Jaime Espin

Rationale: Health technology assessment (HTA) is the principal instrument wealthy health systems use to decide what to fund, and it measures the health a technology adds for treated patients rigorously. Yet population‐health considerations—disease burden, prevention, externalities such as herd protection, fairness between groups, and democratically chosen priorities—are weakly built into how HTA judges value. Aims and Objectives: To specify the nature of this gap and to offer a conceptual framework that clarifies it: to distinguish three paradigms of value assessment (clinical, cost‐effectiveness and public‐health HTA), and to separate two frequently conflated ideas—respecting public‐health priorities versus yielding to political pressure. Methods: A conceptual analysis informed by a narrative reading of HTA methodological guidance and the health‐economics, public‐health and priority‐setting literatures. Results: First, contemporary HTA clusters around two paradigms—clinical assessment and cost‐effectiveness assessment—while a population‐health paradigm is named but instantiated in no major agency's reference case. Second, public‐health objectives (burden, prevention, transmission externalities, distribution, priorities) are only partially operationalised—acknowledged but rarely weighed in the formal value calculation. Third, the three‐paradigm model shows the gap is systematic, not incidental: today's methods ask “how much does it help the patient?” and “how much health per euro?”, seldom “how do we improve population health, fairly?”. Fourth, the gap is tractable—the necessary instruments exist and are compatible with opportunity cost, and two common objections (that QALYs already capture population value, and that priorities equal interference) do not withstand scrutiny. Conclusion: A public‐health‐informed HTA—explicit disease‐burden assessment, transmission modelling, transparent treatment of priorities and formal equity analysis—could better align appraisal with population health, fairly distributed. We call on EUPHA and HTAi to convene a joint working group.

Cardiovascular, renal, and metabolic (CVRM) conditions—including hypertension (HTN), chronic kidney disease (CKD), type 2 diabetes (T2D), heart failure (HF), obesity (OBS), and dyslipidemia (DLD)—s...

Objectives. Regulation (EU) 2021/2282 introduces the Joint Clinical Assessment (JCA), a common descriptive clinical evaluation that Member States must take into account in national appraisal. Because national health technology assessment (HTA) systems have developed distinct methodologies, their alignment with the JCA is uncertain. We characterised it across six European jurisdictions and interpreted how the pattern arose. Methods. Qualitative comparative study combining structured expert-consensus rating with documentary process tracing. Two EU-HTA methodologists derived 35 methodological dimensions in five domains from the Regulation, its Implementing Regulation and HTA Coordination Group guidance, fixing and source-tracing the JCA reference position for each before any national rating. Expert pairs in six jurisdictions (France, Germany, Italy, Spain, the Netherlands and England) rated their system against every reference position by consensus, on an anchored three-point qualitative alignment scale. Reporting follows the SRQR. Results. Germany was aligned on 33 of 35 dimensions, with no substantial divergence. England was most distant (23 substantially divergent), followed by France (18); Italy and the Netherlands were intermediate (14 each). Spain recorded no aligned dimension but the fewest substantial divergences of any non-German system (7), diverging in degree rather than in kind. Divergence concentrated in the domains the JCA excludes and in appraisal posture, yet German proximity persisted once these were removed (27 of 29; 19 of 20). Documentary evidence on the legislative compromise, EUnetHTA21 methodological production and subgroup leadership suggests the JCA has emerged as a German-compatible default rather than a neutral synthesis of national traditions. Conclusions. National methodologies align with the JCA to markedly different degrees, the closest fit being the most codified national model. The alignment finding is empirical, the influence account interpretive; neither ranks any national model as superior. Because divergence concentrates where national appraisal remains sovereign, the framework may displace methodological heterogeneity downstream rather than resolve it.

