ISPOR Europe 2025 – Inside the first plenary: Rethinking patient engagement in healthcare investments

The opening plenary of ISPOR Europe 2025, organized by ISPOR — The Professional Society for Health Economics and Outcomes Research, set the tone for four days of discussion in Glasgow focused on value, access, and collaboration under the theme “Powering Value and Access Through Patient-Centered Collaboration.”

Titled “Patient Engagement in Healthcare Investments—A Promise or a Practice?”, the session brought together stakeholders from across the healthcare ecosystem to examine how far patient engagement has progressed from principle to practice. Here, I share an overview and key talking points from the session. 

This opening plenary session tackled a critical question for the healthcare ecosystem: is patient engagement still a promise or has it become a practice? Moderated by Joep Muijrers (Gilde Healthcare Partners, the Netherlands), the session brought together voices from industry, academia, regulation, and patient advocacy, along with the investor perspective, to explore how patient experience can – and must – be embedded across the healthcare investment and innovation cycle. Panelists included Luc Truyen (Argenx, USA), Carole Longson (University of Manchester, UK), David Haerry (Positivrat, Switzerland) and Steffen Thirstrup (European Medicines Agency [EMA], the Netherlands).

Discussion 1 – The Emerging Trends: Patient Engagement as a Pillar of Innovation

Carole Longson (University of Manchester, UK) opened by stating that meaningful patient inclusion must begin with belief. “You have to believe that engagement and inclusion is the right thing to do,” she said, noting an ongoing gap between the principle and its consistent application. Asking “what patients bring to the table,” she argued, is the wrong question – the focus should instead be on creating space for patients’ voices to shape decisions that affect their lives.

From the biopharmaceutical perspective, Luc Truyen (Argenx, USA) described an early experience developing treatments for myasthenia gravis. Investors and clinicians initially questioned the unmet need, but direct engagement with the patient community revealed a different reality – one that ultimately altered trial priorities around patient-defined outcomes, such as minimal symptom expression. This, he said, exemplified how crucial early and sustained patient input is.

Patient advocate David Haerry (Positivrat, Switzerland) shared his personal journey living with HIV and decades of collaboration with regulators and industry. He emphasized that while some organizations have embraced engagement, others lag behind. “Those who go on the journey don’t turn back,” he said, noting the effort required but underscoring its value: “It is more work than you expect, but it’s worth it.” Haerry highlighted the need for more observational data post-approval and urged greater use of real-world evidence (RWE) to understand how therapies perform in practice.

From a regulatory standpoint, Steffen Thirstrup (European Medicines Agency [EMA], the Netherlands) outlined the EMA’s long history of involving patients, including through its Patients and Consumers Working Party. He cited a powerful example from multiple sclerosis (MS) treatment, where patient representatives’ willingness to accept minimal risk fundamentally changed a regulatory decision. Thirstrup introduced the EMA’s recent reflection paper on patient experience data, now open for public consultation, and advocated for stronger patient representation – including voting rights – on scientific committees. 

Discussion 2 – From Principle to Practice: Embedding Patient Perspectives in Evidence and Access Frameworks

Muijrers offered the investor’s perspective, acknowledging that financial stakeholders wield substantial influence over which innovations advance. He urged investors to embed patient engagement criteria into investment milestones, ensuring companies integrate patient perspectives early – ideally at the stage of indication selection and trial design. “At the end of the day, all we’re doing is involving the end user earlier in product development,” he said.

The panelists agreed that while progress has been made, change and improvement is still needed. Longson called for institutional policies that make inclusion a visible organizational commitment. Truyen emphasized the need for systems capable of capturing the entire patient journey, while Thirstrup and Haerry stressed better integration of patient experience into real-world data frameworks.

During audience Q&A, a question arose about whether small groups of patients can truly represent the broader community. Muijrers countered that this challenge is not unique to patients: “We don’t ask this of physicians”. Thirstrup noted that at the EMA, patient participants are subject to the same conflict-of-interest standards as all experts, and that frameworks exist to balance representativeness with lived experience. Longson added that the goal is to ensure appropriate voices are around the decision-making table, while Haerry observed that such concerns often stem from inexperience—there are already robust tools and methodologies, such as those from PFMD, to guide meaningful participation. 

Call to action

In closing, each panelist issued a clear call to action for advancing patient engagement:

• Truyen: Join forces across sectors to sustain patient engagement efforts beyond individual initiatives.
• Longson: Reflect personally and institutionally – “If you believe this is the right thing to do, you will find ways to do it.”
• Haerry: Convince senior leadership that patient engagement must be a strategic priority embedded throughout the organization.
• Thirstrup: Provide constructive feedback on the EMA’s reflection paper on patient experience data and support stronger formal roles for patients in regulatory decision-making.
• Muijrers: Help build a lasting multi-stakeholder community committed to collaboration, ensuring patient inclusion becomes standard practice. 

Takeaway 

Across all perspectives – regulatory, industry, patient, and investment – the message was consistent: belief must translate into structure. Patient engagement should not be viewed as an optional principle but as a measurable, sustained practice embedded in how healthcare innovation and decision making are done.