The Evidence Base Post 10 November 2025

ISPOR Europe 2025 daily round-ups: Day 1

Joanne Walker, Katie McCool & Laura Dormer

The doors have opened on ISPOR Europe 2025 in Glasgow, where researchers, policymakers, and industry leaders are coming together to explore the ideas shaping health economics and outcomes research (HEOR). Hosted by ISPOR — The Professional Society for Health Economics and Outcomes Research, the conference marks the Society’s 30th anniversary with four days of discussion under the theme “Powering Value and Access Through Patient-Centered Collaboration.”

With The Evidence Base team on site at the Scottish Event Campus, we’re covering some of the key sessions from the multi-track conference program. Here are some of the highlights from Day 1.

Welcome Remarks – Celebrating 30 Years of ISPOR

Opening the conference, ISPOR CEO Rob Abbott welcomed nearly 6,000 attendees and reflected on Scotland’s historic role in health economics. Acknowledging the challenges that are facing the community today – including funding cuts, scientific skepticism and organizational change – Abbott urged the HEOR community to draw inspiration from ISPOR’s 30-year legacy and “step confidently into the future”. Abbott highlighted the field’s real-world impact on health technology assessment (HTA), pharmaceutical pricing, and public health programs, emphasizing the need for enabling system conditions and broader dissemination beyond peer-reviewed articles.

In an exciting development for ISPOR, Abbott announced the launch of the ISPOR Institute for Healthcare Transformation, a new division that will be a “step change” in the relevance, use and impact of HEOR globally, and feeding into ISPOR’s 2030 strategy and its mission to bring science to policy and decision-making worldwide.

Keynote Address – Decision Making in a Complex World

Following the welcome remarks, Day 1 got underway with a keynote address, in which Dave Snowden (The Cynefin Company, UK) explored how complexity science offers a way to understand and manage healthcare systems. Snowden explained that complex systems are defined by numerous interacting elements, or actants, whose localized interactions lead to emergent patterns without central control. Such systems have memory distributed across the network and cannot be understood through linear cause-and-effect models.

Snowden cautioned against “retrospective coherence” – the human tendency to “join the dots” after events and impose false narratives of predictability. Instead, he urged decision makers to map the energy gradients within a system before acting, and to replace rigid outcome-based targets with vector goals that measure direction and speed of progress.

To manage complexity effectively, Snowden identified key principles: focus on proximity (acting where threats or opportunities are closest), pay attention to anomalies (which spark learning), maintain optimal granularity (smaller, more flexible units of action), and embrace obliquity (approaching problems indirectly). He also highlighted disintermediation – removing interpretive layers that distort insight – and abduction, the uniquely human reasoning mode that contrasts with AI’s inductive logic.

Illustrating these ideas with healthcare examples, Snowden described projects in which patients and clinicians simultaneously interpret patients’ real-time narratives, revealing discrepancies that improve empathy, safety and care design. Capturing stories as quantitative data, he argued, creates a more authentic and inclusive evidence base – embodying epistemic justice, the right of individuals to interpret their own experiences.

Snowden concluded that healthcare systems must move beyond mechanical models toward approaches that recognize complexity, uncertainty and human meaning. By integrating narrative data with scientific rigor, he suggested, HEOR can achieve deeper understanding and more adaptive decision-making – turning patient stories into dynamic, real-world evidence (RWE).

Plenary 1: Patient Engagement in Healthcare Investments – A Promise or a Practice?

The first plenary then provided the perfect introduction to the conference theme of “Powering Value and Access Through Patient-Centered Collaboration”, highlighting the importance of belief, structure and shared responsibility in embedding patient perspectives throughout innovation, regulation and investment. Moderator Joep Muijrers (Gilde Healthcare Partners, the Netherlands) was joined by panelists Luc Truyen (Argenx, USA), Carole Longson (University of Manchester, UK), David Haerry (Positivrat, Switzerland) and Steffen Thirstrup (European Medicines Agency [EMA], the Netherlands), spanning the divest perspectives of investors, industry, academia, regulation and patient advocacy.

Read our in-depth summary of Plenary 1 here >>>

Spotlight Session: How Could Patient-Centric HTA Evolve in the Changing Global Pricing Landscape?

Amid rapid policy shifts and growing calls for patient involvement, this spotlight session at ISPOR Europe 2025 examined how HTA can evolve to reflect real-world needs and values. The discussion brought together Sean D Sullivan (University of Washington, USA), Nick Adler (Novartis, Switzerland), Lynda Doward (RTI Health Solutions, UK) and Olivia Wu (University of Glasgow, UK).

