Perspectives: What is the role for comparative effectiveness research?

The Journal of the American Medical Association devoted this week's issue to the topic of comparative effectiveness and how it should be used in medicine.

Journal of the American Medical Association: Is It Time For Medicine-Based Evidence?
Although (evidence-based medicine) involves finding as well as evaluating evidence, and even though more recent considerations of EBM have modified prior rigid approaches, a generation of clinicians has been taught that randomized controlled trials (RCTs) can find truth, whereas observational studies are inherently flawed. ... In a medicine-based evidence approach, the primary emphasis is on clinically relevant issues of who and where were the patients, what and why were the treatments, and when and how were the outcomes assessed-;as well as an assessment of validity and generalizability considered together and denoted as accuracy. Thus, in contrast to an emphasis in EBM on hierarchies of research design, medicine-based evidence is both rigorous and patient-centered (Dr. John Concato,4/17).

Journal of the American Medical Association: Comparative Effectiveness Research
Comparative effectiveness research is not new, even if the term is, but the enthusiasm and funding directed toward CER are well placed. ... Yet, whatever it is called, and whether based on RCTs, observational studies, decision analyses, or other research methods, the basic tenets of making comparisons between viable options rather than inappropriate but marketable comparisons with placebo, trying to estimate effect sizes in realistic settings, and ensuring that the patient perspective is incorporated in research and in treatment, are paramount (Drs. Robert M. Golub and Phil B. Fontanarosa, 4/17).

Journal of the American Medical Association: The Patient-Centered Outcomes Research Institute (PCORI) National Priorities For Research And Initial Research Agenda
The proposition that greater involvement of patients, clinicians, and others in the research process could help reorient the clinical research enterprise, reduce clinical uncertainty, and speed adoption of meaningful findings holds great promise, but remains to be tested. PCORI will test this hypothesis. The underlying imperative is to improve patients' care experience, decision making, and health outcomes (Dr. Joe V. Selby, Dr. Anne C. Beal and Lori Frank, 4/17).

Journal of the American Medical Association: Risk Models And Patient-Centered Evidence
A fundamental contradiction of evidence-based medicine (EBM) is that evidence is derived from groups, whereas medicine is applied to individuals. Inferring individual effects from average group effects is an example of the fallacy of division. Even in a randomized trial, benefit in a summary result does not imply that the probability of benefitting outweighs the risk of harm for all (or even most) patients within the trial (Dr. David M. Kent and Nilay D. Shah, 4/17).

Journal of the American Medical Association: Continuous Patient Engagement In Comparative Effectiveness Research
Experiences with patient engagement in research, including community-based participatory research, suggest that success hinges on patients being interested and emotionally involved in the research question and understanding their role in the (comparative effectiveness research) process (C. Daniel Mullins, Abdulla M. Abdulhalim and Danielle C. Lavallee, 4/17).