CREATIC ATMP newsletter

Who Gets to Decide? Normative Judgements in Health Technology Assessment

By Zainab Afshan Sheikh, Assistant Professor, CeBIL and Jakob Wested, Associate professor of Pharmaceutical law, CeBIL

There are growing tensions and debates around evidence, uncertainty and the authority in healthcare prioritization relating to ATMPs and other innovative therapies. While discussions initially focused on whether such therapies should be accepted on the basis of more uncertain evidence, the debate has increasingly expanded into broader questions about the mandate, transparency and democratic basis of health technology assessment (HTA) systems.

The Danish HTA system and its basis
A dive into the Danish HTA system and recent discussions in Denmark illustrates the tensions. Denmark's national HTA body for hospital medicines, the Danish Medicines Council, is a collaboration between the five Danish Regions responsible for operating Danish public hospitals. The collaboration operates within a politically agreed framework of seven prioritization principles for hospital medicines. These principles include considerations concerning clinical benefit, severity of disease, evidence quality and uncertainty, proportionality between treatment value and costs, equal access, transparency, and responsible use of healthcare resources.

The content of these principles is broadly similar to those guiding HTA boards in, for example, Sweden and the Netherlands, encompassing both health-economic concepts — such as cost-benefit analysis — and references to more politically charged values, such as equality and responsible use of resources. This contrasts with, for example, the German HTA system, where the principles governing HTA stick to health-economic criteria, while omitting references to equality and responsibility. However, the Danish model diverges from all of these countries in its legal basis. Contrary to its European brothers and sisters, the Danish HTA body and the principles under which it operates are established by a political agreement and not by law.

Normative judgements in HTA decisions
While discussions initially focused on whether therapies, such as ATMPs, should be accepted on the basis of more uncertain evidence, the debate has increasingly evolved into broader questions on legitimacy in HTA decision-making. This debate has emerged in the context of discussions about access to innovative and high-cost therapies, including treatments where long-term evidence remains limited and traditional evidentiary standards are difficult to satisfy. In public discussions, commentators and members of the Danish Medicines Council acknowledge that many decisions involve ethical and normative judgements that cannot be reduced to purely technical evidence and financial assessment. Recently, the president of a Danish patient association stated that the Danish Medicines Council's assessments involve ethical and normative judgements that extend beyond purely technical evaluation. He further argued that, given the Council's limited representativeness and vaguely defined mandate, this raises questions about the democratic basis of such decisions.

Ethics and Uncertainty
What makes the Danish debate particularly interesting in a broader European context is that it illustrates how evidentiary uncertainty does not simply create a need for new forms of evidence, including surrogate endpoints, real-world evidence, adaptive evidence generation and patient-reported outcomes. Uncertainty may also expand the space for normative judgements — judgements that are always embedded in decisions — about acceptable risk, unmet need, fairness and access. This is especially visible in discussions concerning therapies where overall survival data may take years to establish. In such situations, HTA bodies must decide how much weight to give surrogate endpoints, how much uncertainty is acceptable, and whether the potential benefits of early access justify the risks of acting on incomplete evidence. These are not merely methodological questions. They are also political and ethical questions concerning how societies prioritize scarce healthcare resources and who has the mandate to make such decisions. When evidence is uncertain and decisions involve balancing competing values — such as severity, rarity, cost, quality of life and unmet need — expert assessment inevitably becomes intertwined with normative prioritization.

A starting point for understanding HTA assessments for ATMPs
Across Europe, regulators have increasingly embraced adaptive approaches to evidence generation, uncertainty management and risk-sharing. However, HTA systems often remain anchored in more traditional hierarchies of evidence and more rigid expectations concerning certainty and demonstrated value. This raises important empirical and conceptual questions that deserve closer attention. How are judgements about uncertainty, severity and unmet need articulated and justified? What forms of expertise are considered legitimate in these deliberations? And how do patients, clinicians, economists and regulators differently understand the role of evidence in patient access decisions?

HTA is, fundamentally, a process of prioritization. Its recommendations or decisions will therefore never satisfy everyone: patients, clinicians, industry and decision-makers all have something significant at stake, and every decision will leave someone disappointed. If such agreement were anywhere within reach, a prioritization board would not be needed in the first place. This makes it important to examine how prioritization is structured and justified in practice. The legal basis, the content of prioritization principles, and the composition of the competent body are key parameters for analyzing how HTA systems manage uncertainty, value judgements and accountability in decisions on recommending use of ATMPs.

Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them.