We previously reported on Amarin’s lawsuit against the FDA, arguing that its restrictive interpretations of off-label speech would prevent the company from sharing “truthful and non-misleading information with healthcare professionals”. The lawsuit continues, and there has been significant back and forth between the FDA and Amarin on the issue, with oral arguments in early July. The lawsuit raises important questions about off-label speech and healthcare communications in general.
Background
In an editorial by Dr. Scott Gottlieb at Forbes, the former senior FDA official discusses the negative impact of FDA labeling regulations on prescription drug competition and patient health. Over at the Pharmaceutical Research and Manufacturers of America’s (PhRMA) blog, Catalyst, they raise an interesting point: as HHS and other stakeholders dedicate themselves to shift U.S. healthcare to a system rewarding value-based care, is that aligned with biopharmaceutical companies’ ability to support this shift to value? Especially when FDA regulations specifically limit the companies’ ability to communicate information like cost effectiveness data on approved treatments when the information is not included in the package insert approved by FDA?
Discussion
Gottlieb notes that valuable economic information, like uncontrolled observational studies, cannot be shared by manufacturers, regardless of the quality of the study. This results in less incentive for manufacturers to develop this data in the first place. Gottlieb cites a survey conducted by Avalere Health, indicating how much current regulations dissuade drug makers from developing information they cannot promote or speak about.
The study found that 86% of pharmaceutical companies indicated they would invest more in studies to support the development of healthcare economic information if provided additional guidance on their ability to use this evidence with external audiences. This information could then be used to help payers and providers make treatment decisions.
Proposed changes in the 21st Century Cures legislation are intended to provide clarity around the evidence manufacturers can communicate with payers and decision makers about the value of products. Avalere suggests this may “result in better evidence on cost-effectiveness, comparative benefit, and real-world outcomes for payers determining patient access to these medications.”
Currently, laws and FDA regulations govern pharmaceutical company communications to physicians and consumers. Companies’ clinical claims must be substantiated by two well-controlled trials, provide fair balance, and be consistent with FDA-approved labeling regulations. Additionally, Section 114 of the FDA Modernization Act sets different standards for how health economic evidence may be presented to payers. Without additional guidance, permissible evidence and possible audiences for communication may be left out due to varying interpretations of the law across the industry.
“Setting aside the question of whether the current rules restricting this speech are constitutionally permissible, or violate First Amendment protections, the fact is that the rules and enforcement chill the free exchange of health economic information,” writes Gottlieb. “Drug companies can only make claims around benefits, and usually relative to a placebo. As a consequence, resources are plumbed into trials that will enable the kinds of information and results that government rules allow drug makers to share.”
This results in a less competitive market because there is less data and information reported, skewing debates about relative value. Ultimately, manufacturers cannot put forward data that argues the true value of their product, whereas, those with “economic incentive” to control access can do so, writes Gottlieb. He also notes Section 6301 of the Affordable Care Act’s specific instruction to government agencies to disseminate research “with respect to the relative health outcomes, clinical effectiveness, and appropriateness of … medical treatments [and] services.”
Supporters of the Affordable Care Act note that physicians often practice medicine without knowing the comparative effectiveness of medical interventions. For instance, one of the leading medical textbooks writes that it is not known whether drug treatment works better than removing certain heart cells to treat a common type of heart arrhythmia. While comparative effectiveness research is conducted in other countries, it is less prevalent in the United States. The purpose of the Patient Centered Outcome Research Institute (PCORI) was designed to fill this research gap. Gottlieb points out PCORI “creates the strange specter of the federal government funding the creation of information that the feds then bar certain parties from speaking about,” as PCORI creates standards for the conduct of real-world evidence studies and other trials that likely will not meet FDA standards, including system reviews and observational studies.
Conclusion
Gottlieb concludes by reminding policymakers they want cost and value to be factors in how decisions are made in American healthcare. “Should they be comfortable with a landscape where sponsors are discouraged from generating this data about their products, and as a consequence prohibited from competing on these important domains? Or where the information and speakers are biased in favor of one side of that debate?”
A lack of information reduces competition which leaves the patient and their physician with less information to make informed decisions. If we want greater value in the pharmaceutical market, more information is one way to start.