Research Article: Informed Consent and Shared Decision-Making: A Requirement to Disclose to Patients Off-Label Prescriptions

Date Published: November 11, 2008

Publisher: Public Library of Science

Author(s): Michael Wilkes, Margaret Johns

Abstract: Michael Wilkes and Margaret Johns argue that the doctrine of informed consent should require doctors to disclose to patients when they are prescribing a drug off-label.

Partial Text: A 9-year-old with cerebral palsy received an injection of the neurotoxin “Botox” to relieve muscle spasms. This off-label use was legal but not approved by the US Food and Drug Administration (FDA) for this indication. People with headaches have also received Botox injections as a legal, but unapproved, treatment—in this case the FDA is investigating whether the manufacturer actually promoted the drug for this indication. In fact, the drug has some significant dangers leading to hospitalizations and deaths [1].

Because a basic premise of the US Federal Food, Drug, and Cosmetic Act is that manufacturers are prohibited from marketing drugs or devices without FDA approval, the public commonly assumes that all uses of prescription drugs have been approved by the FDA. However, after a drug is approved for one set of indications, researchers and doctors often discover new applications for it. Even when the FDA approves a drug for a single, specific use, doctors may legally prescribe the drug to any patient for any use. Physicians are not restricted to prescriptions that comply with the FDA approval. The FDA considers such treatments “off-label” because substantial evidence regarding their safety and efficacy has not been presented or evaluated. But such uses are perfectly legal. In fact, FDA policy explicitly states that “once a [pharmaceutical] product has been approved for marketing, a physician may prescribe it for uses in treatment regimes of patient populations that are not included in the approved labeling” [2]. Indeed, as the Supreme Court has recognized, off-label prescribing “is an accepted and necessary corollary of the FDA’s mission to regulate in this area without directly interfering with the practice of medicine” [3].

Given that off-label prescribing of drugs may expose patients to unnecessary risks and may result in the prescribing of expensive new drugs when older ones are equally effective, cheaper, and safer, it is reasonable to apply the ethical mandate for SDM to doctors and require that health care providers disclose off-label prescribing to patients and seek their consent to the off-label use. In the US there are two grounds for requiring these discussions, one legal (informed consent) and one ethical (SDM).

Patients need information about off-label uses to make well-informed health care decisions. The legal doctrine of informed consent should be expanded to require disclosure of off-label prescribing where the drug has not been proven safe and effective for the condition, especially where scientific evidence is inadequate and risks are substantial or unknown. The ethical requirement of SDM should be expanded to require discussions of off-label uses under the same circumstances. Requiring disclosure will protect patient autonomy and educate patients about alternatives and risks, leading to improved health care decisions.



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