Trends of Clinical Trials for Drug Development in Rare Diseases.

ABSTRACT:

Background

Drug development for rare diseases is challenging because it is difficult to obtain relevant data from very few patients. It must be informative to grasp current status of clinical trials for drug development in rare diseases.

Objective

Clinical trials in rare diseases are to be outlined and compared among the US, EU and Japan.

Method

ClinicalTrials.gov (NCT, National Clinical Trial), EU Clinical Trials Register (EUCTR) and the Japan Primary Registries Network (JPRN) were analyzed. Clinical trials involving information on rare diseases and drugs were extracted by text-mining, based on the diseases and drugs derived from Orphanet and DrugBank, respectively.

Results

In total, 28,526 clinical trials were extracted, which studied 1,535 rare diseases and

SUBMITTER: Sakate R 

PROVIDER: S-EPMC6327118 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature