Skip to main content
Search
Search
Advanced Search
Skip main navigation
The Evidence Base Post

ISPOR Europe 2024 daily round-ups: Day 1

  • Joanne Walker, Katie McCool & Laura Dormer

The much-anticipated ISPOR Europe 2024 conference, organized by ISPOR—The Professional Society for Health Economics and Outcomes Research, taking place in Barcelona, Spain, from November 17–20, 2024. The Evidence Base team is on site, bringing you comprehensive coverage, including daily round-ups and key insights from the plenary sessions. Here, we share highlights from Day 1.

Welcome Remarks 

Rob Abbott (CEO & Executive Director, ISPOR) opened ISPOR Europe 2024 by welcoming over 6300 delegates from 120+ countries to Barcelona, making it ISPOR's largest conference yet. He recognized the hard work of all the ISPOR staff as well as the the three program chairs: Yannis Natsis (European Social Insurance Platform [ESIP]), Patrice Verpillat (European Medicines Agency) and Anne Willemesen (Dutch National Healthcare Institute).

Abbott likened the evolution of health economics and outcomes research (HEOR) to the building of the Sagrada Familia—a project requiring time, collaboration, and gradual appreciation. Although HEOR is still not fully appreciated as a discipline, Abbott highlighted its journey to addressing critical healthcare challenges like affordability and complexity.

Abbott noted ISPOR's history in engaging specific stakeholders, such as researchers and healthcare providers, and emphasized the need to broaden focus to emerging stakeholders like regulators, payers, investors, and tech companies. He underscored ISPOR's new strategy: leading the definition and application of healthcare value in decision-making, and being a trusted authority influencing health policy globally.

He called on delegates to "move the needle" in their countries by applying science intentionally and communicating its relevance, impact, and value. Abbott urged all attendees to engage with stakeholders to make healthcare effective, affordable, and accessible. He concluded by empowering delegates to realize, "This is our time."

Keynote Address – Translating from EU to Africa to Empower Patient Involvement and Access to Healthcare

Rob introduced Mimi Choon-Quinones of Partners For Patients NGO, who delivered a keynote on translating healthcare strategies from the EU to Africa, focusing on patient involvement and access. Mimi highlighted that healthcare costs remain a significant burden for many in Africa and Europe, sharing insights from her 30+ years in the field.

Each year, Africa loses 227 million years of healthy life, according to WHO. In September 2023, the NGO was chosen by the Pan African Parliament of the African Union to co-create a strategy for patient access across Africa, aiming to accelerate health equity by 27 years over the next decade. The NGO’s three goals are to improve skills shortages in decision-making, generate evidence, and harness the power of partnerships.

The NGO is also collaborating with the Pan African Parliament on, “Africa’s Beating Cancer Plan,” inspired by Europe's initiative. Over its 34-year history, the 100% volunteer-based NGO has served over 100,000 patients and caregivers, trained 3000 doctors and students, and worked with over 1000 volunteers. This year, they launched an e-medical school in Africa and are establishing six cancer centers in Ghana. Starting in 2025, the NGO will explore funding models to support future activities. She concluding by stating:

“I look forward to continuing our fight to accelerate equitable access to medicines for all patients, no matter where they live.”

Plenary 1 – The Evidence-Price Conundrum: What Is the Way Forward for Patient Access?

The opening plenary of the ISPOR Europe 2024 conference tackled one of the most pressing challenges in healthcare today: how to balance the need for robust evidence, fair pricing, and equitable patient access. Moderated by Yannis Natsis, the session brought together leading experts—Valerie Paris (OECD), Pedro Facon (Belgium's National Institute for Health and Disability Insurance), Birgitte Klindt Poulsen (Danish Medicines Council), and Richard Bergstrom (IQVIA)—to explore the tensions between accelerated drug approvals, rising pharmaceutical costs, and the sustainability of healthcare systems.

