Background: The therapeutic landscape for celiac disease is rapidly evolving, with multiple investigational therapies advancing through clinical development. While regulatory approval represents a critical milestone, it does not ensure patient access. Unlike many chronic diseases with established coverage pathways, celiac disease lacks a coordinated framework to support payer decision-making, creating a substantial risk that effective therapies may face inconsistent or restrictive insurance coverage following approval.
Methods: A multidisciplinary policy symposium convened experts in gastroenterology, pediatrics, pathology, laboratory medicine, genetics, regulatory science, health economics, payer policy, and patient advocacy to examine barriers to therapeutic access. Discussions focused on the evidence, infrastructure, and policy considerations necessary to support equitable insurance coverage for future celiac disease therapies.
Results: Panelists identified several key gaps that should be addressed to facilitate patient access to therapeutics. Diagnostic variability across international guidelines may create inconsistent eligibility determinations. Limited standardization and patient access to diagnostic documentation and celiac associated HLA-DQ Alpha 1 and DQ Beta 1 reporting could complicate treatment qualification. There is currently no consensus definition of treatment-eligible celiac disease that incorporates symptoms, histology, serology, and patient burden. Existing clinical trial endpoints may not fully align with payer evidence requirements, while health economic data demonstrating the value of therapy remain limited. Finally, experience from other disease communities demonstrated that payer engagement, guideline development, physician education, and coordinated patient advocacy should begin well before regulatory approval.
Conclusions: Achieving equitable access to future celiac disease therapeutics will require proactive development of the clinical and policy infrastructure that supports insurance coverage. Establishing harmonized diagnostic standards, defining treatment eligibility, generating health economic evidence, and engaging payers early represent essential next steps. The unique value of this symposium lies in its multidisciplinary, cross-sector approach; lessons learned from this can be applied to other disease states with similar barriers.