The value of Real-world data from Early Access Programs

Reflections from ISPOR Europe 2025 

The ISPOR Europe 2025 conference, held in Glasgow, Scotland, this year, brought together global leaders in health economics and outcomes research (HEOR) to discuss how evolving evidence sources are shaping healthcare decision-making.

Clinigen’s panel session posed the question:
'Data from Early Access Programs: Essential HTA Evidence or Misaligned Value Tool?’

On our panel, representing the views of Clinigen, industry and regulators, we had
- Kieron Lewis, Director of Strategic Consulting at Clinigen
- Stephen Maddocks, Medicines and Healthcare products Regulatory Agency (MHRA)
- Em Jennings, Global Executive Director of Operational & Strategic Pricing at Novartis
- Susanne Michel, Head of HTA & Market Access at Clinigen

The panel discussion was extremely well attended, with participants from industry, patient advocacy groups, HTA bodies, regulatory agencies, and other HTA consultancies.

Together, we explored the rapidly evolving use of Real-world data (RWD) and Real-world evidence (RWE) in health research, which has been accelerating over the last decade, highlighted by key milestones in both the US and EU, such as:

21st Century Cures Act
https://www.mintz.com/insights-center/viewpoints/2146/2016-12-19-_1st-century-cures-act-requires-fda-expand-role-real

Framework Program for FDA RWD
https://www.federalregister.gov/documents/2018/12/07/2018-26546/framework-for-a-real-world-evidence-program-availability

FDA Guidance for Industry on RWE and RWD
https://www.federalregister.gov/documents/2023/08/31/2023-18841/considerations-for-the-use-of-real-world-data-and-real-world-evidence-to-support-regulatory

CIOMS Report on RWD and RWE in decision-making
https://cioms.ch/working-groups/real-world-data-and-real-world-evidence-in-regulatory-decision-making

EMA roadmap for regulatory guidance on RWE
https://www.ema.europa.eu/en/about-us/how-we-work/data-regulation-big-data-other-sources/real-world-evidence

EMA reflection paper on the use of RWD in non-interventional studies (NIS) https://www.ema.europa.eu/en/reflection-paper-use-real-world-data-non-interventional-studies-generate-real-world-evidence-scientific-guideline

We also explored how Early Access Programs (EAPs), which provide patients with life-threatening or seriously debilitating conditions access to medicines prior to formal authorisation, can generate high-value RWD. When designed thoughtfully, these programs can deliver meaningful insights into how treatments perform in clinical practice, patient characteristics at the point of early access, and real-world patterns of use that may not be fully captured in pre-approval trials.

Supporting HTA through RWD from EAPs

For manufacturers, one of the most compelling opportunities lies in the potential to combine EAP-derived RWD with clinical trial data to support HTA submissions. Such data can:

• Provide early insights into effectiveness and safety in real-world populations, beyond the controlled environment of pivotal trials.
• Inform cost-effectiveness models with real-world resource utilisation, adherence, and discontinuation data.
• Support unmet need and disease burden assessments, particularly when registry or claims data are limited.
• Help contextualise uncertainty identified by HTA bodies, for example, by demonstrating consistency (or divergence) between clinical trial and early access outcomes.

Key considerations for integration of RWD from EAPs

The panel emphasised that incorporating EAP data into HTA and broader evidence packages requires careful planning. Several important considerations emerged:

• Data quality and governance: Real-world data from EAPs must meet acceptable standards of completeness, accuracy, and traceability to be credible for regulatory or HTA use.
• Alignment with evidence strategy: EAPs should be designed in coordination with the overall evidence generation plan, ensuring relevant endpoints and data structures are defined from the outset.
• Ethical and operational oversight: Patient consent, data privacy, and compliance with local regulations are essential when collecting and linking patient-level data.
• Stakeholder engagement: Early dialogue with regulators, HTA agencies, and data partners helps ensure expectations are clear and methods are transparent.

A growing opportunity

As health systems increasingly value real-world evidence to complement clinical trials, the ability to generate and utilise RWD from EAPs represents a meaningful opportunity to enhance the evidence base for new treatments. Below are links to several literature reviews and peer-reviewed publications that use RWD from EAPs to inform decision-making.

Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals
https://bmjopen.bmj.com/content/12/1/e052186

Long-term effects of volanesorsen on triglycerides and pancreatitis in patients with familial chylomicronaemia syndrome (FCS) in the UK Early Access to Medicines Scheme (EAMS)
https://pure.qub.ac.uk/en/publications/long-term-effects-of-volanesorsen-on-triglycerides-and-pancreatit/fingerprints/?sortBy=alphabetically

Real-world treatment duration in patients with non-small cell lung cancer (NSCLC) with EGFRExon20 Insertion (EGFRex20ins) mutations receiving mobocertinib through the globalEAP
Victor Lee1, Huamao M. Lin2, Eileen Curran2, Yu Yin2, Eric N. Churchill3, Susan Allen2, Jennifer Abovich2, Natasha B. Leighl4 1

Patients with EGFR mutant (m) MET-altered NSCLC receiving tepotinib with an EGFR tyrosine kinase inhibitor (TKI): A case series
https://ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.9070

The discussion at ISPOR highlighted that, despite ongoing methodological and governance challenges, Real-world data from EAPs can be instrumental in informing access, reimbursement, and ongoing evidence generation.

ISPOR Europe proved to be an invaluable platform for advancing these conversations. The strong engagement during the session reflected both the complexity of the topic and the collective recognition that early, collaborative approaches to RWD are critical to enabling faster, evidence-based access to innovative medicines.

At Clinigen, we work with clients to design and optimise evidence strategies that integrate real-world data generation across the product lifecycle, from early access through to post-launch. Our experience in HEOR, market access, and data strategy enables us to help clients translate complex real-world insights into credible, decision-ready evidence for regulators and HTA bodies. As the role of RWD continues to expand, we see growing opportunities to harness EAPs not only as mechanisms of access, but as valuable sources of early, high-impact evidence to support patients, payers, and healthcare systems alike.

Contact us to learn more or to discuss how Clinigen can support your evidence and access strategy.

Following up on these insights, we are also hosting a related webinar in January, ‘Navigating the path from clinical trials, orphan drug designation to patient access’, where Kieron Lewis, Susanne Michel and Delphine Wagner will explore how to defeat uncertainty across orphan drug development journey. Join us on 27 January 2026, 14:00–15:00 GMT, to gain practical, real-world insights you can apply to your own programmes – register HERE to secure your place.