Expert public only
Intensive Short Course
This course introduces participants to design such trials, focusing on the pragmatic approach and trials aiming to support decision-making in the real world. It will consider emerging technologies and routinely collected data that are increasingly shaping the future of randomized trial evidence generation. The course is intensive, experiential, and intended for individuals or small teams who are interested in real world evidence.
Course description: There is an increasing interest in providing real world evidence for health care decision-making. Randomized trials often have characteristics that make them not useful to guide such decisions. There are two approaches to design of randomized trials that can help to solve such problems and provide more useful trials. They were first described by Joseph Schwartz and Daniel Lellouch, two French statisticians who wrote a seminal paper in 1967: Pragmatic trials are aimed at informing a health care decision, namely which of several treatment options to choose in a real world situation, while explanatory trials are more like laboratory experiments, testing whether or not a particular mechanism of action is responsible for a change in outcome.
While we take the perspective of those who design trials, the course will also be helpful for those who use randomized trials for example in systematic reviews, health technology assessment, regulatory or funding decisions. Exposure to basic theory or practice of randomized trial design is required, e.g. from an introductory epidemiology course or through participation as a coinvestigator in a completed, not necessarily pragmatic randomized trial.
With full engagement, those who bring a prepared research question to the course can expect to produce a well-considered 2 page outline proposal for a pragmatic randomized trial, which could become the basis for writing a full submission for funding. For participants who do not have a specific intervention in mind, you will join a group with others who do, and apply your learning to their proposed intervention, or work on example projects provided by the lecturers.
The course will last 3 full days (from 9 am to 5 pm). To get the most out of it, preparation before and during the 3 days will be beneficial. The format for each session contains a didactic lecture on pragmatic attitudes and real world evidence, with plenary questions and discussion, followed by small group meetings, in which each group writes that section of their own protocol, followed by plenary discussion of a couple of groups’ written protocol sections. Over the 3 days everyone attending will present sections of their groups protocol and take part in plenary discussions of theirs and others protocols.
Do you have a conviction that a particular therapy or intervention would benefit patients, communities or healthcare delivery, and the open-mindedness to want to know if your conviction was valid under real world conditions? If yes, you can learn how to design a randomized trial that evaluates your conviction.
The randomized trial you have in mind may be of a drug, a surgical or other clinical procedure, a digital health app, a medical technology, a change in how one or other health care service is delivered. Alternatively, you may not know what kind of intervention you will one day want to evaluate, but you urgently want to learn how to evaluate interventions under real world conditions in randomized trials.
Pre-Course readings: Info to come
- Course language: English
- ECTS points: 2,5
The completion of an assignment after the course is mandatory for the award of ECTS credits. The assignment will be the development of a draft outline proposal, either on an own project idea or based on an example case.
- Course Fee: € 625, € 425 for students (proof required)
Organizational and administrative matters
Merrick Zwarenstein, MBBCh, MSc, PhD is an internationally-recognised primary care and health services researcher, who received his PhD in Epidemiology at the Karolinska Institute (Sweden), an MSc in Community Health at the London School of Hygiene (UK), and an MScMed in Microbiology and a medical degree (MBBCh) from the Witwatersrand University Medical School (South Africa). He is a full Professor in the Centre for Studies in Family Medicine, at the University of Western Ontario in Canada and a senior scientist in IC/ES, the research organization using Ontario’s electronic administrative data sets on health and health care for policy and clinically relevant analyses. He holds honorary positions at the Universities of Cape Town and Stellenbosch in South Africa, and the Karolinksa Institute in Sweden. He has led international research consortia focussing on overcoming limitations with the use of randomised trials in support of policy decision making.
Lars G. Hemkens, MD, MPH is Senior Scientist and Deputy Director of the Basel Institute for Clinical Epidemiology and Biostatistics (ceb), Department of Clinical Research, University Hospital Basel. His key interest is to inform health care decision-making and includes answering health care questions with data that were not made for answering those questions. His work focuses on routinely collected data and big data, pragmatic trials and real world evidence, and meta-research. He co-designed various clinical studies in a wide range of medical fields. He is board member of the Network for Evidence Based Medicine, associate editor of Trials, sits in the editorial board of BMJ Evidence-Based Medicine and in the working committee of the RECORD reporting guideline group. He is QUEST visiting fellow at the Berlin Institute of Health and coordinator of the RCD for RCT initiative, which aims to explore the use of routinely collected data for clinical trials.
Berlin School of Public Health
Institute of Public Health, Charité - Universitätsmedizin Berlin
9:00 - 17:00h