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Posted on 27. May 2021

What Do I Do When I Receive a Denial for an Off-Label Medication?

By Abbie Cornett

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The other day, I received a phone call from a patient asking for help with an appeal. When she explained her situation, my heart sank. Her physician had prescribed intravenous immune globulin for off-label use, and the insurance company had denied it based on the grounds that its use for the condition is "experimental."

Like many newly diagnosed patients, she didn't know what the insurance company meant by off-label use. The U.S. Food and Drug Administration (FDA) approves drugs for specific health conditions. When a doctor prescribes a drug off-label, he or she prescribes it for a different condition or at a different dosage than the FDA has approved.1 But just because the drug has not been approved for the condition the physician is prescribing it for doesn't mean it won't be effective. An off-label medication is often beneficial for patients with rare diseases who have exhausted all other approved options.

She then asked me: If it works, why is it off-label? Drug manufacturers frequently don't try to get a new FDA approval for a medication because of the time and cost involved. To obtain additional indications (approvals) for a drug already approved requires the manufacturer to submit a supplemental drug application.2 And, many manufacturers don't feel the expense of the additional approval is worthwhile.

For patients, the biggest problem with an off-label medication prescription is getting the insurance company to reimburse it! Many insurance companies will not pay for an expensive drug that's used to treat a condition that is not on the approved drug label.3

When an insurance company denies a medication, an appeal has to be filed. The appeal process is common among all insurance plans:

  • First Appeal. During the first appeal, the patient or his or her doctor can contact the insurance company to ask it to reconsider the denial. Additionally, the physician can request a peer-to-peer review with the insurance company's medical reviewer to discuss the decision.
  • Second Appeal. If the first appeal is still denied, it is typically reviewed by a medical director at the insurance company who was not involved in the claim decision. The goal of this appeal is to prove the request should be accepted within the coverage guidelines.4
  • Final Appeal. If the medication is again denied, the patient has the right to have an independent external medical reviewer and another doctor who specializes in the disease determine if the medication will be approved.

Receiving a denial letter can be frustrating, particularly when dealing with a chronic illness. But a denial letter doesn't mean there are not options. Everyone has the right to the appeal process. The insurance company's requirements for an appeal are stated in the explanation of benefits.


1  MedicalNewsToday. What to Know About Off-Label Drug Use. Accessed at
2  Wittich, CM, Burkle, CM, and Lanier, WL. Ten Common Questions (and Their Answers) About Off-Label Drug Use. Mayo Clinic Proceedings, 2012 Oct; 87(10): 982–990. Accessed at
3  American Cancer Society. What Is Off-Label Drug Use. Accessed at
4  JDRF. Denials/Appeals: What to Do When Your Insurance Company Denies You Coverage. Accessed at


Comments (2) -

Mark Leventhal
10:28 AM on Friday, August 20, 2021

from my experience, the doc has to find any published article where the drug has worked for the diagnosis they are trying to get the exception for. I even one patient has had success they generally will approve it.

William E. De Lorenzo
3:43 PM on Sunday, August 22, 2021

I have a "Cousin" in Seattle, WA who worked for an Insurance Company in the "Claims Review and Approval" Area.  She told me several years ago that many times, JUST "ONE" REVIEWER,"who obviously is "Detached" from the Claim Situation and the "Claimant,"  "Approves" or "Denys" the Claim. Obviously, the "RED FLAG" is almost ALWAYS the "AMOUNT" of the Claim!!! If I'm relatively accurate, IVIG can cost over $300,000 a year, or more, depending on the Hospital , and the Frequency of the Treatments!! She said to ALWAYS APPEAL it at the "Highest" LEVEL, because sometimes they  don't have the "Time" to read EVERYTHING!  

Twenty-two years ago, when I needed "Prostate Cancer Radiation," and my Medical Insurance turned down my Request for  the "Safer", "Much Improved" and "More Effective" Radiation Treatment for me at that time, at Johns Hopkins, because they determined that it was an "EXPERIMENTAL" Treatment! Well I knew OTHERWISE!! So I IMMEDIATELY called my Carrier''s Company, and asked how they came to THAT conslusion? I also asked if they had  spoken with  the Dr. who "Ordered" and was HIGHLY involved in my "Treatment Plan, as I had REQUESTED them to do, BEFORE submitting the Claim!!"  It turned out, that they had spoken to the "Person" behind the "Appointment Coordinator"s Desk," because they couldn't get the Doctor.  That "APPOINTMENT' COORDINATOR," who had NOTING TO DO with the "Treatment" was  the one who TOLD them it was "EXPERIMENTAL!!" She had NO idea that they had been using that Treatment for 8 uears. with VERY HIGH Success!!!

After CALMING down, I told them that conclusion was TOTALLY UNACCEPTABLE, the pers]on to whom they spoke  was just a Hospital EPLOYEE, at the FRONT DESK, and didn't KNOW ANYTHING about the Treatment.  I told her that, they needed to  SPEAK with my "Radiology Doc," after which they needed review my Request once more, and that I wouldn't accept ANY other "Response," other than "IT WAS APPROVED!!"  ULTIMATELY; "IT WAS!!!  Sometimes, we have to FIGHT  for what WE know is RIGHT!

---------- Forwarded message ---------
From: Alan & Linda Sinclair <alsin6206@verizon.net>
Date: Tue, Jul 27, 2021 at 5:40 PM
Subject: Fwd: August 2nd, Myasthenia Gravis Support Group of Atlanta, GA Zoom Mfeeting
To: delorenz@umd.edu <delorenz@umd.edu>

Please forward to group.
Thanks, Alan

-----Original Message-----
From: Alexis Rodriguez <mggatalk@gmail.com>
Sent: Tue, Jul 27, 2021 4:42 pm
Subject: August 2nd, Myasthenia Gravis Support Group of Atlanta, GA Zoom Meeting

MG Community,

You are invited to a Zoom meeting.
When: Aug 2, 2021 06:30 PM Eastern Time (US and Canada)

Register in advance for this meeting:

After registering, you will receive a confirmation email containing information about joining the meeting.

Kind Regards,

Alexis Rodríguez
Patient/ Leader/ Volunteer
Georgia MG Support Group

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