ISPOR Europe 2024 daily round-ups: Day 3

As ISPOR Europe 2024—the European conference for ISPOR, The Professional Society for Health Economics and Outcomes Research—comes to a close in Barcelona, Spain, we present our final round-up, summarizing key sessions and presentations from Day 3 of the conference. We extend our sincere thanks to ISPOR for welcoming The Evidence Base as a media partner for this event.

See coverage of Day 1 here See coverage of Day 2 here

Final Address

Rob Abbott highlighted the significance of the conference's closing plenary, describing it as, "tying a ribbon around everything." He formally thanked the conference program committee co-chair—Patrice Verpillat, Yannis Natsis and Anne Willemsen—for curating plenaries that, "really hit the mark in terms of content." Abbott also expressed gratitude to the larger program committee and his staff for working tirelessly behind the scenes.

Reflecting on the conference's theme, Abbott emphasized its focus on, "generating evidence towards health and well-being," highlighting ISPOR's core commitment to rigorous, unbiased scientific evidence generation. He praised the journey from the Real-World Evidence Summit to the third plenary, which aimed to build on the plenaries one and two by providing the regualtory viewpoint. Abbott closed by expressing his excitement for the journey ahead, challenging the community to continue advancing in evidence generation and communication.

He welcomed Laura Pizzi, ISPOR’s Chief Science Officer, who provided an update on ISPOR's science and health policy initiatives for 2024, emphasizing recent progress and future plans. Recently ISPOR has published several highly-cited Good Practices Reports, which are directly influencing global health policies and practices. Their recently published trends report covering topics like, real-world evidence (RWE), AI and patient centricity, provides a comprehensive overview of pivotal trends influencing global healthcare decision-making. Their taskforce pipeline is strong, with initiatives in generative AI, health equity, and more. ISPOR’s strategic plan aims to expand the definition of healthcare value, improve stakeholder relationships, and integrate patient perspectives—culminating in a patient-centered research track at ISPOR Europe 2025. Pizzi highlighted collaboration as key to achieving these goals.

Abbott closed ISPOR by inviting attendees to upcoming conferences – ISPOR 2025 in Montréal, Canada (May 13–16, 2025) and ISPOR Europe 2025 in Glasgow, Scotland (November 9–12, 2025). If you're planning for future ISPOR events, connect with Phil Garner to discover how The Evidence Base can support your communication efforts and help share your scientific research effectively.

Plenary 3 – Reality Check: Are We Bridging the Evidence Gaps for Patients?

Following the payer perspective in plenary one, and HTA viewpoint in plenary two, the third plenary rounded off ISPOR Europe 2024 with a focus on the regulatory landscape by asking, "Reality Check: Are We Bridging the Evidence Gaps for Patients?" Patrice Verpillat (European Medicines Agency) was joined by the previous plenary moderators, Yannis Natsis (European Social Insurance Platform) and Anne Willemsen (Dutch National Healthcare Institute), along with Nikos Dedes (Greek Patients' Association), Peter Mol (College ter Beoordeling van Geneesmiddelen) and Almath Spooner (AbbVie).

Spotlight Session – The Dawn of a New Era: Cross-Border Collaborations—Regional, Pan-European, and Transcontinental—How Will They Shape the Future of Access? 

Neil Grubert began by highlighting significant developments in cross-border collaboration, focusing on Europe’s evolving landscape through initiatives like EU HTAR. He pointed out that these collaborations are increasingly harmonizing efforts in HTA, drug pricing, and innovative therapies, contributing to a more cohesive global healthcare system. Key strategies include horizon scanning, sharing best practices, joint HTA, pricing negotiations, and unified procurement approaches.

