EJP RD Training: Replicated N-of-1 Randomized Controlled Trials for Rare Diseases

An N-of-1 trial is defined as a prospective, multiple crossover trial in a single patient. If randomization of the treatment sequence and replication across patients is included in the trial design, then it results in “replicated N-of-1 RCTs”. These replicated N-of-1 RCTs have been recommended to test evidence-based treatments for patients with rare diseases, but several design and statistical issues have to be taken into account when such a research strategy is chosen. In the webinar, we will demonstrate one approach to the statistical analysis of data collected in N-of-1 RCTs based on the randomization of the treatment sequence, which is both simple and sound. Furthermore, several meta-analytical options for the statistical comparison and aggregation of the replications will be presented. 

The aim of the lecture is:  

• Present replicated N-of-1 RCTs as an option to test evidence-based treatments for patients with rare diseases. 
• Discuss the advantages and disadvantages of replicated N-of-1 RCTs for rare diseases. 
• Demonstrate the use of simple and sound statistical methods for the analysis of data collected in N-of-1 RCTs and replicated N-of-1 RCTs. 

This training is at an advanced level course. While an overview will be given, without all technical details, a variety of statistical modelling and inference concepts will be used.   

 This training is divided in two parts: 

 The first part of the webinar, of about one hour, is devoted to the lecture “Replicated N-of-1 RCTs for Rare Diseases” presented by Patrick Onghena. 

 The second part of the webinar consists of one hour of panel discussion with known experts from different fields bringing various perspectives of the topic. This will include the regulatory, pharma-industrial, academic, and clinical perspective. In particular, questions from the audience will be answered while reflecting these different perspectives

Patrick Onghena is full professor at the Faculty of Psychology and Educational Sciences, KU Leuven, Belgium, and visiting professor at the University of Barcelona, Spain, and the University of Sydney, Australia. He has an academic background in psychology and statistics and teaches quantitative research methods and statistics to undergraduate, master, and doctoral students in the behavioral and educational sciences, speech and language pathology, and master of statistics. His research interests include single-case experimental designs, N-of-1 clinical trials, distribution-free statistical inference, meta-analysis, and mixed methods research, and he authored and co-authored numerous publications on these topics in both methodological and substantial international journals. He was associate editor of Behavior Research Methods, Journal of Statistics Education, and section editor of Intervention studies/Observational studies in The Encyclopedia of Statistics in Behavioral Science for which he provided many entries. Together with Wim Van den Noortgate he elaborated the multilevel approach to meta-analysis, and applied this approach in the area of single-case experimental designs. He is best known for his work on randomization tests and for co-authoring the classical handbook on this topic together with Eugene Edgington (now in its fourth edition). 

The training webinar is organized as a series of lectures presented by experts in the specific topics. Interaction between participants and lecturers will be facilitated by moderated question & answer sessions.  

The workshop is open to the international research community, clinicians, medical specialists, healthcare professionals and advocacy patient groups with knowledge on the RD Clinical Trials. 

The registration is mandatory and until June 29 2023, 11:45pm.

Register here.

The training and registration are free of charge.  

The training organisers will not cover expenses incurred by the participants in any way.  

At the end of the training, participants will be requested to fill an online questionnaire as feedback for learning and impact assessment.  

A certificate of attendance can be requested via email to dbodden@ukaachen.de.

No credits of Continuing Education in Medicine will be issued.  

English.

Online. Details for the connection will be provided the day of the webinar to registered participants.  

This Training webinar is organized by Universitaetsklinikum Aachen (Germany) and Assistance Publique Hopitaux de Paris (France).  

If you have questions, please contact the course organisers through this email address: dbodden@ukaachen.de and maya.dirani@ejprd-project.eu.

Please, indicate in the subject: WP20 June training webinar