What rare disease commercialisation in Europe teaches us - and why biotechs are leaning in

Rare disease launches are the ultimate stress test for European commercialisation. Small, dispersed populations, variable diagnostic pathways, and multi‑country reimbursement frameworks mean every step must be designed, owned, and measured.

As Rare Disease Day approaches, the lessons from recent European launches are clear: success comes from disciplined operating models, disease‑centred engagement, pragmatic evidence strategies, and collaboration across the ecosystem. Europe is demanding - but for biotechs that align to how care is actually delivered, it can also be decisive.

For me, this work is personal as well as professional. As a father to a daughter with a rare condition, I understand firsthand why timely, thoughtful commercialisation matters - and why families cannot afford delays or fragmented strategies.

Why Europe is challenging, and why innovators are leaning in

Europe is not one market. Health technology assessment (HTA) requirements, reimbursement processes, and prescriber norms differ by country; direct‑to‑patient promotion is restricted; and specialist capacity is uneven. That complexity rules out a copy‑and‑paste playbook.

Yet, the region also offers meaningful advantages for rare innovators: public funding and an increasing regulatory and business incentive that priorities high unmet need once value is established; structured access routes; and specialist centres capable of delivering consistent quality. For companies willing to build around clinical pathways, evidence, and coordination - rather than just promotion - Europe is both viable and scalable.

Lesson 1: Discipline is the differentiator

In rare disease, complexity is a constant. That’s why discipline - not tactics - separates success from struggle. Every touchpoint matters: pricing and market access strategy, recognition and referral, diagnostic work‑up, initiation, benefits verification, adherence, and follow‑up. The operating model must make each step explicit and accountable.

In practice, that means standardising what works across geographies (structured onboarding, evidence‑based education, coordinated clinical and access workflows, predictable paths to initiation) and then localising for each system. Teams that rely on improvisation hit bottlenecks; teams that operate from disciplined design absorb complexity without losing momentum.

Lesson 2: In Europe, disease‑centred engagement beats brand‑led models

Direct‑to‑patient approaches don’t translate in Europe. Where promotion is restricted, disease‑centred models become essential: education on recognition and referral, support for pathway navigation, and coordination with clinical teams - all designed around the condition and care ecosystem, rather than the brand.

As programmes scale, the model should flex - high-touch human support for complex cases; digital enablement, automation, and guided self‑service for predictable tasks. That balance protects scarce specialist resources while maintaining quality at higher volumes.

Lesson 3: Value must be simple, explicit, and evidence‑led

Payers have limited time and many priorities. In rare disease - where datasets are smaller and endpoints may be unconventional - the value story must be straightforward and backed by evidence that matters in day‑to‑day care. The effective approach starts early: burden‑of‑illness, treatment patterns, resource utilisation, and outcomes aligned to HTA expectations. Post‑launch, measuring adherence, persistence, and relevant clinical outcomes protects access over time. In small populations, real‑world performance is not a reporting exercise; it is the backbone of sustainable pricing and coverage.

Lesson 4: Localisation and collaboration are non‑negotiable

Care is delivered locally - and rare disease care often involves caregivers, patient organisations, and specialty centres with distinct norms. One‑size‑fits‑all frameworks underperform. The most resilient programmes are modular by design and built to integrate with local practice.

Equally important: collaboration. Disease‑focused ecosystems benefit when manufacturers work with innovative health‑tech and digital health partners to extend capabilities - from patient identification and education to monitoring and coordination - within local pathways. In practice, these programmes are rarely built in isolation; good partners reduce fragmentation.

A preview of what’s next

Rare Disease Day underscores that in rare disease commercialisation decisions directly shape patient access and, in Europe, there is little room for error.

Success depends on discipline from the start: programmes built around real clinical pathways, grounded in locally relevant evidence, and designed to work country by country. Disease‑centred engagement, pragmatic value strategies, and coordinated execution are the baseline.

Europe is demanding, but revealing. Rare disease launches expose every weakness in a commercial model, but, when done well, show what patient‑centred commercialisation must look like.

About the author

Mike Ryan is general manager, Europe, at EVERSANA. With more than 25 years of experience in the life sciences industry, Ryan has held global leadership positions in both clinical development and life sciences software companies. Given this experience, he is highly experienced in the use of technology and understands its impact in simplifying the process of developing and delivering novel therapies and making them more accessible to patients around the world. He has worked with many biotech and pharmaceutical companies to develop clinical pathways that deliver upon the ultimate commercial goals required to ensure success for all stakeholders. Ryan speaks at many global industry events to promote the idea of innovation in healthcare and to encourage a greater level of partnership between healthcare stakeholders.