FDAnews Drug Daily Bulletin
Pharmaceuticals / Regulatory Affairs

Drugmakers Push Back Against Exclusion Criteria for Rare Disease Trial

April 16, 2019

Two drug companies are pushing back against proposed FDA guidance that would require drug sponsors to hold pre-trials to determine whether patients with a nasty form of esophageal swelling could benefit from an alternate therapy.

Eosinophilic esophagitis is a rare disease of the immune system that can cause vomiting, difficulty swallowing, persistent heartburn and food getting struck in a patient’s esophagus. In February, the FDA published draft guidance that, among other things, would require drugmakers to screen out patients who would respond to proton pump inhibitor (PPI) therapy by holding a two-month trial of an “adequate dose” of PPI.

In public comments, both AstraZeneca and Adare Pharmaceuticals asked the agency to reconsider that recommendation.

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