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The Evidence Base Post

ISPOR 2025 daily round-ups: Day 1

  • Joanne Walker, Katie McCool & Laura Dormer

After months of preparation, the leading international conference from ISPOR—The Professional Society for Health Economics and Outcomes Research returns with ISPOR 2025, taking place May 13–16 in Montreal, Canada. This year’s theme, ‘Collaborating to Improve Healthcare Decision Making for All: Expanding HEOR Horizons,’ reflects the growing role of HEOR in shaping policy and practice worldwide.

The Evidence Base team is on site, providing coverage throughout the event, including daily round-ups and key insights from the plenary sessions. Below, we share highlights from Day 1.

Welcome Remarks

ISPOR 2025 opened with a traditional indigenous welcome to Montreal, followed by welcome remarks highlighting the Society’s 30th anniversary. Rob Abbott, CEO and Executive Director of ISPOR, was joined on stage by Tammy Clifford from the Canadian Institutes of Health Research and Carole Jabet from the Fonds de Recherche du Québec, to welcome the more than 4000 attendees to the event and highlight the conference them of, ‘Collaborating to Improve Healthcare Decision Making for All: Expanding HEOR Horizons’. Abbott reflected on ISPOR’s three decades of advancing health economics and outcomes research (HEOR) and highlighted the Society’s evolution into a global leader in improving healthcare decision-making. Clifford and Jabet highlighted Montreal's diversity and contributions to healthcare innovation, including evidence-based medicine, artificial intelligence (AI) and clinical trials. Abbott, Clifford and Jabet then welcomed Gregory Daniel (Eli Lilly) to the stage, as Co-Chair of the conference program and moderator of the first plenary session.

Plenary 1 – Drug Price Controls—What Are the Unintended Consequences to Innovation?

The first plenary session of ISPOR 2025 tackled one of the most politically charged and globally consequential issues in pharmaceutical policy: drug price controls and their potential unintended consequences for innovation. Moderated by Gregory Daniel (Eli Lilly), the session brought together an expert panel – including Virginia Lee Acha (Merck), Graham Cookson (Office of Health Economics), Sean D Sullivan (University of Washington) and Darius Lakdawalla (University of Southern California) – to explore the tension between affordability and future therapeutic development.

The session was framed around three key discussion areas:

  1. What are the key facts about the impact of price setting on pharmaceutical innovation?
  2. Why are those key facts so difficult to convey to policy makers?
  3. International reference pricing – what is it trying to solve, and what are some better alternatives?

The panel examined how policies like the US Inflation Reduction Act (IRA) and proposals for international reference pricing may shift investment behavior – particularly for high-risk, early-stage and small-molecule R&D. Panelists emphasized that while drug pricing reform is politically popular and often driven by public demand, it can have long-term effects on innovation. The session concluded with a call for evidence-based policymaking and for the HEOR community to play a highly active role in shaping future policy.

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

Spotlight Session: Medicare Price Negotiation of Part B Drugs: Implications for Provider Reimbursement and Commercial Spillover

Moderated by Sean D Sullivan (University of Washington), the Day 1 Spotlight Session examined the expanding scope of Medicare drug price negotiations under the IRA, focusing on the forthcoming inclusion of Part B drugs (which include high-cost biologics and other physician-administered drugs) and the far-reaching implications across the healthcare ecosystem.

Sullivan opened the session with an overview of the IRA’s phased implementation, outlining how price negotiation began with Part D drugs and will soon expand to include Part B therapies, beginning with the third negotiation cycle. He highlighted the statutory distinctions between the reimbursement models: while Part D relies on negotiated plan prices, Part B drugs are reimbursed based on the Average Sales Price (ASP) plus 6%. This fundamental difference creates added complexity, particularly around the selection of negotiation-eligible drugs and operationalizing maximum fair prices (MFPs). Sullivan also flagged the evolving debate in Congress over the so-called ‘pill penalty’ versus ‘biologic bonus,’ referring to the differing timelines for small molecules and biologics to become negotiation-eligible.

Kristi Martin (formerly Chief of Staff, CMS; now at Camber Collective) delved into the draft guidance for the third negotiation cycle, IPAY 2028, which includes up to 20 drugs from both Part B and D. Martin emphasized the introduction of two new policy areas: the inclusion of Part B drugs and the process for renegotiation. She explained that CMS maintained a qualitative framework for determining MFPs, though it is now requesting comments on how negotiation factors should be weighted – a move that raised questions, given that the agency has not previously disclosed how the factors were originally weighted. Martin noted the continuation of public engagement mechanisms such as patient-focused roundtables and the need to address complex data issues, especially around the challenge of applying 30-day supply standards to infused therapies.

