Industry research suggests that a top-20 pharma company could unlock more than $300 million per year by deploying advanced real-world evidence analytics across its value chain. Yet the same research acknowledges that most pharma companies still struggle to deploy these capabilities effectively. Data sits in fragmented systems. RWE functions are dispersed across regulatory, commercial, and clinical development teams. And pilot projects that demonstrate value in isolation fail to scale into enterprise-wide operations.
This gap between potential and execution defines the current moment for large pharma and real-world data. The global RWE solutions market was valued at $2.81 billion in 2024 and is projected to reach $6.01 billion by 2033. The FDA's December 2025 decision to accept de-identified real-world evidence without requiring identifiable patient-level records removed one of the most significant regulatory barriers. And across the industry, investment in RWE infrastructure continues to accelerate.
The question for large pharma is no longer whether to invest in real-world data. It is how to move from isolated pilot projects to integrated, production-scale evidence generation that drives value across the entire product lifecycle.
The Scale Advantage
Large pharmaceutical companies operate at a scale that creates unique opportunities for real-world data. Multi-billion dollar portfolios spanning dozens of therapeutic areas generate an enormous volume of real-world interactions: electronic health records from prescribing physicians, insurance claims across millions of covered lives, patient registries tracking outcomes over years, and increasingly, data from wearables and digital health tools.
This scale advantage is significant. IQVIA maintains over 1.2 billion de-identified patient records globally. The Sentinel System, developed in partnership with the FDA, now covers more than 100 million lives. Large pharma companies with established partnerships across these data ecosystems can access longitudinal, linked datasets that would be impossible for smaller organizations to assemble.
But scale also creates complexity. Many pharma companies centralize their RWE functions in global medical affairs, but the actual capabilities for study design and data management are often dispersed across regulatory, commercial, and clinical development teams. Multiple business units may each maintain separate data governance standards. Without a unified strategy, even the most sophisticated data infrastructure can produce fragmented insights that fail to inform strategic decisions.
Where RWD Delivers Value Across the Lifecycle
Label expansion. Perhaps the most commercially significant application for large pharma, RWE can support supplemental applications for new indications without conducting additional clinical trials. A landmark example: Pfizer’s 2019 label expansion of Ibrance for male breast cancer patients was approved based on real-world evidence from electronic health records and post-marketing reports sourced from the IQVIA Insurance database, Flatiron Health Breast Cancer database, and Pfizer’s global safety database. Among 218 labeling expansions granted between January 2022 and May 2024, researchers identified RWE in FDA documents or related publications for 55 approvals.
Comparative effectiveness. Payers increasingly demand evidence that a product performs well not just in controlled trial settings, but in routine clinical practice across diverse patient populations. RWE provides the foundation for demonstrating real-world effectiveness versus competitors, supporting formulary positioning and payer negotiations. As value-based care models expand, this evidence becomes critical for reimbursement and market access.
Post-market safety surveillance. Clinical trials, by design, exclude many patients who will eventually use a product in routine care. Older patients, those with multiple comorbidities, and those on complex medication regimens are frequently underrepresented. Real-world data captures the safety profile across these broader populations. This is particularly important for identifying rare adverse events that may not emerge until millions of patients have been treated.
Clinical trial optimization. RWE is increasingly used to inform trial design before enrollment begins: identifying feasible endpoints, refining eligibility criteria, selecting trial sites based on real patient populations, and constructing external control arms. PwC reports that RWE led to a 40% reduction in the planned sample size of a pivotal Phase III program, translating into six months of time savings. For companies facing patent cliffs, with over $300 billion in sales at risk by 2030, accelerating development timelines has direct financial impact.
Health economics and outcomes research (HEOR). Large pharma portfolios require continuous evidence generation to support pricing and market access across multiple geographies. RWE provides insights into treatment patterns, resource utilization, and economic outcomes that inform health technology assessment submissions globally.
The Regulatory Landscape Is Accelerating
The FDA’s December 2025 announcement represents a watershed moment. By accepting de-identified real-world evidence without mandating identifiable patient-level data, the agency removed what many sponsors and data scientists considered the primary barrier to meaningful RWE use in regulatory submissions. While this initial guidance applies to medical devices, the FDA has stated it intends to extend the approach to drugs and biologics.
This builds on several years of expanding regulatory acceptance. The FDA’s Advancing Real-World Evidence Program now accepts up to four primary meeting requests per submission cycle, providing formal pathways for sponsors to discuss RWE protocols before submission. The FDA’s RWE guidance explicitly lists applications beyond post-market surveillance: training AI/ML algorithms, supporting label expansion, creating historical controls, and generating primary clinical evidence for marketing authorization.
