NCPE Annual Symposium 2025

The 2025 Annual Symposium of Ireland’s National Centre for Pharmacoeconomics (NCPE) in Dublin considered various challenges in health technology assessment (HTA), medicines access, and evidence generation. Our key takeaways from this recent conference are summarized below.

1. Medicines Spend & Value Imperatives

Ireland’s medicines bill is now a major share of total healthcare expenditure – a reminder that every reimbursement decision must balance innovation with demonstrable patient benefit.

In oncology, the majority of recent HTA submissions received positive recommendations, yet only a fraction showed statistically significant survival or quality‑of‑life gains – highlighting the need for stronger clinical evidence before funding.

Delays in decision timelines remain a systemic challenge, impacting patient access.

2. Early Access Programmes – Promise & Practicality

There is strong interest in the discussions currently underway for an Irish early access programme (EAP) for cancer drugs and potentially also for orphan/rare disease medicines. However, key design questions persist in terms of eligibility criteria, governance responsibility, evidence requirements, and funding models.

A consensus is emerging that real-world data capture – both clinical and financial – must be embedded from the start.

Clear principle: access should be temporary until cost-effectiveness is proven, with funding withdrawn if the value case fails.

3. European Union HTA Regulation – Preparing for Impact

Joint Clinical Assessments (JCAs) will reshape evidence expectations from 2026, having been enacted in European legislation in 2025, the real world impact is starting to manifest starting with oncology, advanced therapy medicinal products (ATMPs), and orphan drugs.

The JCA impact on NCPE processes and dossiers remains uncertain. The NCPE has confirmed there are no changes to its rapid reviews. These do not readily accept indirect treatment comparisons as part of the evidence package. Irish companies must still navigate national reimbursement processes post‑JCA – timely submissions will be vital to support efficiencies, avoid delays and outdated data.

The NCPE expects to publish its first JCA report in Quarter 1, 2026. It is leading two JCAs. 1). Tovorafenib in paediatric low-grade glioma with Germany’s Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG, the Institute for Quality and Efficiency in Health Care). 2). Onasemnogene abeparvovec for spinal muscular atrophy 5q with Haute Autorité de Santé (HAS, the French National Authority for Health). A review of the current legislation is expected in 2028; HTA bodies will feed into this review in 2027.

4. Obesity – A Public Health and Economic Priority

In Ireland, separate care models for adults and children are being implemented under the National Clinical Programme for Obesity. Indeed, recurring government funding marks a significant policy shift towards long‑term investment in prevention and pharmacotherapy.

Treating obesity early is not just a clinical imperative – it can avoid downstream costs across more than 200 related diseases.

5. Pragmatic Trials – Aligning Research with Real‑World Needs

There are calls from leading oncology researchers to design trials that mirror clinical practice, not just controlled environments. Tools such as PRECIS‑2 help assess how ‘real-world’ a trial truly is – ensuring endpoints are relevant to both clinicians and payers. More pragmatic trial design can also help to support faster, more meaningful access decisions.

6. Industry & Policy – The Efficiency Challenge

Strong HTA processes underpin Ireland’s outcomes, but gaps remain in capturing all aspects of value. Real‑world delays in reimbursement stem from policy complexity, process bottlenecks, and misaligned expectations.

Predictability, partnership, and adherence to agreed timelines are seen as essential for delivering faster patient access.

7. HTA–Regulatory Collaboration on Evidence Generation & Uncertainty Management Under Health Technology Assessment Bodies (HTAbs)

A progress update on the joint initiative between the European Medicines Agency and heads of HTA agencies group focused on developing practical solutions to strengthen evidence generation for both HTA and regulatory decision‑making.

The working group’s objectives are to:

• Improve mutual understanding of challenges and uncertainties at the time of assessment, and how they are managed
• Identify, explore and discuss feasible solutions to generate better evidence for HTA and regulatory decision making
• Establish groundwork for future collaboration on methodology

8. Enhancing the Evidence Ecosystem

New approaches to enhance the evidence ecosystem are essential to increase the impact on health policy and practice. Living guidelines can enhance the evidence ecosystem by providing updated comparative effectiveness. But living evidence synthesis is critical for living guidelines.

Examples of living guidelines include the WHO Therapeutics and COVID-19: Living Guideline and the BMJ Rapid Recommendations series, for e.g. on risk-stratified cardiorenal outcomes.

Living guidelines rely on global collaboration. There are examples of successful initiatives, but they still need to be further improved. To produce relevant high-quality evidence, the enhanced evidence ecosystem needs to synthesize evidence, produce and disseminate guidance and implement and evaluate its impact on practice.

Conclusion

The NCPE Annual Symposium 2025 called for a more agile, evidence‑driven and collaborative health technology assessment system in Ireland — one that balances rising costs with proven patient benefit, prepares for new EU regulations, and accelerates access through real‑world data, pragmatic trials, and strong partnerships across policy, industry, and clinical care.