New methods and software for designing adaptive clinical trials of new medical treatments / Michael Rosenblum.

Format:

Book

Author/Creator:

Rosenblum, Michael, author.

Language:

English

Subjects (All):

Medical care, Cost of.

Outcome assessment (Medical care)--Methodology.

Outcome assessment (Medical care).

Physical Description:

1 online resource

Place of Publication:

Washington, DC : Patient-Centered Outcomes Research Institute (PCORI), 2019.

Summary:

We aimed to (i) develop new adaptive enrichment designs and prove their key statistical properties; (ii) conduct simulations that mimic features of completed trial data sets in order to evaluate the new trial designs'performance (such as sample size, duration, power, bias); the data sets are from trials involving treatments for HIV, stroke, and heart failure; (iii) develop user-friendly software for optimizing the performance of our new adaptive designs and comparing them to standard designs. The goal was to construct designs that satisfy power and type I error requirements at the minimum cost in terms of expected sample size, ie, average sample size over a set of plausible scenarios. We also considered the maximum sample size, ie, the number of participants enrolled if there is no early stopping.

Contents:

ABSTRACT

BACKGROUND

PARTICIPATION OF PATIENTS AND/OR OTHER STAKEHOLDERS IN THE DESIGN AND CONDUCT OF RESEARCH AND DISSEMINATION OF FINDINGS

METHODS

RESULTS

DISCUSSION

CONCLUSIONS

REFERENCES

RELATED PUBLICATIONS

ACKNOWLEDGMENTS.

Notes:

Description based on publisher supplied metadata and other sources.