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Patients Against Lymphoma


Advocacy > Background

Advocacy Alert: Limiting Off-label Use of Cancer Drugs 

Last update: 08/25/2007

Introduction | Background Articles | Our Letter | Endorse the Letter | Attachments


"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.



The policy would set a dangerous precedent that it would limit 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

  1. Increase mortality, pain and suffering of many patients fighting cancers.

  2. Become an additional disincentive for companies to develop and test new cancer drugs, which we now export proudly to other countries.

  3. Increase the costs as many approved drugs are less specific than new drugs and can therefore sometimes cause undo complications. 

  4. Will slow 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.  

  5. Will slow discovery of "new standards of care" as described below.)

  6. Will demoralize patients facing cancer and lower the morale of oncologists who treat these individuals.  

Joanne 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 overwhelming."


"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 indication." 

"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 drugs. "  

"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.

Source: 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?


We will also send a copy of the letter, including the personal stories and endorsements, to our representatives who have oversight authority over CMS: 

Health Committee - House Ways and Means

Health, 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.  



Karl Schwartz
Patients Against Lymphoma


Disclaimer:  The information on Lymphomation.org is not intended to be a substitute for 
professional medical advice or to replace your relationship with a physician.
For all medical concerns,  you should always consult your doctor. 
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