The July 2011 issue of Scientific American ran a rather interesting article on comparing the effectiveness of medical therapies (1). The piece, The Best medicine, is subtitled A quiet revolution in comparative effectiveness research just might save us from soaring medical costs. I doubt the savings in medical costs — every dollar spent on medical services is a dollar earned by someone providing those 'services', and those providers aren′t going to give up their slice of the pie without a fight. But I like the 'quiet revolution' in comparative effectiveness research (CER).

What′s so revolutionary about it? It doesn′t rely on randomized controlled trials (RCTs), the 'gold standard' that medical therapy is supposedly based on. Instead, investigators take advantage of the availablilty of detailed electronic medical records from millions of patients. Analyzing these data, researchers believe, yields results that are every bit as rigorous, at a fraction of the cost of RCTs.

RCTs are undoubtedly the best tool for the initial assessment of the safety and efficacy of new drugs. Randomly assigning study participants to either a treatment or a control group minimizes confounding differences; patients assigned by chance to either the treatment or the control groups are likely similar in all aspects except the therapy in question. Hiding these assignments from both patients and doctors (blinding) should minimize the placebo effect on the patient and bias on the part of the physician interpreting the 'treatment' results.

But once drugs are approved and prescribed, blinding and randomization become irrelevant. Therapies need to be assessed in real-world clinical settings where both doctors and patients know the treatment protocol. And detailed medical records contain all the relevant health, socioeconomic and other details for comparing patients known to be similar in every respect except the therapy in question.

The limitations of RCTs are well known (2), but they remain the 'gold standard' in medical research, the evidence to be given the greatest weight. Yet, much as Big Pharma would have us believe otherwise, most therapies aren′t supported by RCTs. The majority of drugs are prescribed for off-label use, and the majority of patients are taking drug combinations. Neither off-label use nor most drug combos are supported by RCTs. To quote one investigator, "There is a chasm between what gets done in practice and what science has shown" (1)

The insistence on RCT-based evidence as the ultimate authority is hypocritical and self-serving. It will undoubtedly be used to dismiss CER-based reassessments of existing therapies if those therapies are found to be ineffective. Insisting on RCT-backed evidence is also useful for discrediting 'alternative' therapies as 'unscientific'. Yet, RCTs are simply inappropriate in most areas of health and nutrition research. For example, it makes no sense to study supplementation with single nutrients in isolation; all nutrients are needed in the right amounts for optimal health.

Obviously we need, and should insist on, evidence-based therapies. But it is ridiculous to equate sound scientific evidence exclusively with randomized controlled trials. Let′s hope that this 'quiet revolution in comparative effectiveness research' takes a firm hold and relegates RCTs to the drug approval stage where they truly are the best strategy.

This new type of comparative effectiveness research may or may not alter medical practice and lower medical costs. But at least it should tell us, the patients, which of the available therapies are actually effective. If we are to be partners in making decisions that affect our health and wellbeing we need reliable information untainted by the financial interests of the medical/pharmaceutical establishment. By insisting on therapies that′ll actually do us some good we, the patients, might just help bring down medical costs.

Sources

1. Sharon Begley, The best medicine. A quiet revolution in comparative effectiveness research just might save us from soaring medical costs. Scientific American July 2011, 50-55.
2. Black N, Why we need observational studies to evaluate the effectiveness of health care. Br Med J 1996;312:1215.
   http://www.bmj.com/content/312/7040/1215.full