August 21, 2018
Unlike the regulatory prohibitions against drug and device manufacturers advertising and/or promoting use of their products off-label, doctors are free to responsibly use FDA-regulated products outside of their approved indications as part of the practice of medicine. Prescribing a drug for an off-label use is when a doctor prescribes a drug (1) for different therapeutic reasons, (2) to a patient in a different age group or gender, or (3) in a different dose, mode of administration, or length of duration, than what was designated in the FDA approval process.
To be clear, “omission from the approved label does not mean that the FDA disapproves of an off-label use, it simply indicates that the agency has not reviewed that use.” Furthermore, off-label use is not the same thing as experimental or research use. Once the FDA has approved a drug for a certain on-label indication, that drug can then be prescribed/used legally for any indication. Off-label prescribing is a common part of contemporary medicine, accounting for approximately 10-20% of total prescriptions.
A doctor should balance her patient’s individual needs and characteristics with available scientific evidence when deciding whether to prescribe a drug for an off-label use. This advisory provides several practice pointers for physicians who may engage in off-label prescribing.
There are numerous drivers for the increasing incidence of off-label use, including:
- Medical advances outpacing the regulatory approval route, which is a costly and time-consuming process.
- Limited availability of data for rare conditions or certain populations of patients, who are frequently excluded from clinical trials, such as children, pregnant women, and the elderly, thereby resulting in fewer FDA approved options.
- Patients with life-threatening or terminal conditions, seeking additional life-saving options after having tried all FDA approved options.
- A drug has been approved for a use with similar pathologic or physiologic features to the condition intended to be treated. A doctor may reasonably extrapolate by comparison that the drug could be used effectively in both the on- and off-label ways.
Most Virginia litigation on off-label use involves manufacturers, not providers. While off-label prescribing has fueled some medical malpractice cases, most courts so far have held that off-label use is a matter of medical judgment and not per se prohibited or indicative of malpractice. It is rarely a stand-alone cause of action and usually appears in combination with other malpractice allegations.
A successful informed consent claim requires the plaintiff to establish that the physician breached the standard of care by failing to disclose material risks associated with the treatment, or the existence of any alternatives, thereby precluding the plaintiff from making an informed decision about whether to undertake the particular course of treatment.
Generally, a doctor has no legal duty to inform a patient of a drug’s regulatory status and is obligated only to provide a patient with clinical information. However, to reduce the risk that patients consider themselves under-informed, and to protect against associated liability, doctors should expand their informed consent process to explicitly incorporate any off-label uses of treatments. For example, the process can be supplemented to include:
- Discussion of the risks, benefits, and uncertainties of the proposed off-label treatment. A patient may find it material to know whether a proposed treatment is for an on- or off-label use, and whether or not the off-label use is supported by medical literature.
- Careful documentation in the patient’s chart of the clinical assessment and reasons for recommending the off-label treatment.
Prior to prescribing off label, doctors can ascertain whether published peer reviewed scientific literature supports the off-label use. Medical societies have issued policies on off-label prescribing, stating that such use must be done in the best interests of the patient and be based upon sound medical judgment and scientific evidence. A fair and balanced evaluation of the relevant scientific evidence can help doctors considering off-label prescribing.
Doctors should ensure that their off-label use of FDA-regulated products is informed and well-reasoned. Off-label use should be undertaken with the best interest of the individual patient in mind. The benefits of off-label use should outweigh the risks. Peer reviewed scientific support for the prescribed off-label use, a particularized informed consent for the off-label use, and detailed charting regarding the off-label use can help to insulate providers from malpractice claims.
For more information, please contact a member of Hancock Daniel’s Life Sciences , Medical Malpractice Defense or Risk Management teams.
The information contained in this advisory is for general educational purposes only. It is presented with the understanding that neither the author nor Hancock, Daniel & Johnson, P.C., is offering any legal or other professional services. Since the law in many areas is complex and can change rapidly, this information may not apply to a given factual situation and can become outdated. Individuals desiring legal advice should consult legal counsel for up-to-date and fact-specific advice. Under no circumstances will the author or Hancock, Daniel & Johnson, P.C. be liable for any direct, indirect, or consequential damages resulting from the use of this material.
 See e.g., Understanding Unapproved Use of Approved Drugs “Off Label,” U.S. Food & Drug Administration, available at: https://www.fda.gov/forpatients/other/offlabel/default.htm (last visited August 20, 2018); Katrina Furey, MD & Kirsten Wilkins, MD, Prescribing “Off-Label”: What Should a Physician Disclose?, AMA J. Ethics (2016).
 See e.g., Marc A. Rodwin, Medical Knowledge and Practice: Rooting Out Institutional Corruption to Manage Inappropriate Off-Label Drug Use, 41 J.L. Med & Ethics 654 (2013).
 Rebecca Dresser & Joel Frader, Off-Label Prescribing: A Call for Heightened Professional and Government Oversight, 37 J.L. Med. & Ethics 476 (2009).
 Furey, supra.
 Dresser, supra, at 476; April S. Fitzgerald, MD & Patrick G. O’Malley, MD, MPH, Staying on Track When Prescribing Off-Label, American Academy of Family Physicians (2014).
 Furey, supra.
 Fitzgerald, supra.
 Christopher M. Wittich, MD, PharmD, et al., Ten Common Questions (and Their Answers) About Off-Label Drug Use, 87 Mayo Clin. Proc. 10:982-90 (2012).
 Id.; Fitzgerald, supra; Rodwin, supra, at 655.
 Wittich, supra.
 See e.g., Wittich, supra.
 Allison v. Brown, 293 Va. 617, 628-29 (2017). See also, Wittich, supra (citing Canterbury v. Spence, 464 F.2d 772, 786-87 (1972)).
 Furey, supra; Wittich, supra.
 Elizabeth Stump, How to Avoid Malpractice Risks When Prescribing Off-Label, 8 Neurology Today 20: 25-26 (2008).
 Dresser, supra, at 481.
 Stump, supra.
 Fitzgerald, supra; Wittich, supra; Dresser, supra, at 476.
 E.g., American Medical Association House of Delegates, Patient Access to Treatments Prescribed by Their Physicians, H-120.988, available at: https://policysearch.ama-assn.org/policyfinder/detail/off-label?uri=%2FAMADoc%2FHOD.xml-0-201.xml (last visited August 13, 2018); Committee on Drugs, American Academy of Pediatrics, Uses of Drugs Not Described in the Package Insert (Off-Label Uses), Pediatrics 110, no. 1: 181-83 (2002), available at: http://pediatrics.aappublications.org/content/110/1/181 (last visited August 13, 2018).