Artículo
EU HTA implementation: Learnings from the first year
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Ruairi O'Donnell, PhD
Strategic insights into EU HTA Regulations from year one
The EU Health Technology Assessment (HTA) regulation, launched in January 2025, marks a significant milestone in harmonizing health technology evaluation across Europe.i Its goals are ambitious: streamline assessments, minimize redundancies, and accelerate patient access to innovative therapies. While the regulation has been met with optimism, its first year has highlighted both progress and challenges that demand strategic adaptation, especially for manufacturers navigating this new system.
During ISPOR Europe 2025, the Cencora team hosted interviews with key stakeholders, offering invaluable insights into the EU HTA landscape:
- Niklas Hedberg (Chief Pharmacist at TLV and Co-Chair of the HTA Coordination Group) shared his perspective on Sweden’s evolving role under the regulation, the progress of PICO consolidation, and the importance of predictable processes for member states.
- Antonella Cardone (CEO of Cancer Patients Europe) emphasized the essential need for meaningful patient involvement in HTA processes and highlighted initiatives to improve patient engagement, such as early participation in PICO definition stages.
- Natalie Steck (EU HTA Team Lead, HTA Strategy and Policy Director at MSD) discussed challenges manufacturers face in aligning evidence generation with PICO requirements and called for greater transparency and formalized industry engagement during scoping.
- Alexander Natz (Secretary General, European Confederation of Pharmaceutical Entrepreneurs - EUCOPE) reflected on the broader implications of EU HTA and the need for effective uptake of JCA reports by member states to reduce redundancies and foster innovation.
Their combined expertise provide valuable lessons on the progress, obstacles, and opportunities ahead for stakeholders navigating the EU HTA system. This article distills key learnings from these discussions to offer practical guidance for manufacturers and stakeholders preparing for success under the regulation.
Progress: Early milestones in EU HTA implementation
The first year of implementation has seen several notable achievements:
Joint clinical assessments (JCAs):
Submitting the first JCA dossiers and consolidating PICO frameworks (Population, Intervention, Comparator, and Outcomes) were key milestones.ii These frameworks define the scope of assessments and are fundamental for preparing high-quality dossiers. For manufacturers, aligning evidence generation with PICO requirements is essential to meet the expectations of HTA bodies across member states.
Coordination and capacity building:
Coordination among member states has improved, with experienced HTA bodies supporting less experienced counterparts via tandem assessments, subgroup networks, and formal training programs. These efforts aim to build capacity across all 27 member states and ensure consistent assessment quality.
Stakeholder engagement:
Mechanisms like the EU HTA multi-stakeholder network group have fostered collaboration among patients, clinicians, health technology developers (HTDs), and payers. Proposed initiatives such as plain-language summaries of dossiers aim to make the HTA process more inclusive.iii For manufacturers, early engagement with stakeholders helps ensure their products address unmet medical needs.
These early wins demonstrate the potential to harmonize HTA processes, reduce inefficiencies, and improve patient access. However, there is still work to be done to ensure long-term success, and manufacturers must remain proactive in adapting to the evolving requirements.
Challenges: Key obstacles to address
Despite the progress made, several challenges have emerged:
Complexity of PICO consolidation:
As expected, harmonizing diverse national requirements into a single European PICO framework has proven challenging.iv Manufacturers often face uncertainty about how PICOs are formulated and consolidated, which shape the scope of submission document they need to prepare as part of the JCA. Formalized consultations with HTDs during the scoping process are needed to improve predictability and alignment.
Capacity constraints:
Both HTA bodies and industry stakeholders face resource limitations, particularly in managing the increased workload associated with JCAs and Joint Scientific Consultations (JSCs). Smaller companies may struggle to allocate sufficient resources to navigate the system effectively.
Patient and expert engagement:
Strict conflict-of-interest policies and limited capacity risk creating barriers to meaningful involvement of patients and clinical experts, particularly in rare disease areas where expert availability is limited.v For manufacturers, this can complicate efforts to ensure their products align with patient needs and clinical priorities.
These challenges are not insurmountable, but they require continuous refinement and collaboration among stakeholders to overcome effectively.
Opportunities: Practical steps to enhance EU HTA
The evolving EU HTA system presents several opportunities for manufacturers to optimize their engagement and contribute to its success:
Enhancing transparency and predictability:
Clear communication, timely updates, and formalized consultation opportunities for HTDs are key.iv Continuous process improvements, such as FAQs and feedback mechanisms, can help build trust and predictability in the system. Staying informed about changes will help manufacturers better anticipate requirements and prepare their dossiers effectively.
Leveraging JCAs for national decision-making:
Success will depend on how effectively JCA reports are utilized by member states to streamline reimbursement processes. Manufacturers should work closely with HTA bodies to ensure their JCA submissions are aligned with national needs, avoiding redundancy and maximizing efficiency.
Expanding JSC slots:
JSCs provide manufacturers with an opportunity to engage with HTA bodies early in the development process, ensuring alignment on evidence generation and assessment expectations. Advocating for more JSC slots and prioritizing early engagement can help manufacturers optimize their clinical development strategies.
Strengthening patient involvement:
Early patient engagement during PICO definition stages is key for ensuring products address unmet medical needs. Manufacturers should proactively engage with patient organizations and support initiatives like plain-language summaries of dossiers and JCA reports to make the process more inclusive and transparent, benefiting all stakeholders.
By taking these steps, manufacturers can position themselves as key contributors to the success of the EU HTA regulation while ensuring their products meet the needs of patients and healthcare systems.
