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About 1 million people in the Netherlands have a rare disorder with often profound effects on their quality of life and life expectancy. On June 3, RARE-NL was launched; a collaboration of Dutch initiatives from university medical centers and FAST. RARE-NL focuses on integrated research into the treatment of rare diseases and on drug repurposing: developing existing drugs for new indications.
Development of new therapies for rare diseases is challenging due to complexity, small patient populations, high risks for industry with limited ability to recoup investments, regulatory hurdles and often high costs for society. RARE-NL aims to create a national infrastructure for sustainable and affordable therapies by pooling initiatives. Socially responsible public-private partnerships are central to this. Teun van Gelder hopes that RARE-NL will become the platform for giving concrete direction to therapy development for rare diseases and drug repurposing: repurposing existing drugs for other conditions than those for which they were originally developed.
From case study to system
RARE-NL embraces a “case to system” approach and aims to connect expertise, foster collaboration, and share insights to increase availability of therapies for rare diseases and encourage drug repurposing. Lessons and knowledge from case studies will be pooled to improve sustainable access at the system level as well. 'RARE-NL will provide cohesion and exchange of expertise, both preclinical and clinical, to give patients with rare diseases a real chance,' said Dirk Lefeber.
Working together under social conditions
To promote therapy development for patients with rare diseases, public-private partnerships are essential, integrating not only economic but also social values. Especially for rare diseases, more collaborative efforts will be needed to make accessibility and affordability go hand in hand. 'We as RARE-NL want to collaborate with anyone who wants to develop drugs under social conditions,' says Carla Hollak.
Collaboration Hub
RARE-NL is a collaborative initiative involving several university medical centers joining forces as a hub of FAST, the center for Future Affordable Sustainable Therapy Development. Saco de Visser, scientific director explains: "FAST is an independent national center of excellence for therapy development, focused on working together to better bring innovation and affordability together. FAST brings together knowledge, fosters dialogues and engages a wide range of experts. The center identifies key opportunities and challenges, and addresses them."
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Carla Hollak (Medicine for Society, Amsterdam UMC), Teun van Gelder (Academic Pharma, LUMC and National Pharmaceutical Knowledge Center Foundation), Dirk Lefeber (Therapy Accelerator for Rare Diseases, Radboudumc) and Saco de Visser (FAST) are starting RARE-NL. Representatives from other MUMCs are invited to join. More information Medicijn voor de Maatschappij (in Dutch) and RARE-NL.
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