Ramesh Srinivasan (McKesson) discussed five main implications stemming from Part B negotiations. These included operational uncertainties in effectuating MFPs, margin pressures for both manufacturers and providers, potential commercial spillover due to ASP-linked reimbursement, and payer challenges with cross-benefit management. He explained that providers reimbursed at MFP + 6% could see lower margins if MFPs fall below ASPs, with ripple effects into the commercial sector where ASP is often used as a benchmark. Srinivasan also warned of downstream effects on biosimilar development, citing Humira as a case where biosimilars have struggled to gain market share despite steep discounts. He concluded that while CMS has requested comments on operational models for implementing MFPs in Part B, no detailed framework has yet been proposed, which raises concerns about data lags, cash flow burdens, and the diverse landscape of provider reimbursement pathways.

In the Q&A, the possibility of including international reference pricing (IRP) in the negotiation framework was explored, referencing the much-talked about recent executive order. Martin responded that statutory constraints limit the negotiation factors to US market data, and any move toward IRP would likely require new rulemaking under the Innovation Center’s authority. Audience questions raised further concerns about biosimilar viability, weighting transparency, and challenges in managing dual-eligible products covered under both Part B and D.

Concurrent Sessions

Attendees of ISPOR 2025 had the opportunity to hear from a range of speakers in the concurrent sessions, including issue panels, workshops, forums, and other breakout sessions.

Issue Panel: What’s Next? Year 1 Learnings of Evidence Planning for IRA Drug Price Negotiation

Underscoring a recurring theme at this year’s conference, this Issue Panel brought together experts to reflect on the first year of Medicare drug price negotiation under the IRA. The session was moderated by Taylor T Schwartz (Avalere Health), with insights from Peter Neumann (Tufts Medical Center), Michael Ciarametaro (Avalere Health), and Russ Montgomery (GSK US).

Ciarametaro began the discussion with an overview of the initial MFPs released in 2024 for the first ten selected Part D drugs. He outlined the key evidence components required in the negotiation process, noting that many MFPs were near to their ceiling prices, shaped largely by each product’s economic value proposition and the selected therapeutic alternatives. He also highlighted the impact of CMS’s relatively narrow comparator selection and flagged potential implications for future negotiations.

Montgomery addressed manufacturer perspectives, underscoring the uncertainty around how CMS uses submitted evidence. He stressed the importance of early planning and embedding IRA considerations into wider evidence generation strategies. Emphasis was placed on high-quality comparative data and Medicare-relevant outcomes, particularly for real-world evidence (RWE). He encouraged manufacturers to proactively shape their value narratives, not only within the formal evidence submission but also during the critical post-submission interactions with CMS that occur before the agency issues its initial pricing offer. Montgomery also highlighted the need for manufacturers to clearly tell their value story and reinforce key messages throughout their submissions, ensuring those points stand out amid the large volume of information CMS must review.

Neumann discussed the challenge of interpreting CMS’s rationale documents, which were heavily redacted. He emphasized the complexity CMS faced: “Even if you got all the CMS negotiators in a room… they'd be hard pressed to really tell you [how they weighed the evidence], because Congress gave them a very difficult task here.” He added that demonstrating methodological rigor is essential when submitting RWE.

Neumann also touched on the role of cost-effectiveness analysis. While the use of the quality-adjusted life year (QALY) measure is restricted under the IRA, the panel agreed that broader value-based arguments, such as cost offsets, downstream savings, and productivity impacts, remain relevant, and can be incorporated into submissions.

Audience questions raised the issue of what types of CMS decisions might incentivize manufacturers to invest in head-to-head comparative studies, given ongoing uncertainty around how such evidence is evaluated. Panelists acknowledged that while the direct impact of these studies remains unclear, CMS’s emphasis on comparative effectiveness throughout its guidance suggests they could be influential. They advised manufacturers to start planning early and to incorporate comparative studies into a broader, forward-looking evidence strategy as the negotiation framework continues to evolve.

