The US Food and Drug Administration (FDA) finalized its Drug and Device Manufacturer Communications with Payors, Formulary Committees, and Similar Entities – Questions and Answers guidance (Final Guidance) on June 12, 2018. The Final Guidance is an important step in a broader effort by FDA to address and correct longstanding enforcement policies that restricted manufacturers’ truthful and non-misleading communications about investigational products, clinical outcomes or off-label (unapproved) uses of drugs and devices in many contexts. The Final Guidance confirms and largely reiterates the framework for communicating off-label information to payors that FDA described in the January 2017 Draft Guidance (discussed here) and expands the categories of information manufacturers can communicate without running afoul of FDA’s restrictions.
Historically, FDA has interpreted the drug and device approval and misbranding provisions of the Federal Food, Drug, and Cosmetic Act (FDCA) to prohibit even truthful and non-misleading statements about unapproved uses in promotional or commercial contexts. However, the practical realities and timing of reimbursement and coverage decisions often require payors to evaluate products before such products are approved (or cleared, as applicable) by FDA. FDA’s enforcement policies fueled uncertainty regarding the scope of permissible discussions with both public and private payors. Decades of First Amendment challenges, amendments to the FDCA under section 114 of the Food and Drug Administration Modernization Act of 1997 (FDAMA) and ongoing enforcement actions did little to clarify the boundaries of permissible payor communications.
In the Final Guidance, FDA acknowledges the sophistication of payors as an audience and their need to have access to a range of information on effectiveness, safety and cost-effectiveness of approved or cleared medical products, including information from manufacturers. This information helps support product selection, formulary management, and/or coverage and reimbursement decisions on a population basis, often long before a product is approved or cleared. The agency also clarifies that while the Final Guidance does not apply to communications to health care professionals (HCPs) generally, HCPs who serve in multiple roles, such as on a formulary committee, are covered under the guidance when acting in their professional capacity in selecting drugs for coverage or reimbursement for a payor, formulary, or similar entity. While noting that it remains critical that information provided by manufacturers must be truthful and non-misleading and must be accompanied with appropriate background and contextual information, the Final Guidance contains several important clarifications that do not appear in the Draft Guidance.
In the Draft Guidance, FDA first addressed communication of health care economic information (HCEI) by drug manufacturers to payors regarding FDA-approved drugs, and (2) communications by drug and device manufacturers regarding investigational products. The Draft Guidance was issued after the enactment of the 21st Century Cures Act (Pub. Law No. 144-255), which revised and expanded the extent to which health care economic information (HCEI) may be disseminated to certain stakeholders.
Applicability to Devices
Consistent with the language of FDCA section 502(a), the Draft Guidance only discussed communications of HCEI by manufacturers to payors regarding approved drugs. In response to public comments, however, FDA clarifies in the Final Guidance that its recommendations are also generally applicable to medical device manufacturers’ communication of HCEI to payors.
Communications About Unapproved Products or Unapproved Uses of Approved/Cleared Products
FDA also explicitly creates safe harbors for two additional types of information that manufacturers may provide to payors regarding unapproved products or unapproved uses of approved, cleared or licensed products:
- Factual presentations of results from studies including clinical studies of drugs or devices or bench tests that describe device performance.
FDA reiterates that no characterizations or conclusions should be made regarding the safety or effectiveness of an unapproved product or use and provides examples on appropriate, factual presentations and inappropriate presentations.
For example, a manufacturer may present information that an investigational product demonstrated statistically significant improvement on a primary endpoint versus the placebo. However, it would not be appropriate to make characterizations about the overall efficacy of the investigational product.
Similarly, a manufacturer may state that an investigational product met a primary or secondary endpoint compared to an active control, but it would not be appropriate to make comparative superiority claims about the investigational product over the active control.
FDA recommends that manufacturers communicating factual presentations of results from studies describe the material aspects of study design and methodology; the material limitations related to the study design, methodology and results; and disclose any positive, negative and null findings (i.e., results should not be selectively presented).
- Patient utilization projections (e.g., epidemiological data projection on incidence and prevalence of a disease or condition)
Background and Contextual Information
The agency clarifies that background or contextual information required to be presented may be concise, provided all material information (e.g., source(s) of data used, the outcome measures used, the type of analysis, the limitations of the analysis and the generalizability of the findings) is provided. FDA also acknowledges that other authoritative bodies (e.g., Academy of Managed Care Pharmacy Format for Formulary Submissions) have developed formatting recommendations on how to disclose relevant background or contextual information, and that the agency does not intend for firms to make duplicative disclosures of this information if it is already provided in accordance with these recommendations.
Overall, the Final Guidance does not represent a significant departure from the Draft Guidance, but it lends additional clarity for device manufacturers and provides a clear framework around the dissemination of information regarding prospective patient utilization and disseminating results of studies, bringing both within the scope of FDA’s safe harbors for communications with payors.