IROs Help Decide Medically on Approved Uses of Off-Label Drugs for Cancer

Tue Mar 24, 2009 8:00am EDT

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LANSDALE, Pa., March 24 /PRNewswire/ --- As the Food and Drug Administration
(FDA) approves new cancer fighting drugs for the market, researchers often
study their use for non-approved, or off-label, treatments. When the studies
show promise for combating other forms of cancer, physicians may prescribe
these drugs to patients. By some estimates, nearly 20 percent of all drugs
prescribed in the United States are for off-label use. Oncologists prescribe
cancer drugs off-label about 60 percent of the time.

"Researchers continually explore the effectiveness of off-label drugs on
cancer, which changes the boundaries of the standard of care," said Joyce
Muller, president of NAIRO, a national trade organization of independent
review organizations (IROs). "Because cancer drugs are often expensive,
off-label use requires that health plans look closely at whether these drugs
meet plan language for patient reimbursement or are investigational or

What is Off-label Use?

The FDA approves a drug for a specific treatment and considers any other use
"off-label." Because applying for FDA approval is expensive, it often doesn't
make financial sense for the drug manufacturer to pursue multiple uses.
Although the use of many drugs off-label has been thoroughly researched and
often considered the standard of care, this doesn't mean the FDA approves
their off-label use.

A few promising cancer drugs are approved by the FDA for multiple use,
including Avastin, Revlimid, Imatinib and Erbitux. For example, the agency
initially approved Avastin for metastatic colorectal cancer. Then later it
approved the drug for non-small cell lung cancer (2006) and breast cancer
(2008). Used off-label, however, some doctors see Avastin as a "miracle drug"
and may prescribe it for treating other cancers, including prostate, renal
cell, head and neck, pancreatic, ovarian and hepatocellular.

Determining Appropriate Off-label Use

Deciding whether an off-label drug is medically necessary for cancer, matches
health plan language and is the standard of care requires an oncologist
specializing in that specific treatment. "When a drug's off-label benefits are
supported by the latest peer-reviewed medical literature, it alters the
standard of care," Muller said. "Through an IRO, payers can access oncologists
up-to-date on the latest off-label cancer drug studies."

How a drug was studied can be an issue, however. To determine the validity of
a study, an oncologist must understand its parameters, including its clinical
characteristics, current laboratory studies and monitoring studies. Because
IROs provide oncologists who also work at leading research centers, they can
determine which studies support off-label drug use as the accepted standard of
care for plans. This helps health plans and UR professionals approve or deny
patient reimbursements based on the latest medical evidence, as well as update
their plan language and coverage policy. This may reduce the number of appeals
for off-label drug coverage and any associated administrative costs a plan

"When deciding if it's appropriate to reimburse patients for using off-label
cancer drugs, health plans should tap into the oncology expertise of an IRO,"
Muller said. "Because plans cannot afford to hire every oncology specialist
needed, an IRO can provide supporting evidence that will show whether the
off-label use is experimental, investigational or the standard of care."

For help finding an IRO to review the off-label use of cancer drugs, contact


NAIRO works to promote the value and integrity of the independent medical
review process. Its members embrace an independent, evidence-based approach to
medical review for resolving coverage disputes between enrollees and their
health plans. For more information, visit 


Martin Middlewood of Frontline Strategies Inc., +1-360-882-1164,
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