Achieving Systemic Coherence for Healthcare Decision Making in Europe

An ISPOR Strategic Dialogues White Paper

Achieving Systemic Coherence for Healthcare Decision Making in Europe

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1. Background and Statement of Purpose

Health economics and outcomes research (HEOR) represents the convergence of 2 complementary disciplines and comprising multiple research methodologies to inform healthcare decision making.[1] By generating and integrating clinical and economic evidence from clinical trials or real-world settings, HEOR provides a structured framework to measure value, weight uncertainty, and support resource allocation decisions across health systems.

In the European Union (EU) and European Economic Area (EEA), patient access to new medicines and other health technologies is primarily shaped by 3 interdependent decision-making groups:

• Regulators,[2] member states’ competent authorities[3] which, hosted centrally by the European Medicines Agency (EMA), assess the benefit-risk balance based on efficacy, safety, and pharmaceutical quality and issue opinions for the European Commission’s marketing approvals.
• Health Technology Assessment (HTA) bodies[4] that primarily evaluate comparative clinical effectiveness and economic value—and can also integrate ethical and social value judgments. They are now organized through a Member State Coordination Group on HTA (HTACG) established by the EU HTA Regulation (HTAR).[5]
• Payers[6] and procurers (eg, statutory health insurance funds, national health services, and regional authorities) who determine coverage, pricing, and access conditions within member states’ national healthcare systems while considering short- and long-term affordability constraints.

The mandates for these 3 groups differ, and processes and decisions are mainly sequential. Regulatory approval of medicines and certification of certain medical devices by a Notified Body[7] is most often a prerequisite for HTA; HTA informs pricing and reimbursement (P&R) negotiations; payers consider both regulatory and HTA evaluations with varying emphasis to inform contracts with companies and real-world patient access.[8] Together and while balancing unmet health needs and budgetary constraints, these processes define the speed, predictability, equity, and sustainability of access to innovation. Understanding the connections within and between these European processes may significantly impact portfolio allocation by venture capital and institutional investors, as well as how health technology developers optimize R&D investments to design studies that satisfy evidence requirements across all 3 groups in a timely manner.

Over the past 2 decades, multiple European initiatives have sought to improve coordination among these groups, for example: 

• Parallel joint scientific consultation[9] and early dialogues[10] on evidence generation between regulators and HTA bodies and technology developers

• Collaboration among HTA bodies to harmonize methods and assessments (EUnetHTA Joint Actions[11])

• Structured interactions between HTA bodies and payers to facilitate translation of evidence into coverage decision[12] 

However, these efforts have predominantly focused on bilateral relationships (regulator–HTA bodies, HTA bodies–payer, and regulator–payer[8]). A consistent, systematic approach that convenes all 3 groups remains absent. Such an approach would:

• Align evidence requirements from early development to post-marketing phases
• Coordinate expectations for the quality and generation of clinical, economic, and real-world evidence
• Deploy HEOR coherently to inform decisions across the entire decision pathway 

The implementation of Regulation (EU) 2021/2282 (EU HTA Regulation, EU HTAR) marks a structural shift toward joint clinical assessments (JCAs) and more proactive scoping of evidence requirements, creating a timely opportunity to address these gaps. As joint HTA activities expand, there is an increasing need to view access decision making as a closely linked continuum, rather than as isolated steps within separate institutions across the 3 groups. 

In addition, alignment across the 3 groups is shaped by industrial, innovation, and trade policies at both national and European levels, as well as by comparable policy shifts in major markets like the United States and China. The Draghi report, "The Future of European Competitiveness,"[13] recognized HTA at the European level as a tool for improving efficiency ahead of national pricing and reimbursement decisions and enabling faster patient access to innovative medicines. Yet a critical gap persists: how can better alignment between these groups serve as a strategic lever for strengthening Europe's competitiveness in the global health technology sector?

