Anna Kapuśniak

The EU Health Technology Assessment Regulation makes the joint clinical assessment dossier the foundation of a single European appraisal of the relative effectiveness and safety of new medicines, and Article 9 conditions that foundation on the completeness of the evidence a developer submits. Drawing only on the first publicly observable outputs of the new system — two completeness reviews and one completed assessment — this article asks how the European Commission and the HTA Coordination Group are giving operational meaning to “completeness”. It argues that an exacting interpretation is emerging, closer to the logic of systematic review than of regulatory submission, but that the evidentiary base remains too thin to call it a settled doctrine. Two structural tensions follow: a certainty paradox, in which uncertainty is catalogued exhaustively yet never converted into a graded judgment of certainty; and an evidence-generation paradox, in which developers of orphan and highly innovative therapies may face analytical demands whose outcome is largely foreseeable. The article situates these tensions within the principle of proportionality, the duty to give reasons and the limits of judicial review, and sets out a research agenda for the Regulation’s scheduled 2028 evaluation.

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.

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.