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Randomized Controlled Trial Data for New Drug Application for Rare Diseases in Japan

Yosuke Kubota1,2, Mamoru Narukawa3

  • 1Department of Clinical Medicine (Pharmaceutical Medicine), Graduate School of Pharmaceutical Sciences, Kitasato University, 5-9-1 Shirokane, Minato-ku, Tokyo, 108-8641, Japan. dl20402@st.kitasato-u.ac.jp.

Therapeutic Innovation & Regulatory Science
|April 28, 2022
PubMed
Abstract

Keywords:
Clinical trialsOrphan drugsPrimary endpointRandomizationRare diseasesRegression analysis

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