2014 : WHAT SCIENTIFIC IDEA IS READY FOR RETIREMENT?

Any enterprise has its limits and boundary conditions, and science is no exception. When the reach of science moves beyond these boundary conditions, when it demands respect and obedience that it hasn't earned, the results can be counter-productive. One example is Evidence-Based Medicine (EBM), which is the scientific idea that I think we should retire.

The concept behind EBM is certainly admirable: a set of best practices validated by rigorous experiments. EBM seeks to provide healthcare practitioners with treatments they can trust, treatments that have been evaluated by randomized controlled trials, preferably blinded. EBM seeks to transform medicine into a scientific discipline rather than an art form. What's not to like? We don't want to return to the days of quack fads and unverified anecdotes.

But we should only trust EBM if the science behind best practices is infallible and comprehensive, and that's certainly not the case. Medical science is not infallible. Practitioners shouldn't believe a published study just because it meets the criteria of randomized controlled trial design. Too many of these studies cannot be replicated. Sometimes the researcher got lucky and the experiments that failed to replicate the finding never got published or even submitted to a journal (the so-called publication bias). In rare cases the researcher has faked the results. Even when the results can be replicated they shouldn't automatically be believed—conditions may have been set up in a way that misses the phenomenon of interest so a negative finding doesn't necessarily rule out an effect.

And medical science is not comprehensive. Best practices often take the form of simple rules to follow, but practitioners work in complex situations. EBM relies on controlled studies that vary one thing at a time, rarely more than two or three. Many patients suffer from multiple medical problems, such as Type 2 diabetes compounded with asthma. The protocol that works with one problem may inappropriate for the others. EBM formulates best practices for general populations but practitioners treat individuals, and need to take individual differences into account. A treatment that is generally ineffective might still be useful for a sub-set of patients. Further, physicians aren't finished once they select a treatment; they often have to adapt it. They need expertise to judge whether a patient is recovering at an appropriate rate. Physicians have to monitor the effectiveness of a treatment plan and then modify or replace it if it isn't working well. A patient's condition may naturally fluctuate and physicians have to judge the treatment effects on top of this noisy baseline.

Sure, scientific investigations have done us all a great service by weeding out ineffective remedies. For example, a recent placebo-controlled study found that arthroscopic surgery provided no greater benefit than sham surgery for patients with osteoarthritic knees. But we also are grateful for all the surgical advances of the past few decades (e.g., hip and knee replacements, cataract treatments) that were achieved without randomized controlled trials and placebo conditions. Controlled experiments are therefore not necessary for progress in new types of treatments and they are not sufficient for implementing treatments with individual patients who each have unique profiles.

Worse, reliance on EBM can impede scientific progress. If hospitals and insurance companies mandate EBM, backed up by the threat of lawsuits if adverse outcomes are accompanied by any departure from best practices, physicians will become reluctant to try alternative treatment strategies that have not yet been evaluated using randomized controlled trials. Scientific advancement can become stifled if front-line physicians, who blend medical expertise with respect for research, are prevented from exploration and are discouraged from making discoveries.