Clinical Trials [MSBMM103]

Learning outcomes

• The student will be familiar with the major principles of research adopted in clinical trials;
• The student will be able to recognize the different types of clinical trials and understand the key methodological aspects of these various trial types;
• The student will grasp the various key components of a clinical trial protocol and be capable of critically reviewing it;
• The student will comprehend the main statistical analyses applied in clinical trials and be able to interpret them;
• The student will be able to analyze potential methodological biases that may be present in a clinical trial. Furthermore, the student will be familiar with good research practices (EQUATOR);
• The student will understand the importance of publishing the results of a clinical trial and will have gained a foundation in meta-research in the field of clinical trials.

Objectives

Allowing students to explore fundamental concepts related to clinical trials while maintaining a broad and interdisciplinary approach to clinical trials beyond pure pharmacological studies, enabling the application of these concepts across various fields and disciplines. The course will be designed to equip students with enough knowledge to foster a critical mindset towards the methodologies employed in clinical trials

Content

Methodology of clinical trials; development of a clinical trial protocol ; systematic literature search ; Type of clinical trials (i.e. cross-over studies; equivalence and non-inferiority trials, pragmatic trials, open-single blind, double blind, triple blind trials); real world therapeutic effectiveness, pragmatic trials; statistical power of clinical trials; judgment criteria (primary, secondary outcome, quality criteria to be considered for a relevant outcome, etc.); patient-reported outcome measure (PROM) (HRQoL, psychometric properties of questionnaires; EMA-FDA requirements); statistical analysis of a clinical trial (statistical effect, intention-to-treat analysis, intermediate analyses, subgroup analyses, survival analyses, validation of a surrogate marker, etc.); monitoring of clinical trials (role of sponsor, role of CRA, etc.) ; methodological biases in clinical trials; good clinical practices ; registries of clinical trial (e.g. clinicaltrial.gov) ; critical reading of randomized controlled trials (good research practice, reporting guidelines, EQUATOR network, etc.); non-pharmacological clinical trials; patient's preference for treatment characteristics (example of a Discrete Choice Experiment); cost-effectiveness analyses; publication of a clinical trial ; good research practice (introduction to meta-research, pressure of publication, fraud in clinical trials, etc.); etc.

Exercises description