Workshop: Global Impact of JCA: Evaluating the Implications of the New EU HTA Regulation

Five months into the implementation of the European Union's Health Technology Assessment Regulation (EU HTAR), this session examined the potential impact of the EU-mandated joint clinical assessments (JCAs) on health technology assessment (HTA) systems beyond the EU. Moderator Danny Yeh (Aesara) framed the discussion by asking whether the JCA’s global impact would be, “a ripple, a wave or a tsunami,” and emphasized that its goal is, “to improve patient access and also enhance the quality of HTA assessment.”

Brian O’Rourke (Brian O'Rourke Health Care Consulting, Inc.) called the HTA regulation, “the most influential change we've seen in the HTA community, probably since we started doing HTA,” but also acknowledged, “a lot of trepidation… about how is this going to work.” He noted that countries are closely watching the outcomes of early JCA procedures for oncology and ATMPs, launched in January 2025. O’Rourke added,

“Lots more harmonization, lots of collaboration. That is the new direction, I believe, for the HTA community.”

Anouchka Vidal (Roche) outlined industry concerns, including global development constraints, country-specific evidence demands, and, “the number of PICOs,” stating, “If we want EU HTA to be successful, it needs to be manageable from both sides.” Roche’s survey of 13 non-EU countries and the US revealed interest in JCA principles, such as “robust methodology” and “avoidance of duplication”, even where awareness was low.

Federico Augustovski (Institute for Clinical Effectiveness and Health Policy) highlighted the potential for regional adaptation, noting that, “changes… take a lot of time to happen,” and that, “in many of Latin American countries, there is still a lot of room for improvement.” He cited regional initiatives like RedETSA and BRISA, suggesting that JCA could inspire similar harmonization within and across Latin American systems. All panelists agreed that growing collaboration and methodological convergence will shape the next phase of global HTA evolution.

Workshop: What Causal Inference Teaches Us About the Limitations of Indirect Treatment Comparisons for Health Technology Assessment

This workshop explored how principles of causal inference can improve the validity of indirect treatment comparisons (ITCs) used in HTA. Moderated by Uwe Siebert (UMIT TIROL, Harvard Chan School of Public Health), the session brought together experts Arthur Chatton (Université Laval), Michael Webster-Clark (Wake Forest University) and Harlan Campbell (University of British Columbia).

Siebert opened by emphasizing that while direct head-to-head trials are the gold standard, they are often infeasible, making ITCs necessary but methodologically complex. He introduced causal diagrams, including directed acyclic graphs (DAGs), the target trial approach, and G-methods to frame how observational biases can be mitigated with careful design and analysis.

Chatton spoke next, providing a deep dive into causal estimators, explaining how propensity scores, g-computation and doubly robust methods can simulate counterfactual outcomes in single-arm trials. He stressed the importance of estimands, confounder adjustment and high-quality data in generating credible causal effects.

Webster-Clark shifted focus to external validity, dissecting how elements like population, comparator, index date, and effect measure scale influence generalizability. He highlighted that assumptions valid for internal validity do not always extend externally.

Campbell discussed examples showing how misalignment between study and target populations – or overlooking non-collapsibility – can distort ITC results. He advocated for aligning estimands with decision goals and adjusting for key prognostic variables to ensure interpretability in HTA contexts.

The session underscored that applying causal inference rigorously is essential not only to strengthen the credibility of ITCs, but also to ensure they serve as reliable inputs for healthcare decision-making.

ISPOR Good Practices Task Force on Patient-Reported Outcomes (PROs) in Prospective Real-World Studies: Preliminary Recommendations

Moderated by Konrad Maruszczyk (University of Birmingham), this ISPOR 2025 Forum session explored emerging recommendations and practical challenges in the evolving field of real-world evidence (RWE) generation – specifically, the use of patient-reported outcomes (PROs) in prospective real-world (RW) studies. Maruszczyk provided background on the ISPOR Good Practices Task Force, which was initiated by the ISPOR Clinical Outcomes Assessment Special Interest Group in collaboration with researchers at the University of Birmingham, in response to the lack of formal guidance in this area.

Maruszczyk explained that the task force defined RW-PRO studies as those employing prospective designs, such as pragmatic trials and registries, focused on collecting patient-level data. Its work centers on key areas including study objectives, patient involvement, population selection, data quality, implementation, and regulatory and HTA engagement.

Tom Keeley (GSK) offered an industry perspective, acknowledging both the potential and ongoing uncertainty surrounding the use of RW-PROs. He described their applications prior to pivotal trials, for informing endpoint selection and supporting instrument validation, and following trial completion, including their role in post-marketing commitments, economic models, and enriching the patient experience narrative. He presented a case study on mepolizumab, illustrating how prospective PRO data from multiple countries helped strengthen the product’s value messaging. Keeley concluded by emphasizing that the true value of RW-PROs will only be realized when such data are routinely collected in clinical practice.