At the regional level, collaborations primarily involve neighboring countries, with the Beneluxa Initiative an exception, involving Belgium, The Netherlands, Luxembourg, Austria, and Ireland. The Nordic Pharmaceutical Forum and Joint Nordic HTA Bodies also work closely with Beneluxa to enhance regional cooperation. Another notable collaboration is the Valletta Declaration Group, which focuses on information exchange and is expected to increase its activities. Additionally, the SUSTAIN-HTA initiative aims to support the alignment of HTA methodologies, involving key stakeholders such as NICE.

Pan-European collaborations further extend the reach of cross-border initiatives. The National Competent Authorities on Pricing and Reimbursement (NCAPR), supported by EU4Health and the European Commission, aims to enhance best practices and transparency. EURIPID, involving 28 countries, provides data on drug prices and managed entry agreements. The MEDEV network, comprising 22 national authorities, unites HTA agencies and payers to streamline assessments, while MoCA (Mechanism of Coordinated Access to Orphan Medicinal Products) improves access to therapies for rare diseases. The Novel Medicines Platform, including 53 countries, works on sharing best practices and joint procurement.

On a transcontinental level, Project Orbis and the Access Consortium are instrumental in accelerating drug approvals, while the AUS-CAN-NZ-UK Collaboration focuses on joint HTA collaboration. NICE International plays an active role in the Asia-Pacific region and Latin America, emphasizing the value of global partnerships in advancing healthcare collaboration.

In the ensuing discussion, Roisin Adams (NCPE) emphasized the potential of cross-border collaborations to pool resources, expertise, and manpower, while acknowledging resource limitations as a challenge. She highlighted the legal complexities, particularly in initiatives like Beneluxa, where national regulations dictate what is feasible. Adams noted that collaborations often occur between countries with similar systems to minimize “bumps”, but differences provide valuable learning opportunities to understand shared challenges.

Johan Ponten (TLV) reflected on the Nordic experience, likening its collaborative spirit to the resilience of the Vikings. The Joint Nordic HTA Bodies (formerly FINOSE) emerged from necessity and were shaped by national authorities, granting flexibility but also raising legal concerns. On procurement, countries independently decide, guided by advice from Nordic collaborations. Ponten stressed pragmatism, focusing on shared goals and leveraging minimal common ground, a lesson he suggested could benefit broader EU efforts.

Meindert Boysen (HTAi Global Policy Forum) shared insights from NICE, renowned globally for its influence on policies. International collaboration enables NICE to both share and gain knowledge. He also discussed the AUS-CAN-NZ-UK Collaboration, launched in 2021 to address COVID-19 challenges through horizon scanning, and now focused on regulatory frameworks, digital health, and advanced methodologies.

On the perception by industry of these collaborations, Adams noted mixed reactions, particularly from larger companies hesitant about the value of collaborations like Beneluxa’s ATMP focus. Ponten and Boysen agreed that greater joint applications and partnerships with pharma are crucial to reduce bureaucratic burdens and accelerate patient access to treatments, a shared goal across all initiatives.

Concurrent Sessions

Wrapping up Day 3 of ISPOR Europe 2024, a series of breakout sessions offered additional learning opportunities. These sessions tackled gaps in clinical outcomes assessment in real-world studies, introduced innovative methods in HEOR, examined the global impact of recent legislative changes in the US and Europe, and proposed solutions for challenges faced in RWE research.

Issue Panel – Giving Voice to Rare Disease During Joint Clinical Assessment (JCA)

As the roll-out of Joint Clinical Assessment (JCA) in the EU approaches, with JCA for oncology and ATMP products (which will include some rare diseases) beginning in January 2025 and for orphan designation products coming into force in 2028, stakeholders across the healthcare landscape are grappling with the unique challenges it poses. This panel discussion explored these issues, focusing on how to prepare in the context of rare disease therapies, where low patient numbers, heterogenous populations, and diverse standards of care present significant hurdles.

Moderated by Caroline Ling (RTI Health Solutions), the session brought together experts with diverse perspectives, including: Abigail Stevenson (Chiesi), Emma Hawe (RTI Health Solutions) and Martin Visnansky (Masaryk University Brno). Together, they explored how stakeholders can proactively address the complexities of evidence generation, given the tight timeframes of the JCA process, and ensure that the voices of rare disease patients are heard.

