This project is dedicated to exploring the realm of healthcare through data-driven insights and analysis. This project aims to leverage data science methodologies to understand, interpret, and derive meaningful conclusions from healthcare-related datasets.
This project will start with basic statistical ideas and then step by step discuss various problems and suggest answers related to these topics:
a. Randomization and study blindness; Outcome adaptive randomization; Sample size/power; Hypothesis testing vs. estimation.
b. Data collection and quality control; Statistical methods for trial monitoring; Early termination due to toxicity, efficacy, or lack of efficacy.
c. Phase I – 3+3 design and also traditional 3+3 design and the continuous reassessment method (CRM).
d. Phase II – single arm designs, one-stage and multi-stage designs, designs based on predictive probability, and Phase II – randomized designs.
e. Bayesian Design: Bayesian sample size calculation; Beta-binomial models and methods for binary outcomes.
f. More Bayesian Design: Bayesian sample size calculation; Beta-binomial models and methods for binary outcomes.
g. Adaptive designs using binary endpoints; Response-adaptive randomization;
h. Phase III study: Repeated significance testing
i. More Phase III study: Repeated significance testing
j. Phase III design: Classical sequential methods; Group sequential methods; Interim analysis
k. Sample size calculation in phase III study
This repository will have both theoretical questions as well as analytical questions for better understanding.
I prepared all the answers and R codes mentioned in this project. Obviously, there could be better ways to solve those. Any suggestion will be highly appreciated.
For more details and a better understanding these 2 books can be very helpful:
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Book name: Introduction to Statistical Methods for Clinical Trials by Thomas D. Cook and David L. DeMets (Publication: Chapman & Hall/CRC. ISBN: 978-1-58488-027-1).
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Book name: Fundamentals of Clinical Trials (4th edition) by L.M. Friedman, C.D. Furberg and D.L. DeMets. (Publication: New York: Springer. ISBN: 978-1-4419-1585-6).