EU HTA Conference Reflections

We joined other stakeholders in taking stock of the implementation of the EU Health Technology Assessment Regulation (HTAR) half-way into its first year of application at the “EU health technology assessment: Advent of a new era of collaboration” conference last week, hosted by the European Commission (EC). Reflecting on discussions, there are topics old and new – but the golden thread to all is a clear commitment to making this collaboration a success. Will it be?

“Strong Roots, New Shoots”

While the theme for this conference was “Advent of a New Era of Collaboration”, it is important to remember the existing foundations of this EU HTAR is over 20 years of voluntary collaboration. These are nicely summed up by the phrase “Strong Roots, New Shoots” (one that we cannot claim as our own!).

Representatives for the EC, the HTA Coordination Group (HTACG) and its subgroups, did not hide the fact that the first 6 months of implementation threw up challenges: some anticipated, some unanticipated. While exact details on those unanticipated challenges were lacking, we doubt the fact they were experienced is unexpected to anyone. The practical implementation of this EU HTAR will be a journey of continued learning for us all.

The strength of collaboration both within and across Member States was evidenced throughout the day, with many references to how states with more established HTA systems had been supporting those with less established HTA systems with their own preparations, as well as sharing best practices. The importance of other key stakeholders was also referred to frequently, with the Stakeholder Network and other external experts recognized as a vital part of the governance and application success of the EU HTAR. In particular, the importance of the patient voice was reiterated; similar to other recent conferences, it does feel like we are moving to the end of “patient tokenism” and towards a more robust and scientific approach to incorporating patient evidence in the decision-making process.

Industry has previously voiced concerns over the apparent lack of collaboration with health technology developers (HTDs) in the design and application of the Joint Clinical Assessment (JCA) process introduced under the EU HTAR. While the design phase has passed, there remain opportunities for this to improve in the application phase. The European Federation of Pharmaceutical Industries and Associations (EFPIA) were invited to be a part of discussions, suggesting that the EC do want industry to have a “seat at the table”.

The EFPIA used this “seat” to reiterate their ambition to be a true partner in making the EU HTAR a success. An open “brainstorming platform” was proposed to allow all key stakeholders to contribute to problem solving as we continue our shared journey of learning in this new era. Industry made up approximately half of the in-person attendees at the conference. They want to be informed, but more than that they want to be involved. Like other key stakeholders, industry are committed to making this collaboration a success but how big a part can they play without  more interaction steps being built into the application phase?

“Do more with less”

Capacity concerns have been raised many times since the EU HTAR came into force, in many different guises, and the capacity-building challenge remains at large. The financing of implementation has also been a key concern of Member States, in a time where demand for HTA is ever increasing while funding for HTA is simultaneously decreasing.

While details on the previously mentioned unanticipated challenges were lacking, the sense we got was that at least some of these could be linked back to capacity constraints and financing. There were several references to “bottlenecks” in discussions, as well as hints towards the need for enhanced predictability of demand and better streamlining between and across regulatory and HTA processes. National HTA bodies also referred to challenges faced when financing assessor and co-assessor roles, with local financing needed for up to 2 years (prior to EC reimbursement).

Upskilling and reskilling of all relevant stakeholder groups remains a priority need to ensure the EU HTAR is successfully implemented. For Member States with less mature HTA systems and processes, there is a continued reliance on expert pools outside of a governance structure, with a need to “subcontract” assessor and co-assessor roles while capacity building is ongoing. The EC has committed €4 million to the “Technical Support Instrument” (TSI) project, providing readiness support to 16 Member States. Those with more established HTA systems have designed their TSIs to focus on compliance, awareness, and optimization; those with less established HTA have designed their TSI to focus on systems, methods and process. There is still a high degree of variability in Member State readiness, though Member States with less established HTA may actually have an easier task ahead of them – creating new systems, methods, and processes is in some ways easier than changing existing systems, methods, and processes.

The EC has also committed to an “HTAR Capacity Building Programme”, to provide training for HTA assessors, co-assessors and staff in HTA bodies of EU Member States from now to April 2026 . However, there is still a long way to go to building sustainable HTA capacity. It is important to remember that training of experts includes patient and clinical experts as well as HTA experts. If we truly want to move towards a more robust and scientific approach to incorporating patient evidence in the decision-making process, we must also invest in training patient and clinical experts to be able to input at all stages of the HTA lifecycle – from research design through to dissemination. The “HTA4Patients” project is intended to support such training but early signs are that there is still a long way to go with reports of low patient engagement to horizon scanning efforts to date. These trainings are also very detailed – do we need a simpler way to explain HTA and the patient role in HTA?