Hawe kicked off the discussion with insights on evidence-generation strategies in rare diseases. She highlighted relevant methodologies, including indirect treatment comparisons (ITCs) and the use of external control arms, to fill evidence gaps. In rare disease contexts, such pragmatic approaches are key to demonstrating value when clinical trial data alone may be insufficient. However, Hawe noted that some of the JCA statements relating to, for example, ITCs, show a level of caution regarding their use, which may prove challenging for rare disease assessments.

Stevenson followed by providing the industry perspective, addressing the challenges associated with collecting robust data across the 27 EU member states – a particular burden for small- to mid-sized companies. She emphasized the importance of adapting evidence-generation practices to account for the unique attributes of rare and ultra-rare diseases with their inherent clinical uncertainty and limited datasets. These factors, including the lack of rare disease-specific processes within the JCA, create difficulties, requiring early and collaborative approaches across cross-functional teams to meet JCA's requirements. Stevenson also highlighted opportunities, including the potential for efficiency and alignment across the EU and all stakeholders.

The conversation also underscored the need to prioritize patient voices. Visnansky, representing the patient and caregiver perspective, stressed the significance of ensuring patients' needs are at the forefront of JCA evaluations. Rare disease patients often face long waits for new treatments, and the urgency to consider patient-relevant outcomes was emphasized.

Workshop – Equity in HTA: Exploring Methods and Their Role in Decision-Making

Chaired by Katharine Cresswell (NICE), this session explored integrating equity into HTA. Health equity, defined by the WHO, aims to eliminate unfair and avoidable health differences across populations. While often overlooked in HTA, equity-focused methodologies are gaining traction. NICE’s 2021–2026 strategy prioritizes reducing inequalities, particularly those worsened by the pandemic, by examining disparities across various groups and factors. Cresswell highlighted the first HTA submission to the agency using distributional cost-effectiveness analysis (DCEA) to address inequalities, prompting NICE to release a position statement and plan manual updates by 2025. NICE is also piloting DCEA tools in guideline development for weight management and type 2 diabetes, supporting equitable healthcare outcomes.

Jeroen Janssen (Center for Translational and Policy Research on Precision Medicine [TRANSPERS]) walked attendees through the foundational principles and practical applications of DCEA. DCEA builds on traditional cost-effectiveness analysis by not only comparing the costs and benefits of new interventions versus standard care but also examining how QALYs gained and QALYs lost (health opportunity costs) are distributed across different societal groups. Using a model-based example, Janssen illustrated how DCEA quantifies health inequalities and informs equitable resource allocation. He also addressed implementation challenges, such as data requirements and complexity in estimating subgroup-specific treatment effects, underscoring DCEA’s role in equity-driven healthcare decision-making.

Melanie Whittington (Leerink Center for Pharmacoeconomics) highlighted challenges in conducting DCEA within HTA, particularly in the US context. Prior to 2024, key obstacles included limited availability of subgroup data, prevalence estimates, insights into opportunity costs, and defining an inequality aversion parameter. However, recent advancements, including new QALE data, have helped progress DCEA. Today, challenges are more practical and ethical, which Whittington explored alongside non-DCEA approaches like ICER’s Health Disparity Index (HIDI) for qualitative deliberation. She advocated for less formulaic and more qualitative use of DCEA.

The panelists concluded with a call for early collaboration and careful planning by pharmaceutical companies, regulators, and patient advocates. Clinical studies should be designed to meet evidence requirements and pathways created that give a voice to patients in every corner of the EU.

Issue Panel – Patient-Centered Research: Using Patient Experience Data Generated During Drug Development to Inform Healthcare Decision-Making: What Researchers Need to Know to Generate Fit-for-Purpose Data While Considering the Global Picture

The discussion focused on the evolving role of patient experience data (PED) and its integration into drug development and healthcare decision-making. Contributions from industry, regulators, HTAs, and patient representatives highlighted the opportunities and challenges in generating high-quality, fit-for-purpose PED.

