COMPARATIVE EFFECTIVENESS RESEARCH: JOHN BURKE ON CER AND CYSTIC FIBROSIS
By Jessie Gruman
April 2, 2014
This is the first 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: What are your general thoughts on comparative effectiveness research (CER)?
John Burke: As an experienced patient with two decades as a federal health care regulator, I think of’ CER as a tool designed to improve medical care and at the same time reduce unnecessary expenditures.
For example, right now, I see my treatment options being determined by only two questions: ‘What is the best therapy available that I know of for John?’ and ‘Will his insurer cover the therapy?” I believe CER can provide a third spoke in the wheel of evaluation of treatment options that will inform the first two spokes.’ This is not to say that I don’t see the typical provider adopting a ‘you have to try this drug before that drug’ unless there is a counter-indication, for example, with beta-blockers.
Gruman: Do you distinguish CER from patient-centered outcomes research (PCOR)?
John Burke: I’ve always thought of CER as looking at two or more therapies and comparing their health outcomes and costs through the use of data alone. ‘ PCOR, on the other hand, as envisioned by the Patient Outcomes Research Institute (PCORI) requires that patients’ perspectives are incorporated when designing and conducting studies and encourages the inclusion of the involvement of other stakeholders (clinicians, payers, industry groups, for example) in those deliberations.
Gruman: Do you think the distinction you make between PCOR and CER is widely recognized?
John Burke: No. For example, I served as a reviewer for the first round of PCORI proposals and saw traditional CER and other types of research efforts trying to ‘pass’ as PCOR.’ Clearly the authors of a number of proposals didn’t get it. I think the research community needs a little more time and experience with PCOR before they will truly incorporate the philosophy of PCOR into their portfolio of research tools.
Gruman: OK, so that’s your opinion as a social scientist with 20+ years experience in the art of information collection and as a federal rule maker.’ Does your perspective as a patient lead you to a different conclusion?
John Burke: I would say not.
From a patient perspective, the concern I have is that CER conflicts with the premise of personalized medicine, which strives to give each person the therapy that will optimize his or her outcomes.’ CER doesn’t take the individual into account.
Another concern with CER research is the impact a CER-based policy decision can have on the patients in general, particularly the indigent or uninformed.
Look at it this way: if a clinical trial isn’t done right, people in the study may be hurt; if the drug is approved, it may hurt some more. If this is the case, the therapy is often removed from the market or the labeling is changed to mitigate any negative impact. However, the implications of CER are much vaster because if CER is not done right, it can put the right therapy out of reach of some; it can create false formularies based on research that is not representative or statistically sound, and it can affect the health of many more individuals before any negative implications can’ be identified and corrected. Making this situation even more problematic is the fact that there is no sentinel process in place to monitor the impact of CER research-related policy decisions.
Gruman: As someone who has been treated for cystic fibrosis all your life and has had a double lung transplant, have your treatment choices ever been constrained or improved due to CER?
John Burke: Not that I know of either way. I’ve been fortunate to have access to traditional fee-for- service health care plans so I’ve not been exposed to the heavy hand of the coverage determination process.’ Sometimes, however, I have had enormous co-pays simply because there was no generic version of a drug I needed.
Gruman: Do you see the increased attention to CER/PCOR affecting the care you will receive in the future?
John Burke: I can envision benefiting from CER research’ that is able to identify therapies that are less invasive, provide for better short- and long-term outcomes and are more cost effective. I particularly see the benefit of CER in procedural medicine ‘ laparoscopic or-wait-and-see- before-you-cut procedures, for example.
My concern with CER used for coverage determinations is’ that our health care system is primarily reactive and that it lags behind in the identification and implementation of preventative medical protocols that could extend life and save money.’ Right now, as a patient advocate, I am working on such an effort that, if adopted, could extend lives of CF patients and save insurers millions of dollars.’ But the first hurdle is getting insurers to come to the table and listen to the evidence.
Gruman: You have been associated with a couple of cystic fibrosis patient groups.’ How does their view of CER compare with your personal view?
John Burke: I could be incorrect, but I don’t think the organizations I am familiar with have an opinion on CER per se.
For cystic fibrosis, I believe families want every option available to save their child.’ I imagine that if pressed, they would say that CER could restrict not only the availability of a therapy that their child might need, but it could conceivably limit the availability of promising therapies and thus constrain the ability to learn from them.
Gruman: Many professionals say that patients are opposed to comparative effectiveness research. Do you think this accurately represents the views of the U.S. public?
John Burke: I don’t think this issue is on the general public’s or patients’ radar screens.’ I think the audience for CER is generally inside the beltway.
Gruman: In closing, describe for me what you see as the rightful role for CER.
John Burke: Wearing my patient hat (as opposed to the policy-maker one), I think CER should be informative for the clinicians to make decisions, not as a coverage determination tool used by the policy-maker.’ CER can inform health care providers and provide for a very good means for creating an ‘options tier.’When clinicians are provided comparable treatment options, for example, ‘Here’s the cost, here are the benefits and risks; here’s why you should consider this for your patient,’ I think they will make the right choice.
In short, as a patient, I believe all appropriate therapeutic options should be available.’ I also believe that the time has come for CER. As a powerful tool, it should be used carefully and correctly to provide direction for health care decision makers.
Original blog post by Jessie Gruman. Updated by the GW Cancer Institute June 2016.