Real-World Data from Early Access Programmes Is Reshaping Evidence Generation, Rare Disease Access & Launch Readiness

Early Access Programmes are primarily designed to provide investigational medicines to patients with serious or life-threatening conditions when clinical trial participation is not possible and no satisfactory treatment alternative exists.

Yet as these programmes expand, the real-world data generated through treatment outside the controlled environment of clinical trials is attracting growing attention from pharmaceutical companies, regulators, HTA bodies and market access teams.

According to pharmaphorum, Early Access Programmes can provide additional information on safety and efficacy in real-world conditions, and while this data cannot replace randomised clinical trial evidence, it can support regulatory dossiers and broader evidence packages.

A cited cross-sectional study of FDA and EMA regulatory approval documentation found that data from Early Access Programmes informed clinical efficacy in 39 approvals, and in 13 cases early access data was central evidence for approval; 12 of those 13 products had orphan drug designation.

This is especially relevant in rare disease, where small patient populations, limited treatment options and strict clinical trial criteria can make evidence generation challenging.

Rare disease is a key growth area for Early Access Programmes and these programmes can become the only pathway for some patients to receive investigational treatments before approval when clinical trial access is limited.

Sciensus similarly highlights the importance of Early and Expanded Access Programmes in rare disease, noting that they can help biotechs build supply chains, engage clinicians, understand regional nuances and generate real-world learnings before regulatory approval.

For companies, the opportunity is significant. Real-world evidence from Early Access Programmes may help teams understand how therapies are used in practice, how patients and families manage treatment, where operational friction occurs, and what support models are needed to sustain access beyond launch.

However, the role of real-world data in Early Access Programmes remains complex. The primary purpose of these programmes is patient access, not commercial gain or drug performance monitoring, and that excessive data collection can create administrative burden, delay patient access and raise ethical concerns.

PwC also highlights the need to understand what data can legally be collected in each market, particularly as real-world evidence becomes more relevant to payer value propositions and European HTA processes.

Pharmaphorum notes that the use of real-world data from Early Access Programmes may become increasingly important as regulators and authorities strengthen frameworks around real-world evidence, while the EU’s Health Technology Assessment Regulation and Joint Clinical Assessment process are expected to increase focus on harmonised evidence generation.

The same article reports that more than one in five NICE technology appraisals have used early access data to inform safety, efficacy or resource-use considerations, underscoring the growing relevance of access-generated evidence in reimbursement discussions.

For access leaders, the challenge is to strike the right balance: collecting data that is meaningful, ethical and operationally feasible without turning patient access programmes into burdensome pseudo-clinical trials.

This is one of the key issues that will be examined at the 5th Operationalise: Early Access Programmes Summit Europe, taking place in London in October 2026.

The summit will bring together 140+ Early Access, Medical Affairs, Access, Regulatory, Clinical Operations, Evidence Generation and Supply leaders to explore how companies are designing programmes that deliver earlier patient access while navigating data, regulatory, supply and operational complexity.

With agenda themes spanning real-world evidence, rare disease programme delivery, European access pathways, post-trial access, clinical supply and global programme models, the meeting will provide a dedicated forum for teams to benchmark how early access data can be collected and used responsibly.

As rare disease innovation accelerates and access pathways become more strategically important, real-world data from Early Access Programmes will continue to sit at the centre of regulatory, ethical and operational debate.

For teams responsible for delivering investigational medicines outside clinical trials, the future will depend on designing programmes that remain patient-first while generating the practical insight needed to improve access, support evidence generation and prepare for sustainable launch.

Download the full event guide to explore how we'll help teams navigate the future of real-world data, rare disease access and programme delivery.

 

Sources

1. pharmaphorum. Transformative Trends with Real-World Data from Early Access Programmes. pharmaphorum.
2. Sciensus. Early/Expanded Access Programmes: Making Rare Treatments Accessible Ethically and Efficiently in Europe. Sciensus, 18 November 2025.
3. PwC Belgium. Navigating the Complexities of Early Managed Access Programmes. PwC, 2025.
4. Tarantola, A., Otto, M.H., Armeni, P., Costa, F., Malandrini, F. & Jommi, C. Early Access Programs for Medicines: Comparative Analysis Among France, Italy, Spain, and UK and Focus on the Italian Case. Journal of Pharmaceutical Policy and Practice, 2023.