Formalizing function

Functional GI disorder patients want PROs to assess nuances of symptoms, effect of relief

FDA agreed with patients at a workshop on functional GI disorders that patient-reported outcomes measures should tease out the relationship between symptom relief and corresponding quality of life. But doing so presents a catch-22 for companies, because it takes an effective therapy to validate the sensitivity of a new PRO, and a sensitive PRO to demonstrate the efficacy of a new therapy.

At a patient-focused drug development workshop on May 11, FDA's Laurie Muldowney said PROs are important for functional GI disorders, which have no obvious structural or anatomical abnormality to track and are diagnosed based on symptoms.

"For us, [PROs] can be a direct measure of treatment benefit," said Muldowney, a medical officer in the Division of Gastroenterology and Inborn Error Products.

In a workshop poll, patients said their most troublesome upper GI symptoms were pain (76%) and nausea or vomiting (67%), and their most troublesome

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