From Compassionate Use to Launch Strategy: Early Access Programmes Are Becoming a Critical Part of Drug Development & Market Access Planning

Early Access Programmes provide access to investigational medicines for patients with serious or life-threatening conditions who cannot enrol in clinical trials and have no comparable or satisfactory treatment options.

However, as access pathways mature and companies plan earlier in the lifecycle, these programmes are increasingly being used to support broader organisational goals. This can include physician familiarisation, patient identification, real-world evidence generation, reimbursement preparation and launch sequencing.

PwC describes early managed access programmes as an integral part of the market access continuum, noting that they can shape go-to-market and hospital engagement strategies by generating insights before launch and exposing organisations to the maturity of the markets they are entering.

The same PwC report highlights a growing industry focus on Early Access, citing a 15% year-on-year increase in publications related to early managed access pathways and identifying 523 unique therapeutics associated with early access activity.

This industry momentum reflects a broader change in how access teams view these programmes. Rather than treating them as late-stage exceptions, leading organisations are now asking how Early Access Programmes can be designed earlier, governed more consistently and integrated into clinical, regulatory, medical affairs and commercial planning.

Early Access Programmes are expanding as companies seek to deliver therapies sooner, prepare markets ahead of approval, build physician experience, generate real-world data and, in selected markets, explore commercial early access opportunities.

This is particularly relevant for biotechs and rare disease companies, where patient populations are smaller, unmet need is often high, clinical trial participation may be limited and access decisions can have significant implications for launch readiness.

Sciensus has similarly noted that Early and Expanded Access Programmes in rare disease are moving “from stopgap to strategy,” with forward-thinking biotechs using them to test real-world operations, build supply chains, engage clinicians and understand regional access nuances before regulatory approval.

For smaller companies, this creates both opportunity and pressure. A well-designed Early Access Programme can support patient need, generate operational learnings and build credibility with physicians and patient communities. A poorly designed programme can create reputational risk, supply pressure, inconsistent access and significant internal burden.

PwC recommends that companies assess managed access programmes across four dimensions: strategic alignment, feasibility, viability and risk. These include corporate strategy, patient-centricity, regulatory compliance, product attributes, funding, pricing, real-world evidence and supply continuity.

This level of complexity explains why Early Access Programmes increasingly require input from multiple functions, including Medical Affairs, Market Access, Regulatory, Clinical Operations, Evidence Generation, Supply, Quality and Pharmacovigilance.

The 5th Operationalise: Early Access Programmes Summit Europe has been built for exactly this cross-functional challenge. The summit will unite 140+ leaders across Medical Affairs, Access, Regulatory, Clinical Operations and Supply to discuss how programmes are being designed, adapted and delivered in practice.

With speakers from organisations including UCB, Eisai, Novartis, BeOne Medicines, Alnylam Pharmaceuticals, Genmab, AstraZeneca, Bristol Myers Squibb, Argenx and ACADIA Pharmaceuticals, the summit offers practical insight from teams actively shaping global Early Access Programme strategy.

Across three days, attendees will explore how to plan, launch and scale programmes, navigate European and global access pathways, manage clinical supply, address post-trial access, understand paid early access opportunities and design programmes that remain patient-centred while supporting long-term access strategy.

As Early Access Programmes become more embedded in development and launch strategy, the organisations that succeed will be those that prepare earlier, align internally, understand national pathway differences and learn directly from peers who have already operationalised programmes in complex markets.

Download the full event guide to see how we will help teams turn early access strategy into practical execution.