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.