NEJM Commentary Rekindles Debate on Communicating CER
A commentary published on July 18 in the New England Journal of Medicine is rekindling the debate about comparative effectiveness research (CER) and the pharmaceutical industry’s ability to communicate health information.
Tufts Medical Center economist Peter Neumann, ScD, who authored the commentary, suggests that Section 114 of the Food and Drug Administration Modernization Act of 1997 be “extended to allow drug companies to promote comparative effectiveness research to health plans and other payers. The law would retain its prohibition against such promotions directly to doctors or patients.”
Dr. Neumann’s paper continues the discussion of this issue that kicked off last year with a conference hosted by the National Pharmaceutical Council, a series of articles in Health Affairs, and the federal circuit court of appeals decision on US v. Caronia.
So what does this mean, and why does this debate matter? As NPC has explained before,
To understand industry’s challenge, it is important to recognize two key issues. First, the type of research used by the Food and Drug Administration (FDA) to make drug approval and labeling decisions is more stringent than the information that can be used by other health care decision makers. Second, FDA regulates the type of research that can be discussed by industry in most forums, but not by other stakeholders.
For a new drug to gain FDA approval, a company must provide “substantial evidence” of effectiveness, a standard under which “at least two major clinical trials are required for product approval, although one trial with supporting evidence is sufficient in limited circumstances.” This “substantial evidence” forms the basis for the drug’s labeling, and manufacturers face stiff fines and penalties if they go beyond the labeling claims when marketing or discussing the use of a product outside limited venues recognized to constitute non-promotional scientific exchange.
Although these clinical trials are considered the “gold standard” in research, most of the millions of dollars in CER that will be funded by PCORI will be derived from observational studies, data pulled from claims and payer databases, literature reviews and other public resources, none of which is considered acceptable for approval purposes by the FDA. With an increasing number of payers, academics and government agencies turning to observational methods of conducting CER, the trend leaves industry out of the conversation.
FDA officials have made statements acknowledging industry’s right to dispute unsupported CER evidence as long as the communication is not promotional in nature and reflects the patients, endpoints, and uses specific to the drug’s labeling. With CER studies examining a variety of off-label outcomes and being published in a range of venues outside of scientific journals, the role companies may have in the conversation is significantly reduced.
In a worst-case scenario, a payer could share an inadequate study about off-label use of a drug with its network of health care providers and patients. The results, which have not been subjected to any external oversight or transparency in the research protocol or analysis plan, would likely shape patients’ and providers’ views of the drugs involved and contribute to decision-making. Relying on such an inadequate study could lead to poor patient outcomes and rising costs. Even if the company that produces the drug spots the misleading information, existing communications rules potentially prevent the company from sharing insights on the study’s results and limitations with doctors and patients.
This unequal ability to communicate harms the very people who stand to benefit most from CER findings – the patients and health care providers facing medical decisions. The current communications environment increases the risk that useful details will be withheld or misleading information will go unchallenged. Decision-makers deserve to hear all viewpoints and weigh each source’s potential biases for themselves.
Given the potential penalties that would be incurred for violating the rules, however, companies will be hesitant to participate in open discourse about CER without clear guidance from the FDA. To open communication channels, the FDA will need to articulate standards that encourage dialogue and information-sharing.