In 2025, ISPOR—The Professional Society for Health Economics and Outcomes Research, launched a new initiative, the ISPOR Strategic Dialogues, beginning with the European Union and European Economic Area (EEA). The EU/EEA pilot initiative convened its first Dialogue meeting in late 2025 with the participation of experts from regulatory, HTA, payer, and wider health policy decision-making fields. The primary aim of this initial pilot project is to enhance the understanding and identification of needs, challenges, and alignment opportunities in the regulator–HTA body–payer decision-making process. Particular focus was put on evidence generation and other pertinent issues for improving the relevance and application of HEOR methods and practices. With ISPOR positioned as an impartial convener, these dialogues focus on evidence generation across the full spectrum, from clinical trial data to real-world data (including patient-reported outcomes or PROs) on outcomes and costs, and to patient preferences while exploring practical ways to strengthen collaboration on patient access and treatment optimization decisions, such as for advanced therapies or oncology treatments.

Purpose of this paper:

This white paper synthesizes insights from the meeting discussions and proposes practical ways forward to improve the alignment of evidence generation across the full life cycle of health technologies. It is intended to support both policy development and structured collaboration that enable more coherent, predictable, and patient-centric evidence generation and use across the full range of regulatory–HTA–payer decision-makers in Europe. Beyond offering recommendations, the paper is intended to serve as a catalyst for wider dialogue, and the authors welcome reflections from other stakeholders, including patients, clinicians, academia, industry, and others, to deepen the dialogue and build European multistakeholder capability focused on improved patient access decision making in Europe.

2. Analysis of Why Evidence Misalignment Persists

2.1 Distinct Mandates Coupled With National Healthcare Systems Specificities Result in Different Evidence Priorities

Regulators focus on benefit–risk balance at a population level. HTA bodies assess relative effectiveness and cost-effectiveness against current standards of care. Payers consider affordability, equity, budget impact, healthcare investment priorities, and political feasibility.

These differences reflect legitimate national sovereignty in how healthcare is organized and financed. However, on top of these institutional arrangements, there is wide disparity in access to medicines across the EU and EEA. This reflects, at least in part, how healthcare systems in Europe developed and continue to be administered. The absence of structured tri-partite alignment mechanisms between regulators, HTA bodies, and payers leads to cumulative fragmentation—both at national and European levels.

2.2 Siloed Sequential Processes Fail to Yield Comprehensive and Cohesive Evidence

Current processes are predominantly sequential as well as fragmented rather than integrated (and dynamically adaptive), thus potentially ignoring the overall impact of new technologies in the healthcare ecosystem. Early scientific advice mechanisms remain primarily regulator–HTA driven, with limited payer and procurer involvement.

At the same time, investor and technology developer incentives tend to prioritize market size and commercial return over population health prioritization. The definitions of unmet medical need and comparative evidence requirements are not consistently embedded upstream in development decisions.

2.3 Data Are Fragmented, Lack Interoperability, and Are Difficult to Access

Data fragmentation and lack of interoperability persist across national registries, electronic health records, and claims databases. Interoperability, data quality standards, and semantic harmonization are uneven across countries.

Initiatives such as the European Health Data Space (EHDS)[24] and DARWIN EU[25] create significant opportunities, and HTA and payer communities must define fit-for-purpose requirements proactively to avoid overly regulatory-centric data architectures that inadequately serve HTA evaluation and reimbursement needs. EU-funded joint undertakings such as IHI-GREG project[26] bringing together multistakeholder perspectives and addressing multiple stakeholders needs, build a much-needed foundation for the contemporary RWD/RWE generation practice, including frameworks and tools.

3. Emerging Opportunities in the EU/EEA Policy Landscape

3.1 EU HTA Regulation Enables a Shift Towards Clearer Evidence Needs

The EU HTA Regulation (EU HTAR) introduces joint clinical assessments and formal scoping processes built around PICO[27]-defined evidence requests reflecting Member State needs and (legal) requirements. This facilitates national authorities’ and technology developers’ earlier planning and work on market access in parallel to JCA and regulatory assessment.

This marks a paradigm shift for Europe—from reactive dossier assessment based on general requirements defined in guidance documents and templates to proactive definition of product-specific evidence requirements. If effectively linked to early scientific consultations and health technology life-cycle planning, this has the potential to influence technology developers’ trial design and data collection and reduce downstream uncertainty.

3.2 European Health Data Space and DARWIN EU Hold Potential for Increased Use by Both Regulators and HTA Bodies

EHDS establishes a governance framework for secondary use of health data across Member States. DARWIN EU provides a distributed data network supporting decision-grade RWE while consulting centers of competence in pharmacoepidemiology in Europe and collaborating with real world healthcare databases across the European Union.

