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Entrepreneurship in rare disease space

It’s Rare Disease Day. Since 2008 the last day of February marks the Rare Disease Day to raise awareness about rare diseases and their impact on patients' lives. Today we join the global Rare Disease community to start the conversation on the parent/patient-led drug development initiatives.

“Where do you start?” is a question many rare disease patients and parents must ask themselves when learning about the diagnosis. For us this is a relevant question as well. There are many inspiring stories about patient/parent-led initiatives in the Rare Disease Domain that push research and development for cures, sometimes just fundraising, other times collaborations with biotechs, or even setting up just such a company. But ... where do we start?

The theme of this year’s Rare Disease Day is Research. Rare disease research is crucial to providing patients with the answers and solutions they need, whether it's a treatment, cure or improved care (RDD). More than one third (37%) of respondents of Rare Barometer survey declared that they have already participated in a research study. These studies targeted clinical trials, quality of life studies and research projects on genetic therapies or medical devices. Primarily, in order to help science and the community. With our research we want to start a conversation about developing treatments via and by startups. While numerous startups work in rare disease space, finding a working business model appears difficult. So, is it a viable way? If not, what is a better way? Entrepreneurship in healthcare is not always met with open hands, so, is it an appropriate way to bring treatments to the patients?

We want to ask opinions of a broader community: patients, families, foundations, researchers, drug developers and everybody who cares. What are the best practices, and what are bottlenecks and obstacles to the treatment and startup development in rare disease space?

We truly believe that patient and parent led initiatives are the driving force in rare disease drug development, and as researchers, teachers and enablers of new entrepreneurial activities, we want to contribute! In order for us to be able to fully contribute to the global Rare Disease Community, we could use your help! We have set up a short and open questionnaire and we would love to hear from you. Please, take five minutes for the survey!

Also, on 27 & 28 June we ( UGCE ) organize the High Tech Small Firm conference in partnership with University of Twente, VU-Amsterdam, University of Hull, and SOM Research Institute. During this conference we will pay special attention to business development in the Rare Disease domain, with a separate track for papers talking about drug, treatment and venture development in Rare disease space. We also organize a Rare Disease Drug Development Panel to bring together perspectives of research, philanthropy, regulators and investors. If you would like to participate, feel free to submit your research or practical contribution via our website! Need more information? Please, contact us at

Rare disease day 2018
Rare disease day 2018
Last modified:03 July 2019 4.31 p.m.