ISPOR 2025 – inside the third plenary: Balancing speed, scientific rigor and patient-centered use of surrogate endpoints

ISPOR 2025, the annual conference for ISPOR—The Professional Society for Health Economics and Outcomes Research, took place May 13–16, 2025 in Montreal, Canada. Featuring a comprehensive program centered around the theme, Collaborating to Improve Healthcare Decision Making for All: Expanding HEOR Horizons, the conference closed with the third plenary, entitled, “Balancing Speed and Scientific Rigor—Patient-Centered Methodologies for Surrogate Endpoints in Accelerated Access”. Here, I share an overview and key takeaways from the final plenary session at the conference.

As regulatory pathways increasingly lean on surrogate endpoints to accelerate access to therapies for high-need conditions, tensions are emerging between the push for speed and the demand for robust, patient-centered evidence. The third plenary tackled this critical challenge, asking how stakeholders can ensure that surrogate endpoints used in accelerated approval processes are both scientifically sound and meaningful to patients. Moderated by Elisabeth Oehrlein (Applied Patient Experience, LLC, USA), the panel brought together diverse expertise from across the healthcare landscape: Ebony Dashiell-Aje (BioMarin Pharmaceutical, USA), Yvette A Venable (AstraZeneca, UK), C. Bernie Good (UPMC Health Plan, USA) and Durhane Wong-Rieger (Canadian Organization for Rare Disorders, Canada). Together, they explored practical approaches for selecting, validating and interpreting surrogate endpoints, highlighting the vital role of early and sustained patient engagement.

Structured in three sections – current context, integrating the patient voice and moving to action, and a Q&A – the session explored both the challenges and potential of surrogate endpoints, with an emphasis on early collaboration, continuous patient engagement and actionable evidence generation strategies.

Current context: challenges and disconnections

Opening the discussion, Venable noted that reliance on surrogate endpoints is not new – but it has become more urgent as we seek earlier signals of benefit, especially for chronic and rare diseases. Using chronic kidney disease as an example, she pointed out that while hard clinical outcomes like dialysis are delayed and difficult to measure in trials, patients care most about delaying that progression.

“We need to find another way to identify what good looks like for those patients.” Yvette A Venable

Good, speaking from the payer perspective, acknowledged that surrogate endpoints are often necessary – especially in small patient populations – but added that they come with real concerns. Payers must assess whether the surrogate is merely plausible or actually predictive of meaningful outcomes. He emphasized the difference between efficacy in idealized trial settings and effectiveness in the real world, calling this one of two key hurdles for payers when considering new therapies. The second of these hurdles he described as the challenge of understanding whether approvals based on surrogate endpoints will truly help patients.

Wong-Rieger, offering the patient advocacy perspective, stressed that in rare diseases, time is of the essence. But she warned of risks if surrogate markers aren’t updated in line with evolving evidence, citing cases where outdated criteria have inadvertently excluded patients from accessing life-changing treatments.

Dashiell-Aje took a holistic view, identifying a common goal across stakeholders: to deliver transformative medicines to patients who need them most. However, she observed that there are big gaps between the kinds of evidence stakeholders want, when they want it, and how it’s interpreted. She called for more integrated evidence generation strategies, designed early and collaboratively, to ensure data is fit for all decision-makers – from regulators to clinicians to patients.

Evidence into action: patient-centered approaches

In the second part of the session, the discussion shifted to how patient voices can be better integrated throughout the product lifecycle.

Wong-Rieger remarked that the era of “token” patient involvement is fading, but there’s still work to do. While engagement is improving on the research side, particularly in rare diseases, it’s often lacking in regulatory, HTA and payer settings. She also emphasized the importance of sustained involvement after market approval, arguing that post-approval monitoring is often where patients are forgotten.

Dashiell-Aje discussed the evolution of clinical outcome assessments (COAs) and patient-reported outcome measures (PROMs), noting that robust frameworks now exist to anchor biomarker changes to meaningful improvements in patients’ lives. She advocated for using qualitative research early in development to identify concepts that matter most to patients and to guide endpoint selection.

Good supported this approach, adding that payers increasingly value evidence of patient-preferred outcomes. He cited past experience where patient priorities diverged from physician assumptions and called for more deliberate patient engagement in trial design. On post-approval monitoring, he emphasized that the responsibility to connect the dots doesn’t end with approval. He called for use of registries, real-world evidence and value-based contracts to ensure that surrogate endpoints lead to genuine clinical benefit.

Wong-Rieger echoed this need for sustained patient engagement, particularly in post-market surveillance. She encouraged making patients part of the research team, with education and incentives to support data collection.

“Your job is not done when you get market approval.” Durhane Wong-Rieger

Q&A and call to action

The Q&A segment highlighted discomfort with the term ‘surrogate endpoint’ itself. Venable supported rethinking the language, suggesting that ‘novel endpoints’ or ‘alternative patient outcome measures’ may better capture the evolving scientific rigor and patient relevance behind these metrics. When asked how patients can assist in identifying outcomes and guiding measurement, Dashiell-Aje emphasized early and collaborative engagement. She explained that endpoint development requires cross-functional work between patients, scientists and clinicians to ensure concepts are not only relevant but also measurable and practical within clinical trials.

Returning to the topic of payer–industry collaboration, Good noted that early conversations can help guide evidence development and avoid misalignment. He commented that payers would welcome more engagement upfront, pointing to ‘coverage with evidence development’ models as one promising path.

To close the session, each panelist offered a call to action:

• Dashiell-Aje: All stakeholders want to review robust evidence; the only way to do that is design studies that allow you to measure what matters most, in reliable, valid ways.
• Venable: Science has progressed and transformed patients’ lives, and we need to find better ways to define what ‘good’ looks like for patients; this is an ethical and equity imperative.
• Good: There is a need to complete the cycle; giving payers the evidence that connects surrogate endpoints to real outcomes. Don’t stop at approval.
• Wong-Rieger: Start talking about individualized indicators and outcomes measures. Use patients as their own measure of what they need and what they’re benefiting from.

Moderator Oehrlein closed with a reminder of ISPOR’s ongoing work on surrogate endpoint evaluation via the Surrogate Endpoint Statistical Evaluation for HTA Decision Making Task Force, and invited attendees to stay involved. Overall, the discussion underscored that while surrogate endpoints can dramatically shorten the time to patient access, their value ultimately hinges on the extent to which they reflect outcomes that matter most to patients and caregivers. By integrating patient perspectives early and continuously, the healthcare community can better align regulatory and HTA decisions with real-world needs, even under tight timelines.

As the session drew to a close, Rob Abbott, CEO of ISPOR, returned to the stage to thank the panel and attendees for their engagement throughout the week. He highlighted several upcoming events that will continue the global conversation around health economics and outcomes research, including the ISPOR Real-World Evidence Summit in Tokyo, ISPOR Europe 2025 in Glasgow, and ISPOR 2026 in Philadelphia. With a call to action echoing the collaborative spirit of the conference, Abbott brought ISPOR 2025 to a close – celebrating the shared commitment to improving healthcare decision-making for all.