True Patient Advocates Must Be Students of Evidence-Based Medicine

Vinay Prasad, MD, MPH


December 27, 2018

There is no more important voice in healthcare than that of patients or those who speak on their behalf. Patient advocates play a crucial role in demanding better drugs, devices, surgeries, and treatments, and for therapies proven to work, advocates demand greater access and lower prices. Advocates ideally give voice to patient preferences and may play a prominent role in many national meetings. For instance, at the Drug Advisory Committee meetings of the US Food and Drug Administration, advocates and patients frequently champion drugs under consideration.[1]

Many medical practices have been extolled, advocated, and propagated for decades in the absence of rigorous evidence.

However, for advocates to do what is truly in the best interest of patients, it is imperative that they become students of evidence-based medicine. What does this mean? A student of evidence-based medicine has an important skill set that allows him or her to separate effective therapies from those that are merely hyped. Without this knowledge, advocates may inadvertently use their enthusiasm and energy to demand ineffective products, encourage the pursuit of marginal—rather than transformational—drugs, and recommend debunked screening tests that do more harm than good.

What attributes does evidence-based medicine provide?

First, a student of evidence-based medicine does not become bogged down in bioplausibility. Often, we see patient advocates overly enthusiastic about a drug with a novel mechanism of action, a compelling basic-science story, or seductive preclinical results (ie, those in mice or a petri dish). Students of evidence-based medicine understand that no matter how promising such data are, the chances that any such drug will meaningfully improve outcomes is very, very low. For that reason, a student of evidence-based medicine would not raise or lower the bar based on how well a drug's mechanism has been marketed.

Second, a student of evidence-based medicine knows that just because a clinical trial is positive does not mean that it is a good clinical trial. One must consider whether the patients selected are representative of patients in the real world. Was the comparator medication the appropriate therapy or a straw man? A student of evidence-based medicine understands that there may be biases in trials, even catastrophic ones—for instance, the use of unequal drug run-in periods with massive dropouts. A student of evidence-based medicine knows that for interventions that affect subjective endpoints (those that patients report) or bias-susceptible endpoints (those that rely upon a doctor's discretion), we need blinding. We know that the knowledge of whether a therapy was actually received and enthusiasm for that therapy can lead to a massive placebo effect, and that only blinding can tease this out.

Why do students of evidence-based medicine feel this way? It's because, as the old quote goes, "Those who cannot remember the past are condemned to repeat it." But we remember. In work my colleagues and I have done on medical reversals, we have found that many medical practices have been extolled, advocated, and propagated for decades in the absence of rigorous evidence that they improve patient outcomes. When, at last, brave investigators test these practices against prior or lesser standards of care, they often find these practices provide no net benefit, and in some cases increase net harm.

Occasionally, we have seen patient advocates lobby against these robust randomized clinical trials, believing that the answer is obvious: The new therapy must be better, so why test it? For new medications, we see advocates ask, "Why would any patient want to receive a placebo if a drug sounds promising?" Thomas Chalmers gave the answer in 1968 when he said, "One has only to review the graveyard of discarded therapies to discover how many patients might have benefited from being randomly assigned to a control group."[2]

There is nothing more painful than watching someone with good intentions place a self-inflicted wound.

Without the skills of evidence-based medicine, advocates may become unwitting pawns in a larger commercial agenda. They may inadvertently lobby to approve drugs that do not actually help patients. They may mistakenly accept inadequate or flawed trials. They may promote indication drift and the use of therapies in patients where the harms outweigh the benefits. They may recommend screening campaigns that lead to massive overdiagnosis or where the harms outweigh the benefits. Without the skills of evidence-based medicine, they may not even recognize this. In my mind, there is nothing more painful than watching someone with good intentions place a self-inflicted wound.

How can we make advocates students of evidence-based medicine? Advocates already have the energy and passion to educate themselves, and need only a direction. There is no better starting point than JAMA's Users' Guides to the Medical Literature: A Manual for Evidence-Based Clinical Practice.[3] Books like Overdiagnosed: Making People Sick in the Pursuit of Health[4] and Bad Pharma: How Medicine is Broken, and How We Can Fix it[5] are useful starting points. Once the flame of evidence-based medicine is lit, it is hard to extinguish and will propagate itself.

Patient advocates bring enthusiasm and energy in lobbying for what is best for patients. However, if advocates are unaware of the lessons of evidence-based medicine, then they may paradoxically worsen outcomes in those they seek to help. He who forgets history is condemned to repeat it, and advocates who are unaware of the hard-fought lessons of evidence-based medicine may be condemning patients to repeat errors of the past.


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