As Early Access Programmes continue to expand across Europe, pharmaceutical and biotech teams are facing growing pressure to deliver investigational medicines to patients earlier, while navigating fragmented national regulations, rising real-world data expectations, rare disease access challenges and increasingly complex global programme delivery.
Early Access Programmes, also referred to as Expanded Access, Managed Access, Compassionate Use or Pre-Approval Access Programmes, allow eligible patients with serious, life-threatening or severely debilitating conditions to access investigational therapies outside of a clinical trial when no satisfactory authorised treatment option exists.
Across Europe, these programmes are becoming increasingly important within broader drug development and patient access strategies. However, unlike markets with a single national framework, European early access pathways remain largely regulated at country level. While the European Medicines Agency can issue recommendations for compassionate use under Article 83 of Regulation (EC) No 726/2004, implementation is determined by individual Member States, creating significant variation in eligibility criteria, approval routes, timelines, reimbursement models, physician request processes and supply requirements.
This country-by-country variation is making Europe one of the most complex regions in which to operationalise Early Access Programmes. Companies seeking to launch programmes across multiple markets must now determine where access is feasible, which national pathway is most appropriate, how investigational product supply will be managed, what data can ethically and legally be collected, and how programmes can be aligned with future regulatory, reimbursement and launch strategies.
The strategic role of early access is also changing. Historically viewed as a reactive compassionate-use mechanism, Early Access Programmes are increasingly being planned earlier in the product lifecycle. For pharma and biotech organisations, these programmes can support physician familiarisation, patient identification, supply-chain readiness, market access preparation and real-world evidence generation before formal approval.
This evolution is particularly significant in rare disease, where patient populations are small, clinical trial access may be limited, and unmet need remains high. For some patients, Early Access Programmes can represent the only route to receive a promising investigational therapy before launch. At the same time, rare disease programmes often present additional operational challenges, including dispersed patient populations, limited clinical expertise, urgent timelines, complex eligibility decisions and heightened patient advocacy engagement.
Real-world data is another area drawing increased industry attention. Data generated through Early Access Programmes may help companies better understand treatment use, safety, dosing, patient burden, clinical practice and long-term access needs outside the controlled environment of clinical trials. However, the primary purpose of these programmes remains patient access. As a result, companies must carefully balance evidence generation with ethical, regulatory and operational considerations to avoid increasing burden on patients, families, physicians and healthcare systems.
These challenges will be central to discussions at the 5th Operationalise: Early Access Programmes Summit Europe, taking place 19–21 October 2026 in London, UK.
The summit will bring together 140+ leaders across Medical Affairs, Patient Access, Managed Access, Early Access, Regulatory Affairs, Clinical Operations, Evidence Generation and Clinical Supply to examine how organisations are planning, launching and scaling Early Access Programmes across Europe and global markets.
Across three days, attendees will explore practical strategies for navigating Europe’s fragmented regulatory pathways, designing paid early access models, managing post-trial access, collecting meaningful real-world data, improving clinical supply planning, supporting rare disease access, and building scalable global programme infrastructure.
Senior leaders from organisations including UCB, Eisai, Novartis, BeOne Medicines, Alnylam Pharmaceuticals, Genmab, AstraZeneca, Bristol Myers Squibb, Argenx and ACADIA Pharmaceuticals are among the experts contributing to the 2026 meeting.
With Europe’s early access environment continuing to evolve, the summit provides a dedicated forum for industry leaders to benchmark approaches, share lessons learned and address the practical realities of delivering investigational medicines to patients outside of clinical trials.
Dive into the full agenda here