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,
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.
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 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.
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
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
Another area of concern for the physician engaged in off-label
use of medical devices is informed consent.
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.
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.
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
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.