Jessica Roydhouse (Menzies Institute for Medical Research, University of Tasmania), presenting remotely, outlined the task force’s preliminary recommendations. She highlighted the importance of engaging patients as true partners, selecting fit-for-purpose instruments, and ensuring a balance between data quality and feasibility. Key themes included inclusive study design, technology-enabled data capture, and reducing burden on participants and staff. Roydhouse noted that mode of data collection remains a topic of active discussion and welcomed stakeholder input.

Onyeka Illoh (US FDA) provided a regulatory perspective, underscoring the growing utility of RW PROs for regulatory decision-making, including label expansions and post-market commitments. She stressed the need for appropriate study design, rigorous methods, validated instruments, and proactive strategies to address bias and confounding. Through a hypothetical case study, Illoh illustrated how regulators evaluate PRO evidence, with particular focus on alignment with study objectives, baseline comparability, and interpretability of endpoints in non-randomized settings.

Workshop: HEOR Meets Investing—Why Are Banks and VCs Collaborating With Health Economists?

Moderated by Meng Li (Tufts Medical Center), the discussion spotlighted the role of health economists in venture capital (VC) and investment banking, particularly in navigating the high-risk, high-cost environment of drug development, where only 1–2% of compounds typically reach market approval.

Li began by outlining the biotech innovation process, emphasizing the substantial cost burden, often exceeding $1–2 billion per successful product when accounting for failed assets and opportunity costs. She explained how funding flows through various stages, from early public sector and angel investor involvement to venture capital, private equity, and public markets—with investment banks facilitating IPOs and M&A activity.

The panel featured Richard Xie (RA Capital Management), Melanie Whittington (Leerink Center for Pharmacoeconomics), and William Canestaro (PAC Ventures, University of Washington). Each shared how health economics can inform investment decisions and mitigate policy uncertainty.

Xie described his role in piloting applications of health economics to support investment modeling at RA Capital. He stressed the need for better integration of societal value into decision-making frameworks and highlighted how policy changes, especially the IRA, are prompting investors to reconsider risk.

Whittington underscored her center’s mission to bridge HEOR and finance, especially by raising awareness of policy risks, facilitating stakeholder engagement, and encouraging early-stage innovators to consider market access and value storytelling. She emphasized the importance of translating health outcomes into economic narratives that resonate with investors.

Canestaro reflected on his transition from health economics to VC, advocating for early, simple models to guide indication selection and investment prioritization. He noted that HTA frameworks, which are traditionally used post-approval, can act as a ‘compass’ earlier in the innovation process, helping investors evaluate comparative value and potential market impact.

In the Q&A, the panelists agreed that effective collaboration between health economists and investors requires adapting language, starting earlier in the development timeline, and aligning on the decisions at stake. They called for more systematic, transparent frameworks that integrate HEOR insights with financial modeling, particularly in an era of increasing regulatory and policy complexity.

Workshop: Bridging the Evidence Gap: Integrating Real-World Data With Randomized Controlled Trials

This session explored how integrating real-world data (RWD) with randomized controlled trials (RCTs) can help close evidence gaps by providing deeper insights into treatment effectiveness, supporting broader population applicability, and improving decision-making. Moderator Mary Beth Ritchey (CERobs Consulting) opened the discussion by noting:

“We know that RCTs are the gold standard for doing data collection and generating evidence. However, sometimes they're just not enough.”

Emilie Toresson Grip (Quantify Research) presented a Swedish case study showing how registry data can be matched with clinical trial data to create an external control arm in acute myeloid leukemia. She noted, “the aim for this study was then to use the comparator arm… and match that intensified standard of care in the Swedish context, utilizing a RWD source,” and highlighted that, “we had seven times more patients that were reliable for inclusion compared to the trial,” demonstrating how high-quality Nordic RWD can enhance evidence generation and applicability.

Fei Fei Liu (Bristol Myers Squibb) shared practical lessons from industry, stating that, “you are, finally, able to put that in your statistics program and run your output,” only after months of multidisciplinary effort. She stressed the importance of planning ahead, noting, “do your data model, first and foremost,” and highlighted data quality and matching challenges.

