|Date & Time
|14 December 2021 16:00-18:30 CET
This is an online training course organised by the European Join Programme on Rare Diseases. Rare Diseases (RD) is an area where national and international multidisciplinary collaborations are valuable to overcome some of the present challenges in research. Comparative trials serve as a major basis for the evaluation of new therapies in RD patient care. Randomisation as a tool for treatment allocation in comparative trials and blinding are the accepted standard tools to enable a fair treatment comparison by mitigating bias. The unique challenges of RD clinical trials originate from the limited sample size and heterogeneity in the patient population, so that researchers may think about restricting or totally avoiding randomisation.
Recent findings underpin the view, that randomisation in small populations clinical trials like in RD is different from classical clinical trials. Regardless of this difference in the value of randomisation, in RD clinical trials, more attention should be paid to the selection of the best performing randomisation procedure. This offers on the one hand improved mitigation of bias and on the other hand the potential for an alternative basis for the inference.
The main focus will be on randomisation aspects in comparative clinical trials with a parallel-group design, where no adaptation of the trial based on interim inspections is applied. Therefore, knowledge of common clinical trial terminology is essential to successfully follow the course. The course will teach the ideas and concepts and will try to avoid analytical derivations, if possible.
The aim of the lecture is:
- to learn about other randomization procedures beyond the most frequently applied permuted bock randomisation in a fixed sample scenario,
- to understand properties of different randomization procedures beyond balancing sample sizes,
- to argue, which randomisation procedure fits best in a particular trial setting,
- to know about the potential advantages of randomisation from design to analysis of clinical trials in RD.
This training is an advanced level course.