For HTA bodies and payers, these initiatives create:

• Opportunities to assess comparative effectiveness as well as treatment pathways in real-world settings
• Mechanisms to study small populations and rare diseases
• Infrastructure for conditional and managed reimbursement linked to data generation

However, timely HTA engagement in defining data quality labels, interoperability standards, and access governance is essential and urgent. Without it, the system will underperform—limiting the ability to effectively assess the value of novel medicines with life-saving potential.

3.3 Payers and HTA Bodies Are Collaborating Across Borders

Collaborations such as BeNeLuxA, Joint Nordic HTA Bodies (JNHB),[28] and the Nordic Pharmaceutical Forum demonstrate:

• Joint HTA and larger procurement capacity
• Stronger leverage in negotiations
• Shared learning on uncertainty management

These models provide scalable templates for broader alignment possibly under the EU HTA framework, which also enables horizon scanning for emerging technologies primarily based on information from EMA and the International Horizon Scanning Initiative[29] (IHSI).

3.4 Ongoing Legislative and Public Health Initiatives Present Policy Opportunities for Change

Parallel developments—including the pharmaceutical legislation reform[30], the Critical Medicines Act[31] and Biotech Act[32] discussions—highlight growing recognition of the link between access to affordable therapies and support to the development of innovation in Europe.

There is a policy window to embed life-cycle evidence planning, unmet need prioritization, and cross-border cooperation into these reforms. Parallel EU legislative reforms present an opportunity to institutionalize integrated evidence-generation plans and cross-border cooperation within a coherent legal framework. Success depends on clarifying operational connections between these files, particularly regarding clinical trial pathways and research incentives, to leverage Europe's larger patient base and create the regulatory predictability that attracts global investment.

3.5 Other Actors Have Recognized and Are Working to Improve Regulator-HTA-Payer Alignment

Several European and international initiatives and projects are actively contributing to improved coherence across regulatory, HTA, and payer decision making. While their mandates and geographic scopes differ, each addresses critical components of evidence generation, pricing and reimbursement, methodological development, or policy coordination. Time-limited project-based initiatives provide an opportunity to launch and develop valuable approaches and solutions,[33] but they usually lack sustainability mechanisms beyond the project duration.

Examples of ongoing European-focused initiatives that are built on sustainable financing models and operational structures include:

• The National Competent Authorities on Pricing and Reimbursement (NCAPR) Joint Action CAPRICORD[34] (Competent Authorities for Pricing and Reimbursement to coordinate Involvement of clinical experts and patients, to manage uncertainty and the Challenges associated to ORphan medicinal proDucts), which strengthens collaboration among national pricing and reimbursement authorities in the EU and EEA, including work on dynamic pricing models, early dialogues, uncertainty mapping tools, and methodological development.[35]
• The Heads of HTA Agencies Group (HAG)[36], which promotes strategic coordination among European HTA agencies, including engagement with regulators on evidence challenges and uncertainty management.[37]
• The ISPOR HTA Roundtable - Europe[38], which convenes European HTA bodies and payers in the region to hold discussions focused on advancing scientific methods, value frameworks, and practical policy translation to optimize healthcare decisions and bridge the gap between decision makers.
• The Centre for Innovation in Regulatory Science (CIRS), which works globally to advance evidence-based regulatory science and facilitates dialogue between regulators, HTA bodies, and industry on evidence standards and life-cycle approaches. Since 2024, CIRS has conducted a series of workshops focusing on the Joint Clinical Assessment implementation issues.[39]

Structured cross-initiative visibility and dialogue is clearly needed. To ensure coherence across these initiatives and maximize their collective impact, a light-touch coordination mechanism should be established—periodic joint briefings, shared priority mapping, or a publicly accessible initiative registry can:

• Improve transparency of ongoing activities
• Reduce duplication of analytical work and stakeholder consultation
• Facilitate knowledge transfer and scaling of successful pilots
• Strengthen the cumulative policy impact of individual initiatives

Given constrained public resources and increasing demands on expert participation, coherence across initiatives is not merely a matter of administrative efficiency—it is a prerequisite for delivering tangible improvements in evidence alignment, patient access, and health system sustainability across the EU/EEA.

