As early access programs continue to expand across Europe, pharmaceutical and biotech teams are facing increased pressure to get investigational drugs to patients early while navigating fragmented national regulations, increasing expectations for real-world data, rare disease access challenges, and increasingly complex global program delivery.
Early access programs, also known as expanded access, managed access, compassionate use, or prior authorization access programs, allow eligible patients with serious, life-threatening, or severely debilitating conditions to access investigational therapies outside of clinical trials when no satisfactory approved treatment options exist.
Across Europe, these programs are becoming increasingly important within broader drug development and patient access strategies. However, unlike markets with a single national framework, Europe’s early access pathways remain largely regulated at national level. Although the European Medicines Agency can issue recommendations on compassionate use under Article 83 of Regulation (EC) No 726/2004, their implementation is determined by individual Member States, leading to wide variation in eligibility criteria, approval routes, timelines, reimbursement models, physician application processes, and supply requirements.
This national variation makes Europe one of the most complex regions to implement early access programs. Companies looking to launch programs across multiple markets need to determine where access is available, which national routes are most appropriate, how the supply of investigational drugs will be managed, what data can be collected ethically and legally, and how the program can be aligned with future regulations, reimbursement, and launch strategies.
The strategic role of early access is also changing. Although historically viewed as a reactive, compassionate use mechanism, early access programs are increasingly being planned early in the product lifecycle. For pharmaceutical and biotech companies, these programs can support physician familiarization, patient identification, supply chain preparation, market access readiness, and generation of real-world evidence before formal approval.
This evolution is particularly evident in rare diseases, where patient populations are small, access to clinical trials is limited, and unmet need remains high. For some patients, early access programs may be the only route to receiving promising investigational treatments before they are released. At the same time, rare disease programs often present additional operational challenges, including dispersed patient populations, limited clinical expertise, expedited timelines, complex eligibility decisions, and increased patient advocacy efforts.
Real-world data is another area that is receiving increasing industry attention. Data generated through early access programs can help companies better understand treatment use, safety, dosing, patient burden, clinical practice, and long-term access needs outside of the controlled environment of clinical trials. However, the primary goal of these programs remains patient access. As a result, companies must carefully balance evidence generation with ethical, regulatory, and operational considerations to avoid increased burden on patients, families, physicians, and health care systems.
These challenges will be at the heart of the discussion at the 5th Operationalise: Early Access Programs Summit Europe, to be held from 19-21 October 2026 in London, UK.
The summit brings together more than 140 leaders in medical affairs, patient access, managed access, early access, regulatory issues, clinical operations, evidence generation, and clinical supply to explore how organizations plan, launch, and scale early access programs across Europe and global markets.
Over three days, participants will consider practical strategies for navigating Europe’s fragmented regulatory pathway, designing paid early access models, managing post-trial access, collecting meaningful real-world data, improving clinical supply planning, supporting access to rare diseases, and building a scalable global program infrastructure.
Experts contributing to the 2026 conference include senior leaders from organizations such as UCB, Eisai, Novartis, BeOne Medicines, Alnylam Pharmaceuticals, Genmab, AstraZeneca, Bristol Myers Squibb, Argenx, and ACADIA Pharmaceuticals.
As Europe’s early access landscape continues to evolve, this summit will provide 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.
Take a closer look at the entire agenda here
