This is the 18th in a series of interviews between CFAH President and Founder Jessie Gruman and patient and consumer group leaders about their experiences with and attitudes toward comparative effectiveness research (CER).
Gruman: Tell me about yourself and your organization.
Helen Haskell: I am the founder of Mothers Against Medical Error, a group I started after the death of my son Lewis in November, 2000. For the past 12 years we have worked to improve health care quality, educate the public and provide support for patients who have experienced medical injury.
Gruman: Where do you see comparative effectiveness research (CER) fitting in the effort to improve the effectiveness of health care?
Helen Haskell: I'll use the example of my own family's case. My son went into the hospital for elective surgery to repair a relatively common condition ' pectus excavatum, a sunken breastbone. This was primarily a cosmetic condition for him. We got started on the road to surgery when we read a newspaper article promoting a new minimally invasive operation to correct this defect. We didn't go into surgery lightly. We looked it up on the Internet and we asked many questions of his doctors. The problem was that most of the information online was highly favorable, and when you do that sort of research you can end up believing you know a lot more than you do. And his doctors were more focused on comparing surgical techniques than in considering whether or not Lewis should have surgery at all. Even though we did a lot of research, we did not get the facts we needed to assess whether this surgery was too risky for a healthy child. And it was too risky. Our son died from this elective surgery.
After Lewis died, we discovered that there was a relatively extensive body of literature on his procedure that we had not known existed. I tore into those articles, looking at the numbers. I was surprised to find that the abstracts and conclusions were often inconsistent with the data in the same articles. I have since seen this phenomenon in other areas of medicine. And since most people read only the abstracts, patients and doctors may not be getting the information they need. CER presents an opportunity to change that, if it reinforces the concept of presenting straightforward comparisons in an unbiased way.
Gruman: Tell me how Mothers Against Medical Error views the relationship between CER and patient-centered outcomes research (PCOR).
Helen Haskell: Patient-centered outcomes research really takes CER to its logical conclusion. It is the way that CER can be turned into tools that patients and practitioners can use for individual treatment decisions. The Patient Centered Outcomes Research Institute is building on basic research like that proposed in the Institute of Medicine's 100 Initial Priority Topics for Comparative Effectiveness Research and is casting a wide net to find out what works and what patients want. I think this kind of patient involvement is the essential ingredient in making sure that CER gets into the community and achieves its potential to affect medical care.
Gruman: Can you give me an example of how your constituents have been affected by CER?
Helen Haskell: I think that, in general, patients are more likely to have been affected by the lack of CER, or at least by the lack of its application to practice. Probably the most notorious example is the COURAGE trial showing coronary stents to be ineffective for stable heart disease, which has certainly been more honored in the breach than in the observance. Other examples are studies raising questions about the relative effectiveness and safety of antidepressants and atypical antipsychotics. It is very hard for patients to know what to think when these treatments continue to be widely used.
Informed consent was one of the first rallying cries of patient advocates back in the 1980 and 1990s. When we began at Mothers Against Medical Error, it was one of our first goals as well. We saw informed consent as the ticket to reducing harm by preventing overtreatment. But we quickly came up against a brick wall, because there simply wasn't the information needed to make real informed consent possible. That is beginning to change. A lot of the information that is now becoming available consists of things like the clinician's record or the facility's record, but it is essential to be able to combine that information with reliable CER to arrive at what the patient wants to do. It is the first tool to dismantle the wall. It's not the only tool, but it is critical.
Gruman: How do you see the increased attention to CER affecting the care your constituents will receive in the future?
Helen Haskell: CER will be transformational if done properly. The critical thing is that it be done without built-in bias. If people can learn about all the options that are available to them and get care that is really targeted to their needs, medicine has the potential to become safer, more effective and less costly.
Gruman: Some professionals believe that patients and patient groups are opposed to CER. Do you think this is accurate? ' If so, what do you think is behind this view?
Helen Haskell: I think it is highly politicized. People fear the medical establishment, and in many ways they are right to fear it: The medical establishment has a lot of power, especially over hospitalized patients. People fear that treatment will be withheld because of CER. But it is curious to me that people who favor limited government seem to be the ones who want the government to be required to pay for ineffective treatments or unnecessary screening. In all the shouting, it seems to get lost that this is about payment, not about banning treatments.
Gruman: What are your fears and hopes for CER?
Helen Haskell: My fear is that CER will be construed as being too narrowly comparative and will not include all the possibilities. Presently, many studies begin from an assumption of benefit of treatment, even when a nonmedical approach might be a significant or even superior option. The failure to include nonmedical options is troubling, because all things being equal, it is hard to imagine that a well-informed patient would not usually prefer to avoid the risk and expense of treatment if possible. My concern is that CER will not break out of this approach.
My hope is that the results of CER will be made widely available and that patients will have a central place where they can go to see their options and come up with the plan that is best for them. It is also my hope that rigorous standards for comparative effectiveness will serve to push medical research forward and inspire it to ask questions that we have not thought about before. How good the evidence is will always be a question, but we need to use this opportunity to make the evidence the best it can be.