What is Real World Evidence?

Real World Evidence (RWE) is a term increasingly being used within the healthcare and pharmaceutical industries, shaping critical decisions around drug approvals, patient treatments, and healthcare policies. But what exactly is Real World Evidence, and why has it become so important?

In this blog, we’ll demystify RWE, explore its applications, and highlight why organisations like ICON are pioneering its use in clinical research.

Real World Evidence refers to clinical data collected outside the tightly controlled environment of randomised controlled trials (RCTs). While traditional clinical trials focus on assessing treatments under strict conditions, RWE is drawn from real-world settings such as patient registries, electronic health records, claims databases, wearable devices, and observational studies.

According to the U.S. Food and Drug Administration (FDA), Real World Evidence is defined as clinical evidence derived from analysing real-world data (RWD) regarding the usage, benefits, or risks of medical products. Real World Data itself refers to information related to patient health status and healthcare delivery routinely collected from various sources.

In short, RWE offers insights into how medicines and medical interventions perform in everyday clinical practice rather than in highly controlled environments.

Real World Evidence is generated from diverse sources, including:

• Electronic Health Records (EHRs): Digital patient records containing comprehensive clinical data.
• Patient Registries: Organised systems that collect uniform data to evaluate outcomes for populations defined by a specific disease, condition, or exposure. 
• Claims and Billing Data: Records from insurance providers detailing healthcare services utilised, diagnosis codes, and prescribed treatments. 
• Wearable and Mobile Devices: Continuous monitoring devices, such as fitness trackers and digital sensors, providing real-time health data. 
• Observational Studies and Surveys: Research studies designed to observe and collect health outcomes without direct intervention by researchers.

Each source offers unique benefits, helping researchers understand patient behaviour, treatment effectiveness, long-term safety, and broader health outcomes.

Real World Evidence has become invaluable to healthcare stakeholders including regulators, healthcare providers, pharmaceutical companies, payers, and patients - offering numerous benefits:

Enhanced Decision-Making Real World Evidence complements traditional clinical trial data by providing additional context. Regulators such as the FDA, European Medicines Agency (EMA), and others are increasingly utilising RWE to inform their decisions about drug approvals, label expansions, and post-marketing surveillance.

Accelerated Drug Development RWE can streamline drug development, accelerating the time-to-market for innovative therapies. By leveraging existing real-world datasets, organisations can identify unmet medical needs, reduce the scope and duration of traditional clinical trials, and swiftly identify optimal patient populations.

Personalised Healthcare Real World Evidence facilitates personalised medicine by highlighting how different patient subgroups respond to therapies in real-life scenarios. This can support tailored treatment strategies, improving patient outcomes.

Cost Efficiency Generating Real World Evidence from existing data sources can be more cost-effective than conducting large-scale clinical trials. Pharmaceutical companies and healthcare providers can utilise RWE to optimise resource allocation, reduce healthcare costs, and enhance economic efficiencies.

While Real World Evidence holds tremendous potential, it is essential to acknowledge challenges and limitations inherent to RWE collection and interpretation:

Data Quality and Standardisation: Real-world data sources are heterogeneous, and inconsistencies in data collection methods and reporting can affect the quality and comparability of evidence generated.

Bias and Confounding Factors: Observational data often carries potential biases due to lack of randomisation, patient selection criteria, and missing information, complicating causality assessments.

Regulatory and Ethical Considerations: Regulators have specific requirements for the acceptability and robustness of RWE, necessitating careful adherence to ethical and privacy standards.

Organisations such as ICON play a critical role in addressing these challenges by applying rigorous scientific methodologies and quality standards to ensure reliability and compliance.

The role of Real World Evidence is rapidly expanding, especially with advancements in technology and data analytics. Experts predict significant growth in the utilisation of RWE in areas like oncology, rare diseases, chronic illnesses, and personalised therapies.

Regulatory bodies worldwide including the FDA and EMA are proactively exploring and implementing frameworks to integrate RWE into approval processes and post-marketing assessments. According to Deloitte, 94% of pharmaceutical leaders now consider RWE essential to their strategy, indicating the increasing importance of RWE in shaping future clinical research and drug approvals.

Moreover, digital innovations such as AI-powered analytics, blockchain technologies, and advanced health tracking devices are expected to further enhance the quality, transparency, and accessibility of Real World Evidence.

Real World Evidence is a transformative force in modern clinical research. It bridges the gap between controlled trial settings and real-world clinical practice, offering essential insights that improve patient outcomes, inform regulatory decisions, and advance healthcare delivery.

At ICON, Real World Evidence helps to enable smarter, faster, and more patient-centric approaches to clinical development.

Understanding Real World Evidence is not only crucial for healthcare professionals and pharmaceutical companies but also vital for patients and the wider healthcare community. By embracing the potential of RWE, ICON and the industry at large are better positioned to deliver effective, safe, and personalised treatments - ultimately improving lives around the globe.