Understanding Pediatric Off-Label Drug Use and Insurance Coverage

Among challenges facing people with narcolepsy, securing health insurance to cover expensive medications can be a source of anxiety and fear. My personal experience with health insurance has been a roller-coaster but ultimately, I am thankful to have decent coverage now (knock on wood). 

However, I’ve heard multiple stories recently (in news articles, at conferences and support groups) about insurance companies denying coverage for medication prescribed by doctors for children with narcolepsy because the medication is not FDA-approved for pediatric use

I believe this is unjust. Why? Because the majority of ALL children’s use of medication is not FDA-approved for pediatric use. This is called “off-label” use (meaning it has not been FDA-approved specifically for that patient population). 

Why are most medications not FDA-approved for children and therefore “off-label”? Because experimental testing on children and infants carries extra ethical problems of gaining informed consent from children. Pharmaceutical studies carry risks of physical or psychological harm that may be difficult to quantify. Ethically, the obligation to act in a child’s best interests entails protecting children from both the potential risks of research and the harms produced by the use of inadequately tested drugs, as well as respecting their autonomy (Read more here). 

Similarly, drug testing on the elderly and pregnant women in early clinical trials is not common because of the higher risks associated with these populations. So, clinical trials are usually conducted in adult populations and FDA-approved for adult populations.

Once the drug is on the market, doctors will likely prescribe the treatments for children. This means that the children who are truly experimented on are often those who need the treatment most. Maybe this seems unsettling, but this is the consequence of our nation’s ethical choice not to conduct widespread experimentation on children without their informed consent.

Therefore, “off-label” drug use is absolutely imperative to serve patient populations who are not represented in clinical trials, such as children, pregnant women and the elderly. 

Some pharmaceutical companies pursue supplemental FDA-approval for pediatric use after the drug is already on the market, but this is the exception and not the norm. The decision to pursue supplemental FDA-approval for pediatric use will depend on how many more years of exclusive patent rights the pharmaceutical company has on the drug and other economic and logistical factors. 

In closing, if your child is facing an illness like narcolepsy, it is important to note that some, if not all, of the medications prescribed for your child by a doctor may not be specifically FDA-approved for pediatric use. “Off-label” use does not mean inappropriate or unsafe use. It means that it hasn’t gone through the formal FDA approval process. Often times, there is data gathered and papers written by physicians about best practices for pediatric use of medications, especially the longer the drug is available on the market. 

Of course, there are always risks to taking any drug, even when specifically approved for your target population. FDA approval is not a guarantee of safety, it is a risk/benefit analysis. Many drugs are approved knowing that they can and will harm people, but that the benefits of having the drug available to all will outweigh the risks to some.

Deciding the best treatment for your child is a conversation between you, your child and your treating physicians. In my opinion, insurance companies denying coverage for a child’s medication because it’s not FDA-approved for pediatric use is unjust and unethical.

These companies are well aware that most drugs are not FDA-approved for pediatric use. I believe they are using this tactic when medications are expensive, as an attempt to avoid coverage. Furthermore, it’s my opinion that insurance companies are feeding off of cultural misunderstandings of “off-label” use in order to avoid covering treatments that could be life-saving and life-changing for children.  

In other words, they are hoping that you will give up. Patients, parents and doctors must be determined to appeal these initial denials. It may be helpful for the physican to send the insurance company copies of peer-reviewed journal articles or other respected sources that support the off-label use.


  1. Mark Patterson, MD, PhD on January 30, 2013 at 9:21 pm

    I agree, Julie, it is frustrating when we doctors prescribe what we think is the best medication for a patient, only to have the insurance company deny its use. Often the reason is because the medication is not on the patient’s formulary. Unfortunately, neither the patient nor the doctor has any say in what goes on the formulary. If you are denied a medication that your doctor thinks you need, appeal, appeal, appeal!

    • julie on January 30, 2013 at 9:28 pm

      Thank you for your insightful comment, Dr. Patterson! Your advise to “appeal appeal appeal” reminds me of the extreme importance of being one’s own advocate in the current healthcare system.

