Beyond participation: How EU HTA is turning patient and clinician engagement into evidence architecture

Europe’s new Health Technology Assessment (EU HTA) framework is often discussed in terms of joint assessments, procedural timelines, and methodological convergence. But one of its most transformative shifts is less visible: the way patient and clinician expertise is now woven directly into the architecture of how clinical evidence is framed, interpreted, and ultimately judged at a pan EU level. 

The structural shift in EU HTA: From consultation to methodological input 

Historically, patient and clinician engagement in HTA across EU has been inconsistent and insufficiently integrated across agencies - particularly in early phases such as scoping - and variable in its influence on decision-making. As a result, patient-relevant outcomes and perspectives have not been systematically embedded to reflect a meaningful patient impact, though this is evolving under the evolving HTA and Regulatory frameworks (ISPOR EU, 2025)¹.  

The EU HTA Regulation (Regulation (EU) 2021/2282) (EUHTAR)² is a major structural shift that aims to strengthen and formalise experts’ engagement, particularly by embedding earlier involvement in scoping and evidence generations. Under the EUHTAR, patients and clinicians are now engaged at defined checkpoints across the assessment lifecycle, contributing not just to validation but to shaping scoping, comparators, and outcomes that matter. While this moves engagement upstream into HTA methodology, its impact on final reimbursement decisions remains variable across Member States. 

Where experts shape the assessment: Joint Clinical Assessments (JCA) 

The shift is most visible within JCA, where expert input is integrated at key stages. The first occurs during scoping when the assessment framework is established. Within short consultation windows (typically 7–14 days), patients and clinicians review the draft scope, informing treatment pathways, comparators, and outcome relevance. 

This stage is critical: the Population, Intervention, Comparator, Outcomes (PICO) framework set here underpins the entire assessment. Omissions - whether subpopulations, meaningful endpoints, or realistic comparators - are difficult to correct later and may impact timely access. Early expert input helps ensure alignment with clinical practice and patient-relevant outcomes. 

A second checkpoint occurs during draft JCA review, typically within a 7‑day window, where patients, carers, and clinicians assess alignment with real world care and key patient concerns. 

Together, these structured checkpoints embed expert engagement within the methodology of HTA, shifting it from consultation to a core component of assessment design and validation. 

Shaping evidence generation: Joint Scientific Consultations (JSC) 

Expert input under EUHTAR does not begin with evidence evaluation. In some cases, it starts earlier - during JSC. Patient and clinician experts review the development strategies presented in the company’s briefing package and provide targeted feedback through questionnaires, interviews, or consultation meetings with HTA assessors. Their input focuses on aspects that strongly influence future evidence and will be considered relevant at the EU level, including the suitability of proposed comparators and subpopulations across different Member States, treatment pathways realism, and whether selected endpoints reflect meaningful patient outcomes. As these consultations occur while development plans are still adaptable, experts’ insight can directly influence comparator selection, endpoint strategy, and evidence generation ahead of pivotal studies completion. 

Together, JSC and JCA create a continuum of expert input - from shaping evidence generation to informing how evidence is assessed with HTA. 

What early experience is revealing 

EU HTAR is beginning to show how this new engagement model works in practice. Clinicians and patients provided some first operational views on experts’ involvement during the European Commission’s (EC) webinar in February 2026.³ 

Expert identification draws from multiple EU networks - including the HTA Stakeholder Network, European Medical Agency (EMA) expert pools, EU Reference Networks, Orphanet, and national authorities - ensuring broad representation. From the several hundred contacted through the HTA Stakeholder Network alone, 200 were nominated, with fewer than <40 contributing to JCAs, and about 10 to JSCs (2025 HTACG Annual report).⁴ 

Selection reflects expertise, practice setting, and geographic diversity, with an emphasis on place of practice or residence rather than nationality, to capture real-world EU clinical practice.  

All experts must submit Declarations of Interest covering prior three-year period, listing any financial relationships, investigator roles, intellectual property rights, and other potential conflicts. Early implementation experience suggests that while conflicts are actively screened and managed, exclusion rates remain relatively low - highlighting a pragmatic balance between maintaining transparency and ensuring access to relevant expertise. 

Overall, early signals suggest EU HTA is building a more structured and representative expert ecosystem. 

Scaling the expert ecosystem 

To deliver at scale, EU HTA must expand and strengthen its pool of patient and clinician experts. Identifying suitable patient representatives remains challenging given variability in expertise, HTA familiarity, and confidence in engaging in technical discussions, particularly in some therapeutic areas. 

New infrastructure is supporting this effort. A dedicated EC website centralizes ‘Opportunities for patients, carers and clinicians’ as a central access point, while the EU HTA IT Platform enables secure document exchange and structured input across JCA and JSCs processes.  

Operational coordination is also evolving. The Brussels Centre for Collaboration in Health (BCCH) supports experts’ onboarding, contracting, and logistics. These mechanisms aim to make participation more systematic, scalable and accessible.  

Participation is governed by strict confidentiality, with patients typically anonymized and clinicians named with affiliations. Experts are compensated in line with public-sector standards, reinforcing their role as formal contributors. 

A structural shift in evidence generation 

The EU HTA framework is not simply expanding stakeholder engagement - it is redefining how expertise shapes evidence. By embedding experts input upstream - across scoping, evidence generation, and methodology - engagement is shifting from consultation to structural influence. 

For pharmaceutical developers, this has several clear implications: 

• Integrate expert insight early, ideally before protocol finalization 
• Design evidence for EU-level assessment, not a single market, accounting for cross-country variation 
• Embed patient-relevant outcomes from the outset, strengthening both clinical relevance and value narratives 
• Prepare for ongoing expert interaction, that may shape evidence before dossier submission 

For patients, carers, and clinicians, the opportunity is equally significant: to directly influence how treatments are assessed and how access decisions are informed across the EU. As EU HTA evolves, success will increasingly depend on simple principle: robust evidence must be both scientifically rigorous and shaped by those it is intended to serve. At Parexel, this aligns closely with our long‑standing commitment to patient‑centered development. With Chief Patient Officer role, we have continued to strengthen our frameworks for embedding patient perspectives across the full product lifecycle, from early evidence generation to regulatory strategy and market access.  

As EU HTA moves from concept to implementation, we believe organizations that successfully integrate patient and clinician insights into the foundations of evidence generation will be best positioned to deliver meaningful outcomes for patients and enable sustainable access across EU.  

References:  

1. ISPOR - Enhancing Patient Involvement in European Health Technology Assessment (HTA): From Inconsistent National Practices to Systematic Engagement in Joint Clinical Assessments 
2. The EU HTA Regulation: Implementation of the Regulation on Health Technology Assessment - Public Health 
3. Health technology assessment: Webinar for patients and clinical experts - Public Health 
4. 2025 HTACG Annual Report 

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