prevention of recurrent venous thromboembolism. The second study evaluated dabigatran compared with placebo to assess if it was superior to placebo. The long-term therapy may offer a welcome alternative to difficult-to-manage warfarin.

The results generally paralleled findings for a shorter course of dabigatran treatment: It was effective, with a lower risk of bleeding than warfarin but a higher risk than placebo. But there were also some findings that may raise concerns. The direct thrombin inhibitor barely met the prespecified noninferiority margin, and the dabigatran group had a higher rate of acute coronary events compared with the warfarin group.

Also this week, Filip Van Petegem, PhD, of the Life Sciences Institute at the University of British Columbia in Vancouver, presented a 3-D animated model depicting a genetic mutation that can spark arrhythmia at the annual meeting of the American Association for the Advancement of Science in Boston. His team’s work also was published Feb. 19 in Nature Communications.

How does basic understanding of mechanisms behind disease states apply to improved care? As Van Petegem explained, this 3-D model will be helpful in designing small molecule pharmaceuticals that may serve as next-generation therapies. With the help of sophisticated models, researchers may avoid some of the trial and error in drug development and produce a more tailored product.

Will that produce a “safer” drug? Let the clinical studies decide.

Candace Stuart

Cardiovascular Business, editor