When the public turns its attention to medical effectiveness research, as Miller-McCune.com did earlier this fall, ("For Dying Cancer Patients, Geography is Destiny") a discussion often follows about how this research might restrict access to new medical innovations. But this focus obscures the vital role that effectiveness research will play in evaluating current medical and surgical care.
I am now slogging through chemotherapy for stomach cancer, probably the result of high doses of radiation for Hodgkin lymphoma in the early 1970s, which was the standard treatment until long-term side effects (heart problems, additional cancers) emerged in the late '80s. So I am especially attuned to the need for research that tracks the short- and long-term effectiveness — and dangers — of treatments.
Choosing a surgeon this September to remove my tumor shone a bright light for me on the need for research that evaluates current practices. Two of the three surgeons I consulted wanted to follow "standard treatment procedures" and leave a 6-centimeter, cancer-free margin around my tumor. This would mean taking my whole stomach out, because of its anatomy and arterial supply.
The third surgeon began our consultation by stating that her aim would be to preserve as much of my stomach as possible because of the difference in quality of life between having even part of one's stomach versus none. If at all possible, she wanted to spare me life without a stomach.
But what about the 6-centimeter margin? "There isn't really much evidence to support that standard," she said. "This issue came up and was discussed at a national guidelines meeting earlier in the week. No one seemed to know where it came from. We have a gastric cancer registry at this hospital going back to the mid-1990s and we haven't seen support for it there, either. A smaller margin is not associated with an increased risk of recurrence."
I agonized about different types of uncertainty, which I realize is embedded deeply in many medical diagnostic and treatment decisions, but which most of the time I don't have to confront quite so personally. Then I decided to work with the surgeon who was guided by the evidence (and lack thereof) rather than the "accepted standards" for surgical treatment.
Here's why: The standard radiation treatment for Hodgkin lymphoma that probably resulted in my current stomach cancer has been shown to be overly aggressive and harmful. If at all possible, I did not want to experience the effects of another unnecessarily aggressive medical procedure, one that for years has been the accepted standard but for which little evidence yet exists.
Most of us remain unaware that the evidence base for much of the current practice of medicine is so spotty. Some of the drugs and technologies we use to keep us upright and free of pain do indeed help us, but some of them may make no difference and some may do us serious harm. We believe that our physicians wouldn't recommend them if they weren't effective. We remain optimistic that advances in medicine will be even better in saving our lives. And so we resonate with media messages that focus on the impact of comparative effectiveness research on new approaches to care: messages that don't undermine our trust in current medical practice, but often do make us question our access to more and better treatments in the future.
My experience reminds me of how little the public understands about comparative effectiveness research. We need to realize that:
• Such research determines not only how well new treatments work, but more importantly, which among standard treatments works best and should remain the dominant approach, and what causes harm and should change.
• Producing such evidence is only the first step in changing a given clinical practice; informing clinicians and supporting widespread adoption of a new approach is no small challenge.
• The evidence for different treatments can and should be a topic that patients and doctors discuss together, difficult though that discussion may be for all parties.
• We — patients and families — are the major stakeholders in the comparative effectiveness research enterprise. Such research is not done by white-coated lab scientists. Rather it is done with, to, and for us: we must actively participate in clinical trials and other types of research if we and our kids and grandkids are going to benefit from the best available care.
Sure, the effect of comparative effectiveness research on new developments in medicine is important. But those concerns constitute only one small part of a much larger, more important public discussion about what it is going to take to develop, identify, provide — and to understand — the evidence to support decisions we make with our doctors to improve the length and quality of our lives.