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Avoiding Physician Liability for Off-Label Use of Drugs and Devices

By Lori G. Cohen and Sara K. Thompson
October 28, 2010

Plaintiffs' attorneys continually seek new and novel ways to circumvent the defenses afforded to manufacturers of FDA-approved medical products, such as federal preemption and the learned intermediary doctrine. At the same time, “it is undisputed that the prescription of drugs [and devices] for unapproved uses is commonplace in modern medical practice and ubiquitous in certain specialties.” Washington Legal Foundation v. Henney, 202 F.3d 331, 333 (D.C. Cir. 2000) (internal citations omitted.) The increased level of enforcement activity by the United States Food and Drug Administration (FDA) has focused the attention of consumers and the plaintiffs' bar on the increased frequency with which physicians prescribe FDA-approved drugs and medical devices for unapproved uses. Plaintiffs' attorneys increasingly choose to name prescribing physicians in their products liability suits as co-defendants, pleading both medical malpractice and other associated tort claims against these physicians, and often premising such claims upon the decision to prescribe for a so-called “off-label” or unapproved use. Thus, the continuing expansion of off-label uses of drugs and devices has led to numerous potential legal minefields for the prescribing physicians.

Often, physicians do not realize that, to a lay person, the use of a drug or device for an indication that has not been approved by the FDA may sound as though the physicians are somehow “experimenting” on their patients with off-label uses. While “[a] physician is free to use a medical device for an off-label purpose if, in the physician's medical judgment, he or she believes that use of the device will benefit the patient,” it also stands to reason that “[b]ecause the off-label use ' is a matter of medical judgment, a physician may be subject to medical malpractice liability for the exercise of that judgment.” Alvarez v. Smith, 714 So.2d 652, 653 (Fla. App. Ct. 1998). It is important for prescribing and implanting physicians and their counsel to be aware of the murky and shifting arguments plaintiffs may make against decisions to prescribe off-label. This three-part article explores the potential theories of liability for off-label prescriptions that have been employed, with mixed success. It will also discuss strategies for physicians to use to protect their power to prescribe approved drugs and devices in an off-label manner, in their exclusive medical judgment, while protecting themselves from a threat of potential future litigation or liability.

Off-Label Prescription Has Always Been Here

Since federal legislation first required FDA approval of prescription pharmaceuticals and medical devices, the federal government has made clear that it did not intend through regulation of the approval of drugs and devices to restrict or hamstring physicians' ability to utilize those drugs and devices in ways not specifically approved by the FDA. For example, a 1994 amendment to the Food, Drug and Cosmetic Act stated that “[n]othing in this chapter shall be construed to limit or interfere with the authority of a health care practitioner to prescribe or administer any legally marketed device to a patient for any condition or disease within a legitimate health care practitioner-patient relationship.” 21 U.S.C. ' 396 (2010). The U.S Supreme Court has recognized that “'off-label' usage of [drugs and] medical devices ' is an accepted and necessary corollary of the FDA's mission to regulate in this area without directly interfering with the practice of medicine.” Buckman v. Plaintiffs' Legal Comm., 531 U.S. 341, 350 (2001). While the FDA prohibits promotion of unapproved uses by manufacturers of FDA-approved drugs and devices, it does not regulate or prohibit a physician's decision in his or her independent medical judgment to use an approved drug or device in an off-label manner. Even the general prohibition on manufacturer promotion of off-label uses does not bar the manufacturer from providing reprints of medical journal articles and reference publications regarding off-label uses to physicians, as the FDA recently made clear in its recent revisions to industry guidance regarding dissemination to physicians of medical literature discussing off-label uses. See Guidance for Industry – Good Reprint Practices for the Distribution of Medical Journal Articles and Medical or Scientific Reference Publications on Unapproved New Uses of Approved Drugs and Approved or Cleared Medical Devices, 74 Fed. Reg. 1,694 (Dep't of Health and Human Servs., Jan 13. 2009). The FDA's revised Guidance notes that “off-label uses or treatment regimens may be important and may even constitute a medically recognized standard of care.” Therefore, if the physician requests it, the agency permits dissemination of literature regarding unapproved uses to prescribing physicians, as long as it is truthful and not misleading. Id.

