Reflecting on the discussions from this year’s International Society for Pharmacoepidemiology (ISPE) Annual Meeting and looking ahead to the future of the field, I’m struck by the steady but significant evolution pharmacoepidemiology is undergoing driven in large part by innovations in real-world data (RWD).
As someone deeply embedded in both the scientific and operational dimensions of real-world evidence (RWE) research, I see immense opportunity but also persistent challenges that demand our collective attention and ingenuity.
The Most Pressing Challenges We Face
There are three critical domains where challenges continue to surface: data, methodology, and regulation.
First, data. Pharmacoepidemiologists must continually stay abreast of emerging data sources, from traditional electronic health records (EHRs) and disease registries to more novel inputs like biomarker and genomic data. There is also a growing emphasis on collecting data outside the clinical setting. Wearables and patient-reported outcomes captured via mobile apps are becoming increasingly relevant and valuable.
Second, methodology. ISPE has long been known as a methods-focused organization, and it remains crucial that we continue to innovate in study design, analytics, and validation techniques. With the rise of more complex and heterogeneous data sources, our methods must evolve accordingly.
Third, regulatory evolution. The guidance frameworks provided by regulatory bodies including the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and others are evolving. These agencies are becoming more receptive to RWD and RWE in certain therapeutic areas, but their requirements are stringent. Adapting to these evolving expectations is a constant challenge that requires a close read of guidance documents, reviewing precedence, and a collaborative approach between industry, academia, and regulatory bodies.
While there may be no single “silver bullet” solution to these challenges, the emergence of robust platforms, greater data transparency, and earlier and deeper regulatory engagement are helping our field rise to the occasion.
Transformative Advancements in RWD Access and Use
One area where we’re seeing transformative change is in data access, particularly access to more diverse and rich datasets. The most exciting developments involve moving beyond structured data elements to integrate biomarker and genomic information. This is an area where we at TriNetX are actively investing, with meaningful progress underway in combining such data with existing clinical records.
Equally transformative is the ability to access source-level data for validation purposes. The regulatory importance of this cannot be overstated. Regulators want to know that the datasets used to inform decision-making are traceable and trustworthy. Through TriNetX’s internal data governance processes, we’ve built a structured, compliant way to connect back to source data. This transparency is not just a feature; it’s a necessity for RWD intended to support regulatory decisions.
These advancements in access, paired with responsible stewardship, are shifting the landscape in ways that make pharmacoepidemiologic research more rigorous, scalable, and actionable.
Pharmacoepidemiology’s Expanding Role in Regulatory Science
Although the field of pharmacoepidemiology often moves at a slower pace, given the time it takes to accrue meaningful observational data, the cumulative progress over the past two decades has been remarkable.
Since then, the role of pharmacoepidemiology has expanded both within regulatory agencies and industry. Today, there is growing demand for experts who not only understand data and methodology, but also how to align their work with regulatory frameworks. This intersection of science and compliance is becoming a defining feature of modern pharmacoepidemiology.
Looking ahead, I anticipate more “use cases” where regulators reference RWE in their decision-making. These precedents (FDA approvals based on RWD, for instance) will serve as case studies for the rest of us, helping to build a clearer roadmap for future work.
Progress I Hope to See by ISPE 2026
By the time we reconvene for ISPE 2026, I hope to see the field make meaningful progress in several key areas:
- More regulatory use cases reported in the public domain that demonstrate the validity and impact of RWD.
- Greater adoption of novel data types, particularly those derived from genomics, biomarkers, and patient-reported outcomes.
- Expanded methodological guidance and standards that enable more consistent and reproducible RWE studies.
- Closer collaboration between industry, academia, and regulators, ensuring that scientific integrity and regulatory needs are harmonized.
All of this must occur without losing sight of our core scientific values: methodological rigor, transparency, and patient-centricity.
The Critical Gap We Must Close
Despite the exciting developments, one major gap still limits the full potential of pharmacoepidemiology: the difficulties in accessing the information in the unstructured text of clinical notes and reports.
While we’ve become highly proficient at working with structured data from administrative claims and EHRs, a vast amount of clinically relevant information remains locked within free-text clinical notes. Getting access to notes and extracting this information at scale to convert it into analyzable formats will be a game-changer.
TriNetX is actively working on enhancing our capabilities to harness this unstructured data. Of course, this is a complex effort that must be approached with caution. We remain committed to advancing these tools responsibly, in accordance with all regulatory and ethical standards, and in close collaboration with our healthcare partners.
Once mature, this capability could significantly expand the depth and accuracy of the real-world insights we provide, bringing us one step closer to a more comprehensive and nuanced understanding of patient journeys, treatment responses, and clinical outcomes.
Accelerating Toward the Next Frontier of Pharmacoepidemiology
The future of pharmacoepidemiology is being written right now, shaped by the collective contributions of scientists, clinicians, data stewards, and regulatory partners. Events like the ISPE Annual Meeting remind us of the importance of staying connected, sharing ideas, and pushing the boundaries of what’s possible.
As we move from ISPE to impact, I believe we’re on the cusp of a new era, one in which RWD is not just an adjunct to traditional research, but a foundational element of how we evaluate drug safety, effectiveness, and value in real-world populations. Let’s keep the momentum going.
Ready to lead the next era of pharmacoepidemiology? Explore how TriNetX’s RWE generation solutions and regulatory-aligned consulting services can help you unlock richer insights, accelerate decision-making, and shape what’s next in drug safety and effectiveness.
About K. Arnold Chan, MD, ScD
K. Arnold Chan is an internationally recognized epidemiologist with more than 30 years of global research experience in academia and the private sector, primarily in the post-marketing evaluation of pharmaceutical agents and vaccines. He has served on the Harvard School of Public Health and National Taiwan University faculty. Dr. Chan has authored or co-authored more than 150 peer-reviewed articles on pharmacoepidemiology and clinical epidemiology and co-edited a textbook on pharmacoepidemiology and therapeutic risk management.
