FDA Releases Draft Guidance on Unapproved Use Communications from Firms to Health Care Providers

Recently, the United States Food and Drug Administration (FDA) released a draft guidance document, Communications from Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products Questions and Answers. The FDA is asking for stakeholders to submit comments by December 24, 2023, to ensure that the agency considers the comments before it begins work on the final version of the guidance.

The guidance specifically focuses on firms that share certain types of communications with health care providers, including published scientific or medical journal articles (reprints); published clinical reference resources (including clinical practice guidelines, scientific or medical reference texts, and materials from independent clinical practice resources); and firm-generated presentations of scientific information from an accompanying published reprint. Other communications by firms are not addressed by the draft guidance and the agency does not intend to convey any views on those communications by issuing this draft guidance.

The guidance is directed at “firms,” defined as those legally responsible for the labeling of medical products, including applicants, sponsors, requestors, manufacturers, packers, and distributors of medical products, as well as licensees of any of those persons, and any persons communicating on behalf of those entities.

The guidance includes examples to help illustrate some of the recommendations and general considerations for firms considering – or sharing – these communications with health care providers. Importantly, and as is often the case, examples in the guidance do not describe every aspect of the relevant communications.

Under the Federal Food, Drug, and Cosmetic Act (FD&C Act) and the Public Health Service Act (PHS Act), the introduction of a medical product into interstate commerce that fails to comply with all applicable premarket requirements or is otherwise misbranded or adulterated is prohibited. This includes introducing a medical product that is intended for a use that has not been approved or cleared by the FDA, even if the same product is approved or cleared for a different use.

Notwithstanding that prohibition, health care providers may be interested in scientific information about unapproved uses of approved/cleared medical products to help inform their clinical practice decisions for the care of individual patients. The draft guidance attempts to strike a balance between supporting that health care provider interest and mitigating the potential that the government interests advanced by the aforementioned Acts are undermined.

Therefore, as the agency has indicated in other guidances regarding communications, it notes its belief that these communications be “truthful, non-misleading, factual, and unbiased and provide all information necessary” for health care providers to interpret the strengths, weaknesses, and validity, and utility of the information in the communication. The FDA included some examples of what information should be included in these communications in the guidance to help ensure the truthful, non-misleading, factual, and unbiased presentation. Any study or analysis described in a source publication serving as the basis for one of these communications should be scientifically sound and should provide information relevant to health care providers engaged in making clinical practice decisions for the care of an individual patient.

FDA further notes that if a firm shares one of these communications with healthcare providers in a manner consistent with the recommendations including the guidance the agency does not intend to use such communication standing alone as evidence of a new intended use.

When it comes to what firms should consider when determining whether a source publication is appropriate to serve as a basis for communication covered by the draft guidance, the agency recommends that the source publication describe studies or analyses that are scientifically sound and provide clinically relevant information. This means that at a minimum, the studies or analyses should meet generally accepted design and other methodological standards for the particular type of study or analysis performed, taking into account established scientific principles and existing scientific knowledge. Additionally, the studies or analyses should provide information that is pertinent to healthcare providers who are engaged in making clinical practice decisions for the care of individual patients.

The guidance, once finalized, will only provide the FDA’s current thinking on common questions regarding certain communications by firms to health care providers of scientific information on unapproved use(s) of approved and/or cleared medical products. The draft guidance also does not cover a firm’s communications of scientific information in response to unsolicited requests, those communications are addressed in the draft guidance for industry from December 2011, Responding to Unsolicited Requests for Off-Label Information About Prescription Drugs and Medical Devices.