Date of Award

Summer 8-9-2024

Degree Type

Thesis

Degree Name

Master of Science - Mathematical Sciences

Department

Mathematics and Statistics

First Advisor

Jacob Turner

Second Advisor

Derek Blankenship

Third Advisor

Robert Henderson

Fourth Advisor

Emiliano Giudici

Abstract

Survival analysis is a critical statistical method in healthcare to assess patient treatment effects and disease progression. Another critical area of statistical methodology in health care is the practice of adaptive designs. Adaptive designs allow for interim analyses to take place during a study and various decisions and actions can take place more ethically. This is beneficial for studies that take multiple years to complete and allows administrators and healthcare providers to make sound decisions as early as possible. A challenging aspect of adaptive designs is that the number of interim analyses is known in advance which is applicable in controlled experiments such as randomized clinical trials.

Motivated and highlighted by our collaborations with Fresenius Medical Care, many clinical studies are observational in nature and have no clear endpoint, making it difficult to determine the number of interim analyses that will be conducted. This research considers the application of survival analysis using adaptive designs within observational studies. To do so, we developed a collection of statistical programs to simulate these types of interim analyses while accounting for the additional complexity that survival data exhibits. Simulations summaries were performed and we will summarize some of the key results including investigations of statistical power, Type-I error control, and parameter estimation performance. Additionally, this work aims to assess the necessary conditions to achieve reasonable power at early looks and/or establish general rules of thumb when designing the study.

Creative Commons License

Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.

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