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Issues in the assessment of non‐inferiority: perspectives drawn from case studies
Author(s) -
Ulrich Burger H.,
Beyer Ulrich,
Abt Markus
Publication year - 2011
Publication title -
pharmaceutical statistics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.421
H-Index - 38
eISSN - 1539-1612
pISSN - 1539-1604
DOI - 10.1002/pst.512
Subject(s) - placebo , risk analysis (engineering) , medicine , clinical trial , computer science , intensive care medicine , medical physics , management science , engineering ethics , psychology , alternative medicine , engineering , pathology
Superiority claims for improved efficacy are the backbone of clinical development of new therapies. However, not every new therapy in development allows for such a claim. Some therapies per se do not try to improve efficacy further but concentrate on important aspects in safety or convenience. Such improvements can be equally important to patients, and development strategies should be available for such compounds. A three‐arm design with placebo, active control and experimental treatment may be viewed as the golden standard for such compounds; however, it may be difficult if not impossible to add a placebo arm in certain diseases. In such situations, non‐inferiority designs are the only development option left. This paper will highlight some of the key issues with such designs in practice and will report experience from two studies from different therapeutic areas intended for regulatory submission. Copyright © 2011 John Wiley & Sons, Ltd.