Capacity concern in a different guise relates to the population, intervention, comparator(s), outcomes (PICO) framework – specifically, the requirement for HTDs to consider multiple potential PICOs when developing the evidence dossier for JCA submission. Industry continues to call for a simplified and unified approach to EU PICO creation, while the HTA CG continues to push back, based on the belief that we are not creating more PICOs but rather putting them all in one place. The reality will likely lie somewhere in between. No one wants an excessive number of PICOs; HTDs want to avoid synthesizing unnecessary evidence, and JCA subgroup members want to avoid wasting time reviewing such evidence.

Can evidence generation efforts (another capacity concern) be reduced through earlier, more informative design? The short answer is yes. Randomized controlled trials (RCT) remain the “gold standard” for generating evidence, but non-informative design can still provide low certainty results, when it comes to HTA, and supplementary data generation is commonly required. The European Organisation for Research and Treatment of Cancer (EORTC) are pioneering new trial methodologies to support treatment optimization and evidence generation applicable to real-world settings in collaboration with the European Medicines Agency (EMA); this is another new era of collaboration we should be closely monitoring.

“Can AI be part of the solution?”

Artificial intelligence (AI) was posed as offering the biggest opportunity for health system change ever faced, and there is an enormous potential for AI to help with some of the capacity building challenges discussed.

HealthAI described traditional HTA as “not designed for adaptive, real-time technologies.” This may be true from the perspective of assessing such technologies, but is it true from the perspective of adopting such technologies to aid assessment? AI technologies are rapidly evolving, and our use of them in HEOR is increasing year on year.

The UK-based National Institute for Health and Care Excellence (NICE) released a mission statement last year on using AI in evidence generation and reporting, quickly followed by US Food and Drug Administration (FDA) guidance on the same topic. The FDA has recently announced deployment of its in-house AI system, Elsa, to speed up scientific reviews in the face of increasing demand. Is it time for the HTA CG to follow suit? Both in terms of their own use of AI and provision of guidance to HTDs on how they can acceptably use AI.

Of course, the future is not AI alone, but AI plus human intelligence; we must upskill ourselves in parallel to AI upskilling, and recognizing the limitations of AI is as important as recognizing its potential. Nevertheless, we can certainly see potential for Expert-Directed AI™ to be part of the solution to several capacity building challenges.

“Faster and broader access”

Timing concerns have been raised many times since the EU HTAR came into force, again under many different guises. Member States who start their national HTA processes post marketing authorization are confident that the JCA will speed up their processes, but those who have accelerated processes that currently run in parallel with regulatory processes are yet to explicitly state how they will incorporate the JCA outcomes. Given that no one wants to slow down access, does this render the JCA obsolete for products eligible for accelerated access programs in  Member States with more established HTA systems?

We also heard from non-EU HTA representatives regarding the potential impact of JCA on their HTA systems and processes. For now, agencies including NICE in the UK are closely “watching with interest”, and the timing of JCA publication will be a key factor in its potential use outside of the EU, at least for initial assessment…of course, HTA does not stop at marketing authorization. The strive for “faster and broader access” should still be anchored around getting the right treatment to the right patients at the right time, and often data informing this is collected post marketing authorization.

What was interesting in the final keynote speech was a clear plea to the Member States from the EC to adopt the JCA in the way it is intended: to minimize additional data requests, acknowledging that “one dossier should be enough,” and to do everything they can to ensure that HTDs gain better predictability and a reduced duplication of efforts across the EU. 

So, will the EU HTAR be a success and achieve its ambition to provide faster and broader access? Only time will tell. True innovation and return on investment takes time, particularly when investment is designed to address multiple parallel objectives, as is the case for the EU HTAR. However, there remains a strong belief that economies of scale will make these early “teething problems” worthwhile, and the enthusiasm, engagement and dedication to successfully implementing this regulation observed to date gives us no reason not to share this belief. Together, we will always be stronger.