Biostatistical Challenges of Trials in Rare Diseases

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 to patients faster 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.

In this webinar, featured speakers will discuss this topic, including:

• A brief review of the European and international initiatives on rare diseases
• A description of special statistical designs and analysis methodologies to address the problem of small populations. Innovative methodologies in design and analysis of clinical trials in small population groups will be summarized focusing on results from the IDeAl project (Integrated DEsign 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

Speakers

Prof. Dr. Ralf-Dieter Hilgers, Department of Medical Statistics, RWTH Aachen University, Germany

Professor Hilgers graduated in mathematics from the RWTH Aachen University and received his PhD in statistics at the University of Dortmund, while working at that time at the University of Cologne. He has been Professor in Biostatistics and chair of the Department of Medical Statistics at the RWTH Aachen University since 2001. Professor Hilgers gives biostatistical advice to clinical and experimental trials in all clinical and preclinical areas. From 2013-2017, he coordinated the IDeAl project funded by the European Community (www.ideal.rwth-aachen.de), which established new methodologies for small population group trials.

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Everardo Saad, MD, Medical Director, International Drug Development Institute (IDDI)

Everardo Saad has over 15 years of experience in Medical Oncology and clinical trial designs. He graduated in Medicine and trained in Internal Medicine in Sao Paulo, and did his fellowship in Medical Oncology at the University of Texas M.D. Anderson Cancer Center, in Houston. After practicing for several years, he shifted his professional career towards education and research in Medical Oncology, and has a special interest in clinical trial methodology, the assessment of endpoints, and the development of novel therapies for cancer patients.

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