Ensuring a Free Flow of CER Information

By Dan Leonard, President, National Pharmaceutical Council

By Dan Leonard, President, National Pharmaceutical Council

When a person with a health issue jumps online to research his or her condition, it can be challenging to separate fact from fiction in the sea of available information. There may be a patient forum, a white paper published by a payer, and a local physician’s website that each may provide some degree of information on the health condition and available treatments. But in that mix of information, what is true, what is false and what is missing important nuances that may be important to the patient? And does that mix of information include data and insights from the very people who may know the most about a treatment – those who developed and manufactured it?
In today’s health communications landscape, the growth of digital information has made it possible for a variety of players – including payers, academics, health care providers and patient advocates – to comment on the effectiveness of treatment options. But concern over industry bias has led to restrictive communications rules that limit industry’s ability to share information in many situations when the company is in the best position to provide facts important to health care decision-making.

This is becoming a growing issue as the Patient-Centered Outcomes Research Institute (PCORI) and government agencies invest hundreds of millions of dollars in comparative effectiveness research (CER). Under the current rules, industry could be blocked from participating in the conversation.

Although limits on health care stakeholders’ speech in other contexts are a lightning rod for protest and controversy, to date the issue of restrictions on industry’s ability to communicate freely with payers, patients and providers has generated only a murmur. Growing awareness and understanding of the issues surrounding limitations on communication, however, recently have led to some thoughtful discussion around the importance of access to information. The upcoming issue of Health Affairs focuses on the dynamics of this asymmetry in the communications landscape, including industry’s challenge to correct the spread of erroneous or misleading information and supply the quality research that stakeholders are seeking.
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.
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.

Although new rules would open the door for greater participation in the CER conversation, industry recognizes that all communicators participating in the high-stakes discussion should adhere to some best practices. Voluntary communication standards could help ensure that only the highest quality research data is disseminated to stakeholders. There is too much riding on CER’s ability to improve patient outcomes and help control overall health care costs. If flaws in the current landscape are carried through into the new CER-informed health care environment, the sustainability of the entire enterprise may be put at risk.
Learn more about this important issue by attending the Health Affairs briefing on October 11, beginning at 8:30 a.m. at the Washington Marriott-Metro Center in Washington, D.C.