Statistical Methods for rare diseases and special populations

Monday, 13 November 2017 - Barcelona

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Statistical Methods for rare diseases and special populations

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 Global Statistical Sciences - Bone & New Diseases at UCB Biosciences GmbH
Lisa Comarella - Director Biostatistics at CROS NT Group
Frank Fleischer - Expert Statistician Methodology at Boehringer-Ingelheim
Giacomo Mordenti - Global Biometrics Director at Livanova

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

Pre-Conference Dinner

The dinner will provide an opportunity for you to network informally with your industry peers:

Barcelona - Sunday, November 12th, 7:30 PM (the meeting is set for 7:15 PM at the restaurant)

Restaurant: La Fonda del Port Olìmpic
Moll de Gregal, 7-10
08005 - Barcelona

The price is € 35,00 in addition to the fee for the conference.
Please confirm your interest on the registration form or during the registration online

Please click here to read the code of good conduct

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