Long Follow-up in Short Supply: The Need for Multi-setting, Clinically Rich Longitudinal Real World Data

The long-term safety of medications is a crucial consideration for healthcare providers, regulators, and patients alike. While short-term clinical trials provide valuable insights into immediate drug effects, understanding late effects requires access to longitudinal data spanning several decades and heterogenetic populations.

Claims data sources in the United States are subject to patient churn through different health insurance providers (employer sponsored, Medicare, Medicaid), making tracking patients over extended periods difficult, hindering efforts to identify treatment to assess the long-term effectiveness and safety profile of drugs. While this is improving with tokenization efforts that link together patient records across different databases, the levels of follow-up available are still limited. In contrast, registries and databases in Europe, such as Denmark, Sweden, and the Netherlands, offer extensive longitudinal data, linked EHR with detailed registry in formation in Cancer, Pathology and pregnancy that enable researchers to follow patients for more than 10-20 years in unprecedented detail, providing valuable insights into late and developing drug effects thought the life cycle of your drugs.

In particular, the following research areas can benefit from long follow-up periods:

• Assessment of Late Effects: Many adverse drug reactions and long-term health consequences only manifest years or decades after initial exposure. Longitudinal data are essential for identifying and studying these late effects to inform clinical decision-making and regulatory policies.
• Evaluation of Treatment Outcomes: Longitudinal studies allow for the assessment of treatment effectiveness and safety over time, providing insights into the real-world use of medications and their impact on patient outcomes.
• Identification of Risk Factors: Longitudinal data enable researchers to identify potential risk factors associated with adverse drug reactions, allowing for targeted interventions and personalized medicine approaches.
• Detection of Rare Events: Longitudinal databases with large sample sizes enhance the detection of rare adverse events associated with medications, providing a more comprehensive understanding of their safety profile.

Recognizing the importance of longitudinal data in drug safety research, collaborating with expertise centra all over the world, to leverage these resources to study complex and heterogenic treatment pathways. This heterogeneity is a unique source to understand drug effectiveness and safety in interaction with the different healthcare systems and social beliefs.

Join us on June 18, 2024 in San Diego at the Drug Information Association (DIA) global annual meeting in a forum titled ‘Long-Term Safety of Approved Medicines: Approaches for Identifying, Characterizing, and Quantifying Delayed Adverse Events.’^[1]
– Steven Weisman, Global President, Clinical and Regulatory, Lumanity
– Naomi Boxall, General Manager, PHARMO Institute
– Gerald Dal Pan, Director, Office of Surveillance and Epidemiology, CDER, FDA
– Vera Ehrenstein, Professor, Department of Clinical Medicine, Dept of Clinical Epidemiology, Aarhus University
– Claudia Salinas, Senior Director, GPS Pharmacoepidemiology, Eli Lilly and Company

During the forum we will explore the critical need for longitudinal data in drug safety research, examining the strengths and limitations of different data collection approaches and examine collaborative opportunities with regulators and organizations such as SIGMA to address these research needs and enhance patient care.

About the SIGMA Consortium
The SIGMA Consortium is a contract-based alliance of ENCePP research centers, co-founded by RTI and the PHARMO Institute, to herald a new era of collaboration to better understand safety and effectiveness of medical interventions. SIGMA is designed to be the best ‘go-to’ organization for comprehensive and rigorous methodological research enable multi-country studies using common analyses to study responses to treatments in large populations.

About the PHARMO Data Network^[2]
The PHARMO Data Network is a population-based network of electronic healthcare databases and combines anonymous data from different primary and secondary healthcare settings in the Netherlands. Healthcare settings include general practitioners, out-patient and in-patient pharmacies, hospitals and clinical laboratories. Furthermore, databases are linked with external registries such as the Cancer Registry, Pathology Registry and Perinatal Registry. The dataset covers 40% of the Dutch population and goes back to 1999, with an average of 10-12 years of observation on an individual patient record.

[1] https://www.diaglobal.org/en/flagship/dia-2024/program/schedule/agenda

[2] Kuiper JG, Bakker M, Penning-van Beest FJA, Herings RMC. Existing Data Sources for Clinical Epidemiology: The PHARMO Database Network. Clin Epidemiol. 2020 Apr 29;12:415-422. doi: 10.2147/CLEP.S247575. PMID: 32425609; PMCID: PMC7196787.