For decades, the celiac disease community has been demanding a drug alternative to the always burdensome and often ineffective gluten-free diet. Thanks in part to work done by the Celiac Disease Foundation, dozens of proposed drug therapies are now in the development pipeline at biopharmaceutical companies and academic research centers around the world, with several now in clinical trials. Every drug therapy for celiac disease patients in the United States must obtain approval from the Food and Drug Administration, the federal agency that evaluates and approves our prescription drugs. To date, not a single drug or therapy for celiac disease has been approved by the FDA, with only one in a Phase 3 trial, the final stage before approval.
Periodically, the FDA hosts workshops to help researchers understand exactly what the FDA will be looking for (aka “endpoints”) when evaluating the efficacy of a proposed drug. In recent years, the “patient voice” has been given greater influence in this process as regulators have come to understand that patients and their caregivers know better than anyone what a drug therapy must accomplish to be considered worthwhile. GREAT VI is the second workshop ever held by the FDA for celiac disease. The first, GREAT3, was funded in part by the Celiac Disease Foundation and held in March 2015. GREAT3 spurred biopharmaceutical interest in developing celiac disease drugs, leading to today’s pipeline.
For GREAT VI, the Celiac Disease Foundation was asked by the FDA to identify celiac disease patients and caregivers who would participate in this essential process. Amplifying the patient voice is one of the exact reasons we created and trained Patient Advocates through our Patient Education & Advocacy Summit and our online State Advocacy Ambassador Training Program over the last several years. I am pleased to announce that three members of our Foundation family have been selected by the FDA to participate in the GREAT VI Workshop — Kelsey Smith, Kathy Hardin, and her child Beckett Hardin.
I want to stress that one of the most important jobs at the Foundation is to organize and elevate the voice of the celiac disease patient community in every possible forum and to every relevant audience. By demanding that researchers, clinicians, lawmakers, regulators, and funders listen to the patient, and by making sure patients are well-equipped to present their case, we are finally seeing progress on multiple fronts toward the development of treatments and a cure. The development of the GREAT VI FDA workshop makes it evident that the celiac disease community is being heard and that tangible steps are being taken to relieve the suffering experienced by so many.
I invite you to review the Agenda and Register. There is power in numbers. We will also send you updates about Kelsey and the Hardins’ testimony.
As always, thank you for all you do to support our work and the celiac community.
To Our Health,
Marilyn G. Geller