Off-Label Uses

Off-label or unapproved uses of drugs or devices may take many forms and may vary slightly or greatly from the FDA-approved indications for use of the drug or device. For example, a physician may prescribe an FDA-approved pharmaceutical to a pediatric patient, which is an off-label use if the drug is not specifically approved for use in children. (Many pharmaceutical products are not approved for use by children, simply because children are not usually studied in clinical trials.) Similarly, a physician may employ an FDA-approved implantable medical device to treat the indications for which it is approved, but may implant the device in a different location than where it is generally used, or implant multiple devices even though this is not a specifically approved practice. Another common off-label use is to implant an FDA-approved medical device for the approved indications for use, but use it in conjunction with an FDA-approved medication that is not specifically approved for use with that device. Even varying dosages or combinations of prescription medications in a different way than indicated on the approved labeling may constitute an off-label use of a prescription drug.

Frequency of Off-Label Prescriptions Would Surprise Most Patients

Although these uses of FDA-approved products may be considered off-label or unapproved uses, all are very common within the medical community. Estimates of the frequency with which physicians write off-label prescriptions vary wildly, with up to 60% of all drug prescriptions estimated as off-label uses. See James M. Beck, Elizabeth D. Azari, FDA, Off-Label Drug Use, and Informed Consent: Debunking Myths and Misconceptions, 53 Food Drug Cosm. L.J. 71 (1998). Off-label use of drugs is particularly common in specialties such as oncology, where patients often face life-threatening conditions and their physicians are increasingly willing to attempt unapproved treatments in an effort to prolong their patients' lives. See Bryan Cote, Compendia: The Bridge Between FDA Approved Indications and Off-Label Usage, Oncology Bus. Rev., 10, 11 (2008), http://www.oncbiz.com/documents/OBR_0108_Compendia.pdf (estimating approximately 60% of cancer drugs are used off-label); see also Richard A. Epstein, Cancer Patients Deserve Faster Access to Life-Saving Drugs, Wall St. Jour., May 2-3, 2009, at A11. While there are numerous estimates of the frequency of off-label use of drugs, quantifying the frequency with which medical devices are prescribed off-label has proven more difficult. However, if up to 60% of all drug prescriptions are written for off-label uses, the probability is high that virtually every patient at some point during their life will receive a prescription medical product for an unapproved use, even though many may never realize it.

The General Public

The general public is largely unaware that off-label prescriptions are widespread and permissible when a physician chooses the off-label use in his or her independent medical judgment, without manufacturer promotion. See Beck and Azari, supra at n59 (citing Harris Interactive Poll, U.S. Adults Ambivalent About the Risks and Benefits of Off-Label Prescription Drug Use (Dec. 7, 2006), http://www.harrisinteractive.com/news/allnewsbydate.asp?NewsID=1126). The disconnect between public understanding of the implications of FDA approval and the scope of permissible off-label uses may lead patients who experience an undesirable outcome with an off-label treatment to blame their poor result on the lack of regulatory approval or the physician's decision to recommend an unapproved use. Generally, when this blame results in litigation, the claims alleged are general malpractice or negligence claims, in which the patient challenges the physician's decision to employ the drug or device in an unapproved manner. Sometimes a patient will assert that the regulatory status of the drug or device should have been disclosed before the patient consented to its use. Conversely, in rare circumstances, the decision not to use a drug or device for an accepted and common off-label use may be challenged as outside the applicable standard of care, where the patient alleges the physician should have attempted the treatment because it was a known option within the field.

This discussion continues in next month's issue of Medical Malpractice Law & Strategy.


Lori G. Cohen, a member of this newsletter's Board of Editors, is a shareholder with the law firm of Greenberg Traurig, LLP. She focuses on litigation and trial work for the pharmaceutical and medical device industries, products liability, and medical malpractice. She has been recognized by the National Law Journal as one of “The 50 Most Influential Women Lawyers in America” and “Top 40 Under 40,” which highlights the most successful 40 litigators under the age of 40 in the country. Sara K. Thompson is of counsel with the firm, and focuses her practice on pharmaceutical and medical device products liability litigation, as well as medical malpractice litigation.

