Original post: 11 Aug 2025 | Updated: 21 Apr 2026
Developing a medicine that is accessible for patients remains a big challenge, especially in areas like rare diseases or drug repurposing. Researchers publish new papers with potential treatments every day, but the path from lab to clinic is long, expensive and hard to navigate. Traditionally, academic inventions trigger tech transfer offices to out-license or spin off into a startup. But what if there is another way? One where you can stay involved in the research and increase the chances of success?
At Orfenix we believe that public-private partnerships in drug development offer a powerful alternative for academic drug development. They are one of the practical models within socially responsible drug development, a broader approach that keeps patient accessibility and affordability central from day one. If you want to understand the full picture of that approach, read our guide on socially responsible drug development. This article focuses on how a PPP works in practice, and why, at Orfenix, it is almost always the structure we land on when a venture makes sense.
We involve academics as dedicated participants throughout all stages of medicine development. This way the risk of the development running aground can be reduced, while the researcher stays involved and together we work toward the shared goal: an accessible product. The incorporation of socially responsible terms in novel PPPs helps to ensure that academic endevours ultimately benefit patients and society.
Let’s take a closer look at what PPPs are, how they work and why you should consider them when developing a novel medicine.
What Is a Public-Private Partnership in Drug Development?
A public-private partnership in drug development brings together different stakeholders around a shared goal: developing a therapy that reaches patients. The PPP itself is a structure, often a dedicated legal entity such as a Limited Liability Company, in which all partners share risks, responsibilities, and rewards.
The major players within a PPP are typically academic institutions, pharmaceutical companies of various sizes, patient organisations, governmental funding programmes, and mission-aligned private investors. Each brings something the others lack. That complementarity is the point.
The strength of the PPP lies in its flexibility. For each new development, the agreements underlying the partnership can be tailored to the specific pathway. An academic partner might provide access to key opinion leaders and disease expertise. A patient organisation might lead on clinical study design and patient recruitment. A private partner oversees product development and regulatory strategy. The arrangements can manage dynamic aspects, like overall development progress, while also providing a solid foundation for fixed elements like governance and intellectual property.
In a socially responsible drug development model, these arrangements go further. Pricing principles, access commitments, and affordability assumptions are written into legal documents from the start, so they survive partner changes and later negotiations.
Why Consider a Public Private Partnership Instead of licensing or spinning out?
Before deciding on a PPP structure, the first question is whether your project should become a venture at all. Our guide on the venture decision in socially responsible drug development helps you work through that question carefully.
At Orfenix, when a venture makes sense, it almost always takes the form of a public-private partnership. The reason is straightforward: academia stays involved. In our experience, academic involvement is not a complication, it is a feature. Public sector research institutions contributed to between 9.3 and 21.2% of all FDA-approved drugs between 1990 and 2007 (Stevens et al., New England Journal of Medicine). A PPP is designed to keep that expertise inside the development structure, rather than licensing it away. It keeps the science sharp, the mission intact, and the governance accountable.
Traditional routes offer real value in the right context. Licensing out a discovery can be a quick way to transfer promising results to a party with development expertise. But it often means losing control over pricing, development direction, and ultimately patient access. Spinning out into a new company offers more control, but typically requires academic researchers to become entrepreneurs overnight, building teams, raising funds, and acquiring development expertise from scratch.
A PPP offers a different path:
- Preserve your scientific role. The academic researcher remains fully involved in scientific development without taking on entrepreneurial obligations.
- Access existing expertise. Private partners bring in regulatory, clinical, and commercial development expertise, eliminating the need to build it from scratch.
- Share the risk. All parties carry responsibilities. No one bears the full burden alone.
- Keep patient access central. By aligning goals from the start, PPPs make it structurally easier to prioritise affordability, patient involvement, and long-term accessibility.
This is especially relevant in rare diseases, neglected conditions, and drug repurposing. These are areas where the commercial incentive for traditional investors is limited. By combining academic expertise, public funding, and private development experience, a PPP creates a more sustainable pathway toward medicines that patients can actually access.
How a Public-Private Partnership Works in Practice
Within the broader framework of socially responsible drug development, a PPP is often the practical answer to one of the most important steps: building a consortium around your strengths and gaps, with clear roles, shared governance, and aligned goals. Here is what that looks like in practice.
Define roles and governance early. A PPP only works when everyone knows what they are responsible for and how decisions get made. All parties must agree on governance structures, including voting rights, IP ownership, and data use, before development pressure builds.
Tailor the legal structure to the pathway. Not every PPP looks the same. The contracts underlying the partnership are designed around the specific development needs, regulatory route, and funding mix of that project. Steward ownership structures are gaining ground in mission-driven development. They separate economic rights from voting rights, keeping control with those accountable to the mission.
In addition, securing mission-aligned funding is not optional. It is what makes the PPP fundable and sustainable. The funding mix typically combines public grants, disease-specific funds, and impact-oriented investors. The key is that all funders understand and support the shared goals around access and pricing before they commit.
Finally, aligning clinical and regulatory planning from the start is one of the most important steps. One of the most costly mistakes in drug development is designing studies that do not support the eventual authorisation or reimbursement pathway. Early dialogue with regulators and payers reduces this risk significantly and can shorten the real path to patient access.
A real-world example: GuanaRep BV
A good example of the model in action is GuanaRep B.V., a Public-Private-Partnership we helped launch in 2024 to develop a promising treatment for the devastating rare disease Vanishing White Matter (VWM).
Researchers at the Amsterdam UMC discovered VWM, unraveled it’s mechanism of action, and developed a functional pre-clinical model over the course of many years of research. But they did not stop there, they identified a potential lead and translated the candidate into clinical trials. Rather than licensing out the idea or setting up a spin-off, the researchers chose to form a PPP, with Orfenix taking on the governance, product- and regulatory development responsibilities and Amsterdam UMC maintaining its key scientific role.
The PPP GuanaRep B.V. was structured to allow for:
- Joint decision making through shared governance
- Access to both public funding and private (social) investments
- Clear agreement on IP, data use and responsibilities
- A common strategy that will ensure sustainable access to the treatment for patients
GuanaRep B.V. now functions as the formal legal entity that drives the development. All parties within the partnership invest in the collaboration with their expertise and/or funding and ultimately share all risk and in the future all potential benefits.
How Orfenix Can Help
At Orfenix, we specialise in building public-private partnerships that are professionally managed, mission-aligned, and built to last. We help academic groups turn discoveries into accessible therapies by filling the gaps: from regulatory strategy and clinical trial design to governance setup, business case development, and investor relations.
Before you commit to any structure, we look at the fundamentals: Is the business case sound? Is the development plan realistic? Are the right partners available? We can support you with feasibility assessments, Product Development Plans, governance design, venture development and management, and partnership structure design.
If your goal is to see your science reach patients, and to do that in a way that keeps affordability and access central, we would like to talk.
For further reading, see: https://pubmed.ncbi.nlm.nih.gov/36938792/
