The Power of Study Design

Abstract

The course will guide the students through designing a clinical study and introduces established study designs and study elements, as well as modern study concepts and designs to address clinical questions. Another focus is on patient perspectives and patient preference studies.

Objective

After taking this course, participants will be able to - understand the patient perspective as an integral part of clinical studies and clinical decision-making when planning their own studies - describe the difference between including versus involving patient and family stakeholders in research studies - identify ways to involve patient stakeholders in research. - apply analytical schemes and frameworks to appraise a clinical study - understand the structure and content of clinical trial documents according to Swissethics and apply the knowledge in creating a study synopsis - understand and explain study outcomes, their meaning and different hierarchies - understand the difference between different Clinical Outcome Assessments (COAs) and explain their implications for the results of clinical trials and describe how Patient-Reported Outcomes (PROs) are developed and adapted, tested and applied in a cross-culturally sensitive way - describe and categorise psychometric properties of measurement instruments (reliability, validity, consistency, responsiveness) - explain why study participants are randomly selected in randomised controlled trials, what this means in practice for the conduct of a clinical trial and how the results are to be classified - identify and describe the most common study designs in observational Phase IV drug safety research and discuss the strengths and limitations of each design - identify and describe advanced study designs in observational research - identify and overcome different sources of bias - describe modern alternatives to classic study designs - understand the concept of decentralised clinical trials and its impact on the statistical planning and organisation of a trial

Content

This first module of the CAS ETH in Digital Clinical Trials (CAS ETH DCT) provides an overview of the fundamentals of clinical studies and new study methods (e.g., involvement of patients as partners, decentralised trials) which will be applied in a study synopsis on a self-selected or assigned topic during the CAS ETH DCT programme. Thus, it serves as a starting point for the subsequent modules, where the knowledge will be deepened, and the application of digital technologies (e.g., digital measures, real-world data and remote monitoring) will be introduced. The topics are: 1. Critical appraisal of clinical studies using case studies, application of evidence levels, and utilization of checklists 2. Designing clinical studies and involving patients 3. Planning and conducting clinical studies in an ethical way 4. Outcomes in clinical studies, definition, hierarchies, Clinical Outcome Assessments (COA), psychometric methods for validating Patient-Reported Outcomes (PROs), and cross-cultural adaptations 5. Feasibility of clinical studies 6. Decentralised clinical trials 7. Observational studies and their place in clinical research, and pros and cons of different study designs 8. Bias and confounding factors 9. New study designs: adaptive, basket, umbrella, data enriched design, master protocols

Resources