4. Practical Pathways to Better Evidence Alignment Across the Life Cycle

4.1 HTAR Updating Provides an Opportunity to Require Early Stakeholder Evidence Planning

The EU HTAR implementation process will undergo evaluation in 2028 after 3 years of coming into force.  This would provide an opportunity to improve joint scientific consultations that should:

• Include payer and procurer perspectives systematically
• Clarify post-launch evidence expectations
• Prospectively define uncertainty management strategies

Current regulator-HTA dialogues with technology developers should expand to tri-partite (regulator-HTA-payer) engagement, shifting from advisory exchange to shared lifecycle evidence planning (“co-creation”)

4.2 Differentiating Evidence Requirements by Level of Innovation

Not all technologies warrant the same evidentiary intensity. A structured EU-level framework for categorizing transformational innovation versus those with incremental benefit (including but not limited to “me too” products) could enable differentiated assessment depth and prioritization aligned with unmet population health needs.

4.3 Adaptive Approaches to Facilitate Access While Evidence Continues to Accrue

The updated EU’s pharmaceutical legislation (with expected endorsement in October 2026) introduces regulatory sandboxes to foster innovation by enabling controlled, time-limited testing of novel therapies under regulatory supervision. These sandboxes allow testing of advanced technologies like AI-driven manufacturing and personalized medicine to speed up market access, aiming to ensure safety while reducing regulatory burdens. 

A living HTA approach[40], supported by RWE, should become standard for products entering with high uncertainty.[41]

Adaptive pathways represent a prospectively planned, adaptive approach that allows a medicinal product to be authorized for use in a controlled or limited patient population while additional clinical trial evidence and RWE are being collected and assessed. Once sufficient evidence is gathered, the authorization can be expanded to larger patient populations or, if data do not support the initial findings, adjusted accordingly.

Post-approval evidence generation should be incentivized by linking pricing to the resolution of key uncertainties identified by all stakeholders—rewarding manufacturers with price premiums when evidence conclusively demonstrates value, while applying price adjustments when critical evidence gaps remain unaddressed. This prospective approach aligns manufacturer investment in targeted real-world studies with payer needs for decision-relevant evidence, ensuring fair return on investment for generating high-quality post-launch data while reducing financial risk for health systems and accelerating patient access to innovative therapies under managed conditions

5. Making Real-World Evidence Work for HTA and Payers

5.1 Defining HTA and Payer Requirements for EHDS

HTA and payer stakeholders should:

• Specify core data elements, comparators, endpoints, and economic variables
• Engage in defining the EHDS “data quality and utility label”
• Align with master data management standards to ensure semantic interoperability

5.2 Building Trust in RWD/RWE

Trust requires transparency in methodology, bias assessment and handling, as well as governance. Common European methodological standards for use of RWE in HTA should be developed collaboratively. The ISPOR-ISPE HARPER protocol template[42] could be a starting point for greater transparency and appropriate standardization.

5.3 Using RWE to Address Small Patient Populations and Rare Diseases

Cross-border RWE aggregation is essential for rare diseases and advanced therapies with small target populations. Shared definitions of severity and orphan status,[43] combined with distributed data networks, can address evidence gaps that no single Member State can resolve.[44]

6. Payers and Procurers Would Benefit From Greater Visibility and Participation With Other Stakeholders

6.1 From “Payers” to Strategic Buyers

The term “payer” understates the strategic role of these actors. As buyers and procurers, they shape market incentives, prioritize investment in healthcare, and determine affordability within macroeconomic constraints.

“Buyer” decision making must increasingly integrate:

• Long-term system sustainability
• Ability-to-pay considerations and transparency on these considerations (cfr. Also 8.3)
• Equity and access modalities, including prioritization based on landscape analysis of horizon-scanning insights

Understanding strategic buyers (beyond just "payers") equips health technology developers with critical market intelligence and design priorities (“affordability-by-design thinking” as developing in other sectors of the economy) that dramatically improve the likelihood of adoption, reimbursement, and sustainable market access.

6.2 Cross-Border Cooperation Can Be Scaled to Improved Evidence Generation and Use

Voluntary payer and HTA regional alliances such as BeNeLuxA, Nordic Pharmaceutical Forum, and JNHBA have demonstrated proof of concept. Scaling these models within the European collaboration context, while preserving final decisions at the national level, can:

• Enhance negotiation leverage
• Reduce duplication
• Share analytical capacity
  
  

7. Governance, Communication, and Trust

7.1 Opening Silos Without Undermining Independence

Structured collaboration must respect institutional independence but recognize interdependencies across the 3 decision-making groups. Formal mechanisms with stable financing and legal status for information exchange (while respecting confidentiality) as practiced by, for example, the EMA and the European medicines regulatory network,[45] are preferable to the unpredictability/vulnerability of ad hoc interaction.

