Celiac Disease Foundation Chief Executive Officer, Marilyn Geller, will present at the US Food and Drug Administration’s Patient-Focused Drug Development Conference (PFDD) on May 11, 2015 just outside of Washington, D.C. Marilyn accepted the invitation to speak on behalf of celiac patients and their caregivers. In anticipation of this meeting, we are asking that members of the celiac community complete the linked survey about living with celiac disease as soon as possible. We want to make sure that your voice is heard by the FDA as they make decisions on proposed therapeutic treatments for celiac disease. This is an urgent request, as our written comments are due to the FDA in a few days. Please complete the survey now.
As you are likely aware, the FDA drug approval process is the most rigorous in the world. Until the passage of the 2012 Food & Drug Administration Safety and Innovation Act (FDASIA), a glaring oversight in the process was patient and caregiver input. With the creation of PFDD, that is now changing. The FDA has authorized PFDD for disease areas that meet the following criteria:
- Chronic, symptomatic, or affects functioning and activities of daily living
- Aspects of the disease are not formally captured in clinical trials
- Currently no therapies, very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives
- Has a severe impact on identifiable sub-populations (such as children or the elderly)
Clearly, celiac disease meets each of these criteria.
What the FDA has learned from the limited application of PFDD over the last couple of years is the following:
- Patients with chronic serious disease are experts on what it is like to live with their condition
- Patients’ “chief complaints” may not be factored explicitly into drug development plans, including measures of drug benefit planned in trials
- Patients want to be as active as possible in the work to develop and evaluate new treatments
In other words, researchers and clinicians may have ideas about what symptoms and underlying factors proposed therapies should target, but those who are actually living with the disease have opinions too, and they should be considered.
With your help, CDF is making its presence felt in Washington this year. This will be Marilyn’s third visit to the FDA over a two-month window on behalf of the celiac community. The first was the GREAT3 conference in March. The second is a meeting this week with Dr. Susan Mayne, Director, Center for Food Safety and Applied Nutrition (CFSAN), U.S. Food and Drug Administration. What this tells us is that, thanks in part to persistent, hard work by CDF and its ally organizations in the celiac community, there is real momentum building in drug research toward the introduction of meaningful treatments for celiac disease. The FDA is getting prepared to review applications for therapies at all relevant steps along the development pipeline and we – you and I – are being invited to have our voices heard. That is why I urge you to complete the survey now.
Talia Hassid, Celiac Disease Foundation Community Coordinator