Future Trends in Pharmacoepidemiology: Insights from the 2025 ISPE Conference

The 2025 International Society of Pharmacoepidemiology (ISPE) Annual Meeting provided a glimpse into the latest innovation and future of pharmacoepidemiology and drug safety. Conference insights revealed significant trends and transformations poised to shape the industry due to enhanced data access, evolving technology and growing emphasis on personalized medicine. We are committed to embracing in these developments, helping companies navigate the evolving landscape of epidemiology solutions and real-world data (RWD) and real-world evidence (RWE) generation strategies, ensuring the safety and efficacy of therapies.

The ascendancy of RWD and RWE

The increasing importance of RWD and RWE for safety surveillance and risk management is a clear indicator of where the industry is headed. RWD sources are becoming indispensable to generate evidence that informs decision-making, supports safety insights and improves patient outcomes.

• Inputs for pharmacovigilance efforts: Although not a new area of focus, therapy and diagnostics developers should prepare for a future where RWD is routinely used alongside clinical trial data to generate evidence that supports an understanding of the effectiveness and safety of pharmaceuticals.
  • Assessing comparative effectiveness and safety outcomes: RWE provides valuable insights into the comparative effectiveness, safety outcomes and long-term real-world benefits of therapies. This enhances understanding of a drug’s real-world impact, informing both clinical and regulatory decisions.
  • Enabling proactive monitoring and risk management: RWD allows for proactive monitoring, risk management and timely safety interventions, ensuring patient safety in real-world settings. This proactive approach helps identify potential safety issues early and mitigate risks effectively.
  • Providing a comprehensive understanding of safety outcomes: Analyzing data from real-world settings provides a more holistic view of the impact of therapies on patients. RWD can help identify previously unrecognized adverse effects and inform risk-benefit profiles.
  • Informing post-market surveillance: Longitudinal clinical registries provide a structured method for data collection and extensive real-world data sets for analysis that align with regulatory compliance for post-marketing safety surveillance. This continuous monitoring of a therapy’s safety profile post-approval enhances the ability to detect adverse events or efficacy/efficacy trends from real-world patient care for a more comprehensive understanding of a therapy.
  • Facilitating personalized care: RWD supports treatment optimization, leading to personalized care and improved patient outcomes. Real-time insights from RWD reveal how therapies perform across diverse subgroups that may not have been fully addressed in clinical trials, allowing for tailored treatments.
  • Identifying barriers to patient uptake: RWD helps identify barriers to patient uptake in the real world, allowing sponsors to anticipate and mitigate challenges for optimal use and success. This ensures that therapies are used effectively and reach the patients who need them most.
• Global data sources: The interest and demand for ex-US data sources highlights the need for a global approach to data collection and harmonization. The ability to combine disparate RWD sources and patient insights for integrated analysis is driving the need for comprehensive real-world data strategies that bridge the gap from evidence-generation planning to real-world study delivery. As the scope for augmentation of existing RWD sources grows, technology-enabled approaches such as tokenization, automated EMR extraction and AI and NLP approaches are rising as invaluable tools to deliver best fit data with speed and efficiency.
• Transparency and reproducibility: Conference discussions highlighted the critical importance of transparency and reproducibility in utilizing RWD, along with considerations around data completeness and harmonization. Ensuring research findings are robust, credible and actionable requires comprehensive and high-quality data. Ongoing efforts to leverage proprietary data sources, build RWD networks and frameworks and employ technology-enabled solutions to create reproducible and transparent RWD analytic workflows will accelerate robust analysis.

Advancements in methodological approaches

There was a strong focus at ISPE 2025 on traditional scientific methods to address common challenges in observational studies, such as confounding and bias.

• Enhanced analytical rigor: Quantitative bias methods serve multiple objectives in epidemiological research and provide a means to assess the potential for residual bias in observational studies. Sponsors are striving to ensure their research meets the highest standards of scientific rigor. An increased focus on maintaining standards of good practices of bias analyses indicates a move toward applying fundamental methods to address confounding and bias, enhancing validity and reliability of research findings.
• Target trial emulation design: The emergence of target trial emulation design is transforming how observational studies are conducted. This approach allows researchers to mimic the conditions of randomized trials, thereby reducing bias and improving the credibility of study results. Integrating target trial emulation into research methodologies can help achieve more robust outcomes.

• Revised evidence hierarchies: The debate around evidence hierarchies in epidemiology suggests a future where diverse evidence sources are valued more equally. The advancement of evidence-based medicine depends on the framework for classifying research approaches according to their dependability and quality. Integrating various forms of evidence, modifying hierarchies in response to technological developments and encouraging global collaboration are all key factors in standardizing evidence hierarchies to enable healthcare stakeholders to collaborate and successfully address health-care concerns.

The rise of registries

The buzz around registries at the conference was hard to miss and highlighted their potential to alleviate the burden of RWD generation and revolutionize data collection and analysis. Bespoke and collaborative registries offer a streamlined approach to gathering high-quality data over a long period of time to support safety, efficacy and value-demonstration for a therapy, particularly in small patient populations.

• Collaborative data initiatives: The enthusiasm for patient and disease registries suggests a future where collaborative data collection initiatives become more of the norm. Participating in existing disease registries or establishing bespoke registries can reduce sponsor and patient burden providing valuable insights for all stakeholders into drug safety, product effectiveness, and patient experience in real-world settings to help inform patient care and quality of life. The opportunity to nest safety studies in existing registries can further minimize time, expense and burden for post-authorization safety studies.
• Enhanced data sharing: The interest in registries underscores the importance of data sharing and collaboration on a global scale to unlock superior data and insights more efficiently. Traditionally, challenges related to scope and scale of long-term data collection have hindered the types and amount of data collected. Registries offer an opportunity to overcome long-term data collection challenges and provide holistic evidence to improve patient outcomes.

The open exchange of scientific information at the 2025 ISPE Annual Meeting revealed a dynamic and evolving landscape in pharmacoepidemiology. The growing importance of RWD and RWE, coupled with advancements in data analytics and technological innovations, are shaping the future of pharmacovigilance, drug utilization research and therapeutic risk management.

We are committed to these advancements, continuously embracing emerging trends, investing in cutting-edge technological solutions and fostering collaborative research models. As the field evolves, our collective efforts will advance patient-centric scientific research and improve health care outcomes.