ISPOR Europe 2025 daily round-ups: Day 2

Day 2 of ISPOR Europe 2025 in Glasgow built on the discussions that began the previous day, with sessions focusing on the practical application of evidence to support value, access, and patient-centered decision-making. Organized by ISPOR — The Professional Society for Health Economics and Outcomes Research, the conference continues under the theme “Powering Value and Access Through Patient-Centered Collaboration.”

The Evidence Base team is attending sessions throughout the meeting to capture key insights and emerging trends. Below are some of our highlights from Day 2.

Welcome Remarks, Presidential Address, and Awards

The second day of ISPOR Europe 2025 opened with welcome remarks from Uwe Siebert (UMIT TIROL – University for Health Sciences and Technology, Austria; Harvard Chan School of Public Health, USA) who reflected on the Society’s mission to advance scientific excellence and evidence-based decision-making.

As ISPOR President (2025–2026), Siebert outlined his presidential vision, identifying five key areas of importance for ISPOR and the wider HEOR community: 

• Scientific rigor – upholding transparency, truthfulness and best-practice methodology
• Inclusiveness and sustainability – ensuring diverse voices and environmental awareness are integrated into health technology assessment (HTA)
• Education – expanding global training access through in-person and digital learning
• Connections – fostering collaboration across societies, regions and sectors
• Globalization – deepening ISPOR’s reach across Asia Pacific, Latin America and emerging regions

Siebert also celebrated the 2025 HEOR Award Winners, commending their contributions to advancing the science and practice of health economics and outcomes research (HEOR).

Siebert then welcomed Natasha Azzopardi-Muscat (WHO Europe, Denmark) to the stage, who addressed the growing challenge of misinformation and declining public trust in science, calling for communication that is both technically sound and socially meaningful. She announced a new 3-year Memorandum of Agreement between WHO and ISPOR, focused on:

• A novel medicines platform to unite decision makers and stakeholders
• Capacity building for HTA systems worldwide
• Leveraging real and emerging evidence and AI for faster, more relevant insights
• Joint advocacy for science and evidence as foundations of public trust

Plenary 2: Pragmatic Trials—Bridging Research and Real-World Care

The second plenary of ISPOR Europe 2025 explored how pragmatic clinical trials can bridge the gap between controlled research and real-world practice. Moderated by Denis Lacombe (EORTC, Belgium), the panel – including Antonella Cardone (Cancer Patients Europe, Belgium), Francesco Pignatti (European Medicines Agency, Netherlands), Beate Wieseler (IQWiG, Germany), Natasha Azzopardi-Muscat (WHO Europe, Denmark) and Michael Zaiac (Daiichi Sankyo Europe GmbH, Switzerland) – addressed misconceptions about pragmatic clinical trials, emphasizing their rigor and value in complementing traditional RCTs.

Discussions highlighted the potential of pragmatic clinical trials to answer practical questions around treatment optimization, reduce toxicity and cost, and improve inclusivity. Speakers stressed the importance of collaboration, early stakeholder engagement, and aligning regulatory, HTA and patient perspectives.

Read our in-depth summary of Plenary 2 here >>>

Spotlight Session: Accelerating Patient Access to Innovations in Europe: Is it Possible to Integrate the JCA into Diverse HTA systems?

Moderated by Ruairi O’Donnell (Cencora, UK), this session examined how the newly implemented EU HTA Regulation (EU HTAR) could transform national decision-making and patient access across Europe. O’Donnell opened with an overview of progress to date: from January 2025, joint clinical assessments (JCAs) have become mandatory for new oncology medicines and advanced therapy medicinal products (ATMPs), with orphan drugs to follow in 2028 and all other medicines from 2030. He described the Regulation as the world’s largest example of centralized HTA, designed to reduce duplication, increase standardization, and support faster, more predictable patient access, while acknowledging the complexity of reconciling 27 distinct national systems.

Anja Schiel (Norwegian Medicines Agency, Norway) provided the HTA body perspective, explaining the practical realities of integrating JCA outputs into existing national frameworks. She illustrated the differences between regulatory evidence focused on benefit–risk and HTA evidence focused on comparative effectiveness, noting that while Norway and other Nordic agencies are ready and committed to reusing JCA results, the process will require significant time and adaptation. Schiel urged patience as all parties adjust to new parallel timelines and resource demands, cautioning against premature criticism of early reports.

Professor Rod Taylor (University of Glasgow, UK) explored methodological implications, using surrogate outcomes as an example of where harmonization may prove difficult. He highlighted the variability in how agencies validate such evidence and questioned whether rigid thresholds could hinder practical implementation.