The CDER Center for Real-World Evidence Innovation (CCRI), established in December 2024, further signals that the FDA views RWE not as an alternative to clinical trials, but as a complementary source of evidence that strengthens the overall regulatory framework.
Building Production-Scale RWE Operations
Moving from pilot projects to production-scale RWE requires investment in three areas.
Data infrastructure and governance. Unified data architectures that connect clinical, claims, registry, and digital health data across therapeutic areas. Standardized quality frameworks that ensure consistency across studies. And clear governance models that define data access, lineage, and documentation standards. PwC anticipates spending on data products to increase at an 8% to 10% CAGR through 2028, with integrated, linked datasets leading the segment.
Cross-functional integration. RWE delivers the most value when it spans the entire product lifecycle, from informing R&D portfolio decisions through supporting commercial launch and post-market evidence generation. This requires breaking down silos between clinical development, medical affairs, commercial, and market access teams. Organizations that relegate RWE to a standalone function miss opportunities to feed insights back into earlier-stage decisions.
Strategic partnerships. The RWE ecosystem has expanded significantly, with vendors offering increasingly specialized capabilities by therapeutic area, data type, and analytical methodology. Companies like IQVIA, Flatiron Health, and Datavant continue to invest in integrated platforms. Large pharma should manage a portfolio of RWE partners rather than concentrating relationships, matching specialized vendors to specific therapeutic needs while maintaining vendor-agnostic internal capabilities.
Looking Ahead
The convergence of expanding data sources, advancing analytics capabilities, and growing regulatory acceptance is creating a moment of inflection for large pharma’s RWE strategies. AI-powered platforms are enabling faster and more sophisticated analysis of unstructured clinical data. Linked datasets combining claims, EHR, genomics, and patient-reported outcomes are providing increasingly complete pictures of treatment journeys. And regulators globally are establishing clearer frameworks for how RWE can support decision-making.
For large pharma companies, the competitive advantage will belong to those who treat real-world data not as a regulatory compliance exercise, but as a strategic asset that informs decisions across the entire organization. The infrastructure, governance, and partnerships that companies build now will determine their ability to capture value from this rapidly evolving landscape.
Exploring how real-world data could strengthen your evidence strategy? Schedule a consultation to discuss your specific opportunities.
In the next post in this series, I’ll explore how health technology companies are generating evidence at unprecedented scale through digital health data, connected devices, and patient-reported outcomes.
Sources
- Grand View Research. (2025). Real World Evidence Solutions Market Report (2025-2033). https://www.grandviewresearch.com/industry-analysis/real-world-evidence-solutions-market-report
- McKinsey & Company. (2020). Creating Value from Next-Generation Real-World Evidence. https://www.mckinsey.com/industries/life-sciences/our-insights/creating-value-from-next-generation-real-world-evidence
- McKinsey & Company. (2022). Generating Real-World Evidence at Scale Using Advanced Analytics. https://www.mckinsey.com/industries/life-sciences/our-insights/generating-real-world-evidence-at-scale-using-advanced-analytics
- FDA. (2025). FDA Eliminates Major Barrier to Using Real-World Evidence. https://www.fda.gov/news-events/press-announcements/fda-eliminates-major-barrier-using-real-world-evidence-drug-and-device-application-reviews
- AJMC. (2026). FDA to Accept De-identified Real-World Evidence for Select Medical Device Applications. https://www.ajmc.com/view/fda-to-accept-de-identified-real-world-evidence-for-select-medical-device-applications
- PwC. What Is the Value of Real-World Data in Pharma. https://www.pwc.com/us/en/industries/health-industries/library/real-world-evidence-pharma.html
- FDA. Advancing Real-World Evidence Program. https://www.fda.gov/drugs/development-resources/advancing-real-world-evidence-program
- Deng YF, et al. (2025). Real-World Evidence in FDA Approvals for Labeling Expansion. Ther Innov Regul Sci. 59(5):982-992. https://pmc.ncbi.nlm.nih.gov/articles/PMC12446098/
- Newristics. (2025). How Real-World Evidence Is Transforming Personalized Patient Care and Pharma Strategies. https://newristics.com/how-real-world-evidence-rwe-is-transforming-personalized-patient-care-and-pharma-strategies.php
- FDA. FDA Use of Real-World Evidence in Regulatory Decision-Making. https://www.fda.gov/science-research/real-world-evidence/fda-use-real-world-evidence-regulatory-decision-making
- Evaluate. (2025). 2025 World Preview: Pharma Growth Steady Amid Turbulent Seas. https://www.evaluate.com/thought-leadership/2025-world-preview/
- FDA. (2024). CDER Center for Real-World Evidence Innovation (CCRI). https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/cder-center-real-world-evidence-innovation-ccri
