A More Structured and Predictable Framework: Aligning Regulatory and Health Technology Assessment Processes in Europe

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Niklas Hedberg, MSc, Co-Chair, European Union Member State Coordination Group on Health Technology Assessment (HTACG)

Europe’s long-anticipated shift to a unified health technology assessment (HTA) framework is no longer theoretical; it is now being tested in practice. As the results from the first Joint Clinical Assessments begin to emerge, Niklas Hedberg describes a system balancing ambition with reality, aligning 27 Member States while managing expectations from industry, patients, and payers. At the same time, deeper questions around evidence, speed, and global competitiveness continue to shape its trajectory, positioning the framework as both a technical reform and a structural shift in how Europe evaluates and adopts innovation.

PharmaBoardroom: What are your key reflections on the first phase of Joint Clinical Assessments under the European Union (EU) Health Technology Assessment Regulation?

Niklas Hedberg: The first point is to manage expectations, as we are still at an early stage, and the experience base remains limited, particularly in terms of what can be shared publicly. We have completed a number of Joint Scientific Consultations, but these are confidential given the commercially sensitive nature of early stage data. On the Joint Clinical Assessment side, we are now close to endorsing the first reports [Editor’s note: The first Joint Clinical Assessment report was published June 9].

At this point, the system is still taking shape, and it would be premature to draw firm conclusions. Much of the work so far has focused on establishing procedures and processes, rather than on outputs, which is a natural phase when building a new framework of this scale. The more meaningful insights will come with time and accumulated experience.

The most important development over these first 16 months has been the transition from a largely voluntary system, built over almost 2 decades under EUnetHTA [the European Network for Health Technology Assessment], to a mandatory framework under the EU Health Technology Assessment Regulation (HTAR). This shift has required a sustained effort to build trust across stakeholders, and we are now moving toward building confidence, demonstrating that we can deliver something of value for patients, healthcare systems, and society. That confidence must extend beyond HTA bodies to include companies, clinical experts, patient representatives, and payers, particularly as these assessments begin to inform national and regional decisions.

“As high-quality assessment reports are produced, published in English, and made openly available, they may increasingly serve as a reference point beyond Europe, shaping how other systems approach health technology assessment.” — Niklas Hedberg

Looking ahead, the system will expand gradually, starting with oncology and ATMPs [advanced therapy medicinal products] followed by orphan medicines, and then a broader scope by 2030. Over time, I would expect a more integrated approach, where horizon scanning feeds into Joint Scientific Consultations, and those consultations help guide evidence generation for both regulatory and HTA purposes. If that alignment is achieved early, the evidence base underpinning Joint Clinical Assessments should improve, making it easier for payers to use these outputs in practice.

PB: To what extent are stakeholders aligned today around the direction of the EU HTA framework, and where do differences in expectations still remain?

NH: When speaking with stakeholders, particularly those working at a European level, there is a clear sense that there is only 1 direction of travel. Without a functioning HTA system, we would be left operating in a fragmented and uncertain environment, so there is broad agreement that this is the path to follow. That said, there have always been different views on how best to move forward, and those views have evolved over time.

If we look back 15 years or more, much of the momentum for a more converged European system came from industry itself, with companies often asking why there was no equivalent of the European Medicines Agency on the HTA side. What became clear over time was that this would require a far more substantial legislative effort than many initially expected.

We have seen the more experienced health technology assessment bodies take a leading role in methodological discussions, bringing their experience to the table and, in effect, creating a platform for others to learn from.

Today, we hear a more critical tone from some parts of the industry, although it may still be too early to draw firm conclusions. Some of that criticism relates to the perceived strength of the link to decision makers and whether the framework is sufficiently binding in practice. Ultimately, however, this will be tested through implementation, and it is at that stage that we will need to demonstrate that the system is delivering and build the confidence that stakeholders are looking for.

PB: How has the new framework approached the challenge of bringing together different national HTA systems across Europe?

NH: From our perspective, this has been a valuable and, in many ways, constructive exercise.  We have seen the more experienced HTA bodies take a leading role in methodological discussions, bringing their experience to the table and, in effect, creating a platform for others to learn from. That dynamic has helped to bridge differences in maturity across systems. At the same time, it is fair to acknowledge that, for companies, particularly smaller ones, the process can appear demanding, and in some cases more complex than anticipated.

However, most of these requirements are not new. They already existed at a national level, often in isolation and with limited coordination. What we have done is bring them together within a single European framework. The work on Population, Intervention, Comparator, and Outcomes, the so-called PICO framework, is a good example of this. Where previously there may have been 20 or more national PICOs, we are now working toward consolidating them into a more coherent European approach. That process is still evolving, but it introduces a level of coordination that was not there before and, over time, should reduce fragmentation rather than increase it.

We are seeing contributions from across Member States, which is an important signal that this is becoming a genuinely European process.

Another important shift relates to the demand for faster access to new therapies, which has come from both patients and industry. In response, regulatory and HTA processes are now more closely aligned, meaning that much of the work has to be carried out in parallel rather than sequentially. The overall volume of work may not have increased significantly, but the way it is organized has changed, and this creates real challenges, particularly for smaller companies that may not have the same internal capacity. It is not possible to design separate systems for different types of companies, but we are making a conscious effort to engage more directly and provide targeted support where possible.

