Statistical Methods in Controlled Trials and Survival Analysis

Teachers

Person in charge

• Marta Bofill Roig (marta.bofill.roig@upc.edu)

Weekly hours

Objectives

1. Objective 1 (Methodological Evaluation & Problem Solving): To critically analyze, model, and solve complex technical problems in medical and clinical research by applying fundamental statistical principles, causal inference, and advanced experimental or observational designs.
   Related competences:
2. Objective 2 (Data Management & Survival Analysis): To efficiently manage, structure, analyze, and visualize complex biomedical data, specifically implementing advanced survival analysis techniques and modeling time-to-event outcomes using statistical software.
   Related competences:
3. Objective 3 (Clinical Decision Making & Application): To design and implement advanced statistical (such as group sequential methods and adaptive designs) to optimize decision-making processes in clinical trials, including early termination for efficacy or futility.
   Related competences:
4. Objective 4 (Ethical Judgements & Uncertainty): To integrate incomplete or limited clinical data to make reasoned, critical judgments, taking into full account the social, ethical, and safety responsibilities associated with human clinical trials and medical research.
   Related competences:
5. Objective 5 (Scientific Communication & Argumentation): To reason, argue, and clearly communicate scientific-technical conclusions, study rationales, and statistical reports to both specialized (medical/statistical) and non-specialized audiences without ambiguity.
   Related competences:
6. Objective 6 (Autonomous Learning & Professional Drive): To develop self-directed learning skills and a continuous improvement mindset, enabling the student to autonomously master new advanced methodologies and face future professional challenges in the fast-evolving field of medical statistics.
   Related competences:

Contents

1. Introduction and Statistical Foundations
   Description of the different types of medical studies and conceptualization of the challenges in the field and the scope of the subject. The concepts of bias, confounding, and causal inference are introduced, along with a review of the statistical tools necessary for evidence-based inference. In particular, data types and descriptive statistics, probability and distributions, hypothesis testing, p-values and confidence intervals, and sample size and statistical power.
   
   1. Observational study designs (cohort, case-control, cross-sectional)
   2. Experimental study designs (non-randomized trial, randomized controlled trial)
2. Clinical Trial Design
   This part focuses on clinical trials as a central tool in experimental medical research. The phases and objectives of trials, the main randomization methods, and the statistical techniques that allow for the decision of early trial termination due to signs of efficacy or futility are examined.
   
   1. Phases and objectives of clinical trials
   2. Randomization methods
   3. Group sequential methods for early termination
3. Survival Analysis
   Many medical studies evaluate the time until an event (death, relapse, recovery) instead of simple presence or absence. Survival analysis is the branch of statistics designed for this context, with applications that go beyond medicine: engineering (time-to-failure) or economics (unemployment duration).
   
   1. Observation schemes and censoring
   2. Basic concepts in survival analysis
   3. Estimation of the survival function and cumulative hazard
   4. Comparison of survival curves between treatment groups
   5. Design and sample size in survival clinical trials
   6. Regression models in survival analysis

Activities

Activity Evaluation act

Teaching methodology

Evaluation methodology

Bibliography

Basic

• Group Sequential and Confirmatory Adaptive Designs in Clinical Trials. - Wassmer, G., & Brannath, W. , Springer Cham., 2016. ISBN: 978-3-319-32062-5
  
• Survival analysis : a self-learning text - Kleinbaum, David G; Klein, Mitchel, Springer, cop. 2012. ISBN: 978-1-4419-6645-2
  https://discovery.upc.edu/discovery/fulldisplay?docid=alma991004029919706711&context=L&vid=34CSUC_UPC:VU1
• Fundamentals of clinical trials - Friedman, Lawrence M; Furberg, Curt; DeMets, David L; Reboussin, David M; Granger, Christopher B, Springer International Publishing, [2015]. ISBN: 978-3-319-18538-5
  https://discovery.upc.edu/discovery/fulldisplay?docid=alma991005096357706711&context=L&vid=34CSUC_UPC:VU1
• Statistical Issues in Drug Development - Senn, S. (2021). , John Wiley & Sons., 2021. ISBN: 978-1-119-56554-3
  
• Statistical models and methods for lifetime data - Lawless, Jerald F, Wiley-Interscience, c2003. ISBN: 9781118033005
  https://discovery.upc.edu/discovery/fulldisplay?docid=alma991005236579706711&context=L&vid=34CSUC_UPC:VU1

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