On October 23, the U.S. Food and Drug Administration (FDA) issued draft guidance, Communication from Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products on its website. This guidance supersedes its 2014 draft guidance on the same topic and represents FDA’s current perspective. FDA is accepting comments and suggestions on this draft guidance for 60 days following publication.

FDA’s Goal with the Draft Guidance. The draft guidance notes FDA’s goal of striking a balance between supporting health care professional (HCP) interest in scientific information about unapproved uses of approved/cleared medical products to inform clinical practice decisions for the care of an individual patient, and the various government interests in incentivizing the development of and satisfaction of applicable premarket requirements for medical products. The guidance is presented in a question-and-answer format.

What is Compliant Disclosure? The guidance discusses what constitutes compliant disclosure of scientific information on unapproved uses (SIUU) of approved products. Generally, FDA notes that SIUU should be truthful, non-misleading, factual, unbiased, and provided with all necessary information so that HCPs can interpret the validity and utility of information.

Specifically, the guidance addresses:

(1) what firms should consider when determining whether a source publication is appropriate to be the basis for an SIUU communication;

(2) what information should be included as part of an SIUU communication;

(3) presentational considerations to consider and address for SIUU communications, e.g., refraining from use of persuasive marketing techniques; and

(4) additional recommendations for specific types of materials (e.g., reprints, clinical reference resources, including clinical practice guidelines, reference texts, and independent clinical practice resources, and firm-generated presentations of scientific information from an accompanying reprint).

Applicability. FDA clarifies that the guidance applies regardless of the medium of communication and applies to a variety of different types of communications, including published scientific or medical journal articles (reprints), published clinical reference resources, e.g., clinical practice guidelines, reference texts, and independent clinical practice resources, and firm generated presentations. This represents an expanded scope of applicability compared the FDA’s previous guidance because the previous guidance did not address firm-generated presentations.

New Standard Requiring Communication to be Scientifically Sound and Provide Clinically Relevant Information. Most notably, the draft guidance notes that SIUU communication must meet a newly articulated standard that the study upon which the SIUU is based must be scientifically sound and provide clinically relevant information. The guidance provides definitions for both phrases, and each term is broadly defined.

FDA notes that for human and animal drugs, the most rigorous studies usually are randomized, double-blind, concurrently controlled superiority trials. While these types of studies would certainly meet the scientifically sound standard and provide clinically relevant information, these study characteristics are not required.

FDA also notes that real world data or other types of well-designed, well-conducted studies and analyses may meet this standard. For medical devices, FDA offers that the types of studies, information, and analyses that are considered valid scientific evidence are described in 21 C.F.R. § 860.7 and may include well-controlled investigations, partially controlled studies, studies and objective trials without matched controls, well-documented case histories conducted by qualified experts, and reports of significant human experience with a marketed device.

As for studies that might not meet the standard, FDA elaborates that certain studies without an adequate comparison or control group, isolated case reports about medical products, and other reports that lack enough detail to permit scientific evaluation would generally not be scientifically sound or clinically relevant. Finally, the guidance continually emphasizes the importance of SIUU communication to the care of the “individual patient” rather than the general population.

Significance. This draft guidance is important for members of any pharmaceutical products or medical device sales and marketing team as it provides important guardrails on the dissemination of information for unapproved uses of approved/cleared medical products. Key stakeholders in the space should ensure they comply with these new guidelines, if finalized, and should consider submitting comments during the period for comments. The Foley team is here to assist as firms develop or adapt their marketing strategies.