Current Challenges in Comparative Effectiveness Research

The National Pharmaceutical Council sponsored a cluster of articles in the October 2012 issue of Health Affairs, "Current Challenges in Comparative Effectiveness Research," and a related briefing on October 11, to raise awareness of the uneven playing field in the health communications landscape, including industry’s challenge to correct the spread of erroneous or misleading information and supply the quality research that stakeholders are seeking.

Health Affairs editor Susan Dentzer and NPC President Dan Leonard kicked off the briefing, which had more than 170 participants in attendance. In his opening remarks, Mr. Leonard addressed some of the limitations facing health care stakeholders, particularly the pharmaceutical industry, in their ability to communicate research results to decision makers.

The panel discussions that followed reacted to a hypothetical case study of comparative effectiveness research (CER) involving a fictional migraine drug. Questions raised included how those research findings could be disseminated and communicated by various health care stakeholders, how patient involvement in the study could have improved the usefulness of the results, and whether there are barriers to the uptake of CER in clinical practice.

The first panel featured Dr. Aaron Kesselheim, assistant professor of medicine at the Harvard School of Public Health, Dr. Eleanor Perfetto, senior director of evidence-based strategies at Pfizer Inc, and Dr. Tevi Troy, senior fellow at the Hudson Institute. Dr. Kesselheim explained that because there are not yet adequate procedures in place to evaluate the validity of observational studies, the Food and Drug Administration's (FDA) reluctance to "authorize promotional claims" based on these studies is "understandable." But clarity about how to dispute or discuss these studies is needed, especially if the data is flawed or promoted by non-industry groups, said Dr. Perfetto. She suggested that stakeholders convene to develop voluntary best practice principles for communicating and disseminating CER. This suggestion also was echoed in different ways in by several speakers and authors of articles in the October issue of Health Affairs.

Next up, Marc Boutin, EVP and COO of the National Health Council, and Dr. Rachael Fleurence, a scientist at the Patient-Centered Outcomes Research Institute (PCORI), explained patients’ concerns about CER and how it could be used to make decisions. Both speakers emphasized the importance of getting stakeholder input early in the research process, and Mr. Boutin said that usability criteria would be enormously helpful to patients who are trying to utilize CER in making treatment decisions. Dr. Art Caplan, director of the Division of Medical Ethics at NYU Langone Medical Center, concurred, stating that it is challenging to provide vetted information in a form that is useful to patients.

NPC Chief Science Officer Dr. Robert Dubois and Dr. Michael Fischer, assistant professor of medicine at Harvard Medical School and associate physician at Brigham & Women’s Hospital, then discussed the use of CER by payers and clinicians, and who has the ability to disseminate the information. Dr. Fischer is a proponent of academic detailing, the goal of which is to close information gaps by providing clinicians with needed evidence and how it could be used in practice. Dr. Dubois questioned the ability of academic detailers to remain unbiased in their approach to reviewing and sharing information, and reiterated Dr. Perfetto’s call to develop communications standards for all stakeholders.

Finally, Dr. Harold Sox, editor emeritus of the Annals of Internal Medicine, and Dr. Justin Timbie, associate policy researcher at RAND, looked at future challenges facing PCORI and CER. Specifically, Dr. Timbie said that there are several reasons why CER fails to change patient care and clinical practice, and understanding those challenges could impact PCORI’s research and communications efforts for the better. But Dr. Sox cautioned that for PCORI to succeed before its funding expires in 2019, it must start with a research project agenda that conveys a sense of urgency and strategic direction.