Reflections from the ISPOR RWE Summit 2025: Laura Pizzi on the future of real-world evidence
At the beginning of October, ISPOR—The Professional Society for Health Economics and Outcomes Research brought together experts from across Asia-Pacific and beyond to explore how real-world data (RWD) and real-world evidence (RWE) can drive better decisions in healthcare. Over the course of the three days in Tokyo, discussions at the ISPOR Real-World Evidence Summit 2025 ranged from methodological advances to regulatory perspectives, highlighting both opportunities and challenges in making RWE more robust, transparent, and actionable. The meeting also reflected how the society is considering the role of RWE within its broader mission and strategic priorities.
Following the summit, we caught up with Laura Pizzi (Chief Science Officer, ISPOR) to hear her reflections on the key themes from Tokyo, how these connect to global trends, and what she expects to see on the horizon at from Society at ISPOR Europe in November and beyond.
Thanks for taking the time to speak with us after a busy few weeks. What were your key takeaways from the ISPOR RWE Summit in Tokyo? Were there particular sessions or discussions that stood out to you?
First, it was significant for ISPOR to be back in the Asia-Pacific region with a scientific summit. Around 700 attendees from 43 countries attended, with strong representation from Japan, China, Taiwan, South Korea, Singapore, Australia, and Thailand in particular. The main takeaway for me is that support for RWE is strong in the Asia-Pacific region – clinical trials are still the gold standard but insufficient, especially due to biopharma innovation pipelines that remain concentrated on rare diseases.
The Asia-Pacific perspective brought unique insights into RWE. How did the regional context shape conversations on data access, quality, and infrastructure compared with what we typically see in Europe or the US?
The Asia-Pacific region is similar to Europe and the US with respect to an insufficient number of high-quality data sources to develop RWE. Accessibility of data sources is a challenge in all three regions but is more advanced in Europe where there has been a strong commitment to furthering RWD from the European Commission and European Medicines Agency. Major EU initiatives to increase the availability and use of RWD include the DARWIN EU datasets and ongoing development of the European Health Data Space (EHDS), the latter of which will enable secondary use of patient data for research purposes. That said, DARWIN EU is still in relatively early stages in terms of accruing datasets and uptake, and its use for regulatory purposes is far more advanced than for HTA. HTA authorities in the EU are aware of it but not using it much yet – and we are several years away from EHDS being ready for regulatory and HTA use.
We don’t have a repository like that in the US and, as a result, it can be difficult to find data that are of sufficient quality to answer the research questions at hand. Asia-Pacific doesn’t have a repository like this either, and achieving one would be uniquely challenging given the number of countries in the region and their heterogeneity; Japan, China, Korea, and Australia have healthcare infrastructures that are ripe for furthering RWD, whereas other Asian countries are in earlier stages. The Asia-Pacific region would also benefit from a common data model to allow country-level comparisons.
RWE frameworks are evolving rapidly. How did the Summit address the role of RWE guidance in regulatory or health technology assessments in the Asia-Pacific region?
There’s been a proliferation of RWE guidance across the world for several years now, making it challenging for health technology developers because there are multiple frameworks to navigate. In terms of regulatory frameworks, a logical approach for Asia-Pacific is to participate in the International Council on Harmonization (ICH) and to adopt the resulting ICH guidelines into national requirements; for example, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) participates in ICH and utilizes the resulting regulatory guidelines in their country. ICH M14 is an excellent example of that.
“ISPOR is proud that this guideline, which focuses on drug safety, recommends the HARPER template for RWE studies that was published by a joint ISPOR–ISPE Task Force a few years ago.”
We did hear from some of the smaller Asia-Pacific countries that they are not at a stage of readiness to engage in ICH. We also heard that the need for country-specific guidelines will persist, particularly to build public trust in RWE.
Collaboration was a recurring theme at the Summit. How is ISPOR working to advance collaboration across stakeholders and regions to strengthen the field of RWE?
Given that ISPOR is a scientific professional organization, we focus on collaborating with entities that can further the methods of evidence development. To that end, we are working with ISPE and the Society for Medical Decision Making (SMDM) on different Task Forces, along with RWE academic centers of excellence such as Harvard University and the Duke Margolis Center for Health Policy. We seek similar relationships with Asia-Pacific thought leaders; for example, HTAsiaLink with whom we’ve had a formal collaboration.