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

Spotlight Session – Fit-for-Purpose Real-World Data: Principles and Developments 

Sebastian Schneeweiss (Brigham and Women's Hospital, Harvard University) introduced the spotlight session on data quality for causal inference. He emphasized that data quality is crucial for generating reliable evidence, which healthcare decision-makers demand. Obstacles to causal inference in clinical studies include selection bias, information bias (misclassification of exposure, confounders, and outcomes), and confounding. Schneeweiss introduced the concept of a "data quality map" to help identify these biases and outlined various methods for assessing data quality, such as data curation, validation studies, and quantitative bias analysis.

Jeremy Rassen (Aetion, Inc.) expanded on Schneeweiss's discussion, focusing on leveraging claims data for clinical event identification and validation. He used GLP-1 medications as a case study, illustrating a stepwise approach to validating outcomes: assessing data fitness, identifying outcomes, and validating observed results. Rassen explained that machine learning (ML) and large language models (LLMs) can help identify additional outcomes beyond those initially considered by investigators, enhancing data-driven outcome stratification. He also underscored the importance of validating outcomes using claims histories, especially with new FDA guidance on the role of real-world data (RWD) from electronic health records (EHRs) or medical claims in regulatory decision-making emphasizing the need for outcome validation.

Next, Daniel Prieto-Alhambra (Oxford University) discussed the concept of "fit-for-purpose" data and the importance of phenotyping standards in federated European RWD. He highlighted the importance of data feasibility, capacity, and validity while discussing European Health Data & Evidence Network (EHDEN), a European initiative focused on harmonizing and standardizing data from various sources, such as EHRs and claims data, by mapping them to a common data model (CDM). To date, EHDEN has mapped data from 30 countries, involving 388 million patients. Phenotyping in this context involves identifying patients with specific conditions or characteristics, but it is complicated by different coding systems used across European countries. Prieto-Alhambra emphasized the need for mapping these data to a unified standard, such as the OMOP CDM, and the use of federated analytics in initiatives like EHDEN to enable efficient data access and analysis.

Building on his presentation from the ISPOR Real-World Evidence Summit 2024, held the day before, Prieto-Alhambra also introduced DARWIN EU, the platform supporting reliable and traceable phenotype generation using RWD. He mentioned new tools developed under DARWIN EU for patient-level characterization studies, which aim to standardize phenotyping processes. A recent publication presents a reproducible framework for generating phenotypes based on RWD for use within DARWIN EU, further enhancing the reliability of data analysis in Europe.

Concurrent Sessions

Attendees of ISPOR Europe 2024 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 – Empowering Patients to Be Effective Stakeholders and Partners in the EU HTA Process

Experts and advocates discussed initiatives to prepare patients for meaningful involvement in the European health technology assessment regulation (EU HTAR). Moderated by Maria Dutarte, EUPATI Executive Director, the panel featured Finn McCartney (EUPATI Project Coordinator), Valentina Strammiello (EPF Director of Strategic Initiatives), and Lotte Klim (EUPATI Fellow).

EUPATI, a patient-led educational initiative, empowers patients through resources on HTA, regulatory affairs, and digital health. McCartney introduced the HTA4Patients project, which includes e-learning courses, online training sessions, and HTA Ambassadors to prepare for the EU HTAR, effective in 2025. He highlighted the project’s accessible design: free resources, multilingual materials, and certification options for patients completing the program.

The session also reflected on ISPOR 2023, emphasizing patient education, the 360° HTA Patient Involvement study, as well as the EUCAPA initiative aimed at strengthening patient capacity. Strammiello further detailed the project’s tailored training formats—introductory, fast-track, and extended face-to-face—to help patients better understand HTA methodologies.

Klim noted challenges in developing resources due to the evolving regulation: “It was a moving target... we’ll revisit this in 2030 when all disease areas are included.” Strammiello highlighted the difficulty in engaging patients, citing awareness gaps: “It took 12 months to get 12 patients.” Both emphasized the need to bridge gaps between individual patients, national organizations, and European networks.

In response to an audience query about minimal patient consultation requirements in the regulation, Strammiello highlighted that, “There is a risk that patient engagement doesn’t happen at all, so we’re stressing early engagement and patient experience data.” Klim pointed out disparities in readiness across countries: “The challenge, especially in countries without a strong structure, will be building this up.”

