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BACKGROUND ON OFF-LABEL USE OF PRESCRIPTION DRUGS
"Since the early 1960s, the Food and Drug Administration (FDA) has required that drugs used in the United States be both safe and effective.
The label information -- on the container, in the package insert, in the Physicians Desk
Reference (PDR), and in any advertising -- can indicate a drug's use only in certain "approved" doses and routes of administration for a particular condition.
The use of a drug for a disease not listed on the label, or in a dose or by a route not listed on the label, is
considered to be a "nonapproved" or "unlabel" or "off-label" use of the drug.
Physicians, based on their knowledge and on available current information, may use a drug for a use not indicated in the "approved" labeling if it seems reasonable or appropriate. With the rising costs of health
care and the desire to curb these costs, the issue of coverage and reimbursement for the "off-label" use of drugs has become an issue.
OF PROPOSED CMS POLICY CHANGE:
The policy would set a dangerous precedent that it
a physician's ability to meet the unique needs of individuals
who face the diseases called lymphoma and other cancers. Specifically, adoption of this policy
is likely to:
pain and suffering of many patients fighting cancers.
an additional disincentive for companies to develop and
test new cancer drugs, which we now export proudly to
Increase the costs
as many approved drugs are less specific than new drugs
and can therefore sometimes cause undo complications.
clinical research by increasing the the need for
participation in additional randomized clinical trials in order to
more formerly validate off-label
uses - which are numerous.
slow discovery of "new standards of care" as
patients facing cancer and lower the morale of oncologists
who treat these individuals.
writes: "It is bad enough that I have to fight this
life-threatening disease and the side effects of treatment,
but now I must also continuously fight with my
insurance company for coverage. It gets so
"A 1991 study by the U.S. General Accounting Office found that one-third of the drug treatments performed by cancer doctors were off-label; more than
half of cancer patients received at least one drug for an off-label
"A 1997 survey of 200 cancer doctors by the American Enterprise Institute and the American Cancer Society found that 60 percent of them prescribed drugs off-label.
Frequently the standard of care for a particular type or stage of cancer involves the off-label use of one or more
"Also, the FDA generally does not approve multidrug regimens themselves, in part because such regimens are so numerous as to make
separate approvals impractical.
"Multidrug regimens change over time as doctors try different combinations and observe which regimens seem to produce the greatest benefit for patients."
"Cancer treatment is always evolving.
Researchers continually conduct studies to determine new uses for already marketed drugs and to find effective combinations of drugs for new indications. The results of these studies are published in peer-reviewed medical journals. When a new treatment approach seems to produce better outcomes for patients, other doctors adopt it and it may become a
new standard of care." Such as CHOP + Rituxan has
become for diffuse aggressive lymphomas.
Q&A on Off-label Drugs - Cancer.gov
* What is off-label use?
* Why is off-label use of drugs so common in cancer treatment?
* Can off-label drug use be harmful?
* Will my health insurance cover drugs used off label for cancer treatment?
* What questions should I ask my doctor about off-label drug use?
* What states require insurance coverage of off-label cancer drugs?
HOW TO HELP:
then, please show your support for this effort by
adding your name to
We will also send a
copy of the letter, including the personal stories and endorsements,
to our representatives who have oversight
authority over CMS:
Committee - House Ways and Means
Education, Labor and Pensions Committee - Senate
NOTE: We encourage you to share the letter with
the local media and with your representatives in government as well.
Finally, I want to express my personal thanks
to all the advisors who assisted in drafting the letter, in particular
Mary Hanson and Carol Lee, and to the patients who shared their personal stories
that we will attach to the letter.
Patients Against Lymphoma