HTA Policy Update

Section Editors: Sandra Nestler-Parr, PhD, MPhil, MSc; Ramiro E. Gilardino, MD, MSc

Welcome to the HTA Policy Update, which provides a brief update on notable HTA policy developments from around the globe. We welcome suggestions and guest editorials for future issues. Please contact the Value & Outcomes Spotlight editorial office with your ideas.

From Implementation to Evolution: Early Lessons for the Future of HTA

Recent months have brought important developments in European health technology assessment (HTA). The publication of the first Joint Clinical Assessment (JCA) reports under the European Union HTA Regulation, alongside policy discussions in the United Kingdom on the future scope of HTA, offers an early window into how assessment frameworks are evolving.

Although emerging from different policy environments, these developments raise 2 fundamental questions facing HTA systems: How do we assess increasingly complex evidence? And how do we define the value that should ultimately inform decision making?

Early Lessons from 3 Joint Clinical Assessments

The publication of JCA reports on tovorafenib in June 2026 and lurbinectedin and tarlatamab the following month marked an early milestone in the implementation of EU Regulation 2021/2282, providing the first opportunity to observe the performance of the common European assessment methodology across different evidentiary contexts in oncology.

The 3 assessments illustrate distinct evidence profiles.

Tovorafenib presented the most challenging evidence base: Of the 8 predefined PICOs for pediatric low-grade glioma, only 1 produced comparative evidence, based on an unanchored indirect comparison. After weighting, the effective sample size fell below 7 patients, confidence intervals crossed the null (no clear evidence of a positive or negative effect), and response rate analysis was excluded due to different outcome definitions across studies. As a result, the assessment is transparent about what cannot be established; 7 of the 8 clinical questions remain unanswered, and the report focused less on what can be concluded than what remains unknown.

The lurbinectedin evidentiary profile was more straightforward, with a single population, 1 PICO, and 1 randomized trial providing direct comparative evidence. Although the JCA generated interpretable comparative results, several limitations remain. The overall survival advantage was smaller at a later data readout, the open-label design introduced potential bias across multiple endpoints, and subgroup data required correction during the factual accuracy review.

Tarlatamab occupied an intermediate position. The assessment included 4 populations and 7 PICOs, with direct evidence for 3 comparisons and indirect evidence for 4 others. The indirect analyses relied on methods such as unanchored, matching-adjusted indirect comparisons and Bayesian network meta-analyses. For the 4 indirect PICOs, assessors concluded that key similarity assumptions were substantially violated, limiting confidence in the resulting estimates.

Three observations emerge from these early assessments. First, the JCA framework generates the most robust outputs when clinical development programs align closely with the assessment scope defined by the Member States. Second, indirect comparison methods continue to face substantial challenges that the current guidance does not fully resolve. Third, while the reports provide detailed documentation of uncertainty, they stop short of offering an integrated judgment regarding overall certainty. The task of translating methodological limitation into a decision-relevant conclusion therefore remains largely with each individual Member State. Whether this reflects a deliberate design feature of JCA or an area for future methodological development remains an important question for the next phase of EU HTA implementation—in 2028, when orphan medicines will enter the JCA process.

Broader Value Enters the Policy Discussion

While the European Union experience highlights challenges associated with evaluating evidence, the United Kingdom has focused its attention on a different question: What dimension of value should HTA capture?

Evidence sessions before the House of Lords Science and Technology Committee in June and July discussed whether current assessment frameworks adequately capture the wider societal impacts of healthcare interventions. Testimony from NICE, the Department of Health and Social Care, and NHS England highlighted productivity and caregiver outcomes as potential areas for inclusion into NICE’s HTA methods, while also recognizing associated equity considerations.

These discussions reflect a broader international debate about whether HTA should move beyond direct health outcomes to more systematically capture societal value elements, such as productivity, caregiver burden, insurance value, and the value of hope.

Looking Ahead: Toward Better Alignment

Although these developments emerged from different policy contexts, they point toward a common observation. The first JCAs demonstrate that methodological consistency cannot fully compensate for limitations in the underlying evidence base, while the debate in the United Kingdom highlights that the very definition of value continues to evolve. Together, these experiences suggest that the next phase of HTA development may be less about refining individual assessment methods and more about better alignment of evidence generation, societal value, and healthcare system priorities.

As implementation of the EU HTA Regulation progresses and discussions about broader value continue across jurisdictions, these early experiences provide important signals for policy makers, HTA agencies, and health technology developers. Whether they lead to future methodological reforms remains to be seen, but they are likely to influence how HTA practice evolves over the coming decade.