Vol 22, 2013 Annals of Health Law 421
PROSECUTIONS OF PHARMACEUTICAL COMPANIES
American Society of Clinical Oncology reported that an astounding fifty
percent to seventy percent of cancer treatments are prescribed off-label.91
In pediatrics, it is well known that although many drugs have not been
tested in children and therefore cannot include label directions for use in
children, they nonetheless may be the only effective treatment in the
pediatric population.92 A similar situation exists in elderly populations.
Medicare and private insurers pay for some off-label uses of drugs, and
such can be considered state of the art.93 Without off-label prescribing, key
populations of patients may go without needed medicine.
Just as clearly, however, off-label prescription of drugs can be
dangerous and sometimes is based on unproven assumptions about benefit
and risk.94 For those uninitiated in the complex and expensive New Drug
Application process, it may seem desirable for pharmaceutical companies to
test drugs in all patient populations, at all doses and for all disease
endpoints. It is beyond the scope of this paper to discuss in detail the new
drug approval process.95 But the nature of the rigorous testing the FDA
justly requires to prove both safety and efficacy leads to narrowly-focused
testing protocols where the drug is given to a discrete population of
patients, at specific doses, and for specific diseases or conditions. Once
years of testing have been completed and the drug is approved for sale, it
often is not in the manufacturer’s best interest—nor is it required by any
regulation—to begin again to test the drug in other populations. As aptly
described by one commentator,
[T]hough new indications may be added to a drug’s label through
a supplemental new drug application, this occurs infrequently:
generic drugs lack a corporate sponsor to bear the required
expenses, and for brand-name drugs that are already widely used
off-label, conducting a costly clinical trial that could produce non-
supportive evidence is a potentially risky business decision.96
Thus, whether a drug is effective either for conditions/diseases other than
those approved in the label or in populations other than those in which it
was tested, is often a question left to the medical community to determine
after the drug is on the market.
91. Id. at 235; see also Girard, supra note 10, at 131.
92. Kesselheim, supra note 90, at 225.
93. Burroughs, supra note 29, at 556.
94. Kesselheim, supra note 90, at 235-38; see also Randall S. Stafford, Regulating Off-Label Drug Use—Rethinking the Role of the FDA, 358 N. ENGL. J. MED. 1400, 1427 (2008).
95. See generally Applications For FDA Approval To Market A New Drug, 21 C.F.R. §