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Society/business UGCE Research Center

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.

If you had to develop a new drug and bring it to the market, where would you start? What would you do if it was a drug for a rare disease and barely dozens of researchers around the globe were working on the topic? And if the whole process costed so much, big pharma companies would not dare to step onto this path? And if every day counted for saving a life of somebody who is very important to you, where would you start?

Karen Aiach, Keynote, Precision Medicine 2016
Karen Aiach, Keynote, Precision Medicine 2016

For most of us, the barriers and difficulties to become a biotech entrepreneur would look unsurmountable, but there are people, who dare and succeed. It was an article in Forbes about Karen Aiach and her journey to fight the rare disease her daughter suffers from, that triggered us to look into this. Although she was a merger & acquisition specialist and knew nothing about creating a new drug, she is now a respected expert on Sanfilippo A, the neurodegenerative disease of her daughter, and a founder of Lysogene, a VC-backed company that aims to create solutions for this and similar diseases.

Rare disease infographic
Rare disease infographic

This and numerous other stories we found, inspired us to look into the world of patient/parent driven drug development. Orphan, or rare, diseases are defined as those that affect fewer than 200,000 (USA criteria) or fewer than 50,000 (UK criteria) people. However, for nearly 7000 rare diseases (together accounting for almost 400 million people!), there are only between 250 and 400 (according to different sources) treatments and biologics developed, with only 9 such drugs being profitable. Parents and patients are increasingly taking matters into their own hands in an effort to accelerate research and attempt to discover a treatment for their disease. This is where we want to contribute with our research.

At UGCE we decided to study the stories of parent/patient-led rare disease initiatives from an entrepreneurship perspective. Studying the processes that people went through, we want to know what hurdles they face, in order to raise awareness of the problems and develop tools to overcome them. We want to know how these entrepreneurs create, organize and use their collaborations – team members, partners, stakeholders, associations, agencies, and others. Rare disease space is an active and living organism with many players working together to contribute to new solutions. We want to know how to navigate this space, and where there could possibly be an improvement. We want to know how these entrepreneurs learn about the science behind the drug development, or about the business side to raise finance and bring the solution to the market, in order to develop support programs and help future initiatives advance faster. For example, through business development support as we do with health entrepreneurs in EIT Heatlh VentureLab.

Our current research team consists on prof.dr. Aard Groen, Aniek Ouendag, MSc, and dr. Olga Belousova (PI), who together work on finding a better way to contribute to the global community from both, academic and practical perspectives. Feel free to contact any of us, if you want to know about our research and development, have your story heard or your case written, or want to join the team.

Last modified:19 December 2017 10.05 a.m.