Kids and Off-Label Prescriptions

The practice of writing prescriptions for medications to be used "off-label" is fairly common, but has received increased attention over the last year. Using a medication for a condition or illness for which it was not originally approved, or using a medication on a population (such as children) that it was not tested for, are both examples of off-label use that have come under fire recently.

Findings published earlier this year in the Archives of Pediatric and Adolescent Medicine, as reported in Newsweek, cast serious doubt over this practice, noting that it is very common. Another study published last year in the Archives of Internal Medicine and reported on-line at the Washington Post, noted that often, the off-label prescriptions written are not based on scientific evidence of the drug's effectiveness for the illness or age-range for which it is being prescribed.

What do you do, then if you are the parent of a sick child or of a child battling a frustrating or painful condition, and find that your doctor has prescribed a medication "off-label"?

As the parents of a child with severe atopic dermatitis, also known as eczema, we have faced this question several times over the last five years. The first time we experienced this practice, Larkin was 18 months old. She was prescribed a topical corticosteroid that was not approved in children under two. The pharmacist insisted on calling the doctor's office before filling the prescription. We were shocked.

Should the pharmacist really be questioning the doctor?

Could Larkin wait another 24 hours without the relief the ointment was supposed to give her?

Should we be concerned that this medication was not approved for our baby, who not only was under age for the prescription, but also underweight and below average height for her age, possibly making accurate dosing even more difficult to determine?

The prescription was eventually filled and approved, and has not, as far as we know, caused any irreversible damage to our daughter.

We went through this same experience again and again, on several occasions shocking the pharmacist into listing possible dire consequences, and urging a second medical opinion on some of the drug choices.

In order to avoid withholding medication that has the possibility to help Larkin while still safeguarding her health, we have developed a list of considerations for determining whether to pursue the medication prescribed by the doctor, or to ask for an alternative.

The first consideration is very unscientific; do we trust the doctor? Our daughter's allergist is someone whom we trust implicitly to do what is best for her in treating her eczema. His current medicinal regimen includes a shot manufactured and marketed for severe asthma sufferers over 12. Our daughter does not have asthma, and is five years old. The shot therapy has worked very well for her atopic dermatitis, a condition closely related to asthma.

The second consideration is financial. Will our insurance company approve the medication, since it is being used off-label? If not, are there steps we can take to get it approved, or as a last resort, can we afford the medication out of pocket?

The third consideration is the possible consequences of using the drug. Unfortunately, like the shot for asthma, there are often no studies to determine possible side effects of using medications on children that were intended for adults. Testing new drugs for children is more costly and time-consuming, and the standards of the Food and Drug Administration (FDA) are more rigorous for prescription drugs intended for children. If a pharmaceutical company can gain FDA approval for a new drug, then doctors can prescribe it any way they perceive as useful. It is only the drug companies who are prevented from marketing the drugs for uses other than those they were approved for.

Without studies or even past experiences to learn from, we must sometimes just go with our gut feeling. If we trust the doctor, the first consideration, this is a small leap of faith.

A fourth consideration is the severity of our daughter's eczema at the time. Larkin's eczema is not just a scaly itch. It is, at times, an all-consuming, itching, burning prison comprised of 90 percent of her skin. If she is scratching to the point of drawing blood and risking serious infection, then taking a chance on an off-label prescription is worth gaining some relief for her.

Safeguarding the health of your children is a serious business, and with the multitude of information now available to parents, the right course of action is not always clear. Some parents may, if they find out their child has been prescribed a medication for an off-label use, decide not to administer the medication.

That fact alone, however, should not be used to determine whether the medication is right for the illness or condition it is being prescribed for. Ask the doctor if there is any scientific evidence to back up using the medication in your child's case, and if not, if there is any anecdotal evidence the doctor knows of to justify the use. Most importantly, if you have faith in the doctor's capabilities, be open to trying something new.

Sources:

Sandra B. Boodman, "Off Label, Off Base?". Washingtonpost.com.

Eve Conant, "Adult Drugs for Children: A Growing Problem?". Newsweek.