ISPOR Europe 2024 – inside the third plenary: Balancing costs and evidence for affordable medicines and stronger EU HTA
ISPOR Europe 2024, the European conference for ISPOR—The Professional Society for Health Economics and Outcomes Research, took place November 17–20, 2024 in Barcelona, Spain. Featuring a comprehensive program centered around the theme, ‘Generating Evidence Toward Health and Well-Being’, the conference closed with Plenary 3, entitled, "Reality Check: Are We Bridging the Evidence Gaps for Patients?" Here, I share an overview and key takeaways from the final plenary session at the conference.
The final plenary session of ISPOR Europe 2024, brought together a multidisciplinary panel, including the perspective of regulators, HTA agencies, payers, industry representatives, and patient advocates, to address a pressing question in healthcare: are we bridging the evidence gaps to truly serve patients?
Moderated by Patrice Verpillat (European Medicines Agency), the session aimed to tie together insights from previous plenaries and explore the regulatory role in improving outcomes for patients. The panelists included Yannis Natsis (European Social Insurance Platform), Anne Willemsen (Dutch National Healthcare Institute), Nikos Dedes (Greek Patients' Association), Peter Mol (College ter Beoordeling van Geneesmiddelen) and Almath Spooner (Abbvie). The discussion revolved around two primary themes: identifying evidence gaps across stakeholders; and exploring how these needs could be integrated to enhance patient care.
Key takeaways from the earlier plenaries
Natsis summarized Plenary 1, which featured a candid discussion focused on the payer perspective of regulatory evidence trends and affordability challenges. Key themes included managing uncertainty and the growing challenges linked to fast-track approvals. Willemsen followed with a recap of Plenary 2, which explored the readiness of stakeholders for the upcoming EU Health Technology Assessment Regulation (HTAR), set to take effect on January 12, 2025. Discussions covered both immediate preparations and long-term implications.
Discussion #1 – The gaps between stakeholders and access to innovation
The panel began by exploring the gaps in evidence needs and access to innovation from their respective perspectives.
Providing the patient perspective, Dedes highlighted the persistent inequalities across Europe in access to medicines and diagnostics, drawing on his personal experience of being diagnosed with HIV at a time when treatments were unavailable. He emphasized the critical role of patient advocacy in drug approval processes and called for high-quality data across diverse sub-populations, greater inclusion of patient voices in designing methodologies and collecting data, and the generation of evidence that supports both patient outcomes and the sustainability of healthcare systems.
“Participation of patients in both designing methodologies and in collecting the data is critical.”
Nikos Dedes, ISPOR Europe 2024
Natsis acknowledged the numerous gaps in evidence, with uncertainty being particularly challenging for payers. He emphasized the need for payers to take a leading role in driving discussions around real-world data (RWD), focus on improving the robustness of randomized controlled trials (RCTs), which remain the gold standard, and advocate for alignment of evidentiary requirements to minimize disparities.
Taking the HTA perspective, Willemsen pointed to a misconception within industry about differences between HTA and regulatory data requirements, urging industry to address HTA needs early in the evidence-generation process. She also called for a focus on generating stronger data to reduce uncertainties in decision-making.
Mol stressed that while RWD offers promise, it will not solve all problems. He identified the key gap as understanding what truly matters to patients, advocating for systematic integration of patient input. He also pointed out the value of combining RWD with robust RCTs to ensure methodological rigor.
“RWD will not be a panacea, it will not be the solution for everything.”
Peter Mol, ISPOR Europe 2024
Spooner concluded the first part of the discussion by highlighting several industry challenges. First, there is limited access to data, despite initiatives like the European Health Data Space and Darwin EU. She also felt there was a lack of clarity around acceptance of innovative trial designs. Finally, she called out the overarching difficulty of integrating diverse stakeholder perspectives, emphasizing the value of working toward convergence in evidentiary standards.
Natsis summed up the discussion points by emphasizing how important it is to explain what these evidence gaps are, as patients deserve to know this information.
“Addressing these gaps has real-life implications.”
Yannis Natsis, ISPOR Europe 2024
Discussion #2 – Understanding and integrating the needs between stakeholders
The conversation then shifted to practical solutions for integrating evidence needs across stakeholders.
Dedes emphasized the need for patient involvement at all stages, from evidence generation to HTA processes, paralleling the participatory model established by the EMA. He also raised concerns about transparency in data use, noting that while patients are often co-owners of health data, they have the least access to it.
Willemsen underscored the differing remits of regulators and HTA bodies, which often result in diverging data needs. While HTA bodies are accustomed to dealing with uncertainty, she stressed the importance of exploring alternatives to single-arm trials, which present significant challenges.
A point of discussion emerged from a recent JAMA publication, highlighted by Verpillat, which examined the role of payers in evidence generation. The article argued that payers are crucial in improving post-market evidence through their influence on coverage decisions, healthcare practices, and access to claims data. Natsis and Dedes reflected on the potential for increased payer involvement. While challenges such as limited resources and uncertain outcomes were acknowledged, there was consensus that payer participation could enhance the robustness of evidence.
Mol went on to discuss the potential of pragmatic clinical trials, particularly for late-stage drug development. Such trials combine the rigor of randomization with the real-world applicability of observational studies. However, Mol noted the challenges of ensuring statistical robustness and the need for additional data collection.
Spooner highlighted both opportunities and challenges within the HTAR and ongoing pharmaceutical legislation reforms. She emphasized the importance of dialog to ensure these efforts deliver on their promises of innovation and patient access.
The panel agreed that greater collaboration is essential to overcoming evidence gaps. Spooner outlined five industry priorities, including:
- Integrating patient voices earlier in the drug development process
- Upholding good scientific principles
- Increasing early dialog with regulators, HTA bodies and payers
- Improving transparency in RWD provenance
- Participating in multi-stakeholder collaborations to address shared challenges
Closing remarks: developing the skills to bridge gaps
The panel concluded with reflections on the skills and capabilities needed to eliminate evidence gaps:
- Spooner stated that industry must undergo a mindset shift to fully embrace collaboration.
- Mol saw a need for regulators to expand expertise in fields such as epidemiology, patient preferences, and regulatory science.
- Dedes issued a request for patient representatives to be empowered through training and resources, following the EMA model.
- Willemsen felt that full utilization of legislative frameworks will be critical to addressing gaps.
- Natsis felt that things were going in the right direction, and that ongoing dialog between payers and regulators will be key to achieving alignment on evidentiary requirements.
Conclusion
The session underscored the complexity of evidence generation and the need for collective action to bridge gaps. While progress has been made, the panelists emphasized that work remains to ensure equitable healthcare access and improved outcomes for patients. The conversations at ISPOR Europe 2024 underscored the importance of ongoing dialogue and collaboration between all parts of the healthcare landscape, striving for a future where evidence gaps are minimized, and patient outcomes are maximized.