Health technology assessment is a discipline that has developed, over five decades, an elaborate scientific and institutional identity centered on the evaluation of clinical evidence, comparative effectiveness, and economic value

This methodological commentary examines the Joint Clinical Assessment (JCA) report of lurbinectedin combined with atezolizumab as maintenance therapy for extensive-stage small-cell lung cancer, based on the phase 3 IMforte trial. The JCA's evaluation rests on two overall survival data cuts: a confirmatory, hypothesis testing, statistically significant interim cut (hazard ratio 0.73, p=0.0174) and a mature, hypothesis generation, non-significant cut (hazard ratio 0.81). We identify two main critical methodological issues in the JCA's handling of these results. First, the JCA prefers the mature cut under a treatment-policy strategy that embeds subsequent crossover to lurbinectedin in the control arm, which is unavailable in routine European practice and dilutes the estimated treatment effect. The report lacks a decision-relevant, hypothetical-strategy analysis adjusting for this treatment switching. Second, the JCA discounts the positive interim result by assigning an "Unclear" risk-of-bias rating based on a late alpha-spending protocol amendment. This suspicion was left unresolved despite available data, the power to seek developer clarification, and regulatory findings from the European Medicines Agency (EMA) clearing the amendment as operational. Instead of a reasoned synthesis, the JCA performs a silent substitution of the mature cut for the interim. The report ignores the information both cut bring and questionably assesses the second cut as low risk of bias. To address these concerns, we recommend: complementing treatment-policy estimates with hypothetical-strategy analyses when trial crossover differs from European practice; resolving risk-of-bias concerns rather than leaving them "unclear"; engaging with prior regulatory evaluations; and providing graded certainty statements to reconcile divergent clinical results.

Rationale. Health technology assessment (HTA) is the principal instrument wealthy health systems use to decide what to fund, and it measures the health a technology adds for treated patients rigorously. Yet population-health considerations—disease burden, prevention, externalities such as herd protection, fairness between groups, and democratically chosen priorities—are weakly built into how HTA judges value. Aims and objectives. To specify the nature of this gap and to offer a conceptual framework that clarifies it: to distinguish three paradigms of value assessment (clinical, cost-effectiveness and public-health HTA), and to separate two frequently conflated ideas—respecting public-health priorities versus yielding to political pressure. Method. A conceptual analysis informed by a narrative reading of HTA methodological guidance and the health-economics, public-health and priority-setting literatures. Results. First, contemporary HTA clusters around two paradigms—clinical assessment and cost-effectiveness assessment—while a population-health paradigm is named but instantiated in no major agency’s reference case. Second, public-health objectives (burden, prevention, transmission externalities, distribution, priorities) are only partially operationalised—acknowledged but rarely weighed in the formal value calculation. Third, the three-paradigm model shows the gap is systematic, not incidental: today’s methods ask “does it help the patient?” and “how much health per euro?”, seldom “how do we improve population health, fairly?”. Fourth, the gap is tractable—the necessary instruments exist and are compatible with opportunity cost, and two common objections (that QALYs already capture population value, and that priorities equal interference) do not withstand scrutiny. Conclusion. A public-health-informed HTA—explicit disease-burden assessment, transmission modelling, transparent treatment of priorities and formal equity analysis—could better align appraisal with population health, fairly distributed. We call on EUPHA and HTAi to convene a joint working group.

Background: The EU Health Technology Assessment Regulation (EU-HTA R), effective January 2025, mandates Joint Clinical Assessments (JCAs) to harmonize HTA across Member States. However, its implementation raises fundamental questions about methodological coherence, institutional capacity, and epistemological alignment. Objectives: This manuscript (1) systematically assesses whether the stated strategic and operational objectives of the EU-HTA R are achievable under current implementation conditions; (2) examines the implications for EU institutional legitimacy if these objectives are not met; and (3) proposes an epistemological framework as a prerequisite for developing a coherent joint HTA methodology. Methods: We conducted a critical policy analysis of the EU-HTA R, its implementing guidance documents, and published templates, supplemented by a comparative review of Member State HTA methodologies and their underlying philosophical foundations. Results: The analysis reveals that the EU-HTA R is unlikely to achieve its strategic goals under current conditions. Key findings include: guidance documents of substandard methodological quality; a restricted assessment scope that excludes scientific judgement and contextualization; insufficient resources and additional workload for national HTA bodies without reducing existing obligations; unresolved epistemological divergences among Member States spanning Bayesian vs. frequentist approaches, Fisher vs. Neyman–Pearson frameworks, and utilitarian vs. deontological ethical foundations; and procedural shortcomings in stakeholder consultation and expert involvement. These shortcomings risk undermining the epistemic authority and legitimacy of EU institutions. Conclusions: Prior epistemological and normative alignment across Member States is a prerequisite for any robust shared HTA methodology. Revisions to the EU-HTA R and comprehensive updates of guidance documents are necessary, with concrete safeguards—including independent peer review, identified authorship, and adequate resourcing—to ensure substantive rather than merely nominal implementation. A phased roadmap is proposed: establishing clear objectives, aligning epistemological foundations, developing institutional structures, and creating operationally consistent guidance.

