Goicea, C. (2026) A Comparative Study of Parent-Founded Rare Disease Ventures: Development Over Time and Factors Shaping Progress Towards Market Entry. Bachelor's Thesis, Biomedical Engineering.
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Abstract
An estimated 300 million people worldwide are affected by rare diseases, often with no cure available. In these cases, it is often the parents of affected children who take action, founding their own foundations, patient organizations, or biotech companies. Nevertheless, there is a lack of studies on how these organizations develop and the reasons behind some developing more successfully than others. This paper attempts to fill this gap by studying what drives the variance in development of parent-led initiatives for rare diseases. Specifically, this research explores how these organizations develop by employing qualitative comparative case-study analysis. As a result, a set of 26 organizations was reduced to 10 organizations, which followed one of the four paths: nonprofit research catalyst, biotech company, venture philanthropy, and merger/transfer. The aforementioned 10 organizations were analyzed through the prism of nine factors, such as funding, professionalization, collaboration, scientific and clinical risks, and adaptability to setbacks. It was found out that these paths have different strategic trade-offs but do not represent different success levels of the organizations. Moreover, organizational status cannot be used as an indicator of their success since some active organizations did not get to the stage of having an approved therapy and those organizations, which lost their independence, succeeded in preserving their mission in another organization.
| Item Type: | Thesis (Bachelor's Thesis) |
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| Supervisor name: | Belousova, O. and Groen, A.J. |
| Degree programme: | Biomedical Engineering |
| Thesis type: | Bachelor's Thesis |
| Language: | English |
| Date Deposited: | 16 Jul 2026 14:21 |
| Last Modified: | 16 Jul 2026 14:21 |
| URI: | https://fse.studenttheses.ub.rug.nl/id/eprint/37437 |
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