When the Food and Drug Administration (FDA) first began to oversee biopharmaceutical communications in 1962, neither FDA nor Congress could have predicted the rapid evolution of our health care system. In fact, even as recently as 20 years ago when Congress enacted the Food and Drug Administration Modernization Act of 1997 (FDAMA)—the last legislative action it took to directly address health care communications—health care looked dramatically different than it does today:
- Our health system was focused on rewarding the volume of services delivered, rather than the value of care provided. Many of the health care stakeholders or entities present today (Medicare Part D health plans, health exchanges, or accountable care organizations) did not exist then.
- Personalized or targeted treatments were not yet available to guide providers and patients in identifying treatments that work for their unique circumstances; and
- There were a limited number of sources and stakeholders generating evidence about what works when, for whom, and under what circumstances—compared to the broad array of information sources such as electronic health records, patient registries, and mobile health devices.
As the way we deliver and pay for health care continues to evolve, it’s more important than ever to facilitate an open dialogue about how treatments work in the “real-world,” how they compare to alternatives (their comparative effectiveness), and their related impact on the total cost of care.
The Problem: Who Can Say What, and When?
Unfortunately, despite these significant changes in our health care system and the increasing interest by health care decision-makers to know about “real-world” evidence, the exchange of evidence remains limited. Unlike other stakeholders, biopharmaceutical companies are subject to federal laws and regulations that limit what they can say proactively about their products and under what circumstances.
However, with the advent of new medications treating a range of conditions, the total cost of care was on many stakeholders’ minds and it became clear that additional flexibility was needed to inform formulary committees—not just providers—about the medication benefits, risks, and costs.
As such, in 1997 Congress took steps aimed at establishing a means by which the biopharmaceutical industry can proactively communicate health economic information under certain circumstances by enacting FDAMA. Section 114 of FDAMA created four central provisions:
- What Information Can Be Shared: Manufacturers can share “health care economic information not in labeling.”
- With Whom: Such information can be shared with “formulary committees and similar entities.”
- Under What Circumstances: If the information “directly relates” to the drug’s FDA-approved indication.
- Based on What Standard: The information must be based on “competent and reliable scientific evidence,”—a standard followed by the Federal Trade Commission, which is different from the FDA’s “substantial evidence” standard.
Unfortunately, there are significant ambiguities in the law’s language, how it would be enforced alongside existing and conflicting FDA standards—combined with the risk of penalties for violating such standards—and lack of guidance about its scope. As a result, not only has the Congressional intent of FDAMA 114 never been fully realized; but as stakeholders wrestle with how to deliver the right care to the right patient at the right time in the most appropriate setting, they do so without all the information they may need to make the best coverage and treatment decisions for patients.
Efforts to Encourage Broader Communication: Current Initiatives Under Consideration
As health care continues to rapidly evolve—and with the growing interest in using comparative effectiveness research and large data sets to inform decision making—more and more stakeholders are recognizing the need to provide better guidance for biopharmaceutical companies to facilitate the communication of truthful, balanced and non-misleading information. Today, clarity could be realized via legal challenges in First Amendment cases, Prescription Drug User Fee Act reauthorization, Congressional legislation or FDA guidance documents.
In addition, policy organizations and multi-stakeholder forums have sought to address this issue, including the Academy of Managed Care Pharmacy (AMCP), which hosted a Partnership Forum on FDAMA 114 in 2016. The forum convened stakeholders from managed care, academia, health care providers and patient advocates to identify recommendations on the evidence needed to meet their needs. Recommendations included language which could clarify the definitions for what is included in health care economic information, the standards upon which this information should be based, the relationship of the information within an approved disease indication, and with whom biopharmaceutical companies can share the information. It is unclear at this time how these actions will be considered by other entities.
Key Recommendations: Evolving Communications Laws, Regulations, and Guidance
It still remains unclear which, if any, of the aforementioned paths forward will provide the important clarity and changes necessary to facilitate the open exchange of real-world data. However, regardless of which approach advances, the following recommendations should be included:
- Ensure changes we make today are sufficiently flexible for future health care evolution. Evidence sources and approaches have significantly evolved over time and, along with it, so too has the range of meaningful and scientifically valid evidence available to stakeholders and our ability to leverage this information using professionally accepted standards. As our evidence base continues to grow, we not only need to develop policies that work in 2016—but policies that are sufficiently flexible for the future of health care product and payment innovations.
- Provide effectiveness (rather than just efficacy) information to health care decision-makers. Product label information traditionally is based upon efficacy or how well a treatment can work; however, today’s stakeholders are also interested in effectiveness—meaning “how treatments work in the real-world” and how these treatments will affect quality measures.
- Ensure meaningful evidence is available to health care decision-makers both now and in the future. The range of sophisticated stakeholders making population health decisions—and affecting the care covered and delivered by everything from health exchanges to accountable care organizations and by providers reimbursed through bundled payments—today did not exist five, 10, or 20 years ago. As we look ahead, we need to ensure all decision-makers have the information they need to make the appropriate choices for patients and our health system.
- Communicate early and often with payers. Health plans and those who bear financial risk for health care need to have information about new technologies 12-18 months in advance of launch to incorporate these changes into future health insurance rate reviews and premium changes, which are reviewed by state insurance commissions. Creating a safe harbor to allow communication is needed.
- Identify and allow trusted sources to educate and empower patients to be engaged and informed decision-makers about their treatments and the impact on their financial investment. Patients increasingly are responsible for premiums, deductibles, copays and prescription cost sharing. With the increase in consumer directed health plans, they need to make informed decisions—assessing the benefits, risks, and costs of treatment. Trusted sources can aid in this shift.