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Symposium on New Methodology for Clinical Trials in Rare Diseases

Asterix End Symposium - 18 & 19 September 2017, Zaandam (the Netherlands)

General Information
The final symposium of the EU FP 7 funded Asterix project is an open, interactive conference on innovation of clinical trial methodology for rare diseases. Its objectives are:

  • To share the progress made to advance clinical trial methodology, in the interest of developing new treatments for patients suffering from a rare disease.

  • To share and illustrate proven and potential impact on clinical trials, with a special focus on drug development and regulation.

  • To stimulate further implementation and continued development of methodology.

The symposium follows the approach that is also part of the project: involving and integrating the views of patients, regulators and clinical researchers.


The target audience was wide: Statisticians and (clinical) researchers working in the field of small populations, patient representatives and all others interested in new methodology for clinical trials for rare diseases.


Program & Sessions
The sessions generally focus on one of the broader areas that have the potential to improve the design, analysis and interpretation of clinical trials. Each session covers topics from a methodological, clinical and patient perspective. Innovations arel further illustrated against regulatory experience of orphan drug approvals over the past 15 years. Throughout the Asterix proposed clustering of conditions is used to aid practical guidance for application of the proposed methods.

Details on the program and overview of the speakers can be be found here.

Presentations are availeble at the end of this page.


Date and Venue
Our Symposium took place in the Inntel Hotel in Zaandam, the Netherlands, and started on Monday September 18th, 1.00 pm with a joint reception at the end of the day. After the reception a joint dinner (optional, Costs € 42,-) was organized  from 7.00 pm until 10.00 pm. On Tuesday September 19th our symposium ended around 5.00 pm.



Download the flyer to advertise the Symposium

Program and confirmed speakers



Session 1: Disease clustering to guide trial design and analysis

Clustering of rare medical conditions based on applicability of methods and designs for clinical trials, Caridad Pontes (Universitat Autònoma de Barcelona)

Systematic evaluation of the applicability of novel methods to the study of rare medical conditionsMarian Mitroiu
(University Medical Center of Utrecht) and Katrien Oude Rengerink (University Medical Center of Utrecht, CBG)

Modelling at a product level as a way to test applicability and move forward to future recommendations,  Arantxa Sancho (Instituto de Investigación Puerta de Hierro, Madrid)

Session 2: Fundamental challenges in small populations - Ethical Framework and patient involvement

Rethinking the ethics of rare disease research, Mark Sheehan (Ethics Advisory Board, University of Oxford)

The role of patient engagement in clinical research, Kerry Leeson-Beevers (Patient Think Tank, Alstrom Syndrome, UK)

First experiences with the POWER model to involve patient representatives in choosing trial outcome measures, Charlotte Gaasterland (Academic Medical Center Amsterdam)

Session 3: Fundamental challenges in small populations - Correct evidence: the role of randomization and observational data

A plea to randomization – lessons learned from the case of digoxin, Lukas Aguirre (Hannover Medical School) - presentation will be available after publication of the manuscript

The role of rare disease registries in drug development, Martine Jansen-van der Weide (Academic Medical Center Amsterdam)

Registries do have potential to be used in clinical trialsVincent Gulmans (Dutch Cystic Fibrosis Foundation)

A patient perspectiveElizabeth Vroom (Patient Think Tank, Duchenne Parent Project)

Registries and trial data and the real world, David Haerry (European AIDS Treatment Group)

Session 4: Potential and pitfalls of meta-analysis in small populations

Meta analysis of a small number of small studies: methods and added value, Kit Roes – coordinator Asterix (University Medical Center of Utrecht)

No solution yet – few studies and heterogeneity, Theodor Framke (Hannover Medical School)

Prospective inclusion of historical efficacy data, Stavros Nikolakopoulos (University Medical Center of Utrecht)

Session 5: Improving clinical trial design: sequential, multiple endpoints and multiple treatment comparisons

Advantages of study designs with multiple endpoints or treatment, Gerd Rosenkranz (Medical University Vienna)

Methods to combine functional loss and mortality in clinical trials for amyotrophic lateral sclerosis, Leonard van den Berg (University Medical Center of Utrecht)

A new study design to demonstrate efficacy in cystic fibrosis, Peter van Mourik (University Medical Center of Utrecht)

Session 6: Use of an alternative endpoint in clinical trials

Regulatory view on alternative endpoints in clinical trials in rare diseases, Kit Roes - coordinator Asterix (University Medical Center of Utrecht)

Brief introduction to Goal Attainment Scaling, Charlotte Gaasterland (Academic Medical Center Amsterdam)

Why a drug developer is interested in Goal Attainment Scaling, Edwin Spaans (Khondrion)

Goal Attainment Scaling: pinpointing elusive differences in haemophilia therapy effectiveness, Radoslaw Kaczmarek (PTT, European Hemophilia Consortium)

Session 7: How to justify different evidentiary standards for decision making in rare disease?

Perspective on the minimum evidence to make regulatory decisions, Fernando de Andrés-Trelles (Universidad Complutense of Madrid, SAWP-, PDCO-EMA Member)

Uncertainties and coping strategies in the regulatory review of orphan medicinal products, Nikolaos Zafiropoulos (Medical University of Vienna)

Leverage existing evidence: evidence, eminence and extrapolation, Gerald Hlavin (Sozialversicherungsanstalt Österreichs)

A decision-theoretic value of information approach to the design of clinical trials in small populations, Nigel Stallard (University of Warwick, coordinator INSPIRE)

Session 8: Implementation and Continued Development