Consultancy
Advocacy
Bridging pharma and biotech with patient communities
Overview
Rare disease patient advocacy organisations hold something a sponsor cannot replicate internally: direct, trusted relationships with the people living with a condition, often built over years and sometimes decades. CortexBio bridges pharma and biotech teams with those communities, building partnerships that are genuinely useful to both sides rather than a one-off outreach exercise ahead of a trial.
What we do
- Strategic advocacy planning — engagement strategies tailored to your therapeutic area, culture and stage of development.
- Patient organisation mapping — finding and connecting with the most relevant organisations for your specific focus.
- Coalition building — linking researchers, clinicians, regulators and advocates into a working ecosystem.
- Authentic partnership — long-term, trusted relationships with patient communities, not transactional contact.
Where this creates value
In rare disease, advocacy organisations are frequently the fastest route to patient identification, the most credible source of natural history insight, and — increasingly — a required voice in regulatory submissions. Building that relationship late, only once a trial needs recruiting, tends to be read by the community for what it is. CortexBio's approach starts the relationship earlier and keeps it two-way: advocacy groups get a sponsor who understands their priorities and constraints, and the sponsor gets access to insight and trust that cannot be bought at the point of need.
Why CortexBio
Dima Martini-Drew, MD, has worked directly with rare disease patient organisations across four enzyme replacement therapy programmes at Genzyme, Astellas and Abbott, and is a founding industry member of the Critical Path Institute's rare and orphan disease programmes — a body built specifically to connect industry, regulators and patient communities around shared evidence standards.
Who this is for
Sponsors entering a rare disease area for the first time and needing an introduction to the relevant patient community, teams preparing for a trial who need advocacy input on recruitment and design, and companies approaching a regulatory submission where patient organisation support strengthens the case. This work overlaps closely with CortexBio's patient-focused drug development service, and the two are frequently scoped together.
Frequently asked questions
What does patient advocacy engagement involve for a pharma or biotech sponsor?
Building genuine relationships with the patient organisations relevant to a therapeutic area — understanding their priorities, sharing information appropriately, and involving them early enough that the relationship is a partnership rather than a request for help at trial recruitment.
Why does advocacy engagement matter in rare disease specifically?
With small, dispersed patient populations, advocacy organisations are often the fastest and most credible route to patient identification, natural history insight and regulatory-relevant patient input — and regulators increasingly expect to see that engagement.
How does CortexBio identify the right advocacy organisations to engage?
Through patient organisation mapping specific to the condition and therapeutic focus, drawing on direct experience of the rare disease advocacy landscape built across 30 years in the field.
What is coalition building in this context?
Linking researchers, clinicians, regulators and advocacy organisations into a working ecosystem around a shared disease area, so evidence, priorities and relationships compound over time rather than resetting with every new programme.
When should a sponsor start engaging patient advocacy organisations?
As early as possible — advocacy relationships built before there is a specific ask tend to be more trusted and more useful than those built at the point a trial needs recruiting, when the community can reasonably read the outreach as transactional.
Can advocacy engagement help outside the UK and Europe?
Rare disease advocacy is an international landscape, and many organisations operate across borders or coordinate with international umbrella bodies. Mapping and engagement is scoped to wherever the relevant patient population and its organisations actually are, not limited to a single jurisdiction.
How an engagement works
Advocacy engagement usually starts with mapping the organisations relevant to a specific condition or therapeutic area, understanding each one's priorities, resources and existing relationships with industry. CortexBio then helps design an engagement approach appropriate to the sponsor's stage — early-stage teams often need introductions and a credible first conversation, while later-stage programmes may need structured input into trial design, recruitment support or regulatory submissions. Throughout, the relationship is treated as two-way: advocacy organisations get a sponsor who understands their constraints and shares information appropriately, not just a request for help.
Because trust in this space is built slowly and lost quickly, CortexBio favours a smaller number of genuine, well-maintained relationships over broad, shallow outreach.