Dr. Smith is an Assistant Radiologist at Massachusetts General Hospital; an Assistant Professor at Harvard Medical School; and Director of Regulatory Affairs at the Center for Integration of Medicine and Innovative Technology, Boston, MA.

The Food and Drug Administration (FDA) enjoys a broad legal mandate to ensure that medical products are safe and effective for their intended use--an authority that extends to medical devices routinely used by radiologists. Under the regulatory framework established by the agency, products are approved for market for specific clinical indications that are described on the FDA-approved package insert or labeling. Frequently, radiologists and other medical professionals see clinical uses for medical devices that lie outside of this approved labeling, which are known as "off-label" uses. Accordingly, the legality of such off-label use is a common question in our specialty.

To answer this question, it is essential to understand the operation of the FDA regulatory system. ¹ At a basic level, the agency regulates the marketing of medical devices, not their use by licensed physicians. This concept, reflected in what is known as the "practice of medicine doctrine," is based in the concept of federalism, the idea that certain governmental functions are reserved for the federal government and others for the various state governments. The regulation of interstate commerce, such as manufacturing and nationwide distribution of medical devices, is a federal power. Regulation of medical practice, as seen with the control of physicians through the medical licensure process, is traditionally a power found in the states.

The FDA, through various documents and policy statements issued over the years, has acknowledged that it does not regulate the practice of medicine, such as the use of medical products by a licensed physician in the care of a specific patient. More recently, what is essentially the practice of medicine doctrine has been articulated specifically in statutory law as part of the Food and Drug Administration Modernization Act of 1997. Together, these documents demonstrate long-standing agency deference to a physician's freedom to practice medicine without FDA oversight.

In operation, the practice of medicine doctrine means that the FDA does not regulate the use of any FDA-approved product by a licensed physician in medical treatment. ²

Accordingly, the off-label use of a medical device is, in most cases, completely legal from a federal regulatory standpoint. When such use occurs, the FDA recommends that the treating physician be knowledgeable in that use and keep records as to the outcome. However, as the agency does not regulate the practice of medicine, these recommendations are only that, and do not carry with them the force of law.

Potential regulatory limitations on off-label use

While broad, the ability to use medical devices off-label free from federal regulatory scrutiny under the practice of medicine doctrine is not absolute. One limitation lies in actions taken by a physician that could be seen as commercializing a product. For example, widely publicizing the off-label use, actively and aggressively seeking patients for that use, or treating a large number of patients with that use could be seen as such commercialization, though the actual boundaries are not well defined in practice. Should a treating physician's actions be seen as commercializing a product, the protection afforded by the practice of medicine doctrine is lost and the physician becomes subject to applicable FDA regulations, just as a manufacturer or other commercial entity is.

Another limitation lies in off-label use that is conducted primarily to gather data in a clinical trial. Where research is the primary goal of device use, a regulatory mechanism known as an "investigational device exemption" (IDE) is required. An IDE carries with it extensive federal requirements designed to ensure the safety of patients and the integrity of a clinical study. Importantly, an IDE is not strictly necessary when data is ancillarily collected as part of off-label treatment use, though data gathered in this fashion cannot subsequently be used to support FDA approval for that off-label use.

Additional legal considerations

The practice of medicine doctrine means that radiologists may use FDA-approved products off-label freely for treatment of their patients, provided that the radiologist's actions do not commercialize that off-label use or that its primary purpose is not data collection. However, it is important to realize that the doctrine in no way mitigates other legal exposure that a radiologist may face as a consequence of that off-label use, such as medical malpractice or informed consent actions.

All physicians engaged in medical practice are liable for damages from negligent injury to patients under the legal doctrine of medical malpractice. In these actions, a key question is whether the medical procedure or service at issue satisfied the standard of care--that is, the standard of medical practice expected of any physician. From the standpoint of legal theory, the exposure faced by the physician engaged in off-label use of medical devices is no different from that faced by any physician, i.e., that off-label use must lie within the accepted standard of care. However, there is the possibility that the existence of off-label use may be misinterpreted by both judges and juries as an indication that the standard of care has not been met, potentially influencing judicial outcome.

Informed consent

Another area of concern for the physician engaged in off-label use of medical devices is informed consent. ³ This doctrine mandates that a patient must be told of a procedure's risks, benefits, and alternatives, and holds a physician liable for any patient injury that results from legally inadequate consent. Courts examining informed consent in the setting of off-label use have split on the issue of whether information regarding a device's regulatory status is required as part of the consent process. However, a growing majority of courts have held that the regulatory status of a medical product is not germane to the risk faced by patients, and therefore does not have to be disclosed. ^4,5 Still, informed consent lawsuits are often very fact-specific, and the outcome of any given case is dependent on those facts. Under these circumstances, it is useful to remember that there is essentially no legal downside to providing the patient with more information than courts have generally required.

Institutional issues

There are a variety of institution-specific issues that surround off-label use. Investigational review boards (IRBs)--the local entities that oversee human clinical trials--not uncommonly become involved in the off-label use of medical devices by physicians and other healthcare professionals within their institutions. If gathering data is part of such use, IRBs will almost certainly become involved. In addition, institutional risk management authorities may also be interested in off-label device use, particularly if that use could be seen as increasing a hospital's legal exposure.

Conclusion

Off-label use of medical devices is a common practice and is generally legal under existing FDA regulations, as long as that use does not rise to the level of commercialization or is intended primarily to collect clinical data. However, there are a variety of legal and institutional concerns that impact these activities that should be considered whenever there is ongoing off-label use of medical devices. By fully considering these implications, radiologists may treat their patients successfully while avoiding potential legal, regulatory, and administrative pitfalls. AR