Early/expanded access programmes: Making rare treatments accessible ethically and efficiently in Europe
At the World Orphan Drug Congress Europe in Amsterdam, Sciensus experts Andrew Cummins and Mathieu Loiseau joined Pat Furlong, President and founder of Parent Project Muscular Dystrophy, to explore how biotechs can design and deliver early and expanded access programmes (EAPs) that are ethical, efficient and genuinely patient-centred.
From stopgap to strategy: EAPs as launch enablers
Across Europe, EAPs were once seen purely as compassionate-use measures, temporary stopgaps for patients in need of critical therapies. But today, forward-thinking biotechs are treating them as a key part of their launch strategy.
When they’re planned alongside clinical and commercial strategies, EAPs serve as a proving ground for not only the clinical validity of the therapy, but also the viability of real-world operations. For smaller companies in particular, they offer an opportunity to build supply chains, engage clinicians and understand regional nuances long before regulatory sign-off.
Mathieu Loiseau, Director of Rare Clinical Services at Sciensus, noted that the data and relationships built during these programmes can accelerate readiness for launch:
“An EAP is where a company can learn how its treatment fits into everyday healthcare. You see how families manage dosing and side effects, how hospitals coordinate care and where the friction points are.”
The progression from stopgap to strategic launch component is seen frequently in rare-disease commercialisation, where access, evidence and implementation all begin earlier in the product lifecycle than mass-market drugs.
The power of patient engagement and real-world evidence
At the heart of every effective EAP is the patient experience. In rare diseases, where community networks are typically small but well-connected, the way companies communicate and collaborate can define their reputation for years to come.
“Patients often come last,” said Pat Furlong, reflecting on decades of advocacy in Duchenne muscular dystrophy. “If companies don’t engage with patients during trial design, they risk creating unmanageable obligations for families. It works best when companies make access a continuum from trial to EAP to reduce burden and do right by patients.”
Mathieu added, “Keep agreements simple, support patients where you can and always keep communications open.”
Patient feedback doesn’t just improve experience; it enhances data quality. Mathieu highlighted that real-world data from EAPs often reveals what trial endpoints miss ̶ crucial things such as quality-of-life impact, treatment adherence issues and unmet support needs.
Digital tools are increasingly central to improving two-way communication. From homecare logistics to digital diaries, technology is helping to simplify reporting and reduce administrative burden for families.
“Digital outcome measures are excellent, especially with children,” Pat said. “But AI isn’t ready to replace human review ̶ accuracy and empathy must come first.”
By putting patients first and using technology effectively, EAPs can generate both meaningful evidence and lasting trust.
Using EAP insights to shape future market access
As European market access grows increasingly complex, EAPs provide actionable insights that go beyond launch readiness. By revealing what health technology assessment (HTA) bodies and clinicians value, EAPs can inform regulatory submissions, pricing strategies and reimbursement negotiations before market pressures begin.
“We see a lot of companies say: we want to collect RWD. But they’re often not sure what they want to collect and when to start collecting it,” says Andrew. “It’s not something you can do a year before the product launches; it has to be part of the full product lifecycle.”
Data collected through EAPs supports harmonised evidence generation for processes like the Joint Clinical Assessment, helping companies demonstrate treatment value across multiple countries.
Operational learnings, from supply chains to patient support, also help biotechs build scalable, patient-centred access models that reduce burden and enhance long-term sustainability.
Mathieu added, “Operationalising an EAP teaches you how to deliver treatments reliably across sites and countries. Lessons about supply chains and home support can help reduce patient burden and guide future market access discussions.”
Ultimately, EAPs are no longer simply about early access. They’re about building readiness, credibility and confidence for patients, regulators and investors.
Plan early. Partner smartly. Learn continuously.
Europe’s rare-disease ecosystem rewards thorough preparation. The most successful biotechs are those that design access, evidence and engagement as a single, joined-up strategy.
Success in European EAPs comes down to having a clear and structured plan. Companies that break down the regulatory, evidence and operational complexity across Europe methodically can turn early access into a strategic advantage.
Want to learn more?
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