7.2 Patient and Clinician Involvement

Meaningful patient and clinician engagement requires sustainable financing, training, and safeguarding representativeness, and includes, but is not limited to, the following:

• Patient-reported data and patient-relevant data from clinical trials and real-world settings should be systematically integrated across the spectrum of evidence generation  
• Integrating patients' preference and engaging patients early and often in the process is needed—they are the ultimate user or “customer”—and anything that is developed (including assessment and decision-making mechanisms and processes) should be tested with patients to help ensure it meets their needs
• Mandatory involvement of patients and clinicians in the joint scientific consultations and joint clinical assessment under the EU HTAR and recognition of patient experience data in the EU marketing authorization processes is a development in the right direction

7.3 Communicating Decisions and Rationales

Decision makers must explicitly articulate:

• Context-specific uncertainties
• Value judgments and priority setting
• Budgetary constraints

Clear communication strengthens legitimacy even when outcomes differ across Member States. There is a call for better communication and explanation of how decisions are made, highlighting the differences in national perspectives and the importance of accepting these differences. Context-specific uncertainty that provides a basis for differences in outcomes of decision making on access in various countries should be transparent.

8. Policy Recommendations and Priority Actions (12–36 Months)

To translate the vision of aligned, patient-centered health technology governance into practice, a coordinated set of actions is needed across procedural, structural, and systemic levels. The following recommendations prioritize interventions that can deliver measurable progress within 12–36 months while laying the groundwork for deeper transformation. They are organized by time horizon and complexity: short-term actions focus on establishing processes and generating evidence; medium-term efforts build institutional capacity and methodological harmonization; and strategic initiatives address fundamental misalignments between innovation incentives, societal needs, and health system sustainability.

Short-Term (Procedural)

• Develop concrete proposals for joint scientific consultations that expand beyond the regulator and HTA to include payer and patient perspectives
• Establish structured HTA and payer engagement in EHDS governance and a protected amount of funding to support these stakeholders’ contributions
• Map where access decisions are made across member states to identify potential for scaling and efficiency gains

Medium-Term (Structural)

• Develop EU-level life-cycle evidence frameworks integrating regulatory and HTA milestones
• Continue to harmonize RWE methodological standards across borders building on Darwin, ISPE - ISPOR HARPER template, and reporting standards such as the TARGET checklist, RWE4Decisions, and the IHI-GREG project, among other emerging relevant initiatives
• Institutionalize periodic HTA re-evaluation linked to post-launch evidence generation (living HTA)

Strategic (Systemic)

• Establish structured EU-level unmet need prioritization processes
• Work with investors and industrial policy makers, both international—eg, OECD—and national—eg, ministries of economy/trade and industry—as well as ministries of science, to realign innovation incentives toward high-value areas that also consider agreed-upon unmet societal needs
• Integrate ability-to-pay and sustainability considerations into value frameworks
  
  

9. Role of ISPOR and the Broader Stakeholder Community

ISPOR is uniquely positioned to catalyze the systemic coordination and methodological alignment needed to realize patient-centered, evidence-based health technology governance across Europe. This unique position stems from ISPOR's role as a neutral platform that convenes stakeholders impartially. ISPOR's foundation in rigorous research ensures credibility, while its focus on the critical interface between scientific evidence and policy application means the solutions developed are both methodologically sound and operationally feasible for real-world implementation. Building on the ISPOR Strategic Dialogues initiative and serving as an impartial convener and facilitator, ISPOR can:

• Develop shared language and tools, frameworks with relevant partners linking HEOR methods to policy needs
• Map ongoing initiatives (eg, NCAPR, payer alliances, regulatory data strategies) to avoid duplication of efforts
• Advocate for evidence-informed policy making at EU and global levels.