Plaintiffs' attorneys continually seek new and novel ways to circumvent the defenses afforded to manufacturers of FDA-approved medical products, such as federal preemption and the learned intermediary doctrine. At the same time, “it is undisputed that the prescription of drugs [and devices] for unapproved uses is commonplace in modern medical practice and ubiquitous in certain specialties.” Washington Legal Foundation v. Henney , 202 F.3d 331, 333 (D.C. Cir. 2000) (internal citations omitted.) The increased level of enforcement activity by the United States Food and Drug Administration (FDA) has focused the attention of consumers and the plaintiffs' bar on the increased frequency with which physicians prescribe FDA-approved drugs and medical devices for unapproved uses. Plaintiffs' attorneys increasingly choose to name prescribing physicians in their products liability suits as co-defendants, pleading both medical malpractice and other associated tort claims against these physicians, and often premising such claims upon the decision to prescribe for a so-called “off-label” or unapproved use. Thus, the continuing expansion of off-label uses of drugs and devices has led to numerous potential legal minefields for the prescribing physicians.

Often, physicians do not realize that, to a lay person, the use of a drug or device for an indication that has not been approved by the FDA may sound as though the physicians are somehow “experimenting” on their patients with off-label uses. While “[a] physician is free to use a medical device for an off-label purpose if, in the physician's medical judgment, he or she believes that use of the device will benefit the patient,” it also stands to reason that “[b]ecause the off-label use ' is a matter of medical judgment, a physician may be subject to medical malpractice liability for the exercise of that judgment.” Alvarez v. Smith , 714 So.2d 652, 653 (Fla. App. Ct. 1998). It is important for prescribing and implanting physicians and their counsel to be aware of the murky and shifting arguments plaintiffs may make against decisions to prescribe off-label. This three-part article explores the potential theories of liability for off-label prescriptions that have been employed, with mixed success. It will also discuss strategies for physicians to use to protect their power to prescribe approved drugs and devices in an off-label manner, in their exclusive medical judgment, while protecting themselves from a threat of potential future litigation or liability.

Off-Label Prescription Has Always Been Here

Since federal legislation first required FDA approval of prescription pharmaceuticals and medical devices, the federal government has made clear that it did not intend through regulation of the approval of drugs and devices to restrict or hamstring physicians' ability to utilize those drugs and devices in ways not specifically approved by the FDA. For example, a 1994 amendment to the Food, Drug and Cosmetic Act stated that “[n]othing in this chapter shall be construed to limit or interfere with the authority of a health care practitioner to prescribe or administer any legally marketed device to a patient for any condition or disease within a legitimate health care practitioner-patient relationship.” 21 U.S.C. ' 396 (2010). The U.S Supreme Court has recognized that “'off-label' usage of [drugs and] medical devices ' is an accepted and necessary corollary of the FDA's mission to regulate in this area without directly interfering with the practice of medicine.” Buckman v. Plaintiffs' Legal Comm. , 531 U.S. 341, 350 (2001). While the FDA prohibits promotion of unapproved uses by manufacturers of FDA-approved drugs and devices, it does not regulate or prohibit a physician's decision in his or her independent medical judgment to use an approved drug or device in an off-label manner. Even the general prohibition on manufacturer promotion of off-label uses does not bar the manufacturer from providing reprints of medical journal articles and reference publications regarding off-label uses to physicians, as the FDA recently made clear in its recent revisions to industry guidance regarding dissemination to physicians of medical literature discussing off-label uses. See Guidance for Industry – Good Reprint Practices for the Distribution of Medical Journal Articles and Medical or Scientific Reference Publications on Unapproved New Uses of Approved Drugs and Approved or Cleared Medical Devices, 74 Fed. Reg. 1,694 (Dep't of Health and Human Servs., Jan 13. 2009). The FDA's revised Guidance notes that “off-label uses or treatment regimens may be important and may even constitute a medically recognized standard of care.” Therefore, if the physician requests it, the agency permits dissemination of literature regarding unapproved uses to prescribing physicians, as long as it is truthful and not misleading. Id.

