Endpoint Analysis in Eosinophilic Esophagitis Trials Gets FDA Overhaul
Trials of drugs or biologics to treat eosinophilic esophagitis should develop their own patient-reported outcome (PRO) tools in the absence of well-defined and reliable instruments for the disease, according to a final guidance the FDA released last week.
New instruments should be tested in phase 2 trials to help determine endpoints and scoring algorithms for phase 3 trials, says the guidance that was first released as a draft in February 2019.
Trials should include a treatment period of at least 24 weeks to assess the drug’s effect on signs and symptoms and the related underlying inflammation, followed by an additional 28 weeks of treatment to establish the treatment’s safety and durability of response. Sponsors should gather long-term data before submitting an application to the FDA.
The agency encourages the inclusion of patients age 12 to 17 in trials as long as data from adult patients shows the treatment is safe, and trial designs for patients younger than age 12 may be considered based on safety data from adult and adolescent populations.
Read the final guidance here: https://bit.ly/3kpx5CX.