  2. James Fadden on January 31, 2013 at 11:32 am

    “Off label” use is often the standard of care for many conditions. Not prescribing (and paying for) such off label use would be sub-standard care.

    Off-label use and the medical standard of care


    Off-Label Uses Held “Medically Accepted” As A Matter Of Law


    • julie on January 31, 2013 at 2:25 pm

      Great point, Jim! Thanks for adding this comment.

      In addition to nearly all pediatric patients being prescribed drugs off label – up to 90% of all rare disease patients take drugs off-label. 60% of cancer patients receive at least one drug off-label. 80% of AIDS patients receive off-label treatment. 23% percent of pregnant women in their third trimester take at least one drug off-label. Thus, off-label drug use fulfills substantial interests in our current health care system.

  3. Alex Withrow on January 31, 2013 at 3:23 pm

    This is a fascinating, extremely well articulated post, Julie. A post like this could really help many people (children, their parents, etc) who are struggling to secure specific treatments.

    Well done!

    • julie on January 31, 2013 at 3:29 pm

      Thank you, Alex! If the post helps just ONE family better understand their child’s medication situation – it will be worth it. 🙂

  4. Sanya on January 31, 2013 at 4:57 pm

    I agree. At the same time, we have to be willing to do our part to get these meds approved. My son and I both have narcolepsy. I did not qualify for the adult study in our area, but he qualifies for the pediatric study. We are now in the process of setting up his appointments. I was surprised and disappointed to hear how few participants there are due to their parents’ unwillingness to have them miss sports activities, etc. Having experienced the misery of growing up with narcolepsy, this saddens me…I want there to e an answer for our children…and a better one than Ritalin!

    • julie on January 31, 2013 at 5:26 pm

      Hi Sanya – thanks for your great comment. Thank you so much for actively participating in trials. Once the drug company initiates a drug trial, patient participation is invaluable – so many thanks sent your family’s way!

      Personally, I’ve taken part in a few university-sponsored narcolepsy studies. One study in particular was a bit invasive, but I was determined to participate. It’s been SO rewarding to hear research updates from these scientists later and realize that my contribution is building a brighter future for narcolepsy.

  5. Sanya on January 31, 2013 at 5:39 pm

    Thank you, Julie! I am so grateful for and inspired by your website! I’m a newbie here, but I’ve been living with narcolepsy for 44 yrs…not diagnosed until 35 yrs old…for me, it wasn’t an onset…it was an explanation of my entire life!

  6. chrys on April 26, 2013 at 6:28 pm

    Thank you so much!! My husband and I are going through this exact thing with our now eight year old son. He was diagnosed a year ago and we have since changed insurance companies and he has been denied the use of Modafinil. We are going to continue to fight!

    • julie on April 26, 2013 at 6:46 pm

      I’m so glad this post is helpful to you. Keep fighting!

  7. Eric on May 25, 2013 at 11:14 am

    Thank you Julie for standing up for the people that have narcolepsy. I like to
    let people to know living with sleep disorders is not fun. I was first diagnosed
    with sleep apnea in 2006 thanks to my wife who is a R.N. she saw that was
    stop breathing multiple times though the night so she made me call our doctor
    who made me get a sleep study and failed it. I went to are family doctor who sent
    me to a sleep specialist doctor started me on a c pap machine it help a little but
    for last 6 years I still was falling asleep during the day my wife kept pushing to
    tell the doctor what was happening? Until she called my doctor and tell him what
    was happening he sent for a sleep study at night and the next day failed that study.
    Went to see him after the study I was diagnosed with narcolepsy. Then fun begin
    with the insurance company denied getting Modafinil so I kept calling every 2 to 3 weeks finally they approved me. I my advice when trying to get approved kept
    calling and document each call ask the persons name and case # and if you do not
    get any action ask for a manager or a supervisor to see what they can do. Plus make sure kept calling your doctor office and let them know what is going on
    with the insurance company so you doctor knows what is happening.

    • julie on May 25, 2013 at 12:29 pm

      Thank you for your comment, Eric. I’m so sorry to hear you’ve battled insurance issues too, but your great advice is invaluable for others!

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