Utrecht University develops knowledge agenda on rare diseases
Utrecht University researchers have worked with the Netherlands Organisation for Health Research and Development (ZonMw) to develop a knowledge agenda on rare diseases. This will be used by the Dutch Ministry of Health, Welfare and Sport to guide research and innovation in the field.
The breadth and heterogeneity of rare diseases pose a challenge. Limited patient populations and therefore limited market opportunities means certain types of rare diseases are ignored by research organisations and companies. In addition to this, the field covers a broad range of activities including fundamental research, prevention, diagnostics, therapy and care, some of which get more attention than others. This emphasises the need for additional support for research and innovation.
Expertise from the Innovation Studies group of the Copernicus Institute of Sustainable Development
Guiding future research and innovation
In order to guide the direction and priorities for future research and innovation, the Dutch Ministry of Health, Welfare and Sport asked The Netherlands Organisation for Health Research and Development (ZonMw) to develop a knowledge agenda. ZonMw requested the expertise of researchers from the Innovation Studies group at the Copernicus Institute of Sustainable Development at Utrecht University.
Exploring expert priorities for diverse aspects of rare diseases
Utrecht University researchers used the Delphi method to explore expert priorities for diverse aspects of rare diseases, including diagnostics, fundamental research, clinical research, drug research, care research, social and psychological research. Experts with diverse backgrounds, including patient organisations, general practitioners, companies and academic hospital researchers actively participated in the multiple rounds of surveys that are part of the Delphi method.
The purpose of a Delphi study is to create the most reliable consensus among a group of experts on a given topic. “First we retrieved all possible research areas from documents and from experts. We then categorised the knowledge questions and let the experts prioritise them. Finally, the experts validated the list of prioritised knowledge questions,” say the authors.
You can download the report here.