Pragmatic trials: Study designs for real-world decision-making
There is an increasing interest in providing real world evidence for health care decision-making. Traditional randomized trials, also known as explanatory, often have characteristics that make them not useful to guide such decisions. This course introduces and deepens the concept of pragmatic trials to support decision-making in the real world.
The course is designed as a step-by-step guide providing an in-depth understanding of the historical foundations and philosophy of clinical trial conception and cutting-edge developments in pragmatic trial design. It will provide a solid understanding of how to design a clinical study that is most useful to inform decisions (pragmatic trial).
You will learn to make optimal design decisions and understand the implications of these choices, for example regarding selection of eligibility criteria, recruitment strategies, controls, adherence, measurement- and follow-up-intensity, choice of primary outcomes, and concepts of analytical approaches.
The course will also consider emerging technologies and routinely collected data that are increasingly shaping the future of randomized trial evidence generation.
The course aims to be highly interactive, intensive, and experiential. It targets those with an intention to conduct a randomized trial to inform medical decisions under real world conditions, soon or at some time in their future career.
This 3-week course requires students to:
- complete weekly assignments (1-2 questions related to the specific topic of the week with reading prerequisites) and
- attend a 4-hour in-person session per week
Monday, 8 May 2023, 9.00-13.00, Lecture Hall 5, Klinikum 1, Universitätsspital Basel, Spitalstrasse 21
Monday, 15 May 2023, 9.00-13.00, Lecture Hall 4, Klinikum 1, Universitätsspital Basel, Spitalstrasse 21
Monday, 22 May 2023, 9.00-13.00, Lecture Hall 5, Klinikum 1, Universitätsspital Basel, Spitalstrasse 21
PhD students of the PhD subject Clinical Research enrolled at the University of Basel, Faculty of Medicine.
Moreover, PhD students of the other PhD subjects of the Faculty of Medicine (Biomedical Engineering, Biomedical Ethics, Medicines Development, Nursing Science, Public Health/ Epidemiology or Sport Science) are eligible to register for the course.
Minimum 8 and maximum 15 registered participants.
Prerequisites for attending
Intention to conduct a randomized trial to inform medical decisions under real world conditions soon or at some time in the future career.
Basic theoretical or practical knowledge of randomized trial design is required, e.g., from an introductory course in epidemiology or from participation as a co-investigator in a completed, not necessarily pragmatic randomized trial.
Upon successful completion of this course, the students will be able to
- Understand basic and advanced principles of pragmatic and explanatory trial designs
- Critically assess the generalizability, applicability, and external validity of randomized clinical trials
- Make optimal study design decisions and understand the implications of these choices for support of decision-making in the real world
- Evaluate different research design options for testing treatment decisions under real world conditions in randomized trials.
- 20 hours of workload distributed over 3 weeks
- Written response to all weekly assignments related to the course content
- Active participation in weekly discussions
- Attendance to all sessions is required
PD Dr. med. Lars G Hemkens
PD Dr. med. Lars G Hemkens
Lead Pragmatic Trials and Real World Evidence at RC2NB
Senior Scientist Department of Clinical Research
Perrine Janiaud, PhD
Clinical Researcher at RC2NB
Research Fellow at the Department of Clinical Research
Training and Education, Department of Clinical Research
The course is free of charge.
Course language is English
Certificates will be issued after successful course completion
Registration is now possible here.
Registration deadline: April 24, 2023
You will be informed about admission to the course after the registration deadline.