The EU Health Technology Assessment Regulation was implemented in January 2025 to replace 27 fragmented national evaluations with a single Joint Clinical Assessment, run in parallel with EMA review. The idea was simple enough and aimed at giving European patients faster, more equitable access to innovative medicines.
In practice, the first JCA reports have yet to be published, capacity is being built from scratch, and industry confidence is conditional at best. The framework has attracted support from almost every corner of the pharmaceutical ecosystem – regulators, payers, big pharma, patient groups – yet the nature of that support varies sharply.
For some, the HTAR is a generational opportunity to make Europe a more coherent and competitive market for innovation. For others, it risks becoming the 28th layer of compliance in a system that already struggles to move at the speed of science. With oncology and ATMPs in scope now, orphan medicines following in 2028, and all centrally authorised products by 2030, the window to get the architecture right is narrowing.
Some of the key voices shaping that debate, from the Coordination Group chair to the EMA’s Executive Director, from SME advocates to big pharma’s Brussels representative, reveal a system that is broadly agreed on destination but far from settled on how to get there.
“Building Confidence, Not Just Process”: The View from the Coordination Group
At the centre of the new framework sits the HTA Coordination Group, the body responsible for overseeing JCAs across all 27 Member States. Its Co-Chair, Niklas Hedberg, is candid about where the system stands. 18 months in, the focus has been less on outputs than on architecture – establishing procedures, building trust, and managing a transition that has no real precedent in European health policy. The first JCA reports have yet to be published, and Hedberg is careful to temper expectations while making the case that the groundwork being laid now will matter enormously as scope expands.
“The most important development over these first months has been the transition from a largely voluntary system, built over almost two decades under EUnetHTA, to a mandatory framework under the EU Health Technology Assessment Regulation,” he says. “This shift has required a sustained effort to build trust across stakeholders, and we are now moving towards 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.”
One of the framework’s central methodological challenges – the consolidation of national PICO (Patient/Problem, Intervention, Comparison, Outcome – a standardised framework used to structure clinical research questions) approaches – is an area where Hedberg sees genuine progress, even if the work is unfinished. The argument is that complexity is not new; it has simply been made visible.
“Where previously there may have been 20 or more national PICOs, we are now working towards 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. Looking further ahead, once the system is fully operational beyond 2030, the Joint Clinical Assessment will be conducted once at European level rather than repeated across multiple countries, while national elements such as pricing and health-economic evaluation will remain in place. The more meaningful insights will come with time and accumulated experience – it would be premature to draw firm conclusions at this stage.”
“Still Significant Work to Do”: The Regulator’s Balancing Act
No institution has had to adapt more carefully to the new framework than the European Medicines Agency. As the body responsible for centralised marketing authorisation, the EMA now runs its review process in parallel with the JCA – a structural change that demands close coordination with HTA bodies whose evidence expectations do not always align with its own. Emer Cooke, the agency’s Executive Director, offers a measured but honest appraisal.
“We have four products that have completed the joint clinical assessment process, with the first opinion published in April and three more due shortly. There is still significant work to do on aligning evidence requirements between the regulatory and HTA sides.
“I would push back slightly on the idea that what HTA bodies are asking for today is inherently more robust than what went before – I would say it is different. We look at the quality, safety, and efficacy of a medicine, the benefit-risk balance in the context of the product itself and patient need. When HTA bodies look at a product, they are assessing where it fits within their own clinical care and healthcare systems, which can vary considerably across member states.”
For Cooke, the answer to the evidence gap is not to slow approvals but to use the tools already available – real-world data, registries, post-authorisation studies – to build confidence over time. It is a position that reflects the EMA’s dual obligation: to act with urgency for patients while maintaining standards that are, by her account, already world-leading.
“We cannot require a full comparative assessment for every innovative product. For medicines where there are few available treatments, or where the standard of care differs across member states, that simply is not feasible – and we have a responsibility to patients that means we cannot always wait. By international standards, our evidence requirements are already among the most rigorous in the world. The answer lies in the tools available to us – real-world evidence, additional follow-up studies, registries. These are the means by which we build confidence in new products over time.”
“The Burden Falls on the Smallest”: SMEs Sound the Alarm
EUCOPE represents the interests of medium-sized and research-based pharmaceutical companies across Europe – precisely the cohort most exposed to the operational demands of the new framework. Its Secretary General, Alexander Natz, is not opposed to the regulation’s ambitions; his concern is with execution. The risk, as he sees it, is that a system designed to reduce duplication ends up layering new complexity on top of existing national requirements, with smaller companies bearing a disproportionate share of the cost.
“EUCOPE remains supportive of the central ambition behind the EU HTA Regulation: a more coherent European framework in which a single clinical assessment can support multiple national access decisions. Where further progress is needed is at product level, particularly through Joint Scientific Consultations that allow companies to understand evidence expectations before launching pivotal studies or preparing submissions. Current consultation capacity remains too limited for the needs of the market.”
“The PICO framework, intended to harmonise how patient populations, comparators, and outcomes are defined across Member States, has become a flashpoint for this concern. Rather than producing one consolidated set of requirements, the system risks generating a proliferation of overlapping evidence demands – each technically compliant, but collectively unmanageable for leaner organisations.”
“If innovative developers must respond to multiple PICO frameworks – covering different patient groups, comparators, and outcomes requested by individual Member States – the process risks recreating the fragmentation it was meant to solve. Each additional evidence scenario can mean further analyses, longer timelines, and higher costs, with the burden falling most heavily on smaller companies operating with leaner European teams. The reform has the potential to become a meaningful advance for European market access, particularly as it expands to orphan medicines from 2028. To deliver on that promise, however, Europe will need greater consistency between Member States, more opportunities for early scientific engagement, and a practical commitment to simplicity rather than additional administrative layers.”
“The 28th Compliance Burden – or a Genuine Breakthrough?”: Big Pharma’s Conditional Support
As the voice of Europe’s research-based pharmaceutical industry, EFPIA has long advocated for a more coherent EU-level approach to HTA. Its Director General, Nathalie Moll, remains broadly supportive of the regulation – but frames that support in explicitly conditional terms. The broader context matters to her argument: European healthcare systems are under fiscal pressure, geopolitical competition for pharmaceutical investment is intensifying, and the case for valuing innovation properly has never been more urgent. Against that backdrop, whether the HTAR simplifies or complicates the market access environment is not a technical question – it is a strategic one.
“The European Union Health Technology Assessment Regulation represents a potentially important opportunity to improve the way innovation is assessed and valued across Europe. The original objective was to create a more coordinated framework and avoid the duplication created by 27 different national evidence requirements and timelines, which today adds complexity and delay to the system. We now have the regulation in place, but it still needs to demonstrate that it can work effectively in practice.”
Moll is careful to situate the HTA debate within a wider shift in how European policymakers are beginning to think about pharmaceutical value – moving, if slowly, beyond short-term budget impact towards a broader accounting of what innovative medicines contribute to health system sustainability. But that shift makes the stakes around the regulation’s execution higher, not lower.
“We remain hopeful about the direction of travel, provided the framework evolves in a streamlined and pragmatic way, with meaningful involvement from industry and methodologies that genuinely focus on assessing the added clinical value of medicines at European level. Policymakers are beginning to look beyond pharma budgets alone and consider the role innovative medicines can play in keeping people out of hospital, supporting workforce participation, and improving the long-term sustainability of healthcare systems. Ultimately, the key issue for us is whether the system becomes ‘the 28th thing’ companies need to comply with, or whether it genuinely succeeds in reducing fragmentation and replacing the existing 27 national approaches.”
