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More Can Also Be Less: We Need a More Complete Public Discussion about Comparative Effectiveness


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 1 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. Thus 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 at 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.

More Blog Posts by Jessie Gruman

author bio

Jessie C. Gruman, PhD, was founder and president of the Center for Advancing Health from 1992 until her death in July 2014. Her experiences as a patient — having been diagnosed with five life-threatening illnesses — informed her perspective as an author, advocate and lead contributor to the Prepared Patient Blog. Her book, AfterShock, helps patients and caregivers navigate their way through the health care system following a serious or life-threatening diagnosis. The free app, AfterShock: Facing a Serious Diagnosis, offers a pocket guide based on the book. | More about Jessie Gruman

Tags for this article:
Evidence-Based Medicine   Jessie Gruman   Communicate with your Doctors   Inside Healthcare  

Comments on this post
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ElaineSchattnerMD says
December 1, 2010 at 11:27 AM

Thanks for this thoughtful post. I agree that it's patients who have most at stake in this, and that the public, like many doctors, has a skewed perception. Unfortunately the press tends to present data as if they were true/false or works/doesn't work, but most medical value and findings are nuanced, particularly when it comes to applying aggregated information to individual persons.

Hard to know how to "sell" CER to doctors and patients, without oversimplifying in either direction.

More Can Also Be Less: We need a more complete public discussion about comparative effectiveness research. | Care And Cost says
December 3, 2010 at 12:10 AM

[...] Originally Published 12/1/10 on the Prepared Patient Forum [...]

Goldie Pyka says
December 3, 2010 at 4:59 PM

Related article from Health Affairs: "Adding The Patient Perspective To Comparative Effectiveness Research"

Summary: Comparative effectiveness research generates evidence that helps consumers, clinicians, purchasers, and policy makers make better decisions about health care. Capturing the patientâ??s perspective is central to this research because it provides a complete picture of treatment impact. This can be done with standardized questionnaires that ask patients to report on their functioning, well-being, symptoms, and satisfaction with care. These data, however, are not collected routinely in either clinical research or practice. Strategies and incentives to link patient-reported outcomes to data from conventional sourcesâ??including clinical research, electronic health records, and administrative dataâ??will accelerate the development of useful evidence.

To view the article:

raywerntz says
December 6, 2010 at 5:18 PM

As a lawyer and health benefit plan designer, the most frustrating aspect of plan writing is establishing a (regretably rigid) standard of medical necessity for purposes of separating covered from excluded expenses. Ideally comparative effectiveness research along with personalized (genomics based tailorized) medicine will allow us to establish more flexible approaches than historicly rigid appraches to medical necessity. Also, Jessie's references to the uncertainty in medicine suggest that a new--and more realistic--standard for malpractice that holds doctors less accountable for treatment when there is evidence based uncertainty should also be investigated. No matter the outcome, this is a very important post.

Jim Jaffe says
December 7, 2010 at 6:08 PM

this is a provocative, powerful and sensitive piece that raises an important issue. many have an "if you build it, they will come" approach to the issue, assuming that research will point to an optimal way of dealing with any medical problems. Others take the "if you provide the light, the people will find their own way," an optimistic perspective that's been proven wrong repeatedly. Its probably too much to suggest a doctor/deity begin the conversation by saying, "here's how I'd handle your problem, but others might take a very different approach and you should be aware of their arguments..." In the absence of that, though, it is hard to know how to begin he conversation.