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Current Status of Celiac Disease Drug Development.

The American journal of gastroenterology
June 1, 2016
Manida Wungjiranirun et al. (3 authors)
Journal ArticleReviewHuman Study
Study Details

Study Goal

The researchers aimed to review the current understanding of celiac disease (CeD) treatment, focusing on the limitations of the gluten-free diet and the regulatory pathway for potential new therapies.

Results Summary

The study found that while the gluten-free diet is the only current therapy for CeD, it incompletely controls symptoms and disease activity. The abstract highlights the need for new therapies and discusses the challenges in using biomarkers as primary outcomes for drug approval.

Population

Individuals with celiac disease (CeD).

Effective Dosage

Not specified

Duration

Not specified

Interactions

None mentioned

Extracted Claims (3)
InterventionDirectionEndpointPopulationDosageImpactClaim #
gluten-free diet
no change
symptoms and disease activity
patients with celiac disease
-
incompletely controlled
#1
biomarkers including histology and serology
no change
improved patient outcomes
patients in therapeutic clinical trials
-
lack the necessary evidence linking them to improved patient outcomes
#2
patient-reported outcomes
increase
Phase III CeD trials
patients with celiac disease
-
will likely be primary end points
#3
Abstract

Celiac disease (CeD) is one of the most common immune-mediated diseases. Symptoms and disease activity are incompletely controlled by the gluten-free diet, which is currently the only available therapy. Although no therapies are yet approved, there is a growing field of candidates and an improving understanding of the regulatory pathway. In this review, we briefly discuss the epidemiology, pathophysiology, and current treatment paradigm for CeD. We also review the major classes of therapies being considered for CeD and discuss extensively what is known and can be surmised regarding the regulatory pathway for approval of a CeD therapeutic. The coming years will see an increasing number and diversity of potential therapies entering clinical trials and hopefully the first approved agents targeting this significant unmet medical need. Although biomarkers including histology and serology will always be important in therapeutic clinical trials, they currently lack the necessary evidence linking them to improved patient outcomes required for use as primary outcomes for drug approval. For this reason, patient-reported outcomes will likely be primary end points in Phase III CeD trials for the foreseeable future.

Medical Subject Headings (MeSH)
BiomarkersCeliac DiseaseCombined Modality TherapyDiet, Gluten-FreeDrug DiscoveryGastrointestinal AgentsHumans
Study Links
Quality Scores
SafetyNot Assessed
Efficacy60/10
Quality75/10
Citation Metrics
Total Citations14
Citations/Year1.6
Relative Citation Ratio0.64
NIH Percentile34.2%
Research Impact Scores
APT Score0.50
Weight Score1.74
Normalized Score0.59
Related Supplements
Current Status of Celiac Disease Drug Development. | Panacea Index