Dutarte emphasized the importance of patient involvement in her interview with The Evidence Base, stating,

“Empowering patients with the necessary knowledge will enable them to contribute effectively, ensuring that the HTA process reflects the voices of those it serves.”

The panelists offered key recommendations to empower patient involvement, including:

  1. Strengthen networks: Connect with European organizations, stakeholder networks, and EUPATI national platforms.
  2. Build capabilities: Learn HTA fundamentals, the PICO framework, and access case studies and regulatory updates.
  3. Engage patient organizations: Participate in consultations, training programs, and develop regulatory knowledge.

The panel concluded with a call for sustained training, collaboration, and evaluation. Strammiello emphasized inclusivity: “We shouldn’t just target an elite of patient advocates who can make time for this,” and urged stakeholders to, “focus on the practical aspects,” including connecting with HTA bodies and establishing clear communication channels. Dutarte summed up the message: “The work must continue.” The session highlighted the importance of empowering patients to contribute meaningfully to HTA processes while addressing challenges of inclusivity, awareness, and sustainability.

Learn more about the pivotal role patients can play in shaping the new EU HTA process in our interview with Maria Dutarte.

Issue Panel – Will Europe’s Pharmaceutical Policy and HTA Stakeholders Respond to Medicare Price Negotiation

In this Issue Panel, Sean Sullivan (CHOICE Institute, University of Washington) moderated a discussion on the ripple effects of the US Drug Price Negotiation Program, introduced under the Inflation Reduction Act (IRA), on European healthcare systems and pharmaceutical policy. Sullivan was joined by Inma Hernandez (University of California San Diego), Ansgar Hebborn (F. Hoffmann-La-Roche AG) and Jens Grueger (Boston Consulting Group).

Sullivan outlined the IRA's provisions, including drug price negotiations for Medicare, aimed at reducing spending and improving affordability for patients. He explained that the DPNP would be implemented in phases, beginning with the first 10 drugs identified for negotiation, which represent a significant portion of Medicare's spending on prescription medications. Hernandez then provided insights from the initial negotiations, highlighting several key findings: the price ceiling set during negotiations was significant, which helped establish a benchmark for other pricing decisions; comparative effectiveness played an important role in determining fair prices, showing that evidence-based assessments were crucial for negotiations; and although the negotiation process generated only modest savings, it demonstrated the potential for future cost reductions. She also noted that while the outcomes were somewhat predictable, they did help to marginally reduce price disparities between the US and international markets, indicating progress towards more equitable pricing.

Grueger explored the DPNP's impact on global access strategies, drawing on interviews with senior executives from 11 major pharmaceutical companies. Findings revealed a shift in global portfolio decisions, with an increased focus on comparative effectiveness and real-world evidence (RWE). Companies have become more cautious about smaller indications, and there has been less interest in orphan drugs. Strong clinical evidence will play a larger role in differentiating products, especially in becoming 'first-in-class' or 'best-in-class'. The IRA was seen to reduce incentives for orphan drugs and smaller indications, potentially affecting innovation.

Hebborn concluded with the implications for European policy, questioning whether Europe contributes its fair share to global R&D. He pointed out differences in drug launches, with the US significantly ahead. The EU is working towards improving affordability, access, and availability through EU HTAR and pharmaceutical reform. However, the IRA has sparked concern in Europe, which may lead to a renewed focus on industrial strategy and the need to better understand the societal value of innovative medicines, tackle disparities in healthcare access, and support sustainable pricing models.

Issue Panel – Harnessing the Power of AI in RWD: Enhanced Insights Through Machine Learning and Causal Inference Advances

The multistakeholder panel, moderated by Katja Hakkarinen (Parexel), provided a rich exploration of the evolving intersection between technology, methodology, and regulation in healthcare. Featuring insights from Patrice Verpillat (European Medicines Agency [EMA]), Andy Wilson (Parexel), and Ipek Özer Stillman (Takeda), the discussion highlighted key regulatory, epidemiological, statistical, and industry perspectives.

