ISPOR Europe 2024 – inside the second plenary: Ensuring readiness for EU HTAR

Following a successful Day one, the second day of ISPOR Europe 2024, the European conference for ISPOR—The Professional Society for Health Economics and Outcomes Research, continued to deliver insightful sessions and discussions. The second Plenary session, 'Ready, Set, Go: The Last Sprint for the EU HTA,' built on the discussions from the first plenary by exploring the readiness of stakeholders for the new EU Health Technology Assessment Regulation (HTAR). With just 55 days to go, this landmark reform promises to reshape how Europe assesses health technologies, aiming to harmonize clinical evaluations, enhance efficiency, and improve patient access across member states.

Moderated by Anne Willemsen (Dutch National Healthcare Institute, The Netherlands), the session focused on HTAR’s goals, short- and long-term challenges, and the collaborative effort required for its success. The panelists included Roisin Adams (National Centre for Pharmacoeconomics, Ireland), Vanessa Schaub (F. Hoffmann-La Roche, Switzerland), Michal Stanak (National Institute for Value and Technologies in Healthcare, Slovakia), and Valentina Strammiello (European Patients’ Forum, Belgium).

The promise of HTAR: opening perspectives 

Willemsen began by outlining HTAR’s goals; namely, increasing the quality and efficiency of assessments while reducing duplication across Europe. She emphasized that the regulation focuses exclusively on clinical domains, leaving reimbursement decisions and value assessments to individual member states. 

Kicking off the discussion, the panelists each shared their hopes for HTAR. Strammiello, who was representing the patient perspective, emphasized its potential to foster trust and collaboration. For patients, HTAR represents the possibility of faster, wider access to high-quality care, built on a foundation of trustworthy, transparent processes. 

Schaub, speaking from an industry perspective, continued by highlighting the need for a unified, high-quality assessment system to replace Europe’s current fragmented approach, which hinders its competitiveness as a hub for R&D and innovation. 

Stanak, representing HTA agencies, particularly those from Eastern Europe, hoped HTAR would reduce decision uncertainty and 'level up' access for patients in late-access countries like Slovakia. With tight budgets, small countries need smarter ways to discern true innovation and allocate resources effectively. 

Adams framed HTAR as a chance to refocus on evidence and transparency in decision-making. She stressed the need to address questions relevant to healthcare systems, driven by the collective goal of providing effective treatments to patients when they need them most – something she suggested should be a 'North Star' for all stakeholders.

Discussion #1 – Short-term implementation preparation

The first topic of discussion of plenary two focused on short-term preparations for HTAR’s January 2025 implementation. 

Adams began by declaring Europe to be ready, pointing to the development of implementing acts, IT systems, and operational financing. Approximately 30 assessments are planned for 2025, with outputs expected by late 2025 or early 2026. 

Stanak noted the varying readiness levels across member states. While well-established HTA bodies will need to focus on harmonizing processes, newer ones face a steep learning curve but also have a unique opportunity to develop pipelines and expertise. 

Schaub shared Roche’s proactive approach, having been preparing for HTAR since its adoption in 2021. With two oncology products slated for joint clinical assessments in 2025, Roche has overhauled internal processes, created cross-functional teams, and invested in training. “This is a massive change management process,” Vanessa explained, emphasizing the importance of treating HTA as equal to regulatory functions. 

Strammiello went on to highlight efforts to equip patients for meaningful contributions to HTAR processes. Programs like European Capacity Building for Patients (EUCAPA) and HTA4Patients, funded by the EU4Health Programme, train patient representatives from diverse backgrounds. Projects like Empowering Patients in HTA – a joint initiative between the European Patients’ Forum, OPEN Health and the European Alliance for Neuromuscular Disease – also aim to identify unmet needs and support patient involvement at both European and national levels.

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Discussion #2 – Long-term implementation preparation

Looking ahead, the panel explored the broader implications of HTAR beyond its initial implementation. 

Stanak raised concerns about comparators and timelines in late-access countries like Slovakia. He questioned whether assessments conducted under HTAR would remain relevant years later, urging the industry to reduce submission delays for smaller markets. 

Adams underscored the importance of robust evidence generation and early horizon scanning to identify healthcare system needs proactively. While European-level buying remains a longer-term goal, HTAR’s focus on shared evidence lays the groundwork for greater collaboration. 

Schaub noted that while PICOs (population, intervention, comparator, outcome) present a challenge – especially in oncology with its numerous subpopulations and comparators – early preparation and pre-specified plans can mitigate risks. She predicted that AI and digital tools will be leveraged in the future to streamline processes, although noted it would be important to keep the related legal and ethical considerations in mind. 

Strammiello stressed the importance of sustainability in patient involvement. This will require resources, updated tools, and a trained pool of patients ready to collaborate when needed. She also called for HTA bodies to strengthen their capacity to engage meaningfully with patients. 

Closing statements

In concluding the plenary session, each panelist offered a final reflection on HTAR’s potential: 

• Strammiello’s wish was that the process runs smoothly, and without bottlenecks. She highlighted that HTAR’s success depends on the collaboration of all stakeholders, and called on the ISPOR audience to think what they can do for HTAR to make it work smoothly, bearing in mind that patients are the ultimate recipients.
• Stanak saw HTAR as an opportunity for countries to make use of the momentum in the short term to allow levelling up in the long term of countries that currently experience later access to innovative treatments. He emphasized the role of HTA agencies role to help decision-makers to help patients.
• Schaub described her ‘blue sky’ dream that in 5–10 years’ time, all the stakeholders in Europe (including patients, caregivers, HTA authorities, policy makers, industry, and clinicians) are advocating for JCA for other jurisdictions around the world, because it has supported equitable access across Europe.
• Adams concluded by commenting that it is often discussed that Europe is falling behind; and that HTAR is Europe’s chance to lead. She also emphasized that it is imperative that HTAR improves evidence generation so that effective treatments are available to patients and that we are addressing questions that healthcare systems need to be addressed.

“This is a landmark reform for Europe, and the world is watching us.”
Vanessa Schaub, ISPOR Europe 2024

Conclusion 

As Willemsen aptly concluded, HTAR is like planting a tree – it will take time to grow, but with shared commitment it will bear fruit for patients across Europe. With its focus on evidence, collaboration, and equity, HTAR represents a bold step toward a more unified and efficient healthcare system. The countdown is on – there are just 55 days to go before the start of HTAR. Willemsen emphasized that now is the time to act, prepare, and ensure HTAR achieves its transformative potential.