Real-World Evidence – A comprehensive guide to its application

Real-world data is a term that is used to refer to data that is sourced from a wide variety of channels, including electronic health records, patient and disease registries, insurance claim submissions, reports from patients and clinicians, connected health devices, wearables and digital therapeutic solutions (DTx). The data that is collected from these sources has unstructured qualitative information, which includes both narrative notes and patient-reported experiences. The input data reveals how treatments affect the daily lives of patients by showing the effects of treatment that go beyond conventional safety and efficacy assessments. Medical therapy assessment now requires complete evidence according to regulatory agencies and reimbursement bodies and health technology assessment organizations.

Understanding the value of real-world evidence for key stakeholders

Real-world evidence (RWE) reveals entirely new demographic findings and treatment options from real-world data that standard clinical trials miss. A well-known example is the breast cancer drug Ibrance’s label, which received expanded into male patient approval through real-world data analysis. RWE reveals unmet medical requirements and shows different treatment applications and enables betterment of current medical guidelines.

From the clinical and healthcare management perspective, RWE analyzed in real or near-real time can expose weaknesses or inefficiencies in established treatment pathways. These insights enable providers to optimize processes and elevate patient outcomes through more targeted care strategies.

Investors and pharmaceutical developers also significantly benefit from RWE. By signaling assets that may deliver low or marginal clinical value before major investments are made, RWE can help prevent costly development failures. Since private capital plays a crucial role in bringing new therapies to market, minimizing risk can be a decisive advantage.

Additionally, real-world evidence strengthens the potential for positive reimbursement decisions, including inclusion in preferred formularies. This can be particularly critical for innovative treatments and digital health technologies that rely on payer support for commercial success. Real-world evidence plays an increasingly critical role in shaping commercial decisions, strengthening payer negotiations and informing sales effectiveness consulting strategies that drive competitive advantage.

Designing and applying an effective real-world evidence strategy

A strong RWE plan begins with a comprehensive scenario assessment to identify needs that are not met within the target population and outline initial economic model priorities. Based on this, measurable endpoints can be defined. For example, when developing digital therapeutics or connected devices, relevant endpoints can reduce patients’ disease burden or improve physicians’ workflow efficiency.

Once these metrics demonstrate value, economic models can be refined and pricing evaluations completed. A critical success factor is ensuring the value proposition resonates across all stakeholders, not just one segment. Engaging expert communication teams early can help articulate cross-stakeholder value effectively.

Leadership considerations in real-world evidence execution

Implementing a real-world evidence initiative is inherently complex. Large organizations often involve multiple global and regional teams with differing priorities, budgets, structures, vendor contracts and access rights. RWE programs might be driven by clinical, regulatory, commercial, medical affairs or safety groups and alignment across these functions can be difficult.

Despite these challenges, the benefits far outweigh the hurdles. Successful RWE leadership requires resilience, collaboration and strategic vision.