Lessons on patient-centricity
One of the recurring themes in the interviews was the importance of embedding patient voices into the EU HTA process. Patients bring unique perspectives rooted in their lived experiences with diseases, offering valuable insights into unmet medical needs, clinical trial design, and treatment adherence.
However, patient organizations often face challenges in building the capacity and capabilities needed to participate effectively. While initiatives like ad-hoc training programs help smaller patient organizations prepare for involvement, more systematic approaches are needed to ensure meaningful engagement. Manufacturers can play a role in supporting patient engagement by collaborating with organizations, providing resources for training, and ensuring their products align with patient priorities.
Promising developments include the European Commission’s establishment of a dedicated working group on patient recruitment and contracting. This shows a willingness to listen to feedback and co-create solutions, which is important for embedding patient voices throughout the HTA process.
However, patient organizations often face challenges in building the capacity and capabilities needed to participate effectively. While initiatives like ad-hoc training programs help smaller patient organizations prepare for involvement, more systematic approaches are needed to ensure meaningful engagement. Manufacturers can play a role in supporting patient engagement by collaborating with organizations, providing resources for training, and ensuring their products align with patient priorities.
Promising developments include the European Commission’s establishment of a dedicated working group on patient recruitment and contracting. This shows a willingness to listen to feedback and co-create solutions, which is important for embedding patient voices throughout the HTA process.
Looking ahead:
What success looks like
By 2028, the EU HTA regulation is expected to deliver a substantial body of JCA and JSC reports, providing a clearer picture of its lifecycle impact. Success will be measured by:
For manufacturers, success means ensuring their products are assessed efficiently and fairly, with JCAs and JSCs enabling faster access to markets across Europe. To achieve this, manufacturers must remain proactive in addressing challenges, engaging with stakeholders, and advocating for continuous improvements to the system.
- Improved patient access to innovative therapies across Europe
- Reduced disparities between member states
- Streamlined reimbursement processes that minimize bureaucracy and redundancy
For manufacturers, success means ensuring their products are assessed efficiently and fairly, with JCAs and JSCs enabling faster access to markets across Europe. To achieve this, manufacturers must remain proactive in addressing challenges, engaging with stakeholders, and advocating for continuous improvements to the system.
In closing
The EU HTA regulation represents a transformative opportunity to harmonize HTAs across Europe. While significant progress has been made during its first year, challenges remain, particularly in areas like PICO consolidation, capacity constraints, and meaningful stakeholder engagement.
For manufacturers, understanding the evolving system, addressing challenges, and leveraging opportunities are essential to ensuring success under the new regulation. By focusing on transparency, collaboration, and patient-centricity, stakeholders can help make the EU HTA a success, delivering better outcomes for patients, supporting sustainable healthcare systems, and fostering innovation across Europe.
As the EU HTA evolves, collaboration and expert support will remain crucial to realizing its full potential. Engaging with an experienced partner can provide manufacturers with the expertise and resources needed to navigate the complexities of EU HTA. With deep HTA knowledge and specialist capabilities, Cencora’s EU HTA Center of Excellence supports manufacturers in optimizing JCAs and preparing for national-level implementation. From early evidence planning to post-JCA execution, Cencora offers the capacity and strategic guidance essential to succeed in this evolving landscape.
For manufacturers, understanding the evolving system, addressing challenges, and leveraging opportunities are essential to ensuring success under the new regulation. By focusing on transparency, collaboration, and patient-centricity, stakeholders can help make the EU HTA a success, delivering better outcomes for patients, supporting sustainable healthcare systems, and fostering innovation across Europe.
As the EU HTA evolves, collaboration and expert support will remain crucial to realizing its full potential. Engaging with an experienced partner can provide manufacturers with the expertise and resources needed to navigate the complexities of EU HTA. With deep HTA knowledge and specialist capabilities, Cencora’s EU HTA Center of Excellence supports manufacturers in optimizing JCAs and preparing for national-level implementation. From early evidence planning to post-JCA execution, Cencora offers the capacity and strategic guidance essential to succeed in this evolving landscape.
*Fuentes que continúan a continuación
About the author:
Ruairi O’Donnell is Vice President, Market Access Europe, and EU HTA Lead, at Cencora. A 20-year veteran of the global market access and HEOR consulting services sector, Ruairi has held numerous leadership positions in leading global consulting organizations, with a strong background in value strategy, evidence development, and market access pathways.
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Sources:
i. European Union. Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (Text with EEA relevance). Accessed on November 28, 2025. https://eur-lex.europa.eu/eli/reg/2021/2282/oj/eng
ii. European Commission. Guidance on the scoping process. Accessed on November 28, 2025. https://health.ec.europa.eu/publications/guidance-scoping-process_en
iii. European Patients Forum. 10 Key Recommendations from Patient Organisations on Joint Clinical Assessments under the EU HTA Regulation. Accessed on November 28, 2025. 20240610-10-key-recomendations-from-patient-organisations-on-jcas---disclaimer.pdf
iv. EFPIA – European Federation of Pharmaceutical Industries and Associations. From guidance to implementation: EFPIA’s reflections on EU HTA scoping & PICO exercises. Accessed on November 28, 2025. https://www.efpia.eu/news-events/the-efpia-view/blog-articles/from-guidance-to-implementation-efpia-s-reflections-on-eu-hta-scoping-pico-exercises/
v. European Commission. Health technology assessment – procedural rules for assessing and managing conflicts on interest - Feedback from: Cancer Patients Europe. Accessed on November 28, 2025. https://ec.europa.eu/info/law/better-regulation/have-your-say/initiatives/13751-Health-technology-assessment-procedural-rules-for-assessing-and-managing-conflicts-of-interest/F3470140_en