Opening the session, Sullivan outlined the shifting global landscape for pricing and reimbursement, noting that traditional cost-effectiveness frameworks are under increasing pressure to incorporate broader elements of value. He traced how international price referencing has shaped markets over the past 25 years, driving price convergence, confidential discounts and delayed product launches, and described how the US is now adopting similar strategies under its emerging “most-favored-nation” (MFN) approach to drug pricing. Recent developments include the newly announced Medicaid-focused GENErating cost Reductions fOr U.S. Medicaid (GENEROUS) Model, alongside two rumored initiatives: GLOBE (Global Benchmark for Efficient Drug Pricing), thought to relate to Medicare Part B, and GUARD (Guarding U.S. Medicare Against Rising Drug Costs), expected to involve Medicare Part D. He noted these efforts signal a wider shift toward international benchmarking of prices and stronger federal oversight of affordability. Sullivan noted that this evolving policy environment sets a complex backdrop for HTA agencies seeking to maintain methodological rigor while adapting to changing patient and policy expectations.

Adler described how HTA systems are increasingly moving toward models that balance clinical benefit, economic efficiency and patient relevance. He noted growing recognition of the need to better capture utilities, caregiver impacts and broader dimensions of disease that extend beyond traditional measures such as the EQ-5D. Emphasizing the importance of maintaining incentives for progress, he proposed introducing an “innovation modifier” within assessment frameworks to ensure that patient benefit, unmet need and the value of novel therapies are appropriately recognized.

Doward explored the evolving role of patient experience data (PED) within HTA, emphasizing that while agencies increasingly request such evidence, its impact on final decisions remains uneven. She observed that submissions often focus narrowly on patient-reported outcomes rather than richer qualitative insights that capture lived experience, treatment burden or adherence. Examples from Germany and Canada illustrated how the presence, or absence, of PED can directly influence outcomes. She urged both sponsors and HTA bodies to integrate these data earlier and more strategically to demonstrate patient value.

Wu concluded with a forward-looking perspective on how HTA methods could evolve to better capture patient value. Drawing on a UK policy lens, she highlighted NICE’s ten priority areas for methodological development, including improved utility measurement, the integration of qualitative research and the use of real-world data (RWD). She noted that strengthening the scientific foundations of HTA will be essential to ensure fair and transparent decisions, and envisioned a future of adaptive, “living” HTA processes and cross-border collaboration where patient-defined value is embedded at the core.

Concurrent Sessions

Issue Panel: External Control Studies - What Does it Take to Get Real?

Moderated by Mariam Bibi (GetReal Institute, UK) and building on discussions from the GetReal Conference 2025, this Issue Panel explored the GetReal Institute’s new External Comparator Best Practices Framework for external control (EC) arm studies and examined its application from HTA, industry and methodological perspectives.

Wim Goettsch (Utrecht University; Zorginstituut Nederland, the Netherlands) opened with the HTA viewpoint, noting that while randomized controlled trials (RCTs) remain the gold standard, EC studies are increasingly proposed when RCTs are infeasible or unethical – particularly in rare diseases and advanced therapies. However, many HTA bodies remain skeptical, often rejecting studies based on aggregated data and finding wide variation in acceptable methodologies. Goettsch emphasized early dialogue through joint scientific consultations and called the GetReal framework a valuable starting point for structured decision-making, though it needs more regulator and HTA input.

Offering an industry perspective, Anke van Engen (IQVIA, the Netherlands) highlighted strategic, operational and design challenges limiting the routine use of EC studies – particularly uncertainty about return on investment and differing national expectations of “what good looks like”. She described the GetReal framework as a unique opportunity to build shared standards and pointed to the EU HTA Regulation (EU HTAR) as a driver for harmonization, especially as unanchored comparisons become more common.

From a methodological lens, Seamus Kent (Erasmus University Rotterdam, the Netherlands) discussed the target trial approach for mitigating bias in EC studies. He argued that ECs are often undervalued in HTA, and that while challenges such as confounding and differential censoring persist, richer longitudinal data and quantitative bias analyses could improve credibility.

In closing, Bibi stressed that RCT alternatives must be anticipated early, with proactive planning, fit-for-purpose data and multi-stakeholder dialogue to strengthen confidence in EC evidence.

