By JESSIE GRUMAN, PhD

Media coverage of the government’s new investment in comparative effectiveness research leans heavily toward the effects of such research on new drugs and technologies: Will such evaluations lead to restricted access to the latest innovations?  Will insurance no longer cover a drug that might give my aunt another year to live? Will such research hinder the development of a drug that could cure my nephew of type I diabetes?

The focus on how results from comparative effectiveness research might affect new approaches  obscures for the public and for policymakers the vital role of such research in evaluating current approaches to diagnosis and treatment that may not only be ineffective but in fact harmful to us.

I am now slogging through chemotherapy for stomach cancer almost certainly caused by receiving high doses of radiation for Hodgkin lymphoma, which was the standard treatment until long-term side effects (heart problems, additional cancers) emerged in the late 1980s.  So I am especially attuned to the need for registries and trials to track the short- and long-term effectiveness of treatments.

So choosing a surgeon in September to remove my tumor shone a bright light for me on the importance of research to evaluate current practices. Two of the three surgeons I consulted wanted to follow “standard treatment procedures” and leave a six-centimeter cancer-free margin around my tumor. This meant taking my whole stomach out, because of the anatomy of my stomach and its arterial supply.

The third surgeon began instead 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 possible, she wanted to spare me life without a stomach.

But what about the six-centimeter margin?  “There isn’t really much evidence to support that standard,” she said.  “That issue came up at a national guidelines meeting earlier in the week and we discussed it.  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 the essence of medicine, but which most of the time I don’t have to confront quite so personally.  I decided to work with the surgeon who wanted to be guided by the evidence (and lack thereof) rather than the “accepted standards” for surgical treatment.

Here’s why: my current stomach cancer was, very likely, caused by what at the time was standard radiation treatment for Hodgkin lymphoma but which evidence now shows was overly aggressive and harmful.  If at all possible, I did not want to experience daily 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 are 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 the media’s focus on the impact of comparative effectiveness research on new approaches: It doesn’t undermine our trust in current practice, only in our ability to get more and better treatments in the future.

My experience reminds me of how poorly the public has been informed 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.

Jessie Gruman, PhD, is the founder and president of the Washington, DC -based Center for Advancing Health. She is the author of Aftershock: What to Do When You or Someone you Love is Diagnosed with a Devastating Diagnosis. She blogs regularly on the Prepared Patient Forum.