Kirk Geale (Quantify Research) presented methods for integrating RWD with RCTs to extend trial results to real-world populations, using a case study on anticoagulation during percutaneous coronary intervention. Emphasizing the value of this approach, he stated, “I think we should almost always be trying to extend inference as best we can from trials to target populations.”

Educational Symposia: Bringing Us Together or Pushing Us Apart: Will JCA, HEMA, and Other Cross-Border Collaboration Initiatives Improve Patient Access?

Moderated by Erika Wissinger (Cencora), this session explored whether JCA, the Health Economics Methods Advisory (HEMA) collaboration, and other international initiatives can improve patient access to therapies by addressing fragmentation in HTA across borders.

Wissinger opened with an overview of three core aims of collaboration: sharing workload (e.g., through JCAs), sharing methods (e.g., via HEMA), and sharing intelligence (e.g., through joint horizon scanning initiatives). She emphasized the potential of harmonized processes to accelerate access and reduce duplication, but acknowledged that local economic assessments and pricing decisions remain outside these collaborations.

Michael Drummond (University of York) focused on JCAs as a tool for ‘levelling up’ HTA capabilities across the EU. He discussed challenges such as heterogeneity in standard of care and preferences for clinical and patient-reported outcomes across jurisdictions. While JCAs reduce redundancy in evidence generation, he cautioned that their impact on access may be limited due to persistent national differences in willingness to pay and local data requirements.

Jon Campbell (National Pharmaceutical Council) discussed method sharing, emphasizing that HTA relies not only on rigorous science but also on contextual judgment and transparency. He highlighted HEMA’s work to define best practices in HTA methodology and urged stakeholders to recognize how values and funding sources shape assessments. He concluded that HTA methods should be seen as tools not rules and must be applied with nuance.

Eldon Spackman (University of Calgary) provided a Canadian perspective, describing how inter-provincial and international collaborations, such as those between CDA, NICE and ICER, optimize resources without eliminating local complexity. He advocated for collaborative modelling efforts while maintaining critical thought.

A final audience poll echoed cautious optimism: while cross-border HTA efforts may not transform patient access overnight, they are viewed as a meaningful step forward.

Other Breakout Session: Real-World Life-Cycle Evaluation for Precision Medicine: From Conceptualization to Successful Implementation

Advances in precision oncology challenge traditional regulatory and reimbursement models due to small patient populations and lack of randomized comparisons. Dean Regier (BC Cancer) introduced a life-cycle HTA framework from BC Cancer’s PREDICT program to address this uncertainty through ongoing RWE generation. “Because of small benefiting populations... we need data from around the world,” he said. “What we want to do is we want to standardize normal study design, data and causal analysis to guide the appraisal, reappraisal... and adoption.”

Deirdre Weymann (BC Cancer) shared a case study using target trial emulation to assess entrectinib’s effectiveness by combining single-arm trial data with RWD. “How do we go about using RWD to generate causal evidence for us to understand how well this treatment works?” she asked.

“We go through all these different steps, and it turns out, when we do this really carefully... we’re able to mitigate observational study bias.”

Blythe Adamson (Flatiron Health) discussed the international transportability of RWE, presenting findings from the Flatiron FORUM (Fostering Oncology RWE Uses and Methods) consortium. She stated,

“What I hear when I engage with different HTA bodies and regulatory bodies is that there's always a preference for local data,”

but noted that this data is often insufficient. In studies of breast cancer, she reported “very strong evidence of transportability of survival... between the US and Denmark.” However, she emphasized that treatment context matters:

“It's not just the first treatments that patients receive... but also, what is the sequence of treatments that patients receive, and are those similar between two countries?”

She pointed to multiple myeloma as an example, where “the number one drug... in Germany is something that is very rarely used in the US,” illustrating when cross-country comparisons may not be valid.

Emanuel Krebs (BC Cancer) presented a life cycle economic evaluation of entrectinib versus standard care, showing how cost-effectiveness evolves as new RWD are integrated. “We propose... life cycle economic evaluation,” he said, warning that, “without being able to implement this... we're going to face... incredibly big challenges to the long-term sustainability of our healthcare systems.” 

Other ISPOR 2025 sessions

Continuing the offering from previous conferences, ISPOR continues to offer the popular 'Digital Conference Pass,' granting on-demand access to recordings of most educational sessions, plenaries, spotlight talks, and other content. As we could not attend all the sessions in person, we will be catching up on-demand from Day 1, including some key presentations that we missed.

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