From a regulatory perspective, Verpillat emphasized the critical role of AI in enhancing regulatory processes and evidence generation. He introduced the Reflection Paper on the use of AI in the medicinal product lifecycle, published by the European Medicines Regulatory Network in September 2024. This document underscores how AI and ML can, "support the acquisition, transformation, and interpretation of data." Verpillat stressed the importance of transparency and early collaboration, encouraging open dialogue with regulators: "Engage us early. Please come talk to us... there’s no downside." He also highlighted initiatives such as the EMA's Digital Academy, Big Data Steering Group, and Digital Innovation Lab, which aim to upskill stakeholders and foster innovation.

Katja Hakkarinen provided an epidemiological lens, focusing on causal inference methods and the increasingly popular target trial emulation (TTE). She explained how TTE addresses biases, such as prevalent user bias and immortal time bias, which can otherwise distort real-world study outcomes. While TTE holds promise, Hakkarinen noted that, "many studies are still ongoing," and emphasized the need for deeper expertise in bias mitigation and causal methodologies.

Andy Wilson spoke about bridging methodological gaps from a statistical perspective, underscoring the importance of causal methods in RWD research. He argued that traditional approaches fall short of addressing the "causal gap" inherent in real-world studies. Wilson discussed two dominant frameworks: the Neyman-Rubin counterfactual framework and the Pearl-graphical causal structural framework. He mentioned that methods like targeted maximum likelihood estimation (TMLE) and ensemble approaches, such as super learners, show promise in addressing methodological challenges. He concluded by advocating for uniting diverse approaches, stating, "Causal assumptions don’t live in the data or the method, they live in the verdict."

Ipek Özer Stillman offered the manufacturer's perspective, focusing on strategic evidence generation and the role of AI in optimizing trial designs to meet evolving regulatory demands, including the EU HTAR and pharmaceutical reforms. Stillman highlighted the dual importance of protocol optimization (e.g., eligibility criteria and feasibility analysis) and early decision-making to minimize costs and improve patient outcomes. She emphasized: "Real-world data provides a window to how patients respond to treatments in everyday settings." Stillman also called for cross-disciplinary collaboration, remarking, "Putting it all together will take literally a village."

The audience questions addressed expanding collaborations to include other disciplines, such as engineering and astronomy, and ensuring transparency when AI/ML is used in internal processes. Verpillat noted the importance of continuous updates to the EMA’s AI work plan, reflecting the fast-evolving nature of the field: "It’s ambitious, but we’re revising it regularly. It’s not fixed forever."

The session underscored the immense potential of AI in RWE generation while recognizing the challenges of methodological rigor, bias mitigation, and ethical use. Transparency, early engagement with regulators, and interdisciplinary collaboration emerged as critical success factors. As Verpillat aptly summarized: "Transparency and early dialogue are key to building trust in these fast-evolving methodologies."

Educational Symposium – Ready for 2025? How Lessons Learned From National System Reforms Can Guide Our Response to the New EU HTA Regulations

The Educational Symposium, moderated by Herbert Altmann (Cencora), featured industry experts discussing the readiness of European countries for the new EU HTAR. The regulation aims to improve patients' access, transparency, and resource efficiency in healthcare across Europe. He was joined by Max Brosa (formerly Managing Director, Pharmalex), Iga Lipska (Medical University of Gdańsk), François Meyer (FIPRA Public Affairs), Oriol Solà-Morales (Health Innovation Technology Transfer), and Mihai Rotaru (European Federation of Pharmaceutical Industries and Associations [EFPIA]).

Altmann provided an overview of Germany's readiness. Organizations like Federal Joint Committee (G-BA) and Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) have been involved in developing guiding principles for joint clinical assessments (JCA) and joint scientific consultations (JSC), supporting harmonization across the EU. While Germany does not expect significant changes, there are concerns about the fast timelines and the impact on orphan drug regulations if JCA reports are delayed. The industry is seeking more involvement and additional consultation opportunities.

