Harnessing Real-World Evidence to Transform Clinical Research: An Expert Perspective

As a founder of NWEH and a pioneer behind the Salford Lung Study, Professor Martin Gibson brings deep clinical insight to the evolving landscape of real-world evidence. In this blog, Martin shares his expert perspective on how RWE is transforming clinical research and improving outcomes across the healthcare ecosystem. 

Introduction 

Real-world evidence (RWE) has rapidly emerged as a pivotal component of clinical research, reshaping the landscape of drug development, and patient care. Pharmaceutical and biotech companies, regulatory agencies, healthcare researchers, and digital health innovators increasingly use RWE to complement traditional clinical trials, drive regulatory approvals, and enhance clinical outcomes. However, it is crucial to understand that real-world trials, which incorporate real-world data into clinical trial platforms, differ significantly from traditional RWE studies. 

What is Real-World Evidence (RWE)? 

RWE refers to clinical evidence derived from the analysis of real-world data (RWD), which includes information collected outside traditional controlled trials. Primary sources include: 

• Electronic Health Records (EHRs)
• Patient registries
• Claims data
• Wearable health devices
• Patient-reported outcomes

While RWD comprises raw, unprocessed patient data from these sources, RWE emerges when RWD is analysed to generate actionable insights. Real-world evidence thus provides a comprehensive view of patient experiences, treatment effectiveness, and healthcare delivery patterns beyond controlled environments. 

Real-World Trials vs. Real-World Evidence 

Real-world trials are prospective studies that incorporate real-world data directly into clinical trial platforms, significantly enhancing traditional clinical trials. This contrasts with conventional RWE, which predominantly relies on retrospective data analysis. Integrating RWD directly into trial platforms provides real-time monitoring, drastically streamlining trial safety and measurement of outcomes. Crucially, it reduces the extensive auditing typically required, as data is captured directly from verified sources such as NHS and other healthcare records. 

Real-world trials offer notable advantages over more traditional studies, including substantial cost savings and the potential to increase the patent life of medicines. These trials are specifically designed for practical real-world scenarios, significantly reducing patient burden and thereby improving participant retention. Enhanced retention minimises patient dropouts and loss to follow-up, resulting in more comprehensive datasets to support regulatory submissions. Furthermore, trials designed with real-world data can inherently possess improved internal and external validity, thereby delivering more generalisable data. Such generalisability streamlines healthcare system uptake by providing insights that are relevant for healthcare funding and health technology assessments (HTA). 

Importantly, real-world trials more accurately reflect the broader patient population, including variations in sex, age, social deprivation, and ethnicity. This inclusivity enhances trial outcomes, providing richer insights into treatment effectiveness across diverse demographic groups, ultimately facilitating more equitable healthcare delivery. 

How Regulators Utilise Real-World Evidence 

Regulatory agencies such as the MHRA, EMA, and FDA are increasingly integrating regulatory-grade RWE into drug approval processes and healthcare decision-making. Examples include: 

• FDA's 21st Century Cures Act (2016): This landmark act encourages the use of RWE for regulatory purposes, allowing data from electronic health records and claims databases to support new drug indications and expanded labelling.
• EMA's Regulatory Science Strategy to 2025: Emphasises the use of RWE to monitor post-authorisation effectiveness, safety, and support adaptive licensing pathways.
• MHRA's Innovative Licensing and Access Pathway (ILAP): Incorporates RWE to accelerate drug approvals and ensure patient-centric outcomes by evaluating therapies in real-world settings.

Regulators acknowledge that high-quality, validated RWE can complement traditional randomised clinical trials (RCTs), enhancing the robustness of evidence presented for licensing decisions. 

Why RWE is Critical in Healthcare 

Accelerating Drug Development & Regulatory Approvals 

RWE supports: 

• Post-market drug surveillance and safety monitoring
• Conditional and accelerated drug approvals
• Assessment of drug effectiveness in diverse populations

Enhancing Clinical Decision-Making & Personalised Medicine 

Clinicians harness RWE to deliver personalised treatments tailored to real-world patient outcomes, thus improving overall healthcare quality. Benefits include: 

• Early detection of adverse drug reactions
• Real-time treatment efficacy monitoring
• Tailored therapeutic strategies based on patient-specific data

Improving Access & Equity in Healthcare 

RWE helps address the underrepresentation of diverse patient populations in traditional clinical trials, promoting health equity. It enables: 

• Comprehensive demographic analyses
• Greater inclusivity in treatment evaluations
• Identification of treatment disparities across patient groups

Reducing Healthcare Costs & Optimising Resource Allocation 

Utilising predictive analytics on RWD can significantly reduce healthcare costs by: 

• Identifying cost-effective treatment strategies
• Reducing hospitalisations through early intervention
• Optimising resource allocation based on real-world insights

NWEH's Role in Leveraging Electronic Healthcare Data for Pharma 

NorthWest EHealth (NWEH) is pioneering the secure and compliant integration of NHS and other electronic healthcare data to generate high-quality, regulatory-grade RWE. NWEH’s ConneXon platform is unique in delivering Real-World Trials by directly incorporating RWE from consented participants into a fully functional EDC. This permits pharmaceutical companies to conduct clinical trials that: 

• Securely access and integrate participant data directly from source
• Streamline regulatory-grade RWE submissions by automatically generating data in industry standard STDM formats
• Drive innovative clinical research through trusted real-world insights

NWEH's collaboration in major studies, including GSK’s Salford Lung Studies and Novartis' Victorian-SPIRIT trial, highlights their expertise and track-record in conducting pioneering real-world trials that demonstrate significant clinical benefits, cost savings, and improved patient outcomes. 

Conclusion 

Real-world evidence is fundamentally transforming healthcare, facilitating smarter drug development, improving clinical decision-making, and providing more equitable patient outcomes. Leveraging electronic healthcare data and advanced analytics, organisations like NWEH ensure the robust generation of regulatory-grade RWE, empowering pharmaceutical companies and healthcare providers to deliver safer, more effective, and personalised care. 

By clearly distinguishing between real-world trials and traditional real-world evidence, stakeholders can better harness the potential of RWD, substantially reducing clinical trial costs, enhancing patient safety, extending patent life, and accelerating healthcare innovation. This collectively benefits to the life sciences industry, healthcare providers and, crucially, accelerates the availability of new medicines to patients.