Workshop: Measuring what matters: practical pathways for personalized endpoints and patient-centric approaches in HTA evaluation

This workshop explored how personalized endpoints and patient-centric approaches can strengthen evidence generation and HTA evaluation. Moderator Danny Yeh (Aesara, USA) outlined how aligning outcomes with patient priorities can make evidence more relevant and credible for decision-making, particularly as current surrogate endpoints often fail to reflect patient experiences and real-world benefit.

Gunes Sevinc (Ardea Outcomes, Canada) introduced goal attainment scaling (GAS) as a structured yet flexible tool for defining and quantifying individual treatment goals. The method involves patients and clinicians jointly identifying key priorities, which are scored and aggregated into standardized measures of treatment efficacy. She highlighted GAS as a means to improve sensitivity to meaningful change, capture heterogeneity, and strengthen engagement throughout clinical studies.

From industry, Hannah Staunton (Roche Products Ltd, UK) described how personalized outcomes sit along a continuum between standardized and individualized assessments. She emphasized their particular relevance for rare and heterogeneous diseases where traditional endpoints may not fully capture treatment impact, while noting that development, validation, and adoption remain resource-intensive and time-consuming.

From an HTA methodology standpoint, Dalia Dawoud (Cairo University, Egypt) explained how patient-centered outcomes can already contribute to clinical effectiveness and value assessment under existing frameworks, such as within joint clinical assessment (JCA) (population, intervention, comparator, outcome) PICO structures. She noted that their inclusion as novel value elements will require careful consideration of aggregation methods, societal preferences, and opportunity costs to ensure methodological consistency and transparency in decision-making.

Issue Panel: AI in CEE Health System Decision Making: Can Payers, Patients, and Policy Align for Smarter Access?

This issue panel session, chaired by Vladimir Guzvic (Health Insurance Fund of the Republic of Srpska, Bosnia and Herzegovina), examined how AI is helping to bridge gaps in HTA decision-making and accelerate evidence generation across Central and Eastern Europe.

Opening the discussion, Maciej Dzik (Agency for Health Technology Assessment and Tariff System, Poland) outlined his agency’s evolving approach to integrating AI into HTA. Referencing guidance from NICE, Canada’s Drug Agency and HAS, he noted that while global agencies emphasize responsible and ethical AI use, there is no unified framework. Dzik detailed AOTMiT’s proposed principles, covering human oversight, openness, transparency, explainability, and legal compliance, and described plans to build internal competencies through training and awareness programs.

Maciej Grys (Certara, Poland) then shared practical insights from using AI-driven tools in evidence synthesis and reimbursement projects. He reported up to 85% reductions in workload without sacrificing accuracy, highlighting how automation enables analysts to focus on interpretation and critical judgment. However, Grys identified ongoing barriers including data fragmentation, regulatory uncertainty, and limited AI literacy, calling for clearer methodological guidance and certification systems.

Concluding the panel, Kevin Kallmes (Nested Knowledge, USA) emphasized the need for methodological transparency, traceability, and expert oversight to ensure trustworthy outputs. He illustrated how AI is already supporting systematic reviews, patient phenotyping, and meta-analyses, and predicted that harmonized, reproducible workflows will define the next phase of HTA practice, where AI enhances, but never replaces, human expertise.

Educational Symposia: Strategic Insights From 5 Years of the EU HTA Regulation: Unleashing the Ambition for Orphan Products and Vaccines

This symposium examined how 5 years of EU HTAR strategic experience can guide preparations for upcoming JCAs of orphan medicines in 2028 and vaccines in 2030. Moderator Caroline Delaitre-Bonnin (Thermo Fisher Scientific, France) opened the session by highlighting the relevance of lessons learned from oncology and advanced therapy medicinal products for these upcoming areas.

Martin Parkinson (Thermo Fisher Scientific, UK) presented insights across four key areas: health technology developers, navigating JCAs and joint scientific consultations, country impacts, and evidence and analyses. He emphasized that JCA readiness requires company-wide transformation, with engagement across functions and early alignment between regulatory, HTA and payer strategies. Parkinson stressed that JCAs should be embedded into product development from the outset, supported by clear governance, coordinated evidence generation, and early involvement of country teams. He also noted that JCAs have the potential to improve equity and speed of patient access across Europe.

Samantha James (Thermo Fisher Scientific, France) discussed the unique challenges and opportunities for prophylactic vaccines under the regulation. She explained that while vaccines share many barriers seen in oncology and orphan medicine assessments, they also present distinct complexities in defining populations, comparators, and endpoints across national immunization programs. Despite these challenges, JCAs could help standardize evidence requirements and accelerate vaccine market access in Europe.

