The joint development of (orphan) drugs by physicians, academic researchers, and industry partners is a promising approach to improving the availability of therapies for rare diseases. This requires new forms of collaboration that explicitly prioritize societal value. At RARE-NL, we use a collaboration model that integrates both financial and societal value. Its core pillars are:

Transparency and Fair Pricing: Costs, investments, and pricing are made fully transparent. Prices are based on actual costs plus a reasonable profit margin. Any profits are reinvested into a revolving fund to finance new projects.

Sustainable Risk Sharing: All parties share risks through innovative project structures where they co-own the development. This enables flexibility and customization while external funding is pursued to distribute financial burdens fairly.

Accessibility: All parties commit to making treatments accessible to patients. Development is not halted if there is evidence of patient benefit and a positive business case.

A Socially Responsible Framework for Public-Private Partnerships (PPPs)

A public-private partnership requires contributions from both sides. The academic partner brings knowledge and public funding to accelerate development and reduce costs. The company contributes additional financing, expertise in economic feasibility, regulatory compliance, reimbursement, production, and access to distribution channels.

RARE-NL promotes public-private partnerships that foster collaboration across all stages of development — from basic research to market launch. Governance is carried out by closely involved stakeholders, such as academic researchers. Data and knowledge are shared equitably in line with GDPR and FAIR data principles, enabling all stakeholders to contribute effectively.

Finally, our framework includes eleven concrete recommendations that serve as a checklist during early-stage discussions between partners. These are drawn from literature, real-world experience, and insights from previous projects. They help shape sustainable, productive, and socially responsible collaborations — ultimately getting effective treatments to patients faster.