Off-Label Uses

Off-label or unapproved uses of drugs or devices may take many forms and may vary slightly or greatly from the FDA-approved indications for use of the drug or device. For example, a physician may prescribe an FDA-approved pharmaceutical to a pediatric patient, which is an off-label use if the drug is not specifically approved for use in children. (Many pharmaceutical products are not approved for use by children, simply because children are not usually studied in clinical trials.) Similarly, a physician may employ an FDA-approved implantable medical device to treat the indications for which it is approved, but may implant the device in a different location than where it is generally used, or implant multiple devices even though this is not a specifically approved practice. Another common off-label use is to implant an FDA-approved medical device for the approved indications for use, but use it in conjunction with an FDA-approved medication that is not specifically approved for use with that device. Even varying dosages or combinations of prescription medications in a different way than indicated on the approved labeling may constitute an off-label use of a prescription drug.

Frequency of Off-Label Prescriptions Would Surprise Most Patients

Although these uses of FDA-approved products may be considered off-label or unapproved uses, all are very common within the medical community. Estimates of the frequency with which physicians write off-label prescriptions vary wildly, with up to 60% of all drug prescriptions estimated as off-label uses. See James M. Beck, Elizabeth D. Azari, FDA, Off-Label Drug Use, and Informed Consent: Debunking Myths and Misconceptions, 53 Food Drug Cosm. L.J. 71 (1998). Off-label use of drugs is particularly common in specialties such as oncology, where patients often face life-threatening conditions and their physicians are increasingly willing to attempt unapproved treatments in an effort to prolong their patients' lives. See Bryan Cote, Compendia: The Bridge Between FDA Approved Indications and Off-Label Usage, Oncology Bus. Rev., 10, 11 (2008), http://www.oncbiz.com/documents/OBR_0108_Compendia.pdf (estimating approximately 60% of cancer drugs are used off-label); see also Richard A. Epstein, Cancer Patients Deserve Faster Access to Life-Saving Drugs, Wall St. Jour., May 2-3, 2009, at A11. While there are numerous estimates of the frequency of off-label use of drugs, quantifying the frequency with which medical devices are prescribed off-label has proven more difficult. However, if up to 60% of all drug prescriptions are written for off-label uses, the probability is high that virtually every patient at some point during their life will receive a prescription medical product for an unapproved use, even though many may never realize it.

The General Public

The general public is largely unaware that off-label prescriptions are widespread and permissible when a physician chooses the off-label use in his or her independent medical judgment, without manufacturer promotion. See Beck and Azari, supra at n59 (citing Harris Interactive Poll, U.S. Adults Ambivalent About the Risks and Benefits of Off-Label Prescription Drug Use (Dec. 7, 2006), http://www.harrisinteractive.com/news/allnewsbydate.asp?NewsID=1126). The disconnect between public understanding of the implications of FDA approval and the scope of permissible off-label uses may lead patients who experience an undesirable outcome with an off-label treatment to blame their poor result on the lack of regulatory approval or the physician's decision to recommend an unapproved use. Generally, when this blame results in litigation, the claims alleged are general malpractice or negligence claims, in which the patient challenges the physician's decision to employ the drug or device in an unapproved manner. Sometimes a patient will assert that the regulatory status of the drug or device should have been disclosed before the patient consented to its use. Conversely, in rare circumstances, the decision not to use a drug or device for an accepted and common off-label use may be challenged as outside the applicable standard of care, where the patient alleges the physician should have attempted the treatment because it was a known option within the field.

This discussion continues in next month's issue of Medical Malpractice Law & Strategy.


Lori G. Cohen, a member of this newsletter's Board of Editors, is a shareholder with the law firm of Greenberg Traurig, LLP. She focuses on litigation and trial work for the pharmaceutical and medical device industries, products liability, and medical malpractice. She has been recognized by the National Law Journal as one of “The 50 Most Influential Women Lawyers in America” and “Top 40 Under 40,” which highlights the most successful 40 litigators under the age of 40 in the country. Sara K. Thompson is of counsel with the firm, and focuses her practice on pharmaceutical and medical device products liability litigation, as well as medical malpractice litigation.

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