Off-Label Treatments and Medications and Informed Consent

5061-dreamstime_m_28850408-e1680196937118Our healthcare and business law firm often assists provider and medical practice clients with preparing informed consents.  Many clients offer treatments in an off-label use and question whether informed consents are necessary for such use.  Currently, there is no FDA requirement to get a patient’s informed consent, but it may be beneficial for your practice to implement off-label informed consents.  If you have questions about patient consents or would like to discuss this blog post, you may contact our healthcare and business law firm at (404) 685-1662 (Atlanta) or (706) 722-7886 (Augusta), or by email, You may also learn more about our law firm by visiting

What Does “Off Label” Mean?

Off label means providing treatment and prescribing medications for conditions or using a dose different than what the FDA has approved.  To become an approved use of a drug or treatment, the FDA must “conduct a careful evaluation of its benefits and risks for that use” and ensure that use “is supported by strong scientific data.”  “Understanding Unapproved Use of Approved Drugs ‘Off label,’”, available here.  The FDA does not regulate the practice of medicine, so off-label use is allowed and has become common.  With certain populations, it is very common because clinical trials in those populations are less frequently (such as children and pregnant women).

Using drugs for off label uses is not prohibited.  According to the FDA, “once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient.”  Examples of off-label uses include the use of low-does aspirin for diabetes and heart disease and Ketamin infusions for depression.

Does Off Label Use Require Informed Consent?

Currently, the answer is “no.”  Although “[t]he FDA requires explicit written consent for drugs being used experimentally or as a part of research, . . . no explicit consent is required for any off-label drug use if it can be argued, like any other treatment, the drug is being used in the patient’s best interests.”  Z. Mithani, MD, “Informed Consent for Off-Label Use of Prescription Medications,” AMA J. Ethics, available here.

Is There a Benefit to Getting Informed Consent for Off-Label Uses?

It is common for providers to prescribe off-label treatments/prescriptions without informaing their patients that the drug was not approved for that purpose.  But some off-label uses can be dangerous.  For example, fenfluramine was frequently prescribed as a weight-loss medication, an off-label use, but then it was later found to cause heart-valve damage.  Mithani, “Informed Consent for Off-Label Use of Prescription Medications.”  By virtue of the use being “off label,” there is a lack of research on that use of the medication or treatment.  This fact alone may lead many providers to air on the side of caution and require informed consent for “off label” uses given the inherent risk in the lack of clinical evaluation and studies.  However, the reverse argument is that if the provider determines in their medical judgment that the treatment is in the patient’s best interest, providing the patient with information about the FDA’s internal legal and administrative processes may bog the patient down with unnecessary information.  And, with some populations with limited testing, it may not be practically feasible to gain informed consent for off label usage.  Although in some practices, such as pediatric practices, off-label informed consent may not be helpful or feasible, in other practices, it may be beneficial to ensure your patients understands off label treatments.  Whether off-label informed consents may be beneficial and protective for your practice is something to consider with your legal counsel.

If you have informed consent or off-label use questions or would like to discuss this blog post, you may contact our healthcare and business law firm at (404) 685-1662 (Atlanta) or (706) 722-7886 (Augusta), or by email, You may also learn more about our law firm by visiting


*Disclaimer: Thoughts shared here do not constitute legal advice.

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