Closing the session, Delaitre-Bonnin reiterated the overarching message: the importance of early engagement, integrated strategy, and proactive planning to ensure readiness for the next waves of EU HTA implementation.

Workshop: Is Transferring Evidence Across Europe a Herculean Task? The Opportunities and Challenges of Transportability Analyses in the Context of European Union Joint Clinical Assessments

In this workshop, moderated by Grammati Sarri (Cytel, UK), speakers Blythe Adamson (Flatiron Health, USA), Antonio Remiro-Azócar (Novo Nordisk, Spain) and Stephen Duffield (National Institute for Health and Care Excellence [NICE], UK) examined the complexities of transportability of non-local data, within the context of the EU’s new JCA process.

Sarri opened with an overview of the landscape, highlighting that JCA reports will provide factual evidence summaries rather than recommendations, leaving judgments on external validity to national HTA agencies. She cautioned against the “data perfection trap”, where high-quality RCTs are prioritized despite RWD offering essential contextual insights.

Through a hypothetical case study, Adamson demonstrated the challenges of selecting appropriate evidence sources when trial populations, healthcare settings, and data quality differ across regions. Remiro-Azócar emphasized the statistical and methodological hurdles of transportability, including the limitations of shifted null hypothesis testing, which may disincentivize robust adjustment methods. Duffield presented UK case studies (including NICE appraisals in NSCLC and urothelial cancer) illustrating how RWD are already being integrated into NICE appraisals and underscored the importance of early engagement with HTA bodies.

The session concluded with discussion on incentives, variability in evidence acceptance across agencies, and the need for methodological innovation to ensure that Europe’s evolving data infrastructure supports credible, transferable evidence for decision making.

Issue Panel: The Evidence Was There. So Why Didn’t It Matter?

Moderator Luis Prieto (Novartis, Switzerland) opened this discussion by emphasizing that methodological rigor alone is not enough for RWE to influence regulatory and HTA decisions; data must also be reliable, transparent, and fit for purpose. Despite advances in methods such as target trial emulation and quantitative bias analysis, speakers examined why technically robust studies often fail to achieve decision impact.

Miguel Hernán (Harvard T.H. Chan School of Public Health; Adigens Health) noted that many problems stem from design flaws rather than the absence of randomization. He called for structured integration of RWE and RCTs, emphasizing collaboration rather than competition between study types, and highlighted benchmarking as a practical approach to strengthen confidence in observational findings.

Mona Khalid (Adigens Health) focused on the persistent trust gap in RWE, stressing that transparency around data selection, pre-specification, and bias assessment remains limited. She argued that without greater openness and early engagement with decision-makers, even methodologically sound evidence may lack credibility or perceived relevance.

From an industry perspective, Clark Paramore (Biogen, USA) described how perceptions of clinical meaningfulness and process dynamics can overshadow robust analyses, particularly in areas of high unmet need or rare disease. He suggested that decision-makers may need to adopt more flexible approaches when assessing evidence supported by long-term or RWD.

Across the session, the discussion underscored the need for early engagement, radical transparency, independent validation, and clear methodological standards to ensure that scientifically sound RWE achieves greater influence in policy and access decisions.

Educational Symposia: Navigating Global Policy Shifts: Implications for Europe’s Pharmaceutical Pricing, Market Access, and HTA Landscape

Moderated by Casper Paardekooper (Vintura, part of Cencora, the Netherlands), the session explored how rapid geopolitical and policy shifts are transforming global pharmaceutical markets and what this means for Europe’s ability to remain competitive in innovation and access. Attracting a capacity audience of more than 300 participants, the discussion highlighted the keen interest in how Europe can navigate, and ultimately benefit from, these global dynamics.

Opening the discussion, Christoph Glaetzer (Chief Global Value and Access Officer, Johnson & Johnson, USA) reflected on healthcare’s dual challenge of maintaining affordability while sustaining innovation amid geopolitical and economic uncertainty. He argued that health should be viewed as an investment rather than a cost, calling for “fit-for-purpose” value assessment frameworks that better capture patient-relevant outcomes and enable faster access to innovation across health systems.

Neil Grubert (Independent Market Access Consultant, UK) then outlined major policy shifts in the US, including the expansion of MFN pricing models, the new Medicaid GENEROUS pilot, and broader use of tariffs and reshoring incentives designed to drive pharmaceutical manufacturing back to the US. Grubert also noted the growing role of China as a more flexible and attractive launch market and discussed how Japan, the Middle East and other regions are adapting their reimbursement systems.

