Refining EU HTA Scoping for Effective Implementation

The European Union’s Health Technology Assessment (HTA) Regulation marks a major step towards unified clinical evaluations across member states. EU HTA scoping forms the foundation of Joint Clinical Assessments (JCAs), which began in January 2025 for oncology medicines and advanced therapy medicinal products (ATMPs). These assessments rely on the PICO framework to outline populations, interventions, comparators, and outcomes. Yet, as the European Federation of Pharmaceutical Industries and Associations (EFPIA) points out in its recent analysis, gaps in this process could hinder timely market access and strain healthcare systems. This article reviews EFPIA’s observations on scoping and PICO exercises, offering perspectives on health economics, policy, and system operations to guide leaders in pharmaceuticals, HTA agencies, and government.

Historical and Current Context

The EU HTA Regulation (EU) 2021/2282 addresses long-standing differences in national HTA methods, which previously slowed therapy approvals and raised costs for developers. Before 2025, efforts like EUnetHTA’s voluntary joint assessments provided partial alignment, but the new rules require JCAs for priority areas. Oncology products, for instance, made up 28% of new approvals in Europe in 2023, according to the European Medicines Agency.

Scoping starts with PICO to merge member state requirements into a single assessment outline. In November 2024, the HTA Coordination Group (HTACG) released guidance on this process. Early in 2025, six PICO simulations followed, testing cases such as durvalumab for hepatocellular carcinoma (PICO MP 01) and etranacogene dezaparvovec for haemophilia B (PICO MP 05). These trials aimed to limit PICOs to the minimum needed, drawing from EUnetHTA 21 pilots (2019–2022) that showed initial oncology proposals reaching 57 PICOs before merging.

Harmonisation offers clear benefits, including potential savings from reduced overlaps. However, it must account for variations in care standards, such as differing comparator choices between Eastern and Western Europe.

Challenges in Initial PICO Development

EFPIA praises the guidance for increasing openness but highlights shortcomings in forming initial PICO proposals. Assessors and co-assessors create these starting points, which then shape responses from member states. Although the process mentions inputs from clinical guidelines, patients, clinicians, and health technology developers (HTDs), details on proposal creation remain vague.

For example, in PICO MP 03 on adagrasib for non-small cell lung cancer, proposals differed sharply on subpopulations, resulting in disjointed scopes. Such ambiguities can add months to preparation times. In rare disease settings, like ATMPs in PICO MP 05, mismatched proposals often demand extra real-world evidence, complicating submissions.

Greater clarity here would help HTDs plan ahead. Without it, the scoping phase risks inefficiency from the start.

Issues in PICO Consolidation

Once proposals emerge, consolidation seeks to combine them efficiently, allowing for elements like multiple comparators or subgroups. EFPIA argues, however, that the approach grants too much leeway without standard procedures or open records of member state discussions.

The simulations demonstrated reductions from dozens to under 10 PICOs in certain instances, but choices frequently favoured larger markets, as seen in outcome adjustments where 40% of elements vanished without explanation. Unclear consolidation could raise dossier costs, particularly for smaller companies.

Moreover, the separate outcomes guidance emphasises ‘final’ measures, such as overall survival in oncology, with strict surrogacy rules. Simulations showed this approach marginalising intermediate endpoints, which feature in many trials. For regions with limited resources, like parts of Eastern Europe, these demands could widen access gaps—ATMP adoption there trails Western Europe.

Broader Impacts on Health Systems and Economics

Unresolved scoping problems affect more than procedures; they influence economic models and system performance. Delays from vague PICOs might push product launches back by 6–12 months, leading to forgone revenues per therapy. Overly broad scopes could also create unwieldy dossiers, reducing national relevance and complicating pricing or reimbursement decisions.

In policy terms, this setup challenges the balance between EU unity and local needs. Globally, it shapes strategies, as EU methods influence bodies like the UK’s NICE or Canada’s CDA, both adopting similar PICO structures. For Eastern European nations, rigid consolidation might overlook budget constraints, potentially slowing innovation uptake.

Practical Recommendations for Improvement

EFPIA proposes targeted changes to strengthen the framework:

• Require structured HTD consultations during PICO drafting, with full records of consolidation reasons—this could cut uncertainty.
• Introduce adaptable surrogacy criteria and routine inclusion of patient-reported outcomes or real-world data to match varied care practices.
• Expand HTACG support through FAQs, training sessions, and a specialised consolidation team. Early scientific advice could align evidence plans, trimming costs.

These measures promote a system that aids innovation without overwhelming stakeholders.

Final Thoughts

EFPIA’s critique reveals the EU HTA’s strengths in fostering transparency alongside risks from unclear scoping and PICO handling. Addressing these through structured input, adaptable rules, and ongoing reviews will help achieve faster patient access, lower costs, and stronger innovation in Europe.

As healthcare pressures mount, collaboration remains essential.

Source

https://www.efpia.eu/news-events/the-efpia-view/blog-articles/from-guidance-to-implementation-efpia-s-reflections-on-eu-hta-scoping-pico-exercises/