Why RARE-NL
Rare diseases affect up to 36 million people in the EU, but often no treatment options are available. The ideas and early stages of development of therapies for rare diseases often originate from academic institutions. But further development often stagnates. This may be due to the complexity of the disease, small patient populations, high risks for industry with limited options for reimbursement, regulatory challenges and high costs for society. In the classic pharmaceutical model, the interests of companies, society, academic institutions and the government regularly conflict. This leads to a system failure: innovations for rare diseases such as ATMPs and/or gene-based therapies, including RNA therapies (ATMP+), and drug repurposing do not optimally progress in development. Either the final price is beyond our willingness to pay or there is no suitable, predictable business case.
Academia-driven drug development as a route to accessible and affordable medicines for rare diseases and drug repurposing.
Our approach
RARE-NL aims to bring together expertise. By joining our efforts, we aim to accelerate progress and improve outcomes for people affected by rare diseases. RARE-NL embraces a 'case to system' approach and connects expertise, promotes collaboration and translates insights from individual cases into systemic solutions for the availability of therapies for rare diseases and the repurposing of medicines. Lessons and knowledge from these cases are disseminated at a systemic level through (academic) publications to improve sustainable patient access to repurposed medicines and treatments for rare diseases.
To promote the development of therapies for rare diseases and drug repurposing, new forms of regulation and collaboration in public-private partnerships (PPPs) are essential, aimed at both financial and social added value. All parties must optimally combine their individual strengths and expertise to create such a value chain that covers the entire path from fundamental research to approved medicines.
RARE-NL's activities are focused on supporting academic parties and socially responsible public-private partnerships and are aimed at:
- Drug repurposing: the development of existing drugs for new indications, rare or more common.
- Therapies for rare diseases: the development of treatments for rare conditions.
RARE-NL; A national hub for the development of drugs for rare diseases and drug repurposing.
A collaborative hub
RARE-NL is a collaborative initiative where various university medical centers join forces as a hub of FAST, the centre for Future Affordable Sustainable Therapy Development. FAST is an independent national centre of expertise and collaboration, devoted to driving innovation in therapy development. Serving as a dynamic network infrastructure, FAST consolidates knowledge, fosters dialogues, and engages an extensive range of experts. The centre identifies key opportunities and challenges, addressing them through comprehensive approaches such as use-cases and pilot studies.
About RARE-NL
Many initiatives in all University Medical Centres in the Netherlands address problems in the development of therapies for rare diseases and the repurposing of medicines. RARE-NL aims to establish an infrastructure to support development of sustainable and affordable therapies through a national approach that brings these different initiatives together with a shared vision on socially responsible public-private partnerships.
Initially, the team consists of:
- Carla Hollak ( Medicine for Society at Amsterdam UMC)
- Teun van Gelder ( Academic Pharma and National Pharmaceutical Knowledge Centre at LUMC)
- Dirk Lefeber ( Therapy Accelerator for rare diseases at Radboudumc)
- Saco de Visser ( FAST)
Representatives of other UMCs are explicitly invited to join this approach to drug development under social conditions.