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Rare Diseases Require Smart Approach (Press Release)


Rare Diseases Require Smart Approach (Press Release)

In collaboration with partners in the Netherlands, Germany, Austria, Spain and Britain, the University Medical Center (UMC) Utrecht is launching a novel research project called ASTERIX. The project focuses on the development of more efficient and effective research designs to study new drugs and treatments for rare diseases. The overall aim is to stimulate the search for treatments for these devastating and largely ignored diseases.

Investing in the patient
The research project aims to develop new analytical methods and uses results of existing clinical trials from the participating academic hospitals from different European countries. Unique in this project is that patients are directly involved in the research process and their input is taken into account in design and analysis of studies. "We want to adapt existing regulations based on the results of this study, ultimately convincing pharmaceutical companies to invest in new drug development for rare diseases, and thus helping the patient," says project coordinator prof. Kit Roes of the Julius Center for Health Sciences and Primary Care at UMC Utrecht.

Grant from the European Union
In order to reliably assess the effectiveness and safety of drugs, a large number of patients is normally required during the development phase. In clinical research on drugs for treating rare diseases such large number are usually not feasible. Thus, such drug development projects are long, highly uncertain and therefore very expensive. For pharmaceutical companies it therefore presents a high-risk investment as compared to the potential return on investment. Clinical studies for the treatment of rare diseases must therefore be designed and analyzed differently. Yet with smaller numbers of patients, the effectiveness and safety of drugs must still be guaranteed. The EU considers this development as an important priority for research and has awarded a grant of € 3 million for the development of innovative methodology for clinical research. Consortium partners UMC Utrecht, AMC Amsterdam, Hannover Medical School , Medical University of Vienna, Autonomous University of Barcelona, the patient organization VSOP and the organization of statisticians in the pharmaceutical industry (PSI/EFSPI) will develop within the ASTERIX project new methods in the coming years, allowing research into medicines for rare diseases to be performed faster and more effective.

Effective and cheaper, with guaranteed quality
Kit Roes: "It is very important to improve our research methods in this field, because the total number of patients suffering from a rare diseases adds up to more than 30 million, and this is only in Europe. We need a smarter approach to find new treatments fast and cost-efficient while the quality of the research is guaranteed. In the end, this will lead to affordable treatments for this great variety of patients."