Biomarkers Consortium - Mucosal Healing in UC: Definition, Treatment Target and Clinical Endpoints

The FDA has recently encouraged the evaluation of histopathology in Ulcerative Colitis (UC) clinical trials in order to make the claim of “mucosal healing” as a treatment goal in a 2016 draft guidance to industry. However, there is no community consensus on the definition of mucosal healing or standardized protocols to measure this treatment target/endpoint. There are many challenges to achieving a standardized assessment of histologic disease activity in a UC clinical trial, including: the heterogeneity in the collection of mucosal biopsies (number, location) from study to study, a lack of fully validated histology scoring systems, and an absence of community standards for definitions of histologic improvement or histologic remission. The lack of standardized measurement of a treatment target creates a major challenge to UC drug development and impacts the lack of treatment option for patients with this serious, chronic, inflammatory bowel disease. This project will address this gap by establishing a common methodology that the scientific community can utilize when developing therapeutics for UC.

The objective of this project is to establish a common methodology for a histologic measurement of a mucosal healing endpoint for treatment of UC that demonstrates clear prognostic value for long-term outcomes for patients. The results of this project will be submitted to the FDA in order to impact the guidance for industry for UC clinical trials.

Program Goals:

This proposal will build a unique dataset to define the best practice for measuring mucosal healing by leveraging existing datasets from at least 3 organizations to:

  • Establish a histolopathologic measurement of mucosal healing that correlates with long-term patient important outcome.
  • Establish a machine learning methodology as a validated objective method for scoring of mucosal healing for use in clinical trials, regulatory approvals, and clinical practice.
  • Establish the number, location, size, and density of biopsies required to capture the variability of inflammation across the colon.

Support

The FNIH currently seeks funding to support this program. For more information on how to partner with us, please contact Joanne Morse, Senior Development Officer at jmorse@fnih.org

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FNIH Contact

Stephanie Cush, Ph.D., Senior Project Manager, scush@fnih.org