Elaine Siegfried, MD: As soon as that medication, dupilumab, was FDA approved in adults, and as a pediatric dermatologist, 90% of the children that I treat, I treat off-label because we don’t have on-label treatments for the great majority of the diseases that we face every day. So I was used to using off-label treatment and fortunately had the opportunity to get access for a pretty big number of kids who had severe atopic dermatitis. My patient population is probably 60% atopic dermatitis. I would say 60% of my patients, 90% of my time, because it’s such a time-consuming disease.
But I now have about probably 60, 70 kids who have been on dupilumab for a couple of years. And everyone that I started on it is still on it and these kids are not cured of their disease, they’re all still on the medication. But for every single one of them it’s made a huge impact on their life. The first kids that I put on it were kids who had evidence of some other immune suppression. Children with moderate to severe atopic dermatitis are really subject to lots of cutaneous infections, and they get lots of problems with recurrent herpes and have strep throat, and sinusitis, and pneumonia sometimes in that population, sort of an immune deficiency population. And that population of kids I had had on intravenous immunoglobulin. And that cost about $10,000 to $15,000 a month. Way more expensive than really the most expensive of the targeted biologic agents. So those were the first kids that I put on this drug, and they’re doing beautifully.
Then subsequently I put other children on it who had been on methotrexate for many years, or who required cyclosporine, who’d been hospitalized repeatedly. I get to see patients back in my office probably every time I have a clinic, so pretty much every day I see somebody in follow-up back on this drug, and it’s life altering. And I tell them frequently, you know part of the measure of the efficacy of any drug is in looking at the impact on quality of life. And we have a lot of validated measures to look at quality of life changes in patients who are on these kind of drugs.
But what we don’t have is a questionnaire that measures quality of life for the provider. And I can tell you that it makes my life way easier to be able to have a medication like this. Because prior to having something available that was targeted like this, I had to wrack my brain about the safety and the effectiveness with a lot of unknown questions and difficulty in even beginning to explain that to patients and their parents about the risk and benefits of these other off-label choices. And now it’s just become so much easier, especially because the drug is very effective.
Julie Block: Since dupilumab has only been on the market a couple of months and approved, at the National Eczema Association, we certainly have many of our members and people who are very near and dear to my heart, families I’ve known for over 12 years that are anxiously awaiting this treatment. And we’re thrilled for that. Anecdotally, we know for many of those who participated in the clinical trial, it’s been nothing but a game-changing, life-altering, have-my-life-back experience. Not for everybody but for those that are successful with this treatment, it is absolutely mind-blowingly life-altering. And we’re thrilled. I would say, too, in my world as the president and CEO of the National Eczema Association, I had a moment about 4 or 5 years ago when I looked at this young boy who I’ve known in this family, and I knew in my heart of hearts that this boy was not going to suffer his whole life, that science, and treatments, and drug development are taking us to new places that give us extreme hope. And for that I’m eternally grateful, and we can’t go fast enough. We have to work really hard to move any barriers to these treatments for our patients.
Elaine Siegfried, MD: Dupilumab, since its FDA approval in March of 2017, I’ve been able to access it for patients with some kinds of insurance. It’s been really variable and difficult to get access, but for the people who have gotten access, it’s been nothing short of miraculous. Since its approval for adolescents just in March of 2019, I think that’s opened the door a little bit for children age 12 and over to get the medication. Frankly, that approval hasn’t…made it enormously easy to get the drug.
But we’re looking forward to having it available for much younger children. The phase 2 clinical trial going on right now to use it in children as young as 6 months of age, that’s going to be wonderful to have access like that. And we’re just wrapping up the phase 3 trial for children aged 6 to 11. Hopefully the FDA approval will be following shortly for that age group.