EBM only works for the mythical “average patient”

Pain-Topics.org News/Research UPDATES: Expect Analgesic Failure But Seek Success – this website is dead, but my post from 2013 still feels pertinent as corporate profit-driven healthcare companies continue their push to standardize our healthcare.

Evidence-Based Medicine (EBM) figures prominently in these efforts and is vigorously pursued and implemented by corporate healthcare (whose prime directive is to create profit for shareholders).

As our healthcare system tries to move toward EBM (evidence-based medicine), it’s critical to remember that there’s no such thing as an “average patient”.

New standards will be created based on statistical evidence – like means and averages – which can be misleading in medicine. This article points out how such statistics may not make sense in the real world (where not one single family has the statistical average of 2.2 children). 

Since no single pain medication seems to work for a majority of patients, pain medications could be excluded from treatment options by the slavish obedience to EBM alone:

If a particular formulation of pain medication only works for 20% of patients, it has a “success rate” of only 20% and would thus not be recommended as a treatment. That’s quite unfortunate for that 20% that did achieve relief since they will not get access to that medication.

Other subsets of patients will find different formulations effective, and there is no method but trial and error to determine what will work for which patients.

The point is:  If one opiate medication does not work, do not assume opiates have failed, but rather try one of the dozen or so other different types and formulations.   It has become clear that patients have very different responses to medications. Yet standards are meant to reduce “trial and error” and will endanger exactly the tailored treatment we pain patients need.

Annotations from the old article:

[The authors] propose a transformation in thinking about how analgesic efficacy and harm should be assessed, and suggest several practical implications of a better understanding and appreciation of therapeutic failure rates:

“No single drug will treat successfully more than a minority of patients with a painful condition.”

Successful pain relief is also likely to improve sleep, depression, fatigue, quality of life, function, and ability to work.

Experience (and some evidence) suggests that failure with one drug does not necessarily mean failure with others, even within a class.

We do not know the best order in which to use drugs, in terms of efficacy, harm, or cost.

Success or failure can be determined within 2-4 weeks, and success, when achieved, tends to be long lasting.

Because success rates are low, a wide range of drugs is needed to do the best for most patients, especially in complex chronic conditions.”

most patient responses are either very good (above 50% pain relief) or very poor (below 15%). Therefore, the frequency distribution curve is more “U-shaped”; rather than the classic bell curve

Patients who get better (responders) typically do well, experiencing improvements in fatigue, depression, and sleep interference, plus better function and quality of life.

Fixed-dose regimens may exacerbate adverse events and discontinuations, resulting in higher failure rates. An alternate approach would be to allow patient-directed drug titration to achieve adequate pain relief with tolerable adverse events;

The authors further observe that guidelines developers often restrict treatment recommendations to 1 or 2 drugs for any pain condition. The developers consider similar drugs to operate as a class, overlooking the fact that there can be important differences in pharmacokinetics or drug interactions across similar medications. Less restrictive guidance recommendations, centered on patient-practitioner interactions

Coupled with the nuances of neurobiological pain modulation, central nervous system transformations, and genetic influences, high failure rates with single pharmacologic interventions are unsurprising.

some current arguments against opioid analgesics for chronic noncancer pain seem to demand that either the therapy works well and for all patients or it is unacceptable — there is no middle-ground or acceptance of failure.

expectations need to be lowered to the level of clinical reality; eg, while long-term opioid therapy may not benefit all patients with chronic noncancer pain, it does help a certain proportion of patients and significantly so.

There is no such thing as an “average” patient, even though research trials tend to define outcomes based on average, or mean, scores on efficacy measures. In doing this, research can be misleading

The most vital question is: what works, for whom, in what circumstances?

A degree of failure should not defeat the pursuit of success when it comes to pain management.