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SeltaSquare reads industry shifts ahead of the curve and delivers meaningful insights to our clients.

The Spring Academic Conference of the Korean Society of Pharmacoepidemiology and Risk Management (KoPERM), held on May 28–29, brought together researchers, regulators, and pharmaceutical industry stakeholders from across Korea. Sessions spanned AI applications in healthcare, causal inference-based treatment effect analysis, RWD/RWE regulatory guidelines, key drug safety issues, signal detection strategies, and region-based active pharmacovigilance systems — offering an in-depth look at the latest research trends and real-world applications across pharmacoepidemiology and pharmacovigilance.

This edition focuses on RWD/RWE regulatory guidelines and global guideline update trends for those working in Clinical and Medical Affairs.

On May 28, a dedicated session titled "Guidelines for RWD/RWE Utilization in Regulatory Science" was held, featuring Prof. Namkyong Choi from the Department of Health Convergence at Ewha Womans University and Prof. Eun-Ah Han from the College of Pharmacy at Yonsei University. The session provided an in-depth discussion on the role of RWD/RWE in drug safety and efficacy evaluation and drug outcome assessment, as well as the current state of domestic guideline development.

 

Background: A Paradigm Shift in Korea's Post-Marketing Safety Surveillance

In February 2025, Korea's post-marketing drug safety system transitioned from a re-examination-centered framework to one centered on the Risk Management Plan (RMP).

The implications for pharmaceutical companies are clear. This shift signals that the systematic use of RWD/RWE is becoming increasingly critical across all aspects of post-approval safety monitoring and additional pharmacovigilance activities. RMP-designated drugs are now required to include either active surveillance (utilizing regional hospitals, patient registry programs, or adverse event-specific monitoring) or comparative observational studies such as cohort, case-control, or cross-sectional designs.

RWD/RWE in Drug Outcome Assessment

At the same time, as the number of high-cost treatments for rare and serious conditions continues to grow, many regulatory and reimbursement discussions are taking place with limited clinical trial evidence. The introduction of Coverage with Evidence Development (CED) has further increased the need for outcome assessments in real-world clinical settings.

RWD/RWE is emerging as a key approach for generating this evidence. The Health Insurance Review and Assessment Service (HIRA) has developed RWE generation guidelines for assessing outcomes of health insurance-listed drugs, published on June 11.

Where Do Global Regulators Stand?

At the conference, Prof. Namkyong Choi of Ewha Womans University reviewed the evolution of RWD/RWE guidelines from the FDA, EMA, and PMDA, and outlined the key strategic directions.

The U.S. FDA has steadily advanced its RWE program framework since the enactment of the 21st Century Cures Act in 2016. With the 2023 release of the Advancing RWE Program, the FDA established a system through which sponsors can proactively discuss specific RWE use cases with the agency and assess the acceptability of study methodologies from a regulatory decision-making standpoint. The FDA's core evaluation criteria are as follows:

• Is the data fit-for-use for the intended purpose?
• Can the study design answer the regulatory question?
• Does it meet regulatory requirements?

The European EMA, through its EMNS 2028 Vision released in 2025, announced a strategic shift toward RWE-based clinical evidence generation frameworks by 2030. EMA's core message is clear: "The research question — not the data source — determines the study design." Regulators are asking not just for analytical results, but for transparency and reproducibility in the design, analysis, and reporting that lead to those results.

Domestic Guideline Development in Korea

In Korea, the Korean Society of Pharmacoepidemiology and Risk Management (KoPERM) is leading a research initiative to develop guidelines for drug safety and efficacy evaluation using RWE. Over three years, six sets of guidelines will be developed sequentially, covering: external comparator selection, registry data evaluation, EMR data evaluation, health insurance claims data evaluation, general considerations, and study design.

SeltaSquare Insight: RWD/RWE Research Starts with the Question, Not the Data

The most important perspective presented at this conference can be summarized in a single sentence:

"The starting point of RWD/RWE research is not the data source — it is the decision question: what uncertainty are we trying to resolve?"

RWE is not a wholesale replacement for RCTs. It is complementary evidence for questions that RCTs struggle to answer — such as real-world effectiveness and safety, outcomes in specific subpopulations, and issues requiring long-term follow-up.

Pharmaceutical companies and research organizations must therefore move beyond "Can we access the data?" and clearly define four questions upfront:

• What uncertainty are we trying to resolve?
• Which data source is appropriate for this question?
• What study design and analysis will answer it?
• Which regulatory decision — approval, safety, or reimbursement — does this evidence support?

For RWE to be accepted by regulators, population definitions, exposure and outcome variable definitions, comparator selection, and the statistical analysis plan (SAP) must be systematically pre-specified and pre-submitted. Furthermore, when RMP-based safety studies and reimbursement outcome studies are conducted in silos, the evidence generated cannot be used in an integrated manner. This calls for a comprehensive Integrated Evidence Generation Plan that considers regulatory approval, safety, and reimbursement objectives together.

With SeltaSquare — From Strategy to Publication

Changes in the RWD/RWE research landscape present new challenges for pharmaceutical companies: clearly defining research questions, assessing data source appropriateness, and building study design, analysis, and reporting frameworks that meet regulatory standards. This requires simultaneous expertise in pharmacoepidemiology, study design, data management, and statistics.

For organizations without a fully staffed CD or Medical Affairs team, designing all of this from scratch is no small task.

SeltaSquare is more than a research outsourcing organization. We translate regulatory questions into study designs and co-develop Integrated Evidence Generation Plans that holistically address regulatory approval, safety, and reimbursement objectives — your End-to-End Evidence Strategy Partner.

From research question scoping → data source review → protocol and SAP development → methodology optimization → analysis and reporting → publication, our team of pharmacoepidemiology, study design, and DM/STAT experts connects every step seamlessly.

The transparency and reproducibility regulators demand. The in-house expertise gaps that are hard to fill. SeltaSquare bridges both.