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Please use this identifier to cite or link to this item: http://hdl.handle.net/1942/14897

Title: A portfolio-based approach to optimize proof-of-concept clinical trials
Authors: Mallinckrodt, Craig H.
Molenberghs, Geert
Persinger, Charles
Ruberg, Stephen J.
Sashegyi, Andreas
Lindborg, Stacy R.
Issue Date: 2012
Citation: Journal of Biopharmaceutical Statistics, 22 (3), p. 596-607
Abstract: Improving proof-of-concept (PoC) studies is a primary lever for improving drug development. Since drug development is often done by institutions that work on multiple drugs simultaneously, the present work focused on optimum choices for rates of false positive (α) and false negative (β) results across a portfolio of PoC studies. Simple examples and a newly derived equation provided conceptual understanding of basic principles regarding optimum choices of α and β in PoC trials. In examples that incorporated realistic development costs and constraints, the levels of α and β that maximized the number of approved drugs and portfolio value varied by scenario. Optimum choices were sensitive to the probability the drug was effective and to the proportion of total investment cost prior to establishing PoC. Results of the present investigation agree with previous research in that it is important to assess optimum levels of α and β. However, the present work also highlighted the need to consider cost structure using realistic input parameters relevant to the question of interest.
Notes: Reprint Address: Mallinckrodt, C (reprint author) - Eli Lilly & Co, Lilly Corp Ctr, Indianapolis, IN 46285 USA. E-mail Addresses:cmallinc@lilly.com
URI: http://hdl.handle.net/1942/14897
DOI: 10.1080/10543406.2011.564340
ISI #: 000304678600011
ISSN: 1054-3406
Category: A1
Type: Journal Contribution
Validation: ecoom, 2014
Appears in Collections: Research publications

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