Target RWE Leads Epidemiology and Analytical Expertise at ISPOR and PMSA 2024 Conferences

Next-generation analytics and real-world evidence (RWE) leader, Target RWE , presented multiple poster presentations at the Pharmaceutical Management Science Association (PMSA) meeting in Denver, Colorado, and ISPOR—The Professional Society for Health Economics and Outcomes Research meeting in Atlanta, Georgia from May 5-8, 2024. From health economics and outcomes research to analytical methods to advance data-driven decisions, both events offered valuable insights into the evolving landscape of healthcare. 

 “Our presentations at both meetings demonstrated Target RWE’s unique ability to bring advanced biostatistics and epidemiology concepts to life in ways that propel the advancement of RWE research,” said Jennifer Christian, PharmD, PhD, FISPE, Chief Scientific Officer. “These principled approaches will transform the field by addressing complexities with more robust and sophisticated methods that are shaping the future of reproducibility. This is an exciting time to have our analytical concepts represented and contribute to improving patient outcomes.” 

Target RWE poster topics included:  

• Negative control outcome studies informing whether to proceed with a comparative effectiveness study  
• Patient cloning as a novel approach to inform clinical treatment decisions 
• The ‘clean room committee’ approach to address post hoc analytic issues  
• Linking medical record data with administrative claims data to study patients with high-risk non-muscle invasive bladder cancer 
• Using electronic medical records to transform Phase 3 clinical trials 
• Prediction in large administrative claims databases 

Redefining RWE through Negative Control Outcome (NCO) Studies 

Unmeasured confounding is often considered one of the primary challenges to performing real-world studies. Negative control outcome (NCO) studies are a valuable tool used by Target RWE to assess whether a study suffers from unmeasured confounding and the comparability of potential treatment groups. Target RWE experts analyzed how conducting an NCO study prior to a comparative safety or effectiveness study can aid in determining whether it is feasible to proceed with a comparative study. 

Comparing Treatment Sequences through Clone Censor Weighting 

Target RWE researchers also presented data of a novel method, known as clone censor weighting, that can evaluate complex clinical questions around optimal disease management over time. In Target RWE’s approach, patients are copied (or “cloned”) into one cohort per treatment protocol that allows for comparing specific treatment sequences observed in the real world that can directly inform clinical treatment decisions.  

Addressing Study Bias with the ‘Clean Room Committee’ Approach   

While there are accepted standards for demonstrating rigor, reproducibility, and transparency in randomized controlled trials, best practices for operationalizing RWE studies intended for regulatory decision-making are still being established. When analyzing RWD, unexpected considerations often arise that require deviation from the pre-specified statistical analysis plan. To address the competing needs of mitigating the potential for bias in these unexpected settings without introducing costly delays, Target RWE has developed and implemented a novel process of an independent Clean Room Committee (CRC). As RWE plays a growing role in regulatory decision-making, principled processes using the CRC are needed to build trust in the evidence without imposing costly delays. 

Examining Healthcare Utilization for High-Risk Non-Muscle Invasive Bladder Cancer 

Research was also presented on real-world evidence in a high-risk non-muscle invasive bladder cancer (HR NMIBC) population using linked registry and claims data. Target RWE’s research uncovered relevant findings applicable to a large number of clinical trials in this space. Specifically, HR NMIBC patients were found to engage extensively with healthcare services, averaging multiple years of follow-up and hundreds of medical and pharmacy encounters per person following the first bladder cancer diagnosis. Linking registry data with administrative claims also provided in-depth insights into HR NMIBC patients’ real-world healthcare utilization over time.  

Utilizing Patient Electronic Medical Records to Transform Clinical Trials  

Phase 3 clinical trials are pivotal in determining the safety and efficacy of new medical interventions prior to their market introduction. However, conventional approaches to these trials often prove to be resource-intensive and time-consuming, failing to fully capture patients' real-world experiences. Target RWE’s presentation explored the transformative potential of integrating Patient Electronic Medical Records (EMRs) into Phase 3 clinical trials, reshaping the landscape of medical research. Target RWE experts discussed the transformation of how electronic medical records are managed, processed, and curated.  

Prediction in large administrative claims databases 

Research also highlighted the challenges and techniques of predictive modeling with large insurance claims databases, using machine learning and AI to address questions such as predicting event likelihood and identifying underdiagnosed patients. The research included case studies on bone fracture outcomes for women aged 50 years or older that informed prediction accuracy metrics and the effectiveness of methods, such as feature construction and inverse-probability weighting to account for missing data. 

Target RWE showcased innovative epidemiologic methods and analyses at both PMSA and ISPOR meetings, offering insights into real-world evidence (RWE) applications across health economics, outcomes research, and data-driven decision-making. From examining healthcare utilization patterns for high-risk non-muscle invasive bladder cancer to exploring the transformative potential of integrating patient electronic medical records into Phase 3 clinical trials, Target RWE's contributions are shaping the future of healthcare research and improving patient outcomes. 

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