François Meyer presented the French perspective, noting that HTA is seen positively as a tool to improve access to new technologies. The French HTA body, Haute Autorité de Santé (HAS), has been highly involved in HTAR activities, and the new process is not expected to bring major changes. France already has a structured early access program, with a focus on evaluating innovation appropriately. Some adjustments include restrictions on requesting data already submitted for JCA and maintaining national early dialogue if the product is not selected for JSC.

Oriol Solà-Morales discussed Spain's progress, noting that authorities have developed a new legal framework expected to be implemented by the end of 2024. Spain's approach is budget-impact focused rather than cost-effectiveness, and companies face challenges understanding the implications of the process at the local level. He emphasized the need for clear communication to help drug developers navigate the changes.

Iga Lipska offered a less optimistic view from Poland, highlighting the challenges for Central and Eastern European (CEE) countries. Poland is not fully prepared for HTAR, but it presents an opportunity to leverage EU-level expertise. CEE countries have diverse healthcare systems and limited financial and human resources, making HTAR a lower priority. Lipska stressed the importance of improving patient access to vital medicines.

Mihai Rotaru highlighted the broader European perspective, noting that while challenges remain, HTAR offers the potential for a more streamlined access environment across the EU, aiming to strengthen evidence generation. Max Brosa added that small- and medium-sized companies face capacity challenges in meeting the tight timelines required for JCA, and there are uncertainties about how local authorities will implement JCA outcomes.

The panel concluded by discussing patient and clinician involvement. Meyer noted the complexities in involving patients and clinicians at the EU level, while Lipska and Solà-Morales emphasized the need for well-structured, unbiased processes. Rotaru stressed the importance of patient-centricity and effective communication as key steps forward.

Hear more from these speakers in our upcoming Webinar:

Forum –  Data Analysis and Real-World Interrogation Network – European Union (DARWIN EU) – Where Are We Now and What Is in Store for the Future?

Moderator Phani Veeranki opened the discussion by introducing Patrice Verpillat (EMA), who explored the regulatory perspective; Shuvayu Sen (Merck & Co., Inc.), who provided an industry viewpoint; and Daniel Prieto-Alhambra (Oxford University), who shared insights on the maintenance and operational aspects of DARWIN EU®. The session focused on the vital role of RWD and RWE in healthcare decision-making, emphasizing DARWIN EU as a federated network designed to support regulatory decisions within the European Union.

DARWIN EU was described as a transparent initiative, with all protocols and reports made publicly available. “For us, what’s important is to be transparent with what we do,” Verpillat explained, adding that data remains local to preserve privacy. The network has already onboarded 20 data partners and aims to expand further by 2025. Highlighting DARWIN EU's foundational principles, Verpillat noted, “As a regulator, we will never tell you, ‘For that question, you must use this method.’ It can be quite diverse.” He emphasized that DARWIN EU is not meant to replace clinical trials but to complement them by addressing limitations such as smaller sample sizes and limited statistical power in certain studies.

Prieto-Alhambra outlined DARWIN EU’s use of the Observational Medical Outcomes Partnership (OMOP) common data model to ensure data interoperability and scalability. The coordination center has established a catalog of standardized tools for rapid and reproducible studies. “We don’t need to reinvent the wheel every time,” he said, explaining how standardized analytics streamline the process. He highlighted notable studies, including the prevalence of valproate use and the management of juvenile systemic lupus erythematosus, and acknowledged challenges such as the limitations of rare disease datasets:

"The dream of having very granular, very valid, and very big data at the same time is probably never going to happen… We need to live in a world where we work with the strengths and limitations of different datasets and make the best of what each can offer."

Sen emphasized the value of DARWIN EU for multi-stakeholder collaboration, transforming patients from information receivers to research contributors and enabling payers to transition toward funding value-based care. He likened DARWIN EU to the “Cat in the Hat,” stating it should be, “fun, inclusive, and collaborative.” Addressing potential future collaborations, he added,

“If the same data is used, then we have common currency when it comes to evaluation of it, so it becomes more consistent and rational and transparent.”

