From John Ioannidis in JAMA. The new guidelines from the American College of Cardiology and the American Heart Association indicate that perhaps one billion people worldwide are in cardiovascular risk groups suggesting that they should take statins.
The ACC/AHA guidelines demonstrate that even in a topic area with extensive amounts of data and published clinical trials, crucial evidence is still missing. The definitive way to test the recommendations is to subject them to randomized experimentation. The new proposed model could be compared against other models or approaches in its ability not only to predict risk accurately but also affect patient outcomes. The proposed strategy could also be compared against different strategies where treatment is recommended at different thresholds. With potentially more than 1 billion people caught in the statin dilemma, there should be hundreds of thousands of interested participants for such trials. With expanded target populations and more affordable generic prices, the cumulative global sales of statins may approach $1 trillion by 2020. Lipitor sales alone exceeded $120 billion between 1996 and 2011. Therefore, funding for trials to demonstrate the best predictive model and treatment threshold should be negligible compared with the accumulated profit from statins and the millions of lives and deaths at stake. [emphasis added]
TIE authors have written a lot about randomized clinical trials, including warning that they do not provide definitive answers for every question. But sometimes RCTs are the best way to answer a question.
Moreover, the argument that “we cannot afford the research” is absurd. If we don’t do this, it’s an institutional failure, not a fiscal necessity.
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