Concluding the session, James Ryan (AstraZeneca, UK) delivered the industry perspective with humor, sharing his self-diagnosed “JCA anxiety disorder” – a tongue-in-cheek reference to the uncertainty surrounding implementation. He observed that while the JCA aims to streamline evidence generation, it also brings unpredictable and complex PICO (population, intervention, comparator, outcome) requirements that vary across member states, alongside earlier timelines, additional analyses, and higher reporting standards. Ryan outlined the recent publication of a seven-step “therapy plan” for manufacturers, stressing early preparation, cross-functional collaboration, proactive dialogue with HTA bodies, and maintaining focus on national decision-making to maximize the real-world value of JCA outputs.

Concurrent Sessions

Issue Panel: Data From Early Access Programs: Essential HTA Evidence or Misaligned Value Tool?

Early access programs (EAPs), often referred to as compassionate use or expanded access depending on national regulations, offer patients with serious or rare diseases the opportunity to receive promising therapies ahead of formal authorization. This expansion has brought renewed scrutiny over how EAP data should be interpreted, whether as meaningful evidence to inform HTA or as information that risks being misapplied in value assessments.

Moderator Kieron Lewis (Clinigen Healthcare Limited, UK) opened by noting that as real-world data (RWD) collection evolves, there is growing interest in how, when, and by whom EAP data can be used to inform decision-making.

Stephen Maddocks (Medicines and Healthcare products Regulatory Agency, UK) provided a regulatory perspective, reflecting on lessons from the UK’s Early Access to Medicines Scheme (EAMS). He acknowledged that while EAMS has not always delivered on its promise, often being implemented too late in development, the MHRA is now piloting a continuous regulatory approach and encouraging earlier data generation. He highlighted the new RWD Scientific Dialogue Programme, new position paper setting out its intentions to reform the regulatory framework for rare disease therapies and ongoing collaboration with NICE to better integrate regulatory and HTA advice.

Susanne Michel (Ascenian, Germany) examined the European experience, outlining how EAP data can vary in usefulness depending on the treatment, indication, and methodology. She noted that while such data are rarely a substitute for randomized controlled trials (RCTs), they can meaningfully complement them, particularly in orphan indications and when defining PICOs under the EU HTAR.

Em Jennings (Novartis, UK) offered the industry viewpoint, emphasizing that EAPs remain primarily patient-focused initiatives, with data collection a secondary benefit. She described how early planning, strong internal coordination, and structured RWD frameworks are key to ensuring these data are credible and ethically collected. Jennings also pointed to the potential role of AI in improving data consistency across global submissions.

Issue Panel: Outcome Assessment in Rare Diseases: Is JCA Breaking the Silos or Perpetuating the Chaos?

With orphan drugs entering the JCA process from 2028, the panel explored how outcome assessment in rare diseases could either streamline or complicate access. Moderator Laura Sawyer (Symmetron, UK) outlined how the EMA, JCA and national HTA bodies interact, describing the JCA as the evidence bridge connecting regulatory evaluation to national reimbursement. She noted that while the framework aims to improve consistency and transparency, defining appropriate outcomes in rare diseases remains one of the toughest challenges due to small populations, limited data and symptom variability.

Milad Karimi (BioMarin, UK) discussed the growing complexity sponsors face when developing a single outcomes plan that satisfies regulators, HTA bodies, clinicians and patients. He suggested that JCA may standardize processes but not goals, and called for flexibility where multiple scientific approaches are valid.

Steffen Thirstrup (EMA, the Netherlands) emphasized that the JCA system is still in its early stages and that expectations should remain realistic. He pointed to joint scientific consultations (JSCs) as a key tool to align on comparators, endpoints and analysis plans for orphan products.

Olivier Chassany (Assistance Publique–Hôpitaux de Paris – AP-HP, France) highlighted major inconsistencies in how HTA bodies assess patient-reported outcomes, including validation requirements, proxy use and endpoint hierarchies. He stressed that flexibility in these areas is essential to avoid penalizing innovation in ultra-rare conditions.

Exhibit Hall Theater: The Real-World Mosaic: Uniting Data to Illuminate the Patient Experience

Moderated by Bruce Feinberg (Cardinal Health, USA), this session explored how integrating real-world datasets can create a more complete picture of patient outcomes across community practice settings. Feinberg opened by highlighting the limitations of RCTs, including duration, and narrow patient populations, and emphasized the need for RWE that is representative, relevant, and rapidly actionable. He described how connecting multiple data sources forms a “mosaic” in which each tile adds clarity to the overall view of the patient journey.

Nicholas Lazarou (Cardinal Health, USA) discussed the challenges of building this mosaic, including fragmented data systems, inconsistent coding, and patient attrition. He explained how tokenization links structured and unstructured data to create a unified evidence framework. Lazarou introduced SoNaR (Specialty Networks Registry), launched in 2022 and developed through partnerships with group purchasing organizations (GPOs) and specialty physician networks. The platform harmonizes data from more than 38 electronic medical record and practice-management systems across over 7000 providers. By connecting clinical, genomic, claims, and patient-reported information, SoNaR reduces information gaps and supports robust longitudinal analyses.