During the session, the speakers addressed questions about DARWIN EU’s integration with the European Health Data Space (EHDS) and its capacity to scale. Verpillat explained that integration is still premature, as lessons from a recent pilot study are being assessed. Looking ahead, he expressed the hope that DARWIN EU will eventually face “too many requests,” reflecting its success. Prieto-Alhambra emphasized the importance of public health impact, while Sen highlighted the need for consistency and transparency to support rational decision-making.

Issue Panel – Innovation or Stagnation? Unpacking Issues and Solutions in the Uptake of Advanced Methods for Real-World Evidence

The session examined the challenges and opportunities in adopting advanced methods for RWE. Stephen Duffield (National Institute for Health and Care Excellence [NICE]) led the discussion, focusing on the use of new methods in decision-making and barriers to their adoption. NICE is striving for more adaptable, timely guidance, including for digital health technologies, to address pressures like shared decision-making and data growth. Duffield also referenced the Sudlow Review, which highlighted the vast potential for data in the UK. Despite some success in methods such as multiple imputation, challenges remain for others like Bayesian borrowing. Duffield emphasized the need for practical, well-supported methods, transparency, and continued enhancements to guidelines, such as NICE’s RWE framework, which is a living document updated regularly.

Duffield highlighted ongoing gaps in adopting advanced methods such as TTE and quantitative bias analysis at NICE. Addressing these involves using pilot studies, enhancing frameworks, and increasing engagement with stakeholders. He also noted that AI-based methods present a particular challenge, with ongoing pilot projects like SYNTHIA exploring potential use.

Barbara Dickerman (Harvard TH Chan School of Public Health) discussed executing advanced methods like TTE, which helps reduce bias in observational studies and provides actionable evidence. She shared case examples of TTE in action, including a study to emulate a target trial of the comparative effectiveness of mRNA-based COVID-19 vaccines. Dickerman also outlined challenges in TTE, such as identifying appropriate data sources and feature extraction, along with potential solutions like leveraging existing phenotype libraries and designing data pipelines.

Grace Hsu (Cytel) provided insights from a consultancy perspective, emphasizing that advanced methods could support robust studies but face challenges in execution, communication, and establishing precedents. She highlighted Bayesian borrowing and quantitative bias analysis as tools that, while useful, face adoption hurdles due to complexities in data access, statistical plans, and stakeholder communication. ML and AI also pose questions about implementation, validation, and stakeholder engagement.

Jonas Haggstrom (Cytel) presented remotely on synthetic data, describing its potential for addressing privacy concerns, bridging data gaps, and complementing clinical trials. However, challenges include data validation, regulatory acceptance, and integration with real-world datasets. Haggstrom proposed frameworks for privacy, tools for data generation, and regulatory collaboration to overcome these obstacles, stressing the need for broader stakeholder engagement.

ISPOR’s 2030 Strategy

The ISPOR senior leadership team – including Brian O’Rourke (2024–2025 Past-President), Eberechukwu Onukwugha (2024–2025 President), Rob Abbott (CEO and Executive Director), Laura Pizzi (Chief Science Officer) and Mitchell Higashi (Associate Chief Science Officer) – provided an overview of ISPOR’s new strategic plan, launched at the US meeting in Atlanta. The plan emphasizes ISPOR’s vision of making healthcare accessible, effective, efficient and affordable for all.

Developed over 2 years, the strategy focuses on three key trends:

  • Whole health and the science of well-being
  • Better health in a digital world
  • Affordable health globally

The plan outlines two major goals:

  1. Lead the definition, measurement, and use of value for health decision making
  2. Be the trusted HEOR authority that leads change in health policy worldwide

These goals aim to expand the concept of value to include public health and well-being, making ISPOR more relevant to a wider range of stakeholders.

Central to the strategy's success is its implementation plan, which involves engaging stakeholders, improving policy responses, and leveraging technology. The leadership team emphasized the importance of transparency, equity, and patient engagement, with a vision of transforming healthcare decision-making globally.

On demand ISPOR Europe 2024 sessions

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. While we couldn't attend all the sessions in person, we'll be catching up on-demand from Day 1, including some key presentations that we missed.

Register for free today to become a member of The Evidence Base and receive the latest news straight to your inbox.