Real World Evidence Revolutionizing Drug Development and Healthcare
Real world evidence (RWE) has become increasingly important in the pharmaceutical and life sciences industry in recent years. With large volumes of real-world patient data now available from electronic health records, claims and billing data, patient registries, and other healthcare data sources, companies are able to generate RWE to supplement results from clinical trials. This data provides insights into how treatments are used and their effectiveness outside of tightly controlled clinical trial settings.
RWE is being utilized across the product lifecycle from drug discovery and development through commercialization. In the early R&D phase, RWE can help identify new targets and biomarkers. It also allows companies to evaluate the benefits and risks of interventions in broader patient populations than typically enrolled in clinical trials. Regulators such as the FDA also accept RWE for expanding drug labels, demonstrating long-term safety and effectiveness, and supporting reimbursement decisions.
On the commercial side, RWE provides insights into treatment patterns, adherence rates, and health economic outcomes important for reimbursement, pricing, and access strategies. It allows pharmaceutical companies and payers to evaluate the real-world benefit of therapies compared to alternatives. As the use of value-based contracts grows, RWE demonstrating improved clinical and economic outcomes is essential for pharmaceutical companies.
Growth Outlook
Growth is being driven by the increasing adoption of RWE by pharmaceutical companies, payers, and regulators globally. Many of the world's largest pharmaceutical firms such as Pfizer, Roche, Johnson & Johnson, Novartis, and Merck have significantly increased their investments in internal RWE capabilities as well as partnerships with data and analytics companies. Large contract research organizations including IQVIA, PPD, Syneos Health, and Parexel have also built out RWE practices and capabilities to serve the needs of biopharma clients.
On the payer side, insurers are recognizing the value of RWE to understand variations in care, evaluate treatment effectiveness, and inform coverage and reimbursement decisions. Groups such as Aetna, Cigna, and UnitedHealth have invested heavily in data and analytics capabilities incorporating real-world health data. Regulators around the globe have also provided increased acceptance of RWE to support regulatory submissions and decision making.
Data and Technology Transforming RWE Landscape
The growth of RWE is being enabled by the exponential growth and availability of healthcare data as well as advancements in data analytics technologies. Today there are over 1,500 known data sources globally containing real-world data, compared to just several hundred five years ago. Within the United States alone, over 80% of physicians use electronic medical records systems producing vast amounts of structured and unstructured patient data.
Technological advances especially in the fields of artificial intelligence, machine learning, and natural language processing have made it possible to generate insights from the petabytes of data being generated. These technologies power solutions for tasks such as identifying patient populations, data extraction, outcome identification, pattern detection, predictive modeling, and generating evidence reports. Partnerships between analytics companies, health systems, and data organizations are increasing amount of data available for RWE generation.
RWE Use Cases Expanding
As RWE methodologies mature and data availability expands, the scope of RWE use cases is growing across the product lifecycle:
- Drug Development - Identification of targets, biomarkers, and generation of real-world evidence for labeling expansions.
- Pricing & Access - Evidence of clinical and economic value for reimbursement, formulary listing, and value-based contracts.
- Safety Surveillance - Long-term monitoring of adverse events for risk identification and management.
- Clinical Trial Recruitment - Identification and targeting of appropriate patient populations.
- commercialization - Evaluation of treatment patterns, adherence, effectiveness and impact on outcomes.
- Product comparisons - Head-to-head assessments of treatments relative to standard of care.
- Healthcare Policy - Public health evaluation, quality measurement, and health system optimization.
Growing Acceptance by Regulators
All major global regulators, including FDA, EMA, and PMDA, have established frameworks and guidance accepting RWE to varying extents. In the United States, FDA has established mechanisms such as Real-World Data Plan, RWE Program, and use of data from the Sentinel System and FDA's National Evaluation System for health Technology (NEST). The 21st Century Cures Act has also allowed use of RWE for pediatric studies and label expansion in certain circumstances.
The European Medicines Agency (EMA) initiated its Parallel Scientific Advice program in 2018 to provide a platform for concurrent advice from multiple EMA committees on RWE strategy and methodology. The Pharmaceuticals and Medical Devices Agency (PMDA) in Japan similarly provides scientific advice on use of RWE for regulatory submissions. These global guidelines solidify the role of RWE as a critical data source for both regulatory decision making and post-approval evaluation of therapies.
Future Outlook
Going forward, pharmaceutical companies are expected to significantly expand their use of RWE with life sciences leaders dedicating 5-10% of total R&D budgets specifically to RWE generation by 2025. Advancement and standardization of methodologies as well as new data sources will increase reliability and trust in RWE. However, challenges around data quality, contextualization, and real-world versus controlled study design will require ongoing attention. Partnerships between industry, academia, healthcare systems, and technology leaders will be critical for maximizing RWE potential to transform drug development and optimize patient care.