Appeal letter sample: Off Labeling

Template letter for professionals to complete when using a medication to treat IBD that is considered off-label. Healthcare provider completes letter and patient submits to their insurance company providing documentation of the medical necessity of the drug being prescribed to the patient.



RE:      Patient:



            Dates of Service: Ongoing

Dear Sir or Madam:

I am writing on behalf of ______________________ to appeal your non-coverage of MEDICATION.

Essentially, INSURER’s rationale for denying coverage is that MEDICATION is not FDA approved for the treatment of _______________.  This is not a credible rationale.  First, I am enclosing copies of peer-reviewed medical literature that demonstrates that the use of MEDICATION is well-established for the treatment of _____________.  Second, this patient has been tried and failed on all other treatment options, which have failed to control her _____________.    Thus, your non-coverage decision should be reversed.


In 1982, the FDA issued a Drug bulletin addressing the prescribing of medication for “unlabeled” or off-label uses.  The FDA itself states that the Food, Drug and Cosmetic Act “does not, however, limit the manner in which a physician may use an approved drug. Once a product has been approved for marketing, a physician may prescribe it for uses or in treatment regimens or patient populations that are not included in approved labeling. Such “unapproved” or, more precisely, “unlabeled” uses may be appropriate and rational in certain circumstances, and may, in fact, reflect approaches to drug therapy that have been extensively reported in medical literature”.

The term “unapproved uses” is, to some extent, misleading. It includes a variety of situations ranging from unstudied to thoroughly investigated drug uses. Valid new uses for drugs already on the market are often first discovered through serendipitous observations and therapeutic innovations, subsequently confirmed by well-planned and executed clinical investigations. Before such advances can be added to the approved labeling, however, data substantiating the effectiveness of a new use or regimen must be submitted by the manufacture to the FDA for evaluation. This may take time and, without the initiative of the drug manufacturer whose product is involved, may never occur. For that reason, accepted medical practice often includes drug use that is not reflected in approved drug labeling.

With respect to its role in medical practice, the package insert is informational only.

FDA Drug Bulletin, Volume 12, Number 1, pages 4-5 (April 1982).  If the FDA itself states that its’ labeling is not intended to limit the prescribing of medications for off-label uses, then insurers should not be permitted to refuse coverage of off-label uses solely based on the fact that the use is off-label.  Clearly, such a result would run contrary to the FDA’s own intent regarding the effect of its labeling.

Many drugs, from 6MP to Asacol to Methotrexate, are used to treat Crohn’s disease are used off-label, without FDA approval for this use.  Yet, these drugs are covered by INSURER and every other insurance company or payment source including Medicaid and Medicare.  Therefore, since INSURER has covered other medication not expressly FDA approved to treat Crohn’s, the INSURER should continue to do so in the case of Mr. Patient

In fact, MEDICATION has been used to treat DISEASE.  I enclose copies of medical journal articles that support its use.  To summarize [SUMMARIZE AND ATTACH COPIES]. 


My patient suffers from DISEASE.  As demonstrated by the enclosed medical records [colonoscopy reports, barium studies, office notes indicating medications that were tried and failed, office notes showing weight loss], my patient requires aggressive treatment.  She has been treated by each of the following, all of which have failed to alleviate her symptoms: _______________________________________________________________




The MEDICATION I propose to use at this time may have a beneficial outcome.  Unlike the other medications we have tried, MEDICATION may control her symptoms because




In each case, PATIENT’s condition was not improved.  However, when we tried a sample of MEDICATION, it was extremely effective. 

Thus, it is my opinion, based on my ___ years as a specialist in inflammatory bowel disease, that MEDICATION may be PATIENT’s best and perhaps only chance for remission.

III. Conclusion

For all of these reasons, I urge you to reverse your non-coverage decision.


Dr. X

Contact Information

For further information, call Crohn's & Colitis Foundation's IBD Help Center: 888.MY.GUT.PAIN (888.694.8872).

The Crohn's & Colitis Foundation provides information for educational purposes only. We encourage you to review this educational material with your health care professional. The Foundation does not provide medical or other health care opinions or services. The inclusion of another organization's resources or referral to another organization does not represent an endorsement of a particular individual, group, company or product.

About this resource

Published: May 1, 2012

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