Saturday, February 20, 2010

Comparative Effectiveness Research: The Debate Continues

In the NEJM there is an article describing the next steps that are to be taken with CER. They state them as follows:

Institute of Medicine’s Recommendations for a National System of Comparative-Effectiveness Research (CER).

1. Prioritization of CER topics should be a sustained and continuous process, recognizing the dynamic state of disease, interventions, and public concern.

2. Public participation (including participation by consumers, patients, and caregivers) in the priority-setting process is imperative for ensuring that the process is transparent and that the public has input into the delineation of research questions.

3. Consideration of CER topics requires the development of robust, consistent topic briefs providing background information, an understanding of current practice, and assessment of the research status of the condition and relevant interventions.

4. Regular reporting of the activities and recommendations of the prioritizing body is necessary for evaluating the portfolio’s distribution, its effect on discovery, and its translation into clinical care in order to provide a process for continuous quality improvement.

5. The secretary of HHS [Health and Human Services] should establish a mechanism — such as a coordinating advisory body — with the mandate to strategize, organize, monitor, evaluate, and report on the implementation and impact of the CER program.

6. The CER program should fully involve consumers, patients, and caregivers in key aspects of CER, including strategic planning, priority setting, research-proposal development, peer review, and dissemination.

7. The CER program should devote sufficient resources to research and innovation in CER methods, including the development of methodologic guidance for CER study design — for instance, on the appropriate use of observational data and approaches to designing more informative, practical, and efficient clinical trials.

8. The CER program should help to develop large-scale clinical and administrative data networks to facilitate better use of data and more efficient ways of collecting new data to inform CER.

9. The CER program should develop and support the workforce for CER to ensure that the country has the capacity to carry out the CER mission.

10. The CER program should promote rapid adoption of recommendations based on CER findings and conduct research to identify the most effective strategies for disseminating new and existing CER findings to health care professionals, consumers, patients, and caregivers and for helping them to implement changes based on these results in daily clinical practice.

The analysis of these objectives leads to further insight as to where these folks are going. To reiterate, CER, as best as I understand their meaning, albeit inferentially, since one cannot find a delimited definition, it is expansively defined by what it does, a typical Government program, is a Government program targeting clinical studies, with the participation of a broad based of interested parties, who will in some undefined manner develop and recommend, perhaps mandate, clinical procedures related to the delivery of health care to Americans.

Frankly this is the antithesis of how medicine or any science is practiced. Imagine is we have had such a group in physics, chemistry, engineering, a centralized Government entity telling us what the problems are that we should consider and then seeking the input from many third party interest groups who may totally lacking in any expertise and then setting up what the truth is. Would we have an Einstein, a Schrodinger, a Feynman, a Wiener, or perhaps a Banting, where would those ideas come from that were initially non-conformists? Frankly are these people just plain Orwellian!

The authors, clear supporters of this plan, state:

First, the national CER program must develop an overall funding strategy. It could follow the traditional biomedical research model by inviting proposals on any of the 100 high-priority topics and awarding grants to the scientifically strongest proposals. However, the research interests of individual investigators would then define the national priorities. Instead, we believe that the national CER program should decide on a coordinated portfolio consisting of research on priority topics, infrastructure enhancement, and studies of translation and adoption.

Medical research has been around for over a century and it continues to evolve as we learn more. It is iterative and it modifies itself as we learn more. Some studies are well posed at their initiation but flawed by the time they are completed. I come back to the classic prostate cancer studies. They were started when a PSA of 4.0 was considered the gold standard. Over the years we have found that a PSA of 2.0 is as important for a younger man as 4.0 is for an older and also that PSA velocity is more a predictor. It is iterative and in some ways combative. A national CER program is consensus driven, worst of all worlds.

Second, the CER program should establish an initial list of priority topics and evaluate the current state of knowledge about each. For the first of these tasks, it should build on the priority-setting work of the IOM committee. It could develop a portfolio chosen from the top 25 IOM topics by applying the already-published prioritization criteria of the IOM

The portfolio is already there as a matter of ongoing research. Why redo the effort? Is this nothing more than justification for billions of more dollars spent by the Government. The money os spent well now why do we need change.

Third, the CER program, with the help of expert advisory committees and the research community, should choose the research methods that will fill gaps in the evidence for a specific topic. In an investigator-initiated research program, the grant applicant typically chooses the methods. The cost of studies using the methods of CER (whether clinical trial, observational study, or qualitative research) varies widely.

Evidence is always changing. Back to my prostate example. We know also that 5-10% of prostate cancers are highly aggressive. The question is why? Perhaps the four or five gene hits, ultimately knocking out PTEN, leads to the aggressiveness. Perhaps many men have genetically had the hits and they are predisposed, possibly there are epigenetic factors as well. These are the issues we should be working on, and these are the issues which the highly motivated and competent researchers are already working on. Why do we need another group? That question has never been answered. Perhaps to create approved methods to just "kill of the old folks" and replace the "death panels" with "death procedures".

Fourth, the program should strive for a balanced portfolio of high-impact research topics. Although it could simply rank topics in order of importance and fund them in ranked order until the money ran out, we recommend developing a portfolio that addresses a balanced distribution of topics, outcomes, and target populations, as well as keeping the total portfolio cost within budget and producing a body of evidence sufficient to influence health care decisions.

The nature of the portfolio changes as we learn more each step. Dynamic portfolios are common in the way we do research now. The "hot topic" appears and researchers follow the path. Having a bunch of Government chart preparers do this is frankly insane!

Fifth, the CER program should evaluate progress and report to the public. To meet this obligation, it should do large-scale, ongoing observational research and evaluation to measure CER’s effects on clinical practices and patient outcomes.

This I really do not understand. Medical research is always publicly available, NEJM is on line, as is JAMA and the list continues. Clinical trials are an every day affair, just read NEJM and JAMA and the hundreds of other journals. So what is the point? Just spending more money.

The only possible reason for CER is Government control. Control over what the Government will pay for and worse the control over what physicians can do. This is not the code of civil procedure used in Federal Courts, this is science, and as such changes. Having the Government as the regulator of change is not just stupid it is immoral.