By: Janet Woodcock, M.D., Principal Deputy Commissioner, and Sandra Retzky, D.O., J.D., MPH, Director, Office of Orphan Products Development, Office of Clinical Policy and Programs
The rare disease community includes diverse perspectives from patients, families, caregivers, and advocates. Each of these perspectives is important and can contribute to the work of advancing rare disease treatments. Input from the rare disease community is crucial to the development of medical products to diagnose and treat conditions, as well to as maintain or increase quality of life.
This year, the U.S. Food and Drug Administration is part of the worldwide community observing Rare Disease Week—February 28th through March 4th. Our participation includes hosting FDA’s Rare Disease Day on March 4th. Our theme is: “Sharing Experiences in Rare Diseases Together.”
What do we mean by “Sharing Experiences in Rare Diseases Together?” We know how important it is to hear and understand the patient voice in all drug development activities. Ultimately, in the review of new medical products, what really matters is the patient perspective—does a drug or device improve how the patient feels, functions, or survives? Notice the word “review.” That’s one small word for everything the FDA does to approve new medical products. “Review” at the FDA encompasses widely disparate activities across many scientific disciplines.
So, at this year’s Rare Disease Day event, we want to share our perspectives alongside those of patients. To accomplish this, members of the FDA’s review teams will share their experiences working on rare diseases. They will explain why their work is so important and how they collaborate across FDA centers. Most importantly, they want to share how it feels to be working on such important products—with the goal that everyone in this country with an illness has access to safe and effective medicines.
At this year’s meeting, four panels of reviewers will share their journeys in the development of new medical products to treat rare diseases. They are:
- A Glimpse into the Reviewer’s Journey Leading to Approval of Two Drugs to Treat Rare Tumors.
- Working Together and Sharing Experiences in the Review of Gene Therapies for Neurocognitive Disorders in Children.
- Our Journey to Enhance Product Development Using A Public-Private Partnership Approach.
- Our Approach to Rare Diseases and The Patient Voice. Reviewer Experiences with Orthopedic Humanitarian Device Exemptions.
In addition to our reviewer journeys, a panel of patients will speak about their experiences interacting with the FDA and how to get involved with our agency. Our final panel is the Future Journey—how we at the FDA can do more to promote drug development for rare diseases. Listeners will hear experts from all FDA centers speak about initiatives to enhance product approval for rare diseases.
Looking ahead, the FDA will continue to seek out and compile valuable stakeholder input to inform the complex medical product regulatory process. From feedback about health and quality of life factors to critiques of clinical trial design from the participant perspective, patient voices provide essential data that the FDA uses to achieve its public health mission. We will continue to hold Patient Listening Sessions, including those with a rare disease focus, as they provide critical information from patients and caregivers about their experiences living with rare diseases.
The stories shared during our annual Rare Disease Day meetings and in other engagements with patients shine a light on the experiences of those in the rare disease community. We were invited and are pleased to participate in this year’s “Light Up for Rare” campaign, organized by the National Organization for Rare Disorders in the United States. By illuminating the FDA headquarters, we also hope to shine a light on the experiences and needs of the rare disease community, on the collaborative efforts being made in this space, and on the strength and resolve of those who are working to bring solutions to the public.
Please join us for this virtual event. There is no cost and registration is open to everyone.