Background: This study aimed to estimate the prevalence of coded diagnoses of COPD and to describe the real-world demographics, clinical profile, pharmacological management, and disease severity, focusing on dual (DT: LABA+LAMA) and triple (TT: LAMA+LABA+ICS) therapy. Methods: This retrospective, descriptive, cross-sectional study analysed anonymised electronic health records from the BIG-PAC® database, covering seven Spanish healthcare regions. For prevalence estimation, adults ≥40 years on 01/01/2023 (index date) were considered, and the presence of a coded COPD diagnosis was assessed. Demographic and clinical variables, including comorbidities, exacerbation history, and disease severity according to GOLD and GesEPOC classifications, were evaluated during the one-year period prior to the index date. Results: Among 987 453 active adults ≥40 years, 48 451 had a COPD code, corresponding to a prevalence of 4.9%. Treatment patterns were as follows: 28.8% DT, 25.6% TT, 18.7% LABA+ICS, 14.8% LAMA, 1.7% LABA, and 7.3% untreated. A total of 23 953 patients receiving DT or TT were analysed (53.5% DT; 46.5% TT). Mean age was 73 years ( sd : 10); 70.1% were males. Most patients had at least one comorbidity (96.5%). Exacerbations occurred in 44.1% (TT: 53.4%, DT: 36.1%, p versus 4.6%, p p <0.001). Conclusion: This study offers real-world evidence on coded COPD prevalence, treatment patterns, and patient characteristics in a large Spanish healthcare database. Individuals on TT were older, had more comorbidities, and experienced more exacerbations than those on DT.

On 14 May 2026, the Member State Coordination Group on Health Technology Assessment (HTA Coordination Group) adopted Guiding Principles on Data Transparency under Article 3(7), points (c) and (h), of Regulation (EU) 2021/2282. The document opens with a disclaimer of legal force; its operational architecture, a self-authored definition of ‘commercially confidential information’, a categorical declaration that broad classes of clinical data are in general non-confidential, and a recourse procedure run by the same family of actors that produced the rule, has consequences that the disclaimer cannot dissolve. We argue that the Guiding Principles sit uneasily with EU lex specialis on commercial confidentiality and with case-by-case jurisprudence on disclosure; that, in producing them, the HTA Coordination Group has concentrated rulemaking, executive and quasi-judicial functions in a manner that engages the principle of conferred powers, the Meroni doctrine, and Articles 41 and 47 of the Charter; and that the corrective is institutional rather than substantive: the problem lies in how the rules were made and by whom, not only in their content.

The Joint Clinical Assessment (JCA) report on tovorafenib (Ojemda), the first assessment of an orphan oncology medicinal product completed under Regulation (EU) 2021/2282 [1], conformed closely to predictions made across our earlier work. This Perspective argues that the outcome was foreseeable from the framework’s design rather than from any feature of the product. We characterise the JCA as an audit that confronts developer-submitted evidence against a corpus of EUnetHTA methodological guidance whose scientific validity and development process have been widely contested. Four structural features explain the result: the referential cannot function as a settled gold standard; the assessment omits the interpretive, context-dependent judgment that gives evidence meaning, on the assumption — specific to one national tradition — that assessment can be separated from appraisal and conducted free of judgment; products supported only by single-arm trials are predictably assessed negatively, as Ojemda illustrates; and the framework departs from the tripod of evidence-based medicine by marginalising clinicians and patients. By multiplying PICOs, subgroups and outcomes and then criticising the resulting analyses as post hoc, the JCA generates its own limitations and ultimately cannot state a degree of certainty of relative effectiveness. We conclude that single-arm-supported products should fall outside the JCA’s remit and that the framework requires a transparent route from documented uncertainty to a stated certainty.