The Celiac Disease Foundation is in discussion with a team at the University of Colorado, Denver to develop the first-ever, FDA-approved pediatric PRO (patient-reported outcome tool). The Foundation, with your continued support, is proposing to fund the development of the PRO. This is an important and necessary step to accelerate research into treatment options and a cure for children with celiac disease.
The FDA defines a patient-reported outcome (PRO) as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” The FDA requires the use of a PRO instrument at virtually every stage of clinical trials when the concept being measured is best known by the patient or best measured from the patient’s perspective, which, as you well know, includes many celiac disease symptoms.
“Clinical trials often overlook the specific needs of children for a variety of reasons. We cannot assume that children are just smaller versions of adults, so it is important to remove any perceived barriers to testing in pediatrics. A pediatric PRO is a necessary tool for upcoming drug trials to take place in children. We are grateful that the Foundation has not only recognized this, but also has been a key driver in making this entire process happen. They have been a huge catalyst in driving policy and advancing research for the celiac community, and this is just one more example.”
–Dr. Edwin Liu, Pediatric Gastroenterologist, Director of the Colorado Center for Celiac Disease, Children’s Hospital Colorado
As of this writing, there are no clinical trials for celiac disease therapies that involve individuals under the age of 18, even though we know that hundreds of thousands of children in this country suffer both physically and psychologically from celiac disease. In the coming months, the Celiac Disease Foundation will be recruiting for the first online study to capture pediatric experiences in living with celiac disease to inform how new therapies may be developed for children, as well as for adults.
An effective pediatric PRO instrument will not only be a valid and reliable way to collect data directly from patients to evaluate therapeutic intervention, but it will also make a positive contribution to clinical care for our children. Pediatricians and pediatric gastroenterologists can use the PRO instrument to understand symptom/disease progression at the point of care and recommend additional interventions, such as nutritional and psychological support, and, with continued progress, therapeutics for children.
We are working tirelessly toward treatments and a cure for celiac disease. This project to create the first pediatric PRO for celiac disease is another important step in accelerating research. Please let me know if you would like to stay updated on the development of the pediatric PRO.
All of our work on behalf of the celiac patient community, for adults and children, is made possible by the generous support of individuals like you. If you are able to give again, please do.
Warmest regards,
Marilyn G. Geller Chief Executive