The number of voices in the comparative effectiveness research (CER) conversation is continuing to grow, but not all stakeholders have the same ability to share information and data. Regulators, payers, biopharmaceutical manufacturers, health care providers and academics debated the pros and cons of regulating the flow and discussion of CER information during a February 9 conference, Asymmetry in the Ability to Communicate CER Findings: Ethics and Issues for Informed Decision Making. The goal of the event, hosted by NPC, the National Health Council and WellPoint, was to develop a framework for evaluating CER speech issues, said Dr. Robert Dubois, NPC’s chief science officer.
Dubois opened the discussion by noting how radically the information landscape is changing. Instead of a small number of randomized controlled trials being available on given clinical questions, those assessing research will have access to a much larger number of observational studies derived from electronic databases, such as administrative or billing information, post-marketing surveillance or patient registries, among others. The question in the CER landscape is how to apply what we know and how we share that information, Dubois noted.
Dr. Scott Gottlieb, resident fellow, American Enterprise Institute, described the regulations that can prevent industry from speaking on topics and how these restrictions create a “tilted” landscape. He noted that some of these restrictions are deliberate, rooted in government distrust of industry. While the government is investing substantial resources in translation and dissemination of data, Gottlieb said that it’s important to recognize that the government, too, has incentives in promoting certain prescribing behaviors and that research sponsored by industry has rigor and value.
Yet, even in the face of these asymmetries, biopharmaceutical companies are not condemned to stay silent on communicating research findings as long as they avoid any promotional behavior, said Dr. Robert Temple, deputy center director for clinical science, Center for Drug Evaluation and Research, Food and Drug Administration (FDA). Manufacturers are free to announce clinical trial results in news releases and publicly rebut findings they dispute, if the information they provide is factual and objective. In addition, Temple said companies must be mindful to avoid contradicting approved labeling or promoting off-label uses.
Even with this guidance, other speakers said that the rules governing CER communication are not always clear. In July 2011, seven biopharmaceutical manufacturers petitioned the FDA for clarity on communication involving responses to unsolicited requests for information, scientific exchange, interactions with payers and dissemination of third-party clinical practice guidelines, said Gerald Masoudi, partner, Covington & Burling LLP. While FDA has not yet delivered this guidance, Masoudi suggested that the resolution of United States v. Caronia, pending in the U.S. Second Circuit Court of Appeals, will likely provide more clarity on these and related questions.
Biopharmaceutical manufacturers are wary of testing regulatory limits because they have limited options for challenging a warning letter from the FDA, said Dr. Tevi Troy, senior fellow, Hudson Institute, and former deputy secretary, U.S. Department of Health and Human Services. Generally, companies can only discuss studies comparatively when there have been at least two robust head-to-head trials. Academics, payers and government officials do not face the same restrictions.
Academics are playing a key role in disseminating CER findings through the practice of academic detailing, said Dr. Aaron Kesselheim, assistant professor of medicine, Harvard Medical School. This involves clinicians providing non-commercial, evidence-based information, which is not product driven, to health care providers. Although biopharmaceutical companies have in-depth product knowledge and tend to employ strong communicators, promotional efforts can drive prescribing behavior that does not match evidence-based practice guidelines or the medical literature, he said.
Dr. Arthur Caplan, director, Center for Bioethics, University of Pennsylvania urged conference attendees to remember the overriding goal of disseminating information in the health care space: to ensure patients can make informed decisions about their treatment and care. Any change in the current rules on CER speech needs to take into account whether the revision will contribute to patient autonomy. That objective should form the ethical framework for evaluating CER speech, he said.
Dr. Stephen Hume, a patient with Alzheimer’s, and his care partner, Candace Darcy, echoed the importance of putting CER to work to meet the needs of patients. They shared their concern that information about effective treatments will not spread as quickly if biopharmaceutical companies cannot freely participate in the CER dialogue. Dr. Hume noted that patients and caregivers can learn about promising off-label uses of medications, particularly for treating terminal illnesses, when there is free flow of information from all stakeholders.
Susan Dentzer, editor-in-chief, Health Affairs, facilitated a provocative discussion about who should be charged with assessing CER. Temple made a tongue-in-cheek suggestion that a new “Agency for Truth” would be up for the task, but the panel broadly agreed that any “assessors” of CER would each bring their own biases. Stakeholders need to collaborate to develop standards for data collection, interpretation and synthesis to ensure decisions are based on quality evidence and information, said Dr. Steven J. Romano, senior vice president, head, Medicines Development Group, Primary Care Business, Pfizer Inc. That work will need to incorporate viewpoints from all stakeholders, he said.
Dr. Joshua Lenchus, associate director, University of Miami-Jackson Memorial Hospital Center for Patient Safety, said that what clinicians need is not more information on prevention and diagnosis but rather better support in integrating good practices into care. He said that, rather than “beating up” clinicians with guidelines, he sees more value in creating incentives for patients to take better care of themselves.
With the adoption of health information technology and evolving payment methods, payers are playing a bigger role in clinical decisions, said Dr. Marcus Wilson, President, Healthcore. As the line between payers and providers blurs, that will increase the demand for CER findings and speed the process of incorporating that information into practice in order to benefit patients.
Additional information on the speakers and slides from select presentations are available on the NPC website; brief video interviews can be viewed on NPC's website and YouTube channel.