Supported by its Institute for Healthcare Transformation and its global membership, ISPOR can bridge scientific rigor and policy pragmatism by:

• Highlighting the value of HTA in achieving the goals of affordability, accessibility, and availability as laid out in the EU Pharma Legislation to ensure the sustainability of national health care systems
• Convening multistakeholder dialogues 
• Mapping gaps, initiatives, and best practices 
• Translating evidence into policy-relevant insights
  
  

10. Conclusion: Toward a More Coherent, Patient-Centric Evidence Ecosystem

Europe has the institutional and scientific capacity, as well as political momentum, to move from fragmented, sequential decision making toward a life-cycle–based, aligned, dynamic, and adaptive evidence ecosystem.

The EU HTA Regulation, EHDS, and a host of legislative measures in the EU/EEA healthcare policy space coupled with expanding cross-border collaborations create a historic opportunity. Realizing it will require proactive engagement, structural reform, and sustained trust-building across regulators, HTA bodies, payers, industry, patients, clinicians, academia, and policy makers.

A more coherent evidence system is not the end. Its purpose is to ensure that patients across Europe receive timely, equitable access to health technologies that deliver meaningful clinical benefit, economic value, and better long-term sustainability for European health systems.

Acknowledgements

A special thank you to the white paper authors-participants of the 2025 ISPOR Strategic Dialogue meeting:

Francis Arickx, PharmD, RIZIV INAMI; Dorthe Bartels, AMGROS I/S; Finn Børlum Kristensen, MD, PhD, University of Southern Denmark; Harald Enzmann, MD, MSc, BfArM; Anja Schiel, PhD, Norwegian Medicinal Products Agency; Tomáš Tesař, PharmD, PhD, MBA, Comenius University; Kärt Veliste, MSc, Ministry of Social Affairs of Estonia; Rob Abbott, ISPOR; Julia Chamova, MBA, ISPOR; Kelly Lenahan, MPH, ISPOR; Laura Pizzi, PharmD, MPH, ISPOR and Rutgers University

Thank you to the additional 2025 ISPOR Strategic Dialogue meeting participants for their contributions:

Mariane Cossito, M.Pharm, INFARMED; Birgitte Klindt Poulsen, MD, Medicinrådet; Saskia Knies, PhD, ZIN; Francois Meyer, MD; Piia Rannanheimo, MSc, FIMEA; Carlos Martin Saborido, MSc, PhD, AETS

Disclaimer

The views expressed in this paper are those of the authors alone and do not necessarily reflect the official positions or policies of their affiliated organizations or institutions.

References

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[2]  https://health.ec.europa.eu/document/download/18a3dc1d-4876-4553-a292-84fc4a7f9e31_en – Key Concepts section, p.13).

[3] https://www.ema.europa.eu/en/partners-networks/eu-partners/eu-member-states/national-competent-authorities-human 

[4] https://health.ec.europa.eu/document/download/18a3dc1d-4876-4553-a292-84fc4a7f9e31_en- Key concepts section, p.13).

[5] HTAR covers comparative effectiveness assessment part of HTA; economic and other aspects are evaluated on the EU Member States level. https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment_en.

[6] Define payers (reference: https://www.yhec.co.uk/glossary-term/payer/#:~:text=A%20payer%20is%20an%20individual,clinics%2C%20and%20pharmaceutical%20companies).

[7] A notified body is an organization designated by an EU Member State (or other countries under specific agreements) to assess the conformity of certain products before being placed on the market. These bodies are entitled to carry out tasks related to conformity assessment procedures set out in the applicable legislation when a third-party intervention is required. (as defined by the European Commission, https://health.ec.europa.eu/medical-devices-topics-interest/notified-bodies-medical-devices_en , accessed April 27, 2026

[8] Access understood as discussed in OECD (2020), Addressing Challenges in Access to Oncology Medicines, Analytical Report.  

[9] https://www.ema.europa.eu/en/human-regulatory-overview/research-development/scientific-advice-protocol-assistance/parallel-scientific-advice-special-development-aspects-or-product-types and relevant activities under the ENCEPP (European Network of Centres for Pharmacoepidemiology and Pharmacovigilance) umbrella https://encepp.europa.eu/index_en

[10] https://www.ema.europa.eu/en/documents/press-release/early-dialogue-between-regulators-and-health-technology-assessment-bodies-key-medicines-development_en.pdf

[11] https://web.archive.org/web/20230118032903/http://www.eunethta.eu/

[12] BeNeLuxA, Nordic Pharmaceutical Forum, JNHB initiatives and others.

