Written by Ben Clark, Paloma Charlesworth, and Kiera Lander
This quarterly update highlights recent and upcoming developments shaping the health technology assessment (HTA) environment across Europe, with a particular focus on rare diseases, EU‑level collaboration, and evolving evidence expectations.
New IPHA framework for rare medicines in Ireland
Ireland has agreed a new Irish Pharmaceutical Healthcare Association (IPHA)–Government framework targeting a 180‑day timeline for HTA and reimbursement decisions, with the stated aim of accelerating access to rare medicines.
Why it matters
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- Historically, the time from National Centre for Pharmacoeconomics (NCPE) submission to reimbursement in Ireland has exceeded 1,000 days, representing a significant access bottleneck for rare disease therapies.
What it means
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- While the framework signals clear intent to improve timelines, it remains uncertain where efficiencies will be realised in practice – whether during the NCPE HTA review itself or primarily in post‑NCPE negotiations between the Health Service Executive (HSE) and companies. Early indications suggest efforts to shorten NCPE review timelines, although capacity constraints may limit the extent of improvement.
- Companies developing rare disease therapies for Ireland may benefit from earlier NCPE engagement and proactive planning around evidence completeness and negotiation strategy to maximise the potential impact of shorter timelines.
2026 candidates for EU JCA
From 12 January 2025, one year into implementation, the EU HTA regulation (HTAR) Joint Clinical Assessment (JCA) programme began preparations to scale in 2026. The JCA Coordination Group has now published a list of emerging technologies expected to be eligible for assessment.
Why it matters
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- The published pipeline points to a substantial increase in both the volume and complexity of JCAs, including oncology products, advanced therapy medicinal products (ATMPs), orphan medicines, and potentially medical devices, as the programme moves beyond its slower‑than‑anticipated first year.
What it means
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- As many as 101 JCAs could initiate in 2026, offering early insight into how JCA requirements will be applied in practice, particularly for orphan ATMPs and, for the first time, medical devices, ahead of mandatory inclusion of all orphan products from 2028.
- The expanding scope reinforces the importance of early JCA readiness, including evidence strategy development, comparative effectiveness planning, and positioning for complex products such as oncology therapies and orphan ATMPs.
- With industry expected to monitor early submissions closely, the coming year is likely to clarify how JCA expectations translate into real‑world submission requirements.
- Manufacturers with products approaching EU submission (particularly in oncology, rare diseases, and advanced therapies) should assess JCA applicability early and consider aligning global evidence and value strategies to anticipated JCA requirements.
Surrogate endpoints in HTA: New ISPOR good practice guidance
An ISPOR Task Force has published a good‑practice framework on the evaluation and use of surrogate endpoints in HTA decision‑making. The framework integrates statistical validation approaches such as multivariate meta‑analysis and Bayesian methods, with guidance on incorporating surrogates into health economic models.
Why it matters
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- Surrogate endpoints are widely used in clinical trials, but validation is often limited, increasing uncertainty for HTA bodies and payers. Expectations for surrogate evidence can also vary substantially across jurisdictions.
What it means
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- The guidance provides a clearer framework for demonstrating surrogate validity and transparently linking surrogate outcomes to final health outcomes within cost‑effectiveness models. This highlights the growing importance of structured surrogacy validation strategies, including appropriate use of advanced statistical methods and real‑world data, alongside clear communication of surrogate‑to‑outcome assumptions.
- For assets relying on surrogate endpoints, early investment in robust validation plans and explicit translation of surrogates into economic models may help reduce HTA uncertainty and support more consistent decision‑making across markets.
‘What to watch’ next quarter
The announcement of the NICE threshold changes in December was also coupled with news on a new value set to be used alongside EQ-5D-5L, with guidance now released. It remains to be seen how this might impact NICE’s current approval of treatments.
If you would like to learn more about HTA submissions (including systematic reviews, health economic modelling, and medical writing), please contact us at Source Health Economics, a consultancy specialising in evidence generation, health economics, and communication.
