Leveling the Playing Field? Could a Landmark Decision Alter the CER Communications Landscape?

A growing chorus of voices is publicly sharing data on the effectiveness of treatment options, but one viewpoint has long been shut out of key areas of the discussion. Restrictive rules hinder the very manufacturers who produce pharmaceuticals from publicly discussing off-label drug uses, including comparative effectiveness research (CER) findings, even when they want to correct erroneous or misleading information patients might be relying on to make medical decisions. But a landmark appeals court ruling could pave the way for industry to more freely share and discuss CER findings that will help patients, caregivers and health care providers make informed choices.

In United States vs. Caronia, a federal appeals court ruled that the government may not prosecute pharmaceutical manufacturers and their employees for truthful and non-misleading speech about off-label uses for approved medications. This could have a significant impact on the CER landscape.

The FDA has required companies to provide “substantial evidence” of a drug’s effectiveness as part of the approval process. According to the FDA standard, “at least two major clinical trials are required for product approval, although one trial with supporting evidence is sufficient in limited circumstances.” This evidence is used to develop the drug’s labeling information. If manufacturers go beyond the labeling claims when discussing the product’s use outside limited, recognized venues for non-promotional scientific exchange, manufacturers potentially face large fines and penalties.

But many CER studies use observational methods to identify real-world trends, rather than the randomized clinical trials that are often considered the “gold standard” in research. As a result, pharmaceutical manufacturers are not in a position to weigh in equally on the growing body of observational CER studies being produced by payers, academics and government agencies. This inequity inspired October’s CER-themed issue of Health Affairs and a related briefing featuring industry experts, including NPC Chief Science Officer Dr. Robert Dubois and Dr. Eleanor Perfetto, Senior Director, Evidence Based Strategies, Pfizer Inc.

The Caronia decision could help address these issues by expanding the ability of industry to discuss CER and other new research findings. The case involved a pharmaceutical sales representative who was convicted of promoting off-label uses of the narcolepsy drug Xyrem. The sales representative, Alfred Caronia, argued his free speech rights were violated and the Second Circuit Court of Appeals decided to vacate the conviction. In the 2-1 ruling, the appeals court noted that preventing industry from communicating about off-label uses does not necessarily advance the FDA’s stated goal of reducing patient exposure to unsafe and ineffective drugs, considering other stakeholders may legally speak about the off-label uses and physicians may lawfully write off-label prescriptions. The court also commented that prohibiting speech on off-label uses interferes with the ability of physicians and patients to receive potentially relevant treatment information.

The appeals court’s ruling might not be the final word on the issue. The Solicitor General could appeal the ruling to the full appeals court (a panel of the court issued the current decision), and also could seek review by the Supreme Court. NPC held a conference in February 2012 to delve into CER communication issues and the landscape’s asymmetry and the issue has remained on the industry’s radar for the past year.

Industry stakeholders recommended potential solutions in the October issue of Health Affairs exploring CER challenges. The issue, sponsored by NPC, recommended exploring voluntary communication standards to govern all stakeholders sharing CER information. This strategy could help level the playing field for communication while ensuring patients have access to the highest quality, most insightful CER findings available – from all sources. Implementing a set of “good communication principles” would guide all communicators in sharing truthful, scientifically sound CER data.

Creating an open environment for sharing the best data and findings ultimately would benefit patients and health care professionals. Expanding the amount of quality data available can help patients and their health care providers make optimal medical decisions. With perspectives from all the experts in hand, patients and providers stand the greatest chance of improving health care outcomes.