[13] https://commission.europa.eu/topics/competitiveness/draghi-report_en

[14] Čechová, Z., Kubátová, J., Bártová, A., Jamárik, J., & Samek, J. (2025). "Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union." Therapeutic Innovation & Regulatory Science, 59, 728–736.

[15] https://beneluxa.org/ 

[16] https://nordicpharmaceuticalforum.com/ 

[17] https://pubmed.ncbi.nlm.nih.gov/36215113/ 

[18] https://www.ema.europa.eu/en/about-us/how-we-work/data-regulation-big-data-other-sources/data-quality-framework-medicines-regulation 

[19] https://target-guideline.org/ 

[20] https://www.darwin-eu.org/ 

[21] https://sustain-hta.org/ 

[22] https://ihi-greg.eu/ 

[23] Patient Reported Outcomes (PROs), Performance Outcome Assessments (PerfOs), Patient Experience Data (PED) 

[24] https://health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space-regulation-ehds_en

[25] https://www.darwin-eu.org/

[26] https://ihi-greg.eu/ 

[27] Population, Intervention, Comparator, Outcome (PICO), EU HTAR Fact Sheet

[28] https://jnhtabodies.org/ 

[29] https://ihsi-horizonscandb.ecri.org/ 

[30] https://health.ec.europa.eu/medicinal-products/reform-eu-pharmaceutical-legislation_en

[31] https://health.ec.europa.eu/medicinal-products/critical-medicines-act_en

[32] https://health.ec.europa.eu/publications/proposal-regulation-establish-measures-strengthen-unions-biotechnology-and-biomanufacturing-sectors_en

[33] https://sustain-hta.shinyapps.io/SUSTAIN-OBS/ 

[34] https://www.isciii.es/en/agencia-evaluacion-tecnologias-sanitarias/capricord/home 

[35] While CAPRICORD Joint Action is a time-limited project, NCAPR is a continuing, voluntary cooperation mechanism rather than a formal permanent EU body with dedicated legal status, staff, or regulatory powers. Its sustainability depends on ongoing political commitment and Commission support.

[36] https://htahag.eu/ 

[37] https://www.ema.europa.eu/en/documents/other/joint-htab-regulatory-perspectives-understanding-evidence-challenges-managing-uncertainties-exploring-potential-solutions_en.pdf

[38] https://www.ispor.org/member-groups/councils-roundtables/health-technology-assessment-council/health-technology-assessment-roundtables

[39] https://cirsci.org/publications/navigating-national-decision-making-post-jca-2025-workshop-synopsis/

[40] https://www.ispor.org/publications/journals/value-outcomes-spotlight/vos-archives/issue/view/preventive-medicine/in-sync-with-technological-innovation-and-ongoing-evidence-generation--the-need-for-an-hta-transformation-through-the-operationalization-of-living-hta 

[41] Vreman, R. A., Geenen, J. W., Knies, S., Mantel-Teeuwisse, A. K., Leufkens, H. G. M., Goettsch, W. G., & on behalf of the HTAi-DIA working group. (2020). Uncertainty management in regulatory and health technology assessment decision-making on drugs: Guidance of the HTAi-DIA working group. International Journal of Technology Assessment in Health Care, 36(4), 346-354.

[42] https://www.ispor.org/heor-resources/good-practices/article/harmonized-protocol-template-to-enhance-reproducibility-of-hypothesis-evaluating-real-world-evidence-studies-on-treatment-effects 

[43] ISPOR report (2015) on rare disease terminology and definition: Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, Hughes DA; International Society for Pharmacoeconomics and Outcomes Research Rare Disease Special Interest Group. Rare Disease Terminology and Definitions-A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group. Value Health. 2015 Sep;18(6):906-14. doi: 10.1016/j.jval.2015.05.008. Epub 2015 Aug 18. PMID: 26409619.

[44] https://www.europarl.europa.eu/thinktank/en/document/EPRS_STU(2026)774708 (EU rare disease action plan- European Added Value Assessment)

[45] Broich, K.; Lobker, W. Towards a Unified European View of Clinical Evidence: What ‘Health Technology Assessment Organizations` Can Learn from Regulatory Experience. J. Mark. Access Health Policy 2025, 13, 19. https://doi.org/10.3390/jmahp13020019 

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