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Optimizing trial designs for targeted therapies
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Ondra, Thomas, Jobjörnsson, Sebastian, Beckman, Robert A., Burman, Carl-Fredrik, König, Franz, Stallard, Nigel and Posch, Martin (2016) Optimizing trial designs for targeted therapies. PLoS One, 11 (9). e0163726. doi:10.1371/journal.pone.0163726
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Official URL: http://dx.doi.org/10.1371/journal.pone.0163726
Abstract
An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment is tested in an overall population and/or in a pre-specified subpopulation. Based on a decision theoretic framework we derive optimized trial designs by maximizing utility functions. Features to be optimized include the sample size and the population in which the trial is performed (the full population or the targeted subgroup only) as well as the underlying multiple test procedure. The approach accounts for prior knowledge of the efficacy of the drug in the considered populations using a two dimensional prior distribution. The considered utility functions account for the costs of the clinical trial as well as the expected benefit when demonstrating efficacy in the different subpopulations. We model utility functions from a sponsor’s as well as from a public health perspective, reflecting actual civil interests. Examples of optimized trial designs obtained by numerical optimization are presented for both perspectives.
| Item Type: | Journal Article | ||||||||
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| Subjects: | R Medicine > R Medicine (General) | ||||||||
| Divisions: | Faculty of Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Medicine > Warwick Medical School |
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| Library of Congress Subject Headings (LCSH): | Clinical trials -- Mathematical models | ||||||||
| Journal or Publication Title: | PLoS One | ||||||||
| Publisher: | Public Library of Science | ||||||||
| ISSN: | 1932-6203 | ||||||||
| Official Date: | 29 September 2016 | ||||||||
| Dates: |
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| Volume: | 11 | ||||||||
| Number: | 9 | ||||||||
| Article Number: | e0163726 | ||||||||
| DOI: | 10.1371/journal.pone.0163726 | ||||||||
| Status: | Peer Reviewed | ||||||||
| Publication Status: | Published | ||||||||
| Access rights to Published version: | Open Access | ||||||||
| Funder: | Seventh Framework Programme (European Commission) (FP7) | ||||||||
| Grant number: | FP7 HEALTH 2013-602144, FP7 HEALTH 2013-6025 52 |
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