On July 3, 2018, the FDA issued draft guidance on Indications and Usage Section of Labeling for Human Prescription Drug and Biological Products. The primary role of the Indications and Usage Section of labeling is to enable health care practitioners to identify appropriate therapies for patients by clearly communicating the drug’s approved indication(s). The draft guidance provides recommendations on general principles to consider when drafting the Indications and Usage section, including what information to include, when to include additional descriptors, limitations, or qualifiers, and how to format the information within the section.

Under 21 CFR 201.56 and 201.57, the Indications and Usage section must (1) reflect the scientific evidence accurately, (2) include the information necessary to clearly convey the safe and effective use of the drug, and (3) use terminology that is understandable to health care practitioners. The section may also cross-reference to a more detailed discussion of the limitations of use and must be updated when new information becomes available that causes the label to be wrong or misleading. Additionally, statutory provisions may require certain products to have recommended language for the Indication and Usage section. Such an inclusion may have benefits in relevant U.S. patent litigation. See Sanofi v. Watson Labs. Inc., 875 F.3d 636 (Fed. Cir. 2017).

The indication should begin with “DRUG-X is indicated” and include the condition being addressed and any other information necessary to describe the approved indication, such as the patient subgroups for whom the drug is approved. In some cases, however, a broad disease indication may not be appropriate because, for example, the drug may affect only certain signs, symptoms, or manifestations of the disease. An indication identifying an outcome or endpoint may be considered when the drug’s effect on the overall disease is not well understood. When a drug is approved for more than one indication, the format of the section should be carefully considered.

Limitations of use, which are presented separately from the indication within the Indications and Usage section, should be included in the section only when the awareness of such information is important for practitioners to ensure the safe and effective use of the drug. Examples of such information includes situations where there is reasonable concern or uncertainty about the effectiveness or safety of the drug in a certain clinical situation, such as adverse effects for a certain demographic or specific conditions that should be met before the drug is used on a long-term basis. The draft guidance also discusses considerations for whether or not to reference the Clinical Studies section in the Indications and Usage section.

Readers are encouraged to read the draft guidance, which is also available on the FDA’s website.