Somewhere between the age of 3 and 4 years old, while playing on the swingset next to the gravel driveway at my grandparent’s house, I heard my Grandfather say:
“There’s more than one way to skin a cat”.
He explained the metaphor, it is playground wisdom still guiding me.
So...if there is more than one way to solve a problem, then is there a way to reduce lifestyle-related health risks that doesn’t require strict adherence to Evidence-Based Medicine (EBM)? Would innovation monitored and followed by research methods would be better than restricting ourselves to EBM standards? You see, the problem is that patients come with need for help, but the EBM often isn't sufficient to guide the intervention.
When is it OK to create programs that go beyond the evidence?
Gaps in the Evidence
As a trained physician-scientist whose clinical investigator training was funded by the NIH 7 full years, I get the EBM concept. Research needs to drive the medical model, and the public shouldn’t be exposed to unproven methods that may not provide benefit and have risk of harm. Got it.
The problem is that life is extraordinarily complex, which creates huge gaps in what we know about community-based lifestyle and physical activity interventions. There isn’t enough evidence.
Lots of people need help in areas that are still in the “grey zone”, particularly in the area of exercise in chronic disease. Many patients - perhaps even most - don’t have solid EBM that applies to them in their environments. Moreover, because most clinical science isn’t planned for the real world, there is often a huge gulf between what is known inside the Marble Halls and Ivory Towers and what is available in Brick and Mortar, 2x4 and Drywall. This is why there is call for more translational and implementation studies, to create patient-centered EBM.
Health plan administrators call for clinicians to stick to EBM, but this approach often leads to offering no help at all and does substantial emotional harm. Many patients have come to me for help on exercise, having been told there isn’t any treatment for their situation, feeling rejected and losing hope. You can see the spirit that lights the soul within grow dim. Worse, I’ve seen the look on the faces of study subjects who reached the end of a particular study protocol, only to see the doctors and scientists walking away. Patients and research subjects find themselves asking:
“Hey, what about me”?
Refraining from creating programs because there isn’t enough EBM to fully justify them is callow, even cruel.
Patients need something to try in their communities, something that at the minimum meets their emotional need to do something. In the case of lifestyle and physical activity, such interventions are nearly always very low risk, so doing nothing for them is probably more harmful than being innovative. There may not be enough science to create an EBM program, but doing something is more ethical than just saying:
“I’m sorry, we just don't know. Come back in...oh, say 10 years”.
My playground wisdom nags at me: is EBM the ONLY way to improve health care?
Maybe we should create patient-oriented interventions in the real world to begin with, and then study how well they work?
In the pharmaceutical industry, the compassionate use doctrine of using available but non-approved experimental treatments is sometimes provided to patients when there is nothing else left to offer. Calling it “compassionate use” is a bit deceiving, the compassion is not in the use of the experimental treatment, but in fulfilling the patient’s desire to try something.
Compassionate use and right-to-try laws have mainly been used for terminally-ill persons, though many right-to-try advocates would allow non-terminal patients as well. These are complex ethical discussions related to whether individual autonomy supercedes governmental regulation, and if so under what circumstances. Fortunately, the risk/cost/benefit situation is not so morally challenging in the case of community-based physical activity and lifestyle interventions.
In lifestyle and physical inactivity interventions, there may be costs in time, money, opportunities or other minor consequences. But most interventions have very little risk of direct harm to the individual - including light-to-moderate physical activity interventions. These are comparable to activities of daily living, and have very little risk.
Patients whose circumstances don’t fit the evidence still need something now. They want and need something to try, based on our best understanding of the existing science. Not trying to help them, when the interventions we could be offering are very low risk, shows no compassion or empathy.
What lifestyle, exercise and community-based realms need is innovative implementation, guided and informed – not constrained – by existing evidence. Such programs should be recorded in registries, should use investigational skills such as proper project design, need to obtain informed consent and should perform objective interval analysis of data for safety and efficacy. We still need to do rigorous systematic science, but in another way. Randomized controlled trials (RCTs) will still be needed, but instead of being designed to drive innovation a priori, RCTs would be used to resolve post-hoc hypotheses.
Experimentational advance via real-world patient-centered problems turns the current EBM process somewhat inside-out. The main advantage is that this approach would give many more people something low risk to try NOW, while they are still alive, would set the stage for RCTs and partially pre-empt the need for translation and implementation research.
Such an approach might be called compassionate intervention. Designed for real-world sustainability, it would give today’s patients something that his or her providers think is the best chance for improvement.
The strongest advocates of EBM are more often administrators than physicians, nurses or allied clinical staff. Administrators don’t see the tears welling up in a patient’s eyes or feel how much it means to patients to do something. Where there is good EBM, we should follow it. But insistence on strict adherence to EBM often fails to provide compassionate care to patients for whom we don’t have good answers. Ranking institutional policy higher than patient needs is a cold-hearted abomination. We must find another way.
Just because we’ve grown up doesn’t mean we can't use a little playground wisdom.
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