To demonstrate its impact, Lazarou and Feinberg presented a case study on Provenge, a personalized immunotherapy for advanced prostate cancer. Using SoNaR data, Cardinal Health showed that patients treated in community settings achieved a 40-month improvement in survival compared with published literature. The speakers concluded that engaging clinicians directly in evidence generation strengthens data quality, enabling richer, regulatory-grade insights that extend well beyond the traditional clinical-trial environment.

Exhibit Hall Theater: MFN pricing: “More Fun to Navigate”—A European roadmap to success

Drawing a capacity crowd, Tim Wright (Genesis Research Group, UK) examined how manufacturers can prepare for the evolving most-favored nation (MFN) pricing landscape in the US and its implications for European pricing strategies – a topic garnering significant attention throughout ISPOR Europe 2025. He emphasized that success depends on understanding the broader political, legal, and economic context, highlighting that MFN pricing remains marked by uncertainty and complex negotiation dynamics.

Wright reviewed lessons from the 2020 Executive Order issued under Trump’s previous term, which encountered significant legal challenges and underscored the limitations of implementing nationwide price controls without Congressional approval. He noted that any future MFN initiative would likely proceed through the Center for Medicare and Medicaid Innovation (CMMI), which permits only limited testing of pricing models across defined drugs and geographies. This makes an immediate, nationwide rollout improbable. He also highlighted the new GENEROUS model, a voluntary CMMI initiative that aims to tie Medicaid drug prices more closely to those paid in other developed countries, as an example of how new pricing approaches may be piloted on a smaller scale.

He described MFN as effectively an international reference pricing (IRP) model that compares US prices with those in OECD countries with at least 60% of US GDP per capita. However, uncertainty persists around which price types to use for comparison, given discrepancies between US published prices, such as wholesale acquisition cost (WAC), average sales price (ASP), and the ‘Big Four’ federal purchasers, and European list or confidential net prices.

Wright advised manufacturers to conduct cross-market price analyses to understand exposure risk, review both the 2020 and 2025 Executive Orders for legal and procedural insights, and incorporate scenario-based planning into strategic assessments. He concluded that while the potential implications are significant, immediate drastic action is not warranted, stressing that measured, informed preparation is the most effective way to navigate continuing policy uncertainty.

Issue Panel: What Lessons Can the US Learn from Europe’s Experience With International Reference Pricing?

Building on the discussion of the MFN policy’s wider implications, the issue panel explored how it may intersect with Europe’s experience of IRP. Moderator Sean D. Sullivan (University of Washington, USA) outlined the MFN framework, which benchmarks Medicaid prices to the second-lowest among eight reference countries, restricts lower pricing abroad, establishes a direct-to-consumer channel, and returns savings to US patients and taxpayers. He also described the GLP-1 pricing agreement with Eli Lilly and Novo Nordisk, which sets negotiated Medicare and Medicaid prices, introduces indicative pricing for forthcoming oral products, and includes a CMMI demonstration model, with coverage expected to begin in mid-2026.

Panos Kanavos (LSE, UK) explained how reference pricing systems function across Europe and highlighted key challenges, including reliance on list rather than net prices, launch delays, price volatility, downward price convergence, and launch sequencing that can disadvantage lower-income markets. He noted a gradual shift toward value-based assessment and outlined best-practice principles for more sustainable pricing frameworks.

From industry, Julie Spiesser (Takeda) called IRP an effective yet blunt cost-containment tool that can distort global pricing, influence launch sequencing, and create risks for trade and supply. She contrasted “price-makers,” which rely on value-based approaches, with “price-takers,” driven primarily by reference pricing, and highlighted Germany’s policy reversals.

Virginia Lee Acha (Merck/MSD, UK) described the MFN proposal as politically durable but still highly uncertain in its details and long-term implementation. She noted that while the approach could reduce revenues and alter investment signals for innovators, its overall impact is likely to vary across therapeutic areas. Acha also discussed how the policy might reshape international trade relationships and influence the strategies of pharmaceutical manufacturers based outside of the US and Europe.

Representing patients, François Houyez (Eurordis, France) emphasized the need for consistent price comparison methods, faster access timelines, and stronger safeguards against shortages. Citing Greece’s post-2008 experience, he called for more coherent economic evaluation practices and greater market diversification.

