Nina Zeldes, National Center for Health Research, September 13, 2019
My name is Nina Zeldes and I am a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data and provides objective health information to patients, providers and policy makers. We do not accept funding from drug and medical device companies, so I have no conflicts of interest.
Thank you for the opportunity to speak here today.
As we have heard, peanut allergy is a common and at times life-threatening condition that can substantially impact patients’ quality of life. We all agree that a treatment to reduce the incidence and severity of allergic reactions is needed.
Our goal today is to determine if this allergen powder [AR101 or Palforzia] has benefits that are clinically meaningful for patients and is proven to be safe. The study results for this drug look promising but there are unanswered questions that are very important to patients with a peanut allergy and their parents.
There was only one study focusing on the efficacy of the drug, and it increased tolerance to low levels of peanut exposure. This would be clinically meaningful, because children would have less fear of exposure to tiny amounts of peanuts that can be in many foods.
However, replication is the key to scientific evidence. Independent clinical trials could have a smaller or larger effect due to differences in the demographics or comorbidities of patients or other factors. This is of particular importance because of the high rate of adverse events and large number of participants who did not tolerate the treatment. Additionally, there was a lack of diversity – the children were predominantly white. As a result, we do not know if this treatment is safe and effective for all children who might consider taking it.
To ensure that the benefits of this drug outweigh the risks we have some questions that we hope you will ask:
- 12 patients developed EOE [eosinophilic esophagitis]– are there solid data to clearly explain the risk of this condition on the label, in terms of incidence and severity? If not, can parents make an informed choice about whether or not to have their children use this medication?
- Are there any data on how many doses can be skipped before a reduction in incidences and severity of allergic reactions are affected? A daily regimen is challenging for many parents of young children, and children will not always follow directions. It is essential to know how important it is to have the children take the exactly correct dose every day.
- What are the potential long-term consequences of exposure to the drug, especially for those children for whom treatment failed? Can the treatment increase their reactivity to peanuts in the real world?
This is the first treatment for peanut allergies and if this drug is approved despite the unanswered questions, it will set a precedent for future drugs to treat food allergies. It is especially important that new classes of products provide strong evidence of safety and efficacy before approval, because it will be difficult if not impossible to obtain it afterwards.
Thank you for your time.