Concluding the presentations, Anja Schiel (Norwegian Medicines Agency, Norway) detailed Europe’s evolving policy response, including the revision of pharmaceutical legislation, the proposed Critical Medicines Act, and the ongoing implementation of the EU HTAR. She stressed the need for predictability, solidarity and sustainability, urging Europe to preserve universal healthcare while accelerating innovation adoption.

In the closing discussion, Glaetzer was optimistic that Europe can regain leadership by accelerating innovation and updating evaluation frameworks. Grubert suggested that pressure from the US and China could spur a more coherent and agile European life sciences strategy. Schiel added that external challenges are strengthening unity across member states, noting that Europe’s predictability and stability remain key advantages for sustainable innovation.

Forum: Modernizing Medical Device Evaluation: Aligning Lifecycle Evidence, HTA Methodologies, and Digital Support Tools

This forum session explored how evidence generation and assessment must evolve to keep pace with rapidly developing medical and digital technologies. Moderated by Lisa Weiss (Stieber Health Consulting, LLC, USA), the session examined how regulators, HTA bodies and industry can align frameworks to support adaptive, real-time evaluation.

Weiss opened by noting that, unlike pharmaceuticals, medical devices evolve iteratively, with continuous software updates and learning effects that challenge static evaluation models. She called for a “continuous evidence ecosystem” that links regulatory and HTA requirements, integrates RWD, and leverages AI and digital tools to enhance transparency and rigor.

Artem Boltyenkov (Siemens Healthcare Diagnostics Inc., USA) outlined the three pillars of market access for diagnostics – coding, payment and coverage – emphasizing the need for alignment across these components and the growing importance of clinical utility evidence in demonstrating value to payers.

From a policy and evaluation perspective, Anastasia Chalkidou (NICE, UK) discussed NICE’s lifecycle approach to digital and AI technologies, including conditional recommendations and early value assessments that balance innovation with evidence generation. This iterative model allows for ongoing data collection, real-world evaluation, and reassessment as evidence matures.

Finally, Rossella Di Bidino (Gemelli Teaching Hospital, Italy) presented the Agile HTA framework, designed to adapt assessment methodologies for fast-evolving digital health technologies. She underscored challenges around bias, evidence timeliness, and sustainability, advocating early stakeholder involvement and realistic, adaptable evaluation frameworks.

Issue Panel: Embedding Effective Patient Involvement in EU Joint Clinical Assessments

Antonella M Cardone (Cancer Patients Europe, Belgium) led the discussion on how to make patient involvement in EU JCAs more timely, structured, and meaningful. Speakers Julie Spony (European Commission, Belgium), Margaret Galbraith (French National Authority for Health [HAS], France) and Jose Diaz (Bristol Myers Squibb, UK) examined current practices, early experiences, and practical ways to strengthen patient participation as JCAs move into full implementation.

Four potential upgrades were discussed: developing an EU-wide patient-led PICO survey to capture outcomes before scoping; requiring multilingual plain-language summaries (PLS) of manufacturer dossiers and JCA reports; involving patients as co-authors for contextual domains such as unmet need and treatment acceptability; and introducing a formal patient and carer involvement policy similar to NICE.

Spony outlined progress in implementing the EU HTAR, including ten ongoing JCAs and more than 40 patients and clinicians already involved. She noted that awareness, conflict-of-interest clarity, and technical support remain areas for improvement. Galbraith described France’s pilot program for patient input into national PICO development, highlighting both its benefits and scalability challenges. Diaz underscored the importance of structured collaboration, transparent communication, and industry’s role in supporting meaningful patient engagement.

Audience polling showed broad support for earlier, structured patient input, with strong backing for patient-led surveys and multilingual PLS to improve transparency and accessibility. Speakers agreed that these measures could help overcome the current back-loaded engagement model, where patient input occurs too late to influence key decisions. Embedding patient perspectives from the outset of PICO development, supported by clear communication, standardized processes, and accessible materials, was seen as essential to achieving consistent, high-impact patient involvement across future JCAs.

Looking ahead to Day 2

Day 2 of ISPOR Europe 2025 promises another packed agenda, with sessions exploring innovation, evidence, and collaboration across healthcare systems. The morning plenary, “Pragmatic Trials—Bridging Research and Real-World Care,” will take center stage, examining how pragmatic approaches can strengthen the relevance and impact of clinical research. Check back tomorrow for our highlights of this plenary and insights key sessions from Day 2.