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Regulatory Focus™ > News Articles > 2019 > 2 > FDA Drafts Guidance on Developing Drugs for Rare Chronic Digestive Disorder

FDA Drafts Guidance on Developing Drugs for Rare Chronic Digestive Disorder

Posted 06 February 2019 | By Ana Mulero 

FDA Drafts Guidance on Developing Drugs for Rare Chronic Digestive Disorder

The US Food and Drug Administration (FDA) issued draft guidance on Wednesday to aid sponsors in the clinical development of drugs and biologics for the treatment of eosinophilic esophagitis (EoE).

The 13-page draft guidance document describes the agency’s clinical trial recommendations for EoE drugs. These relate to trial population and design, safety and efficacy considerations, clinical outcomes assessments, as well as pediatric considerations. The clinical development of drugs for the treatment of non-EoE eosinophilic gastrointestinal disorders falls outside of the scope of the agency’s draft guidance.

FDA recommends sponsors “consider stratified randomization based on the presence or absence of baseline strictures” when making population determinations. Sponsors should also consider potentially including diet as a covariate to evaluate the potential effect of dietary restrictions, according to the draft guidance. This is because “dietary restriction is a known effective treatment for EoE,” the agency said.

For clinical trial designs, the agency recommended conducting “a randomized, double-blind, placebo-controlled trial design with a prespecified screening period before randomization of patients to confirm eligibility criteria.” FDA added that clinical trials intended to support marketing approval of an EoE drug “should evaluate a drug’s effect on both signs/symptoms and the related underlying inflammation.”

The draft guidance outlined five pediatric recommendations as well, citing 2018 research that suggests clinical signs and symptoms in patients with EoE tend to vary with age. “We encourage the inclusion of adolescents (patients 12–17 years of age inclusive) in registration clinical trials, provided that preliminary safety and efficacy data in adult patients support enrollment,” the agency said.

Draft guidance


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