Issue Panel: Harmonizing and Enabling Cross-Border Real-World Evidence Studies in Europe

Moderator Ashwin Kumar Rai (Thermo Fisher Scientific, USA), part of the ISPOR RWE Special Interest Group, began the session by outlining how harmonizing RWE across Europe requires addressing persistent challenges such as data fragmentation, governance variation, and methodological inconsistency across HTA bodies. Rai emphasized the need for a structured, forward-looking data strategy to support cross-border studies and enhance the utility of RWD. He also highlighted collaborative initiatives such as GREG, a multi-stakeholder IHI-funded project aimed at developing common frameworks and best practices for generating robust RWE for HTA.

Sandrine Bourguignon (RWEality, France) highlighted the importance of harmonization in RWE recognition across Europe, calling for interoperable data systems, a minimal common dataset, and the integration of patient experience measures alongside clinical outcomes.

Building on the solutions, Ignacio Medrano (Savana, Spain) showcased how AI-driven natural language processing (NLP) can extract reliable insights from unstructured medical records to build living, “next-generation registries.” He noted that these approaches align with the ambitions of the European Health Data Space (EHDS) to enable secure, federated data sharing and improve evidence generation efficiency.

Offering the industry perspective, Michael Hurst (Bristol Myers Squibb, UK) discussed the challenge of prioritizing evidence generation amid limited resources and heterogeneous national requirements. He explained that industry teams are increasingly required to balance local evidence demands with global strategies, often needing to do more with less. Hurst emphasized that success depends on closer collaboration between industry, HTA bodies, and regulators, as well as the adoption of standardized processes and federated analysis frameworks to achieve consistent, efficient, and high-quality RWE generation across Europe.

Issue Panel: Adding Nature’s Petal to the Value Flower: Can Inclusion of Environmental Impact in HTA Aid the Race to Net Zero?

This issue panel explored whether HTA can and should play a role in addressing the healthcare sector’s environmental footprint. The session was moderated by Grace Hampson (Office of Health Economics, UK), who introduced the session by discussing the vicious cycle of healthcare systems driving climate change, and climate change affecting health. She posed key questions to the panel on whether HTA is the right tool to mitigate environmental impact, or whether other policy levers – such as financial, regulatory, or procurement incentives – would be more effective.

From the HTA agency perspective, Helen Knight (NICE, UK) described NICE’s initiatives to integrate environmental sustainability into decision-making, guided by the NHS principle of achieving “healthy people and a healthy planet”. Public engagement through the NICE Listens project revealed strong support for incorporating environmental considerations, particularly where greener options are equally cost-effective. NICE has since taken practical steps including decommissioning high-emission anesthetics.

Andrew Briggs (London School of Hygiene & Tropical Medicine, UK) questioned whether environmental impact should be addressed within HTA at all, arguing that mitigation should occur earlier in product development and manufacturing. Including environmental effects in HTA, he cautioned, risks diverting limited health budgets from patient benefit.

Finally, Emmanouil (Manolis) Karamalis (IFPMA, Switzerland) advocated a systemic approach, emphasizing that most healthcare emissions stem from supply chains. He outlined guiding principles for progress, including collaboration, flexibility, piloting policies, alignment with broader sustainability goals, and comprehensive frameworks.

Issue Panel: A New Era for EU HTA: What Can the EU HTAR Learn from the EMA’s Path to Harmonization?

Moderated by Wim Goettsch (Utrecht University; Zorginstituut Nederland, the Netherlands), the session compared the EU HTAR’s early implementation with the EMA’s decades-long pathway to EU-level coordination. An opening poll revealed limited confidence in the EU HTAR’s ability to achieve its objectives, with an average score of 5.38 out of 10. Francine Brinkhuis (Utrecht University, the Netherlands) summarized a narrative review and focus-group study comparing trajectories and highlighting four themes: governance and power dynamics, national sovereignty over value judgements, collaboration and trust building across uneven capacities, and differences in regulatory integration and product scope, including multiple PICOs in JCAs.

Tina Wang (Centre for Innovation in Regulatory Science [CIRS], UK) pointed to practical enablers from the EMA model: independent rapporteur and co-rapporteur assessments, transparency through public assessment reports, formal quality management and benchmarking, and multinational teams to grow capacity. She cautioned against judging JCAs too soon and noted access also depends on company submission strategies and national system design.

Anja Schiel (Norwegian Medicines Agency, Norway) underscored Europe’s diversity, patience, and consensus-building, urging balanced engagement with patients and industry and continuous self-assessment. Ansgar Hebborn (Roche Products Ltd, Switzerland) emphasized the EMA’s dual role as protector and enabler, the importance of regulatory science and structured stakeholder engagement, and the need to scale joint scientific advice to ensure decision-relevant evidence, avoiding a decades-long learning curve.

Looking ahead to Day 3

Day 3 of ISPOR Europe 2025 will bring the conference discussions to a close, with the final plenary, “RWE in European Healthcare Decision Making—What’s in it for Patients?” tying together this year’s key themes of collaboration, data, and patient-centered value. Check back for our reflections and closing highlights from Glasgow.