Solange Corriol-Rohou (AstraZeneca) emphasized PED as, “data that are scientifically and robustly collected to inform medicines development with insights from patients.” She noted increasing engagement from the pharmaceutical industry, evidenced by the EFPIA’s May 2022 position paper on transparency in regulatory decision-making and an August 2023 survey that highlighted industry priorities. Key areas included integrating PED into marketing authorization applications, HTAs, and benefit-risk assessments. Corriol-Rohou also discussed the September 2022 EMA multi-stakeholder workshop and FDA-issued methodological guidance, as well as the ICH E22 concept paper, endorsed in June 2023, which outlines harmonized regulatory requirements for patient preference studies. “Involving patients in drug development is of real value,” she said, stressing the importance of collaboration across stakeholders.

Rosa Gonzalez-Quevedo (European Medicines Agency [EMA]) highlighted the importance of optimizing PED collection methods, data quality, and methodologies. PED, she explained, can be collected in both clinical trial and real-world settings, including post-approval phases as real-world data (RWD). Gonzalez-Quevedo announced that the EMA’s reflection paper on PED, set for public consultation in 2025, will define PED, address its use throughout the product lifecycle, and provide guidance for early engagement with regulators. She also noted the EMA’s efforts to increase transparency by updating the CHMP assessment report template to better reflect how PED is used in regulatory evaluations. “We see [PED] as an important contributor to the totality of evidence,” she stated.

Niklas Hedberg (Dental and Pharmaceutical Benefits Agency) discussed the upcoming EU-wide HTA regulation, emphasizing the importance of PED in joint clinical assessments. “We welcome patient experience data... but it must be of high quality,” he said. He highlighted Sweden’s example, where patient preferences are integrated into HTA frameworks, and underscored the need for continuous updates, stating, “Patient engagement is a journey... you will have to keep doing updates to keep up with what is golden standard.”

Julian Isla (Microsoft, COMP member, Dravet Syndrome European Federation, Foundation 29) presented an AI-powered tool, collaborare, that is designed to accelerate PED collection, particularly for rare diseases. “Conventional methods are time-consuming and costly,” he explained, emphasizing that the tool allows patient organizations to validate and refine AI-generated data, making PED collection more accessible. Isla concluded that these innovations could address key gaps in PED generation, particularly for underrepresented patient groups.

The session underscored the need for harmonized frameworks, early dialogue among stakeholders, innovative technologies, and robust methodologies to advance patient-centric drug development. As Corriol-Rohou aptly concluded, “The earlier, the better.”

Issue Panel – Take My Word for It: Can Patient-Centric Social Listening Enrich RWE?

The session explored the potential of social media listening (SML) to enhance real-world evidence (RWE), addressing its promise, challenges, and future directions.

Siddharth Ramanan (Costello Medical) opened the discussion by framing SML as a low-burden, high-impact approach at the intersection of Big Data, AI, and patient centricity. He highlighted its ability to provide a nuanced understanding of patient experiences, particularly in rare diseases where evidence generation is slow. “Patient views are often sought through highly structured methods—this only gives us a snapshot of the lived patient experience,” he said. SML offers a way to broaden this view by analyzing unstructured, real-time data from platforms like forums and social media. However, he challenges with data reliability, representativeness, and privacy, while noting the emergence of evidence frameworks for digital health technologies.

Jackie Cuyvers (Convosphere) described the mechanics and applications of SML, emphasizing its ability to uncover the patient journey and identify unmet needs globally. She explained the process of gathering data with Boolean queries, segmenting it, and analyzing it through a mix of AI tools and human review. By, “using social listening, you can truly understand the impact on patients’ quality of life,” she said, contrasting it with surveys limited by pre-set questions. Cuyvers noted that, “the average social listening project is less expensive than primary market research,” making it a cost-effective method to supplement traditional approaches. Despite its promise, she flagged challenges, including data quality, cultural nuances, and AI’s inability to interpret context such as sarcasm or irony.

