One opportunity. More than one route.
An academic therapy may already have years of research, clinical insight and supporting data behind it. The next question is what is needed to turn that work into a viable development programme. The starting discussion brings together the scientific opportunity, the patient need, the evidence still required, the regulatory and manufacturing work, the intellectual property position and the potential funding routes. The objective is not to create a company for every therapy. A venture is useful when it gives the programme the structure it needs.
The starting question: what needs to be in place for this therapy to progress?
A dedicated venture
Create a focused organisation around the therapy, with a development plan, governance and the capacity to bring funding and specialist partners together.
A separate structure is needed to organise the programme and support its development beyond academia.
What should sit within the venture, and how will academic involvement, development responsibilities and access objectives be reflected?
A coordinated development programme
Bring the necessary work into one development plan, with clear responsibilities across the organisations involved. A new company is not automatically the first step.
The immediate need is coordination of regulatory, clinical, operational or funding work.
Which organisation takes responsibility for each activity, and who coordinates the programme as a whole?
A public-private partnership
Combine academic expertise, public-interest objectives, private capabilities and funding within a shared development structure.
Progress depends on a combination of academic institutions, specialist companies, funders and patient or non-profit organisations.
How will the partners share responsibilities, funding commitments, development risks and access objectives?
Licensing or a pharmaceutical partnership
An existing company may offer an appropriate route to further development and delivery. The relevant question is whether its capabilities, commitments and proposed terms fit the programme.
A suitable partner can take the opportunity forward within its existing development organisation.
What development commitments, licensing terms and patient-access expectations need to be agreed?
Investigator-led or grant-funded progression
Further work may be better carried out within academia before a venture or commercial partnership is appropriate.
The next priority is additional evidence, an investigator-led study or research that can be supported through academic funding.
What evidence is still needed, and what would justify reconsidering the development route?
Pause, reassess or do not proceed
Creating a venture is not appropriate when there is no credible development or sustainable funding case. Further evidence, a different route or a decision not to proceed may be necessary.
The scientific, practical or funding requirements do not yet support progression.
Is there a specific issue that can be resolved, or should effort and resources be directed elsewhere?
These routes can overlap. A venture may sit within a public-private partnership and later enter a licensing agreement. This explorer explains options; it does not determine the right route for a particular therapy.
Four connected parts of one development model.
Venture structure, development planning, funding and access decisions affect one another. Orfenix brings these elements together so that the programme can develop as one coordinated whole.Give the programme a workable structure.
Orfenix helps establish the venture, governance, IP arrangements and responsibilities needed to move the therapy forward while keeping the academic originators involved.
How the institution, venture and other parties work together.
Who leads development, how decisions are made and what sits inside or outside the venture.
Clear roles, governance and a structure for development.
Connect the evidence to the development plan.
Orfenix brings regulatory, clinical, product-development and operational requirements together so the next development steps are clear.
Evidence gaps, regulatory questions, clinical planning, manufacturing, formulation and quality.
What happens next, which activities depend on each other and what expertise is needed.
A coordinated plan with milestones, responsibilities and decision points.
Match funding and expertise to the work.
Funding and partnerships follow the development plan, combining grants, non-dilutive support, investment and specialist capabilities where needed.
Funding requirements, timing, execution capabilities and expectations attached to capital.
Which funding sources and partners fit each stage and whether their expectations align with the programme.
A funding and partnership strategy linked directly to development.
Build patient access into the business model.
Financing, governance, licensing and partnerships can influence future pricing and availability, so access is considered while the programme is being built.
Access objectives, pricing principles, returns and future licensing conditions.
Which commitments matter, how returns are treated and what happens if the programme changes hands.
An access approach built into the programme from the start.
From assessment to patient access
Development is not a straight line. Activities overlap, evidence changes and funding requirements evolve.
Orfenix’ role changes with the programme, from early assessment and structuring to programme leadership, partner coordination and preparation for the next stage of development.
1
Academic science
Understand the starting point. Bring together the scientific rationale, clinical insight, available data and intended patient benefit. What is the therapeutic opportunity, and what evidence already supports it?
Orfenix role: connect the academic opportunity to the wider development questions.
2
Programme assessment
Identify what would make progression viable. Consider the development requirements alongside patient need, intellectual property, the business case, funding options and access objectives. Is there a credible route forward, and which issues need further work?
Orfenix role: bring scientific, development, venture and funding considerations into the same discussion.
3
Route decision
Choose the structure that fits. Compare the possible routes: a venture, coordinated programme, public-private partnership, licensing or further academic development. Pausing is also an option. Which route is proportionate to the opportunity and its development needs?
Orfenix role: define and structure the most appropriate route.
4
Structure & governance
Agree how the programme will operate. Establish the relevant venture, collaboration, intellectual property and governance arrangements. Define the responsibilities of the organisations involved and how key decisions will be made. Who is responsible for which activities and decisions?
Orfenix role: build and coordinate the programme structure.
5
Development plan
Translate the opportunity into defined work. Connect regulatory, clinical, product-development and operational activities with their dependencies, milestones and decision points. What needs to happen next, in what sequence and with which expertise?
Orfenix role: coordinate the development disciplines around one programme plan.
6
Funding partners
Resource the next stage. Link the funding requirement to the development plan and bring in the partners needed to execute the work. Funding may come from several sources rather than a single investment route. Which commitments are required to proceed, and on what terms?
Orfenix role: build the funding pathway and coordinate specialist and funding relationships.
7
Clinical progression
Manage the programme as evidence develops. Clinical, regulatory, product-development and operational activities continue to evolve as new data becomes available. Progress is reviewed against the programme plan and agreed decision points. Does the emerging evidence support the next development step?
Orfenix role: provide programme leadership, coordination and project management as responsibilities evolve.
8
Patient access
Plan for sustainable availability. Development does not stop at approval. The programme also needs a realistic route to supply, reimbursement, licensing or commercialisation that remains compatible with the access objectives established earlier. What needs to be in place for the therapy to reach patients sustainably?
Orfenix role: connect the development and business structure to the agreed access approach.
Access by Design
Carry access terms into future agreements.
A sale or licence can change who controls the development programme. Where appropriate, access principles can therefore be included in the conditions attached to future licensing or transfer.
Make the basis for pricing explicit.
Pricing principles can be considered while the programme is being structured rather than waiting until the product reaches the market. This can include transparency around development costs, expected returns and the basis for a sustainable price.
Align returns with the access model.
The funding structure determines how much capital the programme needs and what investors expect in return. Those expectations need to remain compatible with the affordability objectives of the programme.
Work with organisations that support the model.
Development partners, investors, academic institutions, patient organisations and other stakeholders all influence how the programme evolves. Partner selection therefore matters for both execution and long-term access.
Protect the route to patients.
Access can also be affected by decisions to stop, pause, sell or transfer a programme. The structure can therefore include agreements about what happens when evidence supports continued development.
Consider how future value is used.
Where relevant, part of the economic model can support the development of further therapies or other public-interest objectives.
Not sure which route fits your therapy?
Academic therapies can move forward in different ways. The right route depends on the evidence, development needs, funding and access goals.
Check your programme with our decision tool, or explore how we support other therapies.