Rare diseases affect a small number of patients. As a result, companies and knowledge institutes invest less of their time and money in innovation for rare diseases. To further drive innovation in this area, the European Union and national governments have set up a number of incentives and grant schemes.
The Ministry of Health, Welfare and Sport and the Netherlands Organisation for Health Research and Development (ZonMw) asked researchers of the Copernicus Institute of Sustainable Development to study the Dutch R&D environment; how the Netherlands compares to other European countries; and if there are missing areas the Dutch Government should invest in.
In our final report, we presented an overview of activities that contribute towards diffusion of innovations, focused mainly on the two main categories of rare diseases: ‘metabolic disorders’ and ‘neurological conditions’. Within these two categories, we listed the efforts in acquiring new knowledge, clinical development, mobilising resources, initiating start-ups and patient advocacy. These five areas were subsequently compared to other countries.
Our study showed that The Netherlands currently ranks fifth in terms of academic output and first in output per capita. Proportionally, academic hospitals are the main contributors to rare didease research in The Netherlands. Dutch publications listing clinical trials on rare diseases contribute to 4% of the world output, 1% higher than the Dutch average for all medical publications (3%). In addition, a rare disease with a Dutch orphan drug designation, is studied more often by Dutch knowledge institutes and is of higher interest to Dutch companies. Finally, the report finds indications for the importance of a well-functioning innovation ecosystem in which companies, knowledge institutes, academic hospitals and patient organizations collaborate in creating pharmaceutical products for rare diseases.