Mary Jo Lamberti (Tufts University) shared findings from a Tufts study on AI and ML integration in clinical development, including SML for adverse event reporting. Adoption is in its early stages, but she highlighted its potential to reduce time and costs. Lamberti stressed that building trust in AI/ML requires, “more experience with successful implementations or use cases, clearly articulated ROI, and ethics, transparency, and explainability.” These are essential for scaling SML while addressing data ownership and protection concerns.

Jeanette Kusel discussed SML’s benefits for HTA, emphasizing its ability to capture diverse patient voices often excluded from traditional methods.

“We want well-designed studies that are capturing outcomes that are important to patients. If you don’t have the data on that, committees can’t look at it, and models can’t be designed around endpoints that are actually important to patients.”

Kusel acknowledged limitations, such as social media data’s unrepresentative nature, but emphasized SML’s value upstream in identifying patient priorities and downstream in assessing healthcare recommendations.

Audience polling and the Q&A reflected growing interest in SML, with 88% open to using it for RWE generation. However, the session underscored the need for clear frameworks and methodologies. Kusel concluded that SML’s future lies in aligning with principles of transparency, data suitability, and methodological rigor, making it a valuable supplementary tool for understanding patient experiences and supporting decision-making.

Issue Panel – Bridging Nations, Diverging Views: Exploring the Global Landscape of Indirect Treatment Comparisons Acceptance by Health Technology Assessment Bodies

ITCs, in the absence of head-to-head randomized trials, are a challenge to HTA decision-making. During this panel session, experts explored these challenges and discussed strategies for overcoming them. Moderated by Chris Cameron (EVERSANA), the panelists included Stefan Lange (Dierks+Company), who brought his perspective of his previous role at IQWiG; Olivia Wu (University of Glasgow), bringing her experience at NICE; and Nicholas Adlard (Novartis Pharmaceuticals UK), bringing the industry perspective.

The session highlighted the significant variation in how global HTA bodies evaluate and accept ITC methodologies, leading to inconsistent drug reimbursement decisions and delays in patient access to treatments. This inconsistency is further complicated by the evolving landscape of HTA processes, particularly with the introduction of the EU’s JCA.

Cameron kicked off the session by level-setting with an overview of ITC methodologies, prior to each panelist providing their perspective on the topic. Lange emphasized that multiple PICOs will be common in the future, making ITCs a necessity. There is therefore a need for specific expertise from all stakeholders. Of course, ITCs are also a challenge, as HTA bodies often have concerns over their use. Individual patient data (IPD) and excellent quality data will be key.

Wu continued by reminding the audience that ITCs have been in use for quite some time. She discussed the trade-offs between methodological limitations and decision-making by different HTA bodies, and echoed Lange in highlighting data quality as a key challenge.

Industry sponsors play a vital role in this dynamic. Adlard discussed the rapidly changing evidence landscape for HTA submissions, including the increasing use of indirect evidence, the increase in the number of comparators being considered, and pressures on randomization. He highlighted that it is not possible to please all the HTA agencies all the time, and that pharmaceutical companies need to be proactive in raising the standards of ITCs. He emphasized the necessity of transparency in methodology, and the importance of collaboration between pharma, HTA bodies and academia to reduce uncertainty.

During the subsequent panel discussion key topics were discussed, including the variation in comfort levels of different HTA bodies in dealing with uncertainty, with Adlard advocating for discussion to help standardize expectations, thus making submissions more straightforward for sponsors. Lange brought the discussion back to the data, and the importance of having the highest quality possible. Wu built on this by highlighting the importance of the evolution of trials, and the opportunities this will bring to provide the data quality needed.