In recent years, new anticoagulants such as Pradaxa (dabigatran), Xarelto (rivaroxaban) and Eliquis (apixaban) have been approved by the FDA. They have benefits over older drugs such as Coumadin (warfarin), as they do not require frequent blood monitoring. Apart from their FDA-approved uses, these drugs are being used “off-label” to address conditions for which they have not been formally tested.
Dabigatran, for instance, was approved by the FDA for stroke prevention in patients with non-valvular atrial fibrillation and has been found effective off-label for treatment of venous thromboembolism. Although off-label use is prevalent in clinical practice, the valid question exists whether the patient is required to be informed that the drug has not been approved by the FDA for the prescribed purpose.
It is estimated that over 20 percent of all prescription medications are prescribed for an off-label use.1 “Off-label” use of a medication refers to the practice of prescribing a medication for a use that has not been formally approved by the FDA.2 Off-label use of a drug is prevalent, because the medical community’s clinical experience with the potential benefits of a drug will outpace the speed of the FDA approval process. Recognizing the importance of off label use, Congress amended the Food, Drug and Cosmetic Act (the law empowering the FDA) to state that the law does not limit the ability of a physician to prescribe an approved drug for “any condition or disease.”3
To answer the question of whether a patient should be informed that the medication is being used off-label, it is helpful to consider what FDA approval means. The FDA regulates the marketing of drugs and devices, not the practice of medicine. Some drugs never obtain FDA approval for a legitimate indication because the cost of conducting the clinical trials needed for approval outweighs the benefit. A lack of FDA approval for a particular indication does not mean the drug is not safe, and there has been no clear legal requirement imposed on the clinician to specifically inform the patient that a drug is being used off-label unless it is being used for human experimentation or research.4 If the off-label use is not “experimental,” general rules of informed consent should apply. While the specific requirements of informed consent vary by state, and exceptions apply, generally the physician is required to inform the patient of the risks, benefits and alternatives of treatment so that the reasonable patient can decide in an informed way as to whether to proceed. How much information needs to be provided depends upon the specific treatment, purpose, degree of need, associated risks, benefit to the patient and other variables. The question of the sufficiency of this information may ultimately be decided by a lay jury with the help of the opinions of medical experts. Clinical experience, significant risks not known to the patient and medical judgment, and not the FDA approval per se, should dictate what information is given to a patient.
Peter T. Crean is a Senior Trial Partner at Martin Clearwater & Bell LLP with more than 30 years of legal experience. His practice encompasses all aspects of medical malpractice defense and professional liability defense. Daniel L. Freidlin is a Partner at MCB and focuses his practice on the defense of medical malpractice and professional liability cases.
- Radley et. al. “Off-label Prescribing Among Office-Based Physicians.” Archives of Internal Medicine 166 (9): 1021–1026.
- Buckman Co. v. Plaintiffs’ Legal Comm., 531 U.S. 341 (2001).
- 21 U.S.C. § 396 (1994 ed., Supp. V).
- In noting that neither Congress nor the FDA has attempted to regulate the off-label use of drugs by a physician, the U.S. Court of Appeals stated, “A physician may prescribe a legal drug to serve any purpose that he or she deems appropriate, regardless of whether the drug has been approved for that use by the FDA.” Washington Legal Foundation v. Henney, 202 F.3d (2000).