State Medicaid Directors Share Views on Comparative Effectiveness Research And Its Impact on Policy in National Pharmaceutical Council Survey

For Immediate Release
Contact: Andrea Hofelich,, 202-827-2078

(Washington, DC, April 16, 2015)—A new survey conducted on behalf of the National Pharmaceutical Council (NPC) shows that state Medicaid directors widely expect comparative effectiveness research (CER) to play a positive role in their coverage decisions, which affect over 32 million Americans—including vulnerable populations. Most survey respondents (90%) expected CER will lead to better clinical decision-making and value within five years, but acknowledged that there are barriers to be overcome before Medicaid directors can effectively leverage CER data when setting policy.

“As a factor in Medicaid pharmacy and medical directors’ decisions, CER has the potential to benefit millions of patients. Their expectations for CER’s positive impact is echoed in what we are hearing from other health care stakeholders. These results are also a reminder that, in order for the benefits of CER to be assured, there are several important aspects to address,” said NPC Chief Science Officer and Executive Vice President Robert W. Dubois, MD, PhD.

NPC supported a survey of Medicaid medical and pharmacy directors to better understand how policy makers in the state Medicaid programs view CER and how they use this research in setting coverage policy. The survey details were published as part of a peer-reviewed study, “Translating Comparative Effectiveness Research into Medicaid Payment Policy: Views From Medical and Pharmacy Directors,” in the March 2015 issue of the Journal of Comparative Effectiveness Research.

Sixty medical and pharmacy directors from 46 states responded to the survey and provided their perspectives on CER:

  • Usefulness of evidence. Nearly 90% said they used randomized controlled trials, systematic reviews and consensus statements from national professional societies in setting policy. About three-quarters used expert opinion, and over half used published observational studies from external sources or patient experience/consumer advocacy.
  • Effects of new CER on medical and pharmacy policies. Nearly 95% of respondents were “very” or “somewhat” likely to recommend a change in coverage policy if new CER evidence showed a currently covered product had more harms than benefits or if a new product was found to be less clinically effective than a currently covered one. Ninety-five percent were at least somewhat likely to limit coverage of a new treatment that was shown to be less cost effective than a current treatment.
  • Consideration of heterogeneity. When asked how often they considered heterogeneity (individual patient differences) when setting coverage policies, respondents reported “usually” or “always” half the time or less, with 13%-31% answering “don’t know/not applicable.”
  • Confidence in CER sources. The sources of CER evidence with the highest level of confidence by directors were the National Institutes of Health, the Agency for Healthcare Research and Quality and the Drug Effectiveness Review Project.
  • Barriers to using CER. The two barriers presenting the biggest problems were “lack of high quality research” and “lack of conclusive results.” In addition, approximately half of respondents thought that “legislative mandates” and the “lack of an independent body that provides legitimacy to interpretations of CER results” were important barriers.
  • Effects of CER on U.S. health care policy. Respondents were overwhelmingly in agreement that in the future, CER would result in better clinical decision-making and improved quality and health care value. Only about 50% of respondents thought that CER would actually reduce costs.

“The benefits to building a higher profile for and better understanding of CER among this crucial group of stakeholders is clear,” said Kimberly Westrich, MA, NPC vice president for health services research. “We are seeing high expectations for CER and potential for this research to benefit vulnerable patients, but more work is needed to ensure CER is available, accessible and applicable to Medicaid pharmacy and medical directors as they set policy.”

The study was directed by the Center for Surgery and Public Health, Brigham and Women's Hospital, Harvard Medical School with input from the Institute for Clinical and Economic Review and NPC and conducted by the University of Massachusetts Medical School’s Office of Survey Research between October 2012 and May 2013. The authors included Joel S. Weissman, PhD, and Olubode A. Olufajo, MD, MPH, of the Center for Surgery and Public Health, Brigham and Women's Hospital, Harvard Medical School; J. Lee Hargraves, PhD, of the Center for Survey Research, University of Massachusetts; Steven D. Pearson, MD, MSc, FRCP, and Sarah Emond, MPP, of the Institute for Clinical and Economic Review; and Ms. Westrich and Dr. Dubois from NPC.

About the National Pharmaceutical Council

The National Pharmaceutical Council is a health policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. Founded in 1953 and supported by the nation’s major research-based biopharmaceutical companies, NPC focuses on research development, information dissemination, and education on the critical issues of evidence, innovation and the value of medicines for patients. For more information visit and follow NPC on Twitter @npcnow.