On May 11, 2015, CDF CEO Marilyn G. Geller was proud to represent the celiac community at the public meeting of the U.S. Food and Drug Administration Functional GI Patient-Focused Drug Development Conference (PFDD) at FDA headquarters just outside of Washington, D.C. There are two issues we want to highlight about CDF’s presence at this important conference:

  • Celiac disease does not fall under the functional GI category, which includes diseases like irritable bowel syndrome. Individuals who suffer from functional GI diseases, which impact as many as one in four Americans, may have symptoms similar to celiac disease, but no correlated structural abnormalities in the GI system. As you know, gluten exposure for celiac sufferers causes a structural abnormality – flattened villi – that can be identified through serology and biopsy. Nonetheless, because celiac disease symptoms overlap with functional GI symptoms, CDF was invited to present at the conference.
  • The PFDD process, in which the FDA is now engaged across a number of diseases, is incredibly important to the patient community. This process helps the FDA understand first-hand, independent of the opinions of their staff and the pharmaceutical companies, what types of treatments patients desire to improve their quality of life, especially if the disease is chronic, as celiac disease is. As you can imagine, the symptoms from which a patient might want relief may differ from those for which a pharmaceutical company wants to develop a treatment. Similarly, what a patient might consider a successful treatment may be different from what the FDA or a pharmaceutical company might think to measure. The FDA is being directed by legislation to set benchmarks and endpoints for therapies that reflect patient needs when approving treatments.

CDF’s written submission to the FDA included the results of the community survey we conducted online using the FDA-developed survey questions. The response to the survey, totaling more than 1,200 in just a couple of days, was overwhelming. We learned important details about delay in diagnosis, the serious and often debilitating consequences of exposure to gluten, the breadth of symptoms endured, and even how many of you have missed school and work as a result of the disease. For a more detailed discussion of the results, please click here. Again, we thank you for your participation; the FDA analysts are pleased to have some important information to digest.

Marilyn’s oral testimony at the meeting focused on the impact undiagnosed celiac disease had on her son and her family, the widespread and persistent ignorance in the medical community about the disease, and the probability that a significant minority of those who believe they suffer from a functional GI actually have celiac disease, and can be helped if they were tested for the disease. Her concerns expressed on behalf of the celiac community were well-received.

If you would like to learn more about the public meeting, the FDA has posted a full recording on its website. If you would like to submit your comments to the FDA – and we encourage everyone to do so – you can here. Finally, if you have any questions or comments, you are invited to contact us here.