Developing a medicine that is accessible for patients remains a big challenge, especially in areas like rare diseases or drug repurposing. Every day new papers are published with potential treatments in the scientific literature, but the path from lab to clinic is long, expensive and hard to navigate. Traditionally, academic inventions trigger tech transfer offices to out license it or spin it off to 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 (PPPs) offer a powerful alternative for academic drug development. 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 to the shared goal: an accessible product. The incorporation of socially responsible terms in novel PPPs helps to ensure that academic endeavours 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 Partnerships (PPP) brings together different stakeholders. All share the common goal to develop a novel therapy. The PPP itself is a structure, often a legal entity such as a Limited Liability Company (or ‘Besloten Vennootschap’ in the Netherlands), wherein risks and rewards are shared. The major players within PPPs are the pharmaceutical industry (small, medium, and large pharma), academic institutions, patient organizations, governmental funding programs, and private investors.
The strength of the PPP lies in its flexibility to account for the challenges encountered during the translational stage. For each new development, the contracts underlying the PPP can be tailored to the need of the development pathway. For example, the academic party can provide access to key-opinion leaders or specific equipment, a patient organisation might lead the clinical work, while the pharmaceutical company oversees product development and regulatory strategy. The specific arrangements of the PPP can be used to manage both more dynamic aspects (e.g., the overall progress of the different development stages) as well as provide a solid foundation for aspects that are less changeable (e.g. governance or intellectual property). Finally, the arrangements can detail processes around the topics that are too far out to make solid arrangements on (e.g., pricing and market access).
The PPP is particularly relevant in fields such as rare diseases and drug repurposing. Here the commercial incentive is lacking for traditional investors because of the limited opportunity to generate a substantial return on investment. By combining academic expertise, public funding, and private development expertise, PPPs share risk, attract (social) investors, and create a more sustainable pathway for patient accessible drug development. Therefore, the model offers an integrative approach for academic involvement in medicine development. By sharing risks and rewards, failures in the translational stage can be mutually absorbed.
Why Consider a Public Private Partnership Instead of licensing or spinning out?
Traditionally, tech transfer offices choose to license out academic inventions or spin it off into a new company. Licensing out a discovery can be a quick way to transfer key research results of a potential medicines to a party with development expertise. However, it often means losing control over the product development, its pricing, and, ultimately, the patient’s access to the product. Spinning out into a new company offers more control but often requires academic researchers to become entrepreneurs overnight, capable of building teams, raising funds and gaining development expertise from scratch. In addition, the university, will need to be willing and able to assume the risks of being a shareholder in that specific startup.
PPPs are a great new option, with several benefits:
- Preserve your scientific role. In a PPP the academic researcher remains fully involved in the scientific development, without new entrepreneurial obligations
- Access to existing external expertise. Private parties bring in development expertise, eliminating the need to build new teams and gain expertise from scratch
- Spread risks: All parties in the partnership share responsibilities. No one carries the full burden alone.
- Focus on patient access: By aligning the common goal from the start, PPPs make it easier to prioritize patient involvement, pricing and long-term sustainable accessibility.
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.
Conclusion
Public-Private-Partnerships offer a good alternative to licensing and setting up a spin-off. This is especially relevant if you don’t want to choose between giving up control or taking on everything yourself. At Orfenix, we specialize in building these partnerships. We help academic groups turn discoveries into products by filling in the gaps: from regulatory strategy and clinical trial design to governance setup and investor relations. If your idea has promise and your goal is to see it reach patients, we’d love to talk. If you’re exploring drug development options and want to build a PPP that’s truly focused on patient access, get in touch!
For further reading, see: https://pubmed.ncbi.nlm.nih.gov/36938792/
