Evidence-Based Medicine and the Patient-Centered Outcomes Research Institute: An Update of an Interview with Alfred O. Berg, MD, MPH

 

Alfred O. Berg, MD, MPH; University of Washington

William H. Burnett, Coastal Research Group: In my last interview with you, conducted on behalf of the Student Doctor Network [see Evidence-Based Medicine: Is American medical care based on science or politics?], you described the role of the US Public Health Services advisory committees, and also the role of the Institute of Medicine in recommending national policy. Would you describe how you see the differences in the roles of the policy advisory committees, the “think tanks” that are associated with a particular political philosophy, and the legislative committees that draft legislation?

Alfred O. Berg, MD, MPH: The advisory committees vary tremendously. Some advise on policy. Some have other purposes and audiences, and it is not always easy for the public to track the nuances. For example, one of the things the recent “mammography controversy” revealed to us is that almost everyone misunderstands what the purpose of the task force is that made the recommendation.

WHB: The “mammography controversy” related to a November 2009 recommendation of the U. S. Preventive Services Task Force, which was met by such headline’s as CNN’s “Task Force opposes routine mammograms for women age 40-49”. What is your perspective on the controversy?

AB: The task force’s recommendation simply restated a finding that has been in the literature for two decades – that women in the age category of 40-49 obtain a relatively small benefit from an annual mammography, and that small benefit is offset by some degree of risk. The NIH came to much the same conclusion back in 1997.

The task force making the recommendation was established to recommend to primary care physicians what should be discussed with their patients.  It was not intended to directly advise patients, insurance companies, or policy makers.

WHB: This is a case of a scientific body making a nuanced statement about the evidence they have examined, that the press reported without any of their qualifiers, is it not?

AB: Yes. The language of the recommendation was carefully constructed, but the qualifying statements were not reported by much of the press.

WHB: How should one evaluate health care recommendations from the various bodies that make such pronouncements?

AB: One should ask what is the entity making the statement, and what is the entity tasked to do? What is their charter? To whom do they report? Are they advisory or are they a regulatory body?

I would ask the same questions of any “think tank” or any other such entity: who funded them, what is their charter, and what is the process for them to develop their recommendations?

Often, the body making the recommendation turns out to be constituted by a politically “conservative” or “liberal” entity. It may not be transparent that there is a particular bias to the process by which the recommendation was developed.

This becomes a challenge. Unless you are really into a topic, most students, residents, and practicing physicians do not have the time or resources to find those things out. Fortunately, some organizations obtain reputations for doing things well, such as the United States Public Health Services’ Preventive Task Forces (UDPDTF), the “Bright Futures” initiative, the Advisory Committee on Immunization Practices, and some Institute of Medicine committees. But I think the connections of some entities are so non-transparent that they baffle everybody.

WHB:  Since our last interview a year and a half ago, there have been major developments, including the passage of PPACA, the Patient Protection and Affordable Care Act. One of the entities created by that act is the Patient Centered Outcomes Research Institute (PCORI). You are one of 15 appointees to that entity’s Methodology Committee. How does PCORI work?

AB: The Patient-Centered Research Institute is a very interesting new entity that is a legislatively enacted non-government organization set up by the Affordable Care Act. There is a board of governors that includes the Directors of the National Institutes of Health (NIH) and the Agency for Healthcare Research and Quality (AHRQ). This new organization is supposed to get hundreds of millions of dollars for research through a per person assessment from Medicare and private insurance plans.

PCORI is formally  outside of goverment, but most of its income is from federal dollars and it reports annually to the Secretary of DHHS and Congress. The entity “sunsets” in 2019. There will be several years to see how it works before it has to be reauthorized.

WHB: What is PCORI supposed to do?

PCORI was envisioned to compare treatments in terms that patients understand – patient-centered research. Most medical research studies whether a therapeutic intervention such as a drug is better than a placebo. But comparative studies, such as whether it’s preferable to use a particular drug among several alternatives, or to do something else entirely, are not often supported. There is no incentive for a drug company, for example, to compare its drug in a head-to-head comparison with a competing drug. What if their drug proves inferior?

WHB: What has PCORI done so far?

AB: The initial members of the board and its methodology commitee have been appointed by the General Accounting Office (GAO), as required by the legislation. It had to incorporate, find a location, hire staff, and set up processes and procedures. Substantively it has been working on some reports and priorities due in mid-2012, including definitions, grant programs, and other early deliverables. It has not yet begun to announce grant programs directly funding research. There are still many unknowns.

WHB:  A surprisingly robust movement, from both sides of the political spectrum, has arisen to bring about PPACA’s repeal or its substantial modification, and the PCORI is controversial in some circles. What do you see as the outcome of this controversy?

AB: My expertise in this area is narrow, and focuses on the science and what the legislation may add to an evidence-based system.

I don’t think that patient-centered research should be a partisan issue. Support for some of the principles of PCORI is shared on both sides of the aisle – everyone wants high quality information about what works for patients. Regardless of whether health care reform is implemented or repealed, it makes sense to support this type of research. Many other countries do something like this.

WHB: Some have expressed the idea that public funds should be spent only on therapies and procedures that have been proven to work and more specifically on therapies and procedures that have been defined as cost-effective by scientific bodies free of conflicts of interest. The current national debates have clearly juxtaposed this position with the argument that the government should not be in the business of “rationing care”. How would you respond to this debate?

AB: I am not a health economist, but it seems to me that we can’t have it both ways. We can’t cut costs if everyone can demand anything that they or their physician wants all the time. We already ration care by controlling access.  In the long run we will need to pay attention to costs and cost-effectiveness as one of the important factors to be considered for public funding.

WHB: Returning to the recommendations of the Preventive Task Forces regarding mammographies for women age 40 through 49, did they characterize the intervention as not cost-effective?

AB: The USPSTF up until now has not made its decisions based on cost or cost-effectiveness. And contrary to most media reports, it did not say that women in the 40-49 age range shouldn’t order mammograms. It said that there are small benefits and some risks; therefore, a physician should not routinely order it, but rather talk to the patient to decide together whether mammography is right for her.

Does that mean it should not be paid for? No, only that the the benefit to risk ratio is closely balanced. If the clinician and patient decide to go for it, the USPSTF would probably say it ought to be covered (although insurance coverage is not their issue). I would say that if a woman has to pay for it out of pocket, it might be useful for her to know what the benefits and risks are for her. When you are making a decision, science and economics should be part of it, but there are also ethical issues, feasibilty, preferences and other concerns as well.

WHB: And your final thoughts on PPACA?

AB: I don’t think anyone is happy about everything in the Act. There is something for everyone to like and dislike. Even many of those critics who oppose the bill in its entirety find a few things to like. However, the section on PCORI is the only place in the vast bill that talks about research and evidence reporting. There are still politically-driven constraints, such as prohibiting cost-effectiveness recommendations and recommendations to withhold funding for particular therapies. But in the long run PCORI could be highly influential in reforming the way we use evidence in clinical practice.

WHB: Thank you, Dr Berg.