As part of the EJP RD Training series, an advanced-level training webinar on the topic “Does Randomization matter in RD clinical trials?” (held on 14.12.2021)
The training consists of two parts: a 1.5-hour training in less frequently applied randomization procedures and their performance on the level of evidence, followed by 1 hour of panel discussion with experts from regulatory, pharma-industrial, academic, and clinical fields.
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 evaluation of new therapies in RD patient care. Randomization as 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 randomization. Recent findings underpin the view, that randomization in small populations clinical trials like in RD is different from classical clinical trials. Regardless of this difference in the value of randomization, in RD clinical trials, more attention should be payed to the selection of the best performing randomization 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.