Combined assessment of early and late‐phase outcomes in orphan drug development-Reference-Cited by-同舟云学术

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Combined assessment of early and late‐phase outcomes in orphan drug development

Published:2021-04-04 Issue: Volume: Page:
ISSN:0277-6715
Container-title:Statistics in Medicine
language:en
Short-container-title:Statistics in Medicine

Author:

Pateras Konstantinos¹^ORCID,Nikolakopoulos Stavros¹^ORCID,Roes Kit C. B.²

Affiliation:

1. Department of Data Science and Biostatistics Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht Utrecht The Netherlands

2. Department of Health Evidence Section Biostatistics, Radboud University Medical Centre Nijmegen The Netherlands

Publisher

Wiley

Subject

Statistics and Probability,Epidemiology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/sim.8952

Reference27 articles.

1. Milestones On Orphan Medicinal Products Development: The 100 First Drugs for Rare Diseases Approved Throughout Europe

2. European Medicines AgencyThalidomide pharmion (thalidomide) ‐ assessment report. Technical report; 2008.

3. European Medicines AgencyGalafold (migalastat) ‐ assesment report. Technical report; 2012.

4. Changes in plasma and urine globotriaosylceramide levels do not predict Fabry disease progression over 1 year of agalsidase alfa

5. Increasing Precision or Reducing Expense in Regression Experiments by Using Information from a Concomitant Variable

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. A Study on the Power Parameter in Power Prior Bayesian Analysis;The American Statistician;2022-03-23

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