The discussions also looked to the future of RWE in HEOR. What should we, as HEOR experts, be doing to optimize the use of RWE, ensure it is integrated into decision-making and clinical practice, and leverage tools such as AI to generate valuable insights?
The main tools we need to leverage RWE already exist. These include data-quality checklists, protocol templates (e.g., the HARPER template), and protocol registries for pre-specification of study plans such as the RWE protocol registry hosted by ISPOR, which is free and publicly available. Regarding AI, there’s still a lack of tools guiding its use, at least for producing decision-grade analyses that meet regulator or HTA standards. Best practices on how to validate AI output are needed, with ongoing work in this area to compare AI output using different LLM platforms or with human checks at certain points in the research process.
“There’s enormous promise of AI in terms of harvesting not only study outcome measures but also covariates from large volumes of qualitative data, but the HEOR community should proceed with caution until we have guidance on how to validate AI-assisted RWE that’s been informed by primary research.”
Looking ahead to ISPOR Europe in Glasgow this November, which conversations from the Summit do you expect to carry through? Do you think RWE will remain the dominant trend, or are other priorities, such as digital endpoints, AI/ML, or cross-border collaboration, beginning to take center stage?
The featured topic in Glasgow will be a patient-centered evidence track, which had its own call for submissions and includes research that has been specially curated by a program committee of experts in patient engagement, patient-centered outcomes, and patient preferences. RWE will also be a dominant topic in Glasgow, as well as use of AI in HEOR and broadening the definition of healthcare value to include concepts related to whole health. I encourage readers to visit ISPOR’s website to learn more about whole health as a theme of our Strategic Plan 2030.
Finally, on a more personal note, what moment from the Summit will stay with you the most, and why?
I was truly moved by the beauty of Japanese culture and the warmth that ISPOR summit attendees received from local staffers and scientific contributors. The event volunteers even made origami for ISPOR delegates, which infused colorful art and tradition into the conference. We chose Tokyo as the summit location due Japan’s prominent role in healthcare innovation and use of HEOR, and are left with a lasting impression due to its rich culture and incredible hospitality.
Interviewee
Laura Pizzi
Chief Science Officer, ISPOR—The Professional Society for Health Economics and Outcomes Research
Dr Laura Pizzi is the chief science officer for ISPOR—The Professional Society for Health Economics and Outcomes Research. ISPOR is the leading global professional society for health economics and outcomes research (HEOR). In her role, Dr Pizzi provides strategic scientific leadership and oversees the organization’s major initiatives, including the development of ISPOR’s science strategy, content priorities, and global initiatives such as Special Interest Groups, Patient Council, regional Patient Representative Roundtables, Health Technology Assessment (HTA) Roundtables, and the organization’s AI and Digital Health member initiatives. Her team is also responsible for engaging with global healthcare authorities to support the use of HEOR in healthcare decision making. With a strong passion for education, Dr Pizzi co-led the development of ISPOR ’s first Health Economics and Outcomes Research Competency Framework, which outlines the core knowledge and skills essential to the HEOR profession.
Dr Pizzi brings more than 25 years of academic and research expertise to ISPOR. She has led multidisciplinary teams of methodologists, statisticians, and clinicians in designing and conducting cost and outcomes analyses for both pharmacologic therapies and nonpharmacologic interventions. Much of her work has focused on improving the quality of and access to treatments for older adults in underrepresented communities for diseases of aging such as dementia, diabetes, chronic vision diseases, and vaccine-preventable illness. Her achievements include leading health economic analyses for numerous multicenter clinical trials, testifying before the US House of Representatives Committee on Ways and Means on Medicare spending, authoring a textbook on economic evaluation, and publishing more than 100 peer-reviewed manuscripts and 120 research presentations.
In addition to her leadership role at ISPOR, Dr Pizzi serves as research professor at Rutgers University, Ernest Mario School of Pharmacy. Her academic career has centered on advancing the science of health economics, influencing healthcare policy, and training the next generation of HEOR professionals.
Disclaimer
The opinions expressed in this feature are those of the author and do not necessarily reflect the views of The Evidence Base® or Becaris Publishing Ltd.