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Welcome to the ASTERIX project

Advances in Small Trials dEsign for Regulatory Innovation and eXcellence

“We need to design smarter and effective clinical trials for small populations. We owe it to the millions of children and adults suffering from a rare disease and in need of new, proven treatments.”  
Kit Roes, principal investigator

“This project builds on unique collaboration between statisticians with extensive regulatory and clinical development experience, epidemiologists and -  importantly - patient representatives.”  
Armin Koch, principal investigator 

An estimated 30 million European patients suffer from a rare disease. More than 6000 rare diseases are known, many of them chronic, potentially very disabling and typically affecting children. Since 2000, more than 850 orphan drug designations have been granted by the European Commission, but less than 100 orphan drugs have reached the market. There is a substantial unmet need for drug treatment for rare diseases. If this need is to be met, many clinical trials will need to be performed in small populations.

ASTERIX was a novel EU-funded research project focusing on the development of more efficient and effective research designs to study new drugs and treatments for rare diseases. The overall aim was to stimulate the search for treatments for these devastating and largely ignored diseases.

Objectives

ASTERIX was a novel EU-funded research project focusing on the development of more efficient and effective research designs to study new drugs and treatments for rare diseases. The overall aim was to achieve more reliable and cost-efficient clinical development of treatments for rare diseases and to stimulate the search for treatments for these devastating and largely ignored diseases.

The main objectives were to:

  • Develop design and analysis methods for single trials and series of trials in small populations.
  • Include patient-level information and perspectives in design and decision making throughout the clinical trial process.
  • Validate new methods and propose improvements for regulatory purposes.

Methodology

ASTERIX worked through six highly interactive and interdependent Work Packages ranging from development of methodology, stakeholder participation to the dissemination of the results. Unique in this project was that patients were directly involved in the research process and their input is taken into account in design and analysis of studies.

Results and Reports

ASTERIX results together with reports summaries and related information are available online on the European Commission website. The full list of articles published in a scientific journal can be found in the Publications section.