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Event Details


European Statistical Forum

Statistical Methods for rare diseases and special populations

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Who should attend?

The conference is addressed to statisticians, pharmacometricians, physicians, regulators, academia and other experts interested in the field belonging to: Pharmaceutical, and Biotechnology companies, CROs, Universities/Hospitals, Academic Research.


The development of treatments for rare diseases has been largely boosted by several initiatives from regulatory agencies. For example, the FDA has initiated its rare diseases program with the objective “to facilitate, support and accelerate the development of drug and biologic products for the benefit of patients with rare disorders”. In September 2016, an EU-US collaboration to boost medicine development for rare diseases was announced to achieve a common understanding of each Agency’s regulatory approaches to rare diseases drug development.

Furthermore, special populations like most prominently pediatrics have been promoted as a field of unmet medical need and for further investigations. Also on this topic agencies have initiated programs to enhance research. The EMA initiated the Pediatric regulation in 2007 which led to the establishment of the Pediatric Committee with the objective “to ensure that medicines for use in children are of high quality, ethically researched and authorized appropriately and improving the availability of information on the use of medicines for children”.

One common feature for both areas is the limited availability of patients. Therefore, there is an increasing burden in defining a proper trade-off between the feasibility of these trials and the application of traditional statistical approaches for confirmatory regulatory purposes. This challenge has an increased demand on innovative methods and approaches. Techniques to be mentioned in this context are adaptive designs, meta-analysis, enrichment, randomized withdrawal, Bayesian statistics and use of historical data. Also, decision making under uncertainty and in particular extrapolation play a key role here.

The eighth edition of the European Statistical Forum will be dedicated to the topic of “Statistical Methods for Rare Diseases and Special Populations”. Presentations will focus on

  • Innovative statistical methods for rare diseases and special populations
  • Clinical trials designs, endpoints as well as approaches to evidence synthesis and extrapolation
  • Regulatory views on statistical approaches
  • Practical examples and case studies

Thereby, the conference will promote the exchange between experts coming from statistics, medicine, regulatory and academia interested in the field of rare diseases and special populations.

Scientific Board

Jens-Otto Andreas - Head Statistical Sciences - Bone & New Diseases, UCB Biosciences GmbH
Lisa Comarella - Director Biostatistics, CROS NT Group 
Frank Fleischer - Principal Methodology Statistician, Boehringer-Ingelheim 
Giacomo Mordenti - Global Biometrics Director, Livanova


REGISTRATION FEE FOR THE CONFERENCE: 610,00* euro Early Bird fee until October 13th, 2017; 705,00* euro ordinary fee (after October 13th, 2017); 550,00* euro Patronage members fee; 370,00* euro for Academy, Public Administration, Freelance. The fee includes: seat at the conference, copy of presentations of Speakers who allow the distribution, informative literature for the day, networking lunch, coffee break, organisational office assistance, certificate of attendance. *For Italian companies: + 22% VAT


Stefania Sarga

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