Off-label use

Definition

Off-label use refers to the prescribing or supply of a therapeutic good for a use that is not included in the medicine's approved product information (approved indication). For example, a peptide approved for diabetes might be prescribed off-label for weight management, or an approved peptide might be prescribed at a higher or lower dose than the approved range. Off-label use is legal in Australia and many countries when a registered medical practitioner makes a clinical judgment that off-label use is appropriate for a specific patient. However, off-label use shifts responsibility to the prescriber to justify the use, ensure that evidence supports it, and take responsibility for adverse outcomes. Off-label prescribing is common in medical practice, particularly for conditions where approved options are limited or ineffective. However, promoting off-label use in marketing materials is illegal; promotional claims must adhere to approved indications and approved dosing. Educational content about off-label use must be distinguished from promotional marketing.

Off-label use is not the same as unapproved use. An approved therapeutic good, prescribed off-label by a registered practitioner, retains the status of an approved medicine; the goods are manufactured to regulatory standards and supplied through regulated channels. In contrast, an unapproved therapeutic good (such as a research peptide) has not been approved by the TGA for any indication and is not manufactured under therapeutic goods standards.

Regulatory agencies support responsible off-label prescribing when there is clinical justification, but they restrict marketing and promotion of off-label uses. If off-label use becomes widespread and generates good safety and efficacy evidence, manufacturers may seek formal approval for the additional indication, which would then become an approved indication.