NCHR Testimony at FDA on Clinical Trials for Atopic Dermatitis in Pediatric Patients

My name is Dr. Margaret Dayhoff-Brannigan and I am a senior fellow at the National Center for Health Research. Our research center scrutinizes scientific and medical data and provides objective health information to patients, providers and policy makers. We do not accept funding from pharmaceutical companies, and therefore I have no conflicts of interest.

Thank you for the opportunity to speak here today.

I completed my Ph.D. in Biochemistry and Molecular Biology at the Johns Hopkins School of Public Health.  In addition, my two-year-old has been diagnosed with moderate atopic dermatitis. I bring the perspectives of both a researcher and parent here today.

My child started to have eczema on his face, scalp, chest and back before he was 3 months old. It took months of new lotions, bath soaps, detergents, diet changes, allergy testing and prescription topical corticosteroids to finally get his eczema under control.  I am thankful we were able to treat him, but I understand the impact this disease has on patients with an unmet need.

However, it is critical to ensure proper clinical testing on all age groups before subjecting pediatric patients to treatment options that may not be safe or effective for children their age.

Eczema in children under the age of 2 is a very different disease than older children and adults. It covers a much larger portion of the body, and is often found on the chest, back and face. Eczema in this age group is often related to food allergies. For those reasons, it is inappropriate to extrapolate results from adult clinical trials to this age group.  In fact, subgroup analyses are important for all children.  Metabolism rates change considerably during childhood, so it is important to have clinical trials that analyze dosages for different age groups and weights.

Pediatric patients with eczema are at higher risk for allergic reactions to any exposure, which puts them at risk when trying new treatments. This elevated risk should be weighed when considering the risk-benefit ratio for any treatment.

The bottom line: Pediatric patients with severe disease that do not respond to topical corticosteroids might appropriately be enrolled in a clinical trial for an experimental treatment that has inadequate testing. But these trials should not be open to pediatric patients that have not exhausted safer options such as lifestyle changes and topical corticosteroids.

We ask you to be cautious as you consider systemic treatments for atopic dermatitis in pediatric patients. Atopic dermatitis is non-life threatening, so while it impacts quality of life, children should not be exposed to a treatment that could cause any type of permanent or substantial harm, however rare.

Let’s consider the ethics.  Children are unable to make informed decisions, but it is also difficult to put parents in that position with therapies that haven’t been tested on large numbers of children over a long period of time.

One major problem is that drugs currently used off label to treat atopic dermatitis systemically all carry boxed warnings about side effects and risks, and the long-term effect of the treatments is unknown.  Despite the black boxes and warnings, these drugs are being used in pediatric patients where there is very little data on their safety and efficacy. Patients in these situations would be better served by participating in a clinical trial where parents are made aware of the risks and the patients are carefully monitored by doctors who are knowledgeable about the risks.  Under those very careful conditions, children could be randomized to receive a novel treatment using the standard of care as a control.

Parents may feel desperate to try treatments option for atopic dermatitis for their children, but they may not understand that previous research is often inadequate to prove how safe or effective a new treatment is for their children.  It is the FDA’s job to make sure that treatments are first proven to be safe and effective in animal models for all ages.  There should also be safety data from adults and older pediatric patients before it would be ethical to begin testing younger children.

If a systemic treatment for atopic dermatitis is approved by the FDA, it is essential that all labels should include safety and efficacy information for each age group tested. If a product has not been tested in pediatric populations the label should clearly state that.

Atopic dermatitis affects a very young patient population, so it critical to have safety information for all age groups to prevent dangerous off label use. This is a vulnerable patient population, and the benefits for the treatment must be proven to outweigh the risks.

We urge the FDA not to just grant pharmaceutical companies waivers for clinical trials on pediatric patients.

As a researcher, public health advocate, and mother, I thank you for your consideration of these important issues.