We will start by briefly reviewing the European and international initiatives on rare diseases.
We will describe special statistical designs and analysis methodologies to address the problem of small populations. Innovative methodologies in the design and analysis of clinical trials in small population groups will be summarized focusing on results from the IDeAl project (IntegratedDEsign and AnaLysis of clinical trials in small population groups).
Ongoing efforts to bring the innovative methodology into practice and the next steps will be addressed in the conclusion.
Webinar on Biostatistical Challenges of Trials in Rare Diseases
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ABSTRACT:
The definition of what is considered a rare disease differs all over the world. However, a common estimate is that the number of rare diseases currently amount to 6 to 8 thousand, with these disorders affecting almost 30 million people in Europe alone. Recent initiatives foster cross-border collaboration to bring new treatments faster to the patients, while recognizing the special demands of research on rare diseases. While clinical trials are a key component in drug development, research in rare diseases presents additional challenges compared with more common diseases.
SPEAKERS
Prof. Dr. Ralf-Dieter Hilgers
Everardo Saad, MD
Professor in Biostatistics and chair of the Department of Medical Statistics, RWTH Aachen University, Germany
Medical Director at the International Drug Development Institute (IDDI)
Presentation of the Webinar Biostatistical Challenges of Trials in Rare Diseases
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