Looking ahead, once the system is fully operational beyond 2030, this approach should lead to greater efficiency overall. The Joint Clinical Assessment will be conducted once at a European level rather than repeated across multiple countries, while national elements such as pricing and health economic evaluation will remain in place. At the same time, continued dialogue through the HTA Stakeholder Network will be essential, both to strengthen trust and to refine how clinical expertise and patient expertise are integrated into the process. That ongoing exchange is already proving valuable in improving understanding on all sides.

Over time, this should also contribute to greater alignment in clinical practice across Europe. As clinical guidelines begin to take account of Joint Clinical Assessment timelines, unnecessary variation between countries is likely to diminish, even if some differences will always remain. At the same time, as high-quality assessment reports are produced, published in English, and made openly available, they may increasingly serve as a reference point beyond Europe, shaping how other systems approach health technology assessment.

PB: How is the EU HTA system building the capacity to handle full-scale implementation as all centrally authorized medicines come into scope?

NH: The starting point is that the system must operate on equal terms. We cannot introduce a framework that creates inequality, so the assumption has to be that we will be able to manage the full scope as it expands. Much of the discussion around capacity has therefore focused on Joint Scientific Consultations, where activity is being built up progressively. There is clear feedback from companies that this should move faster, but at the same time there is a need to maintain quality. At this stage, the direction is broadly right, even if the pace remains a point of discussion.

There is also a structured approach to how this is being developed. The HTA Coordination Group publishes an annual work program alongside a report on what has been delivered, allowing stakeholders to follow both ambition and output. While the number of consultations and assessments has so far been relatively limited, the early experience has been positive, particularly in terms of the breadth of participation. One of the initial concerns was that the system might be dominated by a small number of more experienced agencies, but in practice we are seeing contributions from across Member States, whether through assessors, experts, or stakeholder engagement, which is an important signal that this is becoming a genuinely European process. 

There is a clear recognition that real-world evidence will become more important, even if questions around data quality, methodology, and its role alongside clinical trial evidence still need to be resolved.

At the same time, there is a balance to be maintained as the system scales. Numbers will need to increase, but expanding too quickly would risk undermining quality. For now, the priority is to establish a robust and credible foundation, even if that means a more gradual build-up than some stakeholders would prefer.

PB: In areas such as advanced therapies and rare diseases, how is the framework approaching the use of real-world evidence alongside more traditional clinical data?

NH: There are several layers to this. At a methodological level, we provide clear guidance on the type of evidence we would prefer to see and how it should be analyzed, but we also recognize that this is not always available, particularly for newer technologies. In those cases, we expect companies to explain their choices, justify the use of alternative data, and set out how the analysis has been conducted. That inevitably introduces a degree of flexibility, which some stakeholders perceive as a lack of predictability, but it reflects the reality of working with emerging evidence where not everything can be defined in advance.

More broadly, the role of real-world evidence remains an open question. Companies are understandably keen to use real-world data to support earlier access, but that position can become more complex when longer-term data do not fully support the initial assumptions. In that sense, building trust in real-world evidence is an issue not only for HTA bodies, but also for industry. Payers have perhaps moved further in using these data, although even there the overall approach is still evolving and not entirely settled.

At the same time, there is increasing interest in how real-world evidence can be integrated more systematically over time. It can already be discussed within Joint Scientific Consultations, and there is ongoing work on how reassessments might incorporate such data across the product lifecycle. From an HTA perspective, we are actively engaged in this area, including through initiatives such as DARWIN EU, where the HTA community is represented. There is a clear recognition that real-world evidence will become more important, particularly in areas such as oncology, advanced therapies, and rare diseases, even if questions around data quality, methodology, and its role alongside clinical trial evidence still need to be resolved.

PB: What role can the European HTA framework play in strengthening Europe’s competitiveness while maintaining equitable access to innovation?

NH: These are, of course, personal reflections, but one of the more striking contrasts at present is the speed at which policy can shift in other regions. If we look at developments such as Most-Favored-Nation pricing in the United States, the pace of change is extremely rapid, and with that comes a high degree of unpredictability. Europe has taken a different approach, building over several decades an HTA system grounded in evidence, methodology, stakeholder engagement, and value-based decision making. That process has been slower, but it has also created a more structured and predictable framework.

At the same time, it is important not to expect that the HTAR will resolve every challenge, particularly those linked to pricing and the broader global context. Some of these issues are inherently complex and will remain so. What the Regulation can do is strengthen the quality of the evidence base and provide a more consistent foundation for decision making across Europe. In that sense, its role is not to act as a barrier, but to support a system in which innovation can be assessed and adopted in a credible and transparent way, even if that means being less responsive to short-term external pressures.

PB: As the system evolves toward 2030, how should companies be thinking about their approach to the EU HTA framework?

NH: The advice is relatively straightforward, even if the implications are more demanding in practice. Companies need to engage closely with the available guidance, including methodologies and templates, and ensure that these are properly understood, sometimes with the support of those familiar with working within agencies or European institutions. There are also increasing opportunities for engagement through webinars and targeted outreach—including for smaller companies—which are intended to support understanding of the system and how it is evolving.

More fundamentally, preparation needs to begin earlier. The shift towards parallel regulatory and HTA processes means that these activities can no longer be approached sequentially. Regulatory strategy, evidence generation, and market access planning need to be aligned from an early stage across the organization. This reflects a broader change in how the system is designed to function, with the aim of improving patient access by making these processes more coordinated and efficient.