which targets a pathway of which PIK3CA is a part,
and Avastin (bevacizumab), which slows growth of
blood vessels that feed tumors. Torisel is approved
for kidney cancer. Avastin is approved, alone or in
combination with other drugs, for some patients
with colorectal, lung, brain, kidney, cervical and
ovarian cancers. Neither drug has been approved
for head and neck cancer, and the NCCN does not
include these therapies in its guidelines for head
and neck cancer treatment.
A DOSE OF CAUTION
Some patients, like Simpson, receive approved
drugs as part of clinical trials testing whether the
drug could be useful for patients beyond the group
identified in the drug’s FDA label. In other cases,
patients may be prescribed drugs off-label by their
physician outside the context of a clinical trial.
Despite some encouraging outcomes, physicians
and scientists urge caution in choosing off-label
treatments outside of a trial that are not considered
standard and are not listed in the NCCN guide-
lines. “As physicians, we are in a tough spot,” says
Jonas de Souza, a medical oncologist specializing in
head and neck cancer at the University of Chicago
Medicine. “We want to treat our patients, provide
them the best care available. With all the advances
in oncology, there is the potential to start prescrib-
ing things we don’t have evidence for yet.”
Immunotherapy drugs, which unleash the body’s
immune system to treat cancer, are attractive for
off-label use, both because they achieve notable
results in some people and because for many, the
side effects are easier to control than those of
chemotherapy. “There’s a high potential of [saying],
‘Oh, let’s try Keytruda in this cancer’ before the
evidence is there,” de Souza says.
In a poster presented in March 2017 at the
American Society of Clinical Oncology (ASCO)
Quality Care Symposium in Orlando, Florida,
de Souza and his colleagues assessed data from
insurance claims for off-label use of Keytruda,
which is approved for treating advanced NSCLC,
melanoma, classical Hodgkin lymphoma and
squamous head and neck cancer. Looking at
data from private insurance and Medicare and
Medicaid claims from January 2015 to September
2016, the researchers found that 292 out of 871
patients who put in claims for Keytruda were
taking the drug off-label.
The rise of
and targeted agents
is complicating off-
• Seek a second opinion from a specialist in your type of
cancer. If you are considering a targeted therapy, find
someone who has expertise in tumor genomics.
• Ask if the drug you are considering has been tested
in people with your type of cancer. Was it tested in a
randomized clinical trial or just in a few people?
• Look into what is known about the side effects of any
off-label drug or drug combination you are considering taking.
• Get advice on what to do if your insurer denies your claim.
The Patient Advocate Foundation (
helps patients navigate appeals and financial aid.
• Look for clinical trials that include the off-label drug you
are interested in trying.