Challenges to the validity of using medicine labels to categorize clinical behavior: an empirical and normative critique of "off-label" prescribing

This study aimed to determine whether the label status of a medicine penetrates into the clinical reasoning of Australian medical practitioners and to explore the possible reasons for our findings using semistructured interviews with 14 Australian physicians. The interviews revealed 3 broad catalysts for off-label prescribing. The first of these was lack of awareness or understanding of the regulatory process in general and labels more specifically. The second was the perception that labels are not meaningful guides for clinical practice. The third was the recognition of alternative mechanisms for ensuring safe, rational, and evidence-based prescribing occurs. This research suggests that Australian physicians do not consider whether a medicine is off-label to be a reliable measure of the appropriateness of their prescribing practices. Rather, the legitimacy of prescribing practices is determined by the abilities, skills, and knowledge base of particular prescribers by a culture that encourages and supports evidence-based practice, and safe prescribing. Although labels are of minimal clinical significance, there are real conceptual, practical, and moral problems associated with conflating "good" or "better" practice with "on-label" practice, and "bad" or "worse" practice with off-label prescribing as often occurs. To ascribe greater meaning to the term "off-label" than is warranted can have the unintended consequence of casting suspicion on and making it more difficult for physicians to provide appropriate clinical care. We conclude that labeling can, in some cases, provide assurances to both clinicians and patients that their medications have been demonstrated to be safe and effective, but that clinicians should be able to continue to prescribe responsibly off-label without having any stigma attached to their practice.