On September 20, 2021, patients and caregivers managing childhood asthma came together to share their perspectives with the U.S. Food and Drug Administration (FDA). The Little Airways, Big Voices initiative was an externally-led patient focused drug development (PFDD) virtual meeting.
The virtual meeting was divided into two sessions. The first session focused on the patient experience, including symptoms and disease burden. The second session focused on treatment options. Each session featured presentations from patients and caregivers followed by a live, moderated discussion.
We want to thank all of the patients and caregivers who shared their stories during this important meeting with the FDA. By sharing what is important to patients and their families, they help inform the FDA’s drug development process and the approval of new therapies.
You can still be part of this initiative. Here’s how you can get involved:
- Watch the PFDD virtual meeting to hear perspectives from asthma patients
Little Airways, Big Voices is a collaborative effort among four advocacy organizations:
The American Lung Association, American Partnership for Eosinophilic Disorders (APFED), Asthma and Allergy Foundation of America (AAFA) and Allergy & Asthma Network.
What Is a Patient-Focused Drug Development (PFDD) Meeting?
FDA uses the PFDD meetings to collect insights on certain diseases. For example, FDA may want to hear about the impact of childhood asthma on daily life and treatment options.
Some PFDD meetings are led by FDA and others are externally-led by patient advocacy groups. The meetings and resulting Voice of the Patient report help identify what is important to patients. This includes what people want treatment to look like (apart from a cure).
The goal of the Little Airways, Big Voices externally-led PFDD meeting was to gather patient and caregiver insights. These perspectives will:
- inform new treatment strategies;
- engage families in drug development;
- improve health outcomes.
What is the Voice of the Patient Report?
Information gathered from the PFDD meeting, surveys and written comments were summarized in the “Voice of the Patient report” (PDF). The report helps inform FDA, medical researchers and drug development companies about what is important to people living with and managing childhood asthma.
Remarks from Tonya Winders at the Externally-led PFDD Little Airways Big Voices Meeting
What a day this has been. We began by hearing the staggering impact of childhood asthma for over 6 million kids who are living with this chronic disease and the millions more fearful yet hopeful parents and caregivers who are simply praying that their child outgrows their asthma symptoms and condition. But the truth is they won’t outgrow it. The truth is asthma is a chronic condition with a lifelong sentence. The truth is approximately 10 people will die today from asthma alone. And the truth is that the burden heard here today is mind-blowing and absolutely highlights the unmet needs in childhood asthma.
First, we heard unmet needs in prevention. Concerns from people like Charmayne Anderson and Andrea Jensen asking what are the genetic and environmental factors that are driving asthma. No one should deny the disparities that were displayed during our meeting today. What we have seen reflects that African-American children are 2 times more likely to have asthma and 10 times more likely to die from this disease.
Unfortunately, it’s no better in Latinx communities, where over 20% of Puerto Rican children live with asthma. We implore the FDA to continue to research the atopic march, help us prevent asthma in early years of life, especially for those living in our most underserved communities.
Next, we heard the unmet needs in diagnosis. Stories like Ryan Piansky, who experienced 16 years between his initial diagnosis at age 2, and his new specific phenotype-based diagnosis of eosinophilic asthma at 18. But what about the non-eosinophilic asthma kids? What about the kids who never get access to spirometry or valuable diagnostic tools like fractional exhaled nitric oxide? How do we continue to innovate in this space of diagnosis and asthma?
Then we heard so many express the unmet needs in treatment. I’m reminded of Regan Lloyd, Shreaya Madireddy and Max MacDonald who so articulately shared the side effects of their treatment. These side effects included mood disturbances, sleep disturbances, weight gain, bone loss and topical steroid withdrawal.
All of these conditions are so impactful to children’s long term health. It’s not just about until they’re 18. What happens beyond that time and are the current treatments enough? Are they actually, in some cases, harming our children rather than helping them. We ask FDA to explore new routes of administration beyond inhalers, beyond injections, and to consider the massive comorbidity impact on children.
We’ve also heard unmet needs and long-term management and progression of the disease from Javan Allison, Shiv Sewlal, and others. What is the long-term damage that this disease is doing to my lungs? What about the countless ER visits, hospitalizations and ICU stays and the side effects of the treatments that I’m withstanding during those stays.
At the end of today, I really want to stop and just say thank you to each and every one of the speakers who have shared their heartfelt stories. Stories like what we heard from the Rivera family, whose son shared the sentiment, “I just want to be a normal kid. I want to play soccer. I want to run outdoors.”
I want to thank those inspirational testimonies from kids like Regan and Max who share the frustration of the side effects of oral corticosteroids and other treatments, who share the desire to go to school even when asthma forces them to miss over 40 days in a single year.
And I wanted to thank the parents. The pleas of parents who are running out of treatment options. And it is these stories that have brought us here today to be so impactful and to help us share with the FDA, the burden of this disease on our community.
The truth is no child should be forced to avoid playing outside or going to the beach. No child should be sidelined or made to quit playing a sport that they so dearly love. No child should live with the constant anxiety or isolation due to childhood asthma.
No parent or caregiver should have to leave their career or live in fear of the 2:00 am phone call from college saying, “Mom, I can’t breathe,” like I experienced with my son Caleb in 2018. Nor should they be left guessing if the next time that their child has an asthma attack will be the last.
No family should experience the loss of a child or a loved one like what we heard of the Del Principe family on her sister’s wedding day. Yes, our community would love a cure, but until that time we implore the FDA to prioritize childhood asthma by advancing research and expediting reviews of new treatment solutions that help us to breathe better together.