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The value of the information that can be generated : optimizing study design to enable the study of treatments addressing an unmet need for rare pathogens
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Dane, Aaron, Rex, John H., Newell, Paul and Stallard, Nigel (2022) The value of the information that can be generated : optimizing study design to enable the study of treatments addressing an unmet need for rare pathogens. Open Forum Infectious Diseases, 9 (7). ofac266. doi:10.1093/ofid/ofac266 ISSN 2328-8957.
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Official URL: https://doi.org/10.1093/ofid/ofac266
Abstract
In traditional phase 3 trials confirming safety and efficacy of new treatments relative to a comparator, a one-sided type I error rate of 2.5% is traditionally used, and typically leads to minimum sizes of 300-600 subjects per study. However, for rare pathogens, it may be necessary to work with data from as few as 50–100 subjects. For areas with a high unmet need, there is a balance between traditional type I error and power and enabling feasible studies. In such cases, an alternative one-sided alpha level of 5% or 10% should be considered and we review herein the implications of such approaches. Resolving this question requires engagement of patients, the medical community, regulatory agencies, and trial sponsors.
Item Type: | Journal Article | ||||||||
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Subjects: | R Medicine > RA Public aspects of medicine R Medicine > RM Therapeutics. Pharmacology R Medicine > RS Pharmacy and materia medica |
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Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
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SWORD Depositor: | Library Publications Router | ||||||||
Library of Congress Subject Headings (LCSH): | Antibiotics, Antifungal agents, Pathogenic microorganisms, Oncology, Communicable diseases | ||||||||
Journal or Publication Title: | Open Forum Infectious Diseases | ||||||||
Publisher: | Oxford University Press | ||||||||
ISSN: | 2328-8957 | ||||||||
Official Date: | July 2022 | ||||||||
Dates: |
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Volume: | 9 | ||||||||
Number: | 7 | ||||||||
Article Number: | ofac266 | ||||||||
DOI: | 10.1093/ofid/ofac266 | ||||||||
Status: | Peer Reviewed | ||||||||
Publication Status: | Published | ||||||||
Access rights to Published version: | Open Access (Creative Commons) | ||||||||
Date of first compliant deposit: | 29 June 2022 | ||||||||
Date of first compliant Open Access: | 1 July 2022 |
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