When evaluating new therapies for cancer and other diseases, health technology assessment (HTA) bodies are increasingly turning to real-world evidence (RWE) to supplement clinical trial data. However, uncertainties often arise due to limitations in clinical trials, including uncontrolled studies, limited follow-up data, and issues with patent selection and relevance to the target population. Real-world data (RWD) can address these challenges and provide insights into various aspects of routine-care patient populations, clinical effects, disease progression, safety, and cost-effectiveness models. While HTA bodies prefer data from their own regions, the use of international data, especially from the US, has gained acceptance.
In this study, researchers from NICE and Flatiron Health explored critical areas in evaluating new cancer therapies. They first investigated the potential of US electronic health record (EHR)-derived RWD post-FDA approval for treatments evaluated by NICE between 2014 and 2019. The study aimed to bridge evidence gaps and enhance decision-making regarding early drug use.
Subsequently, they conducted a case study showcasing the utility of EHR-derived data in filling HTA evidence gaps. Their objective was not to deepen their understanding of specific drugs but to assess data available to NICE during evaluations and reassessments, gauging its potential to mitigate uncertainties during decision-making processes.
Why this matters
This study uniquely explored the potential of supplementing trial data with real-world insights from US EHR-derived data to apply in decision-making in other countries, offering a nuanced understanding of medication use and outcomes based on earlier access. The research highlights the varying availability of RWD across key healthcare assessment stages, underscoring the need for careful, case-specific evaluation. It also emphasizes the strengths of EHR-derived data, addressing concerns about data relevance and enabling precise patient group categorization. Most importantly, the study showcases the pivotal role of RWE in complementing clinical trial data. It emphasizes the significance of considering population comparability and treatment guidelines in shaping robust healthcare decision-making processes, paving the way for more informed and comprehensive medical choices.