In today's healthcare environment, securing regulatory approval is only the beginning. The real challenge lies in ensuring medicines reach the people and communities who need them the most. Access and reimbursement are where the promise of new therapies is truly put to the test.

Here, real-world evidence (RWE) and patient experience prove invaluable, offering new ways to demonstrate therapeutic relevance in routine care. While randomized controlled trials (RCTs) remain the gold standard for establishing safety and efficacy, they don’t always capture the full complexity of how treatments work in everyday clinical practice. These trials typically include homogenous, carefully selected populations under controlled conditions with brief follow-up periods.

Participants rarely reflect the diversity of those who may ultimately benefit from therapy. In contrast, RWE can help us understand how treatments perform against different comparators and across varied patient populations and real-life settings, including underrepresented groups, people from different socioeconomic backgrounds, rural and remote communities, and people with multiple chronic conditions.

Historically, the strategic value of RWE has been underestimated. RWE must be treated as central to transforming scientific innovation into funded, routine care. When used effectively, RWE informs clinical trial design and regulatory decisions, supports payer and health technology assessments, guides clinical decision-making, and enables expanded access programs. It's the bridge between clinical promise and patient reality.

Redefining RWE

RWE encompasses a wide array of clinical evidence derived from real-world data sources, electronic health records, patient registries, disease surveillance programs, digital health technologies, and patient-reported outcomes.

This integration of diverse data sources often provides a more complete picture of disease burden and unmet needs across actual patient populations. RWE captures the full spectrum of patient heterogeneity, including differences in age, comorbidities, socioeconomic status, and geography that significantly impact how diseases manifest and how patients respond to treatment. RWE sheds light on standards of care and treatment patterns across health systems, showing us how medicine is truly practiced.

At Takeda, large-scale RWE efforts in conditions such as narcolepsy and multiple myeloma have been crucial in informing trial design and endpoint selection, and in deepening our understanding of patient journeys.

RWE enables us to more accurately illustrate how therapies perform in everyday settings across broad populations, thereby creating a fuller picture of a therapy's true clinical importance.

Driving regulatory confidence and clinical insights

RWE serves as a vital complement to RCTs, offering insights that can support clinical practice. Often capturing adherence patterns and real-world utilization metrics that trials cannot fully replicate—such as hospitalizations prevented, quality-of-life improvements, and long-term healthcare resource use—which are essential in evaluating whether a treatment delivers sustainable healthcare value.

Beyond supporting initial approvals, RWE can also support the case for approved label expansions that increase patient access to promising therapies. By combining clinical trial data with real-world data, researchers can fill gaps for patient subgroups not represented in initial studies and uncover unique insights about a treatment’s utility that may not be evident in controlled trials.

RWE also strengthens pharmacovigilance efforts, offering a comprehensive and realistic view of drug safety across larger, more diverse populations than trials could ever capture. In fact, regulators are encouraging adoption of RWE for ongoing pharmacovigilance and post-marketing obligations. Updated FDA guidance and new electronic safety reporting standards aim to strengthen signal detection and transparency, making real-world data a critical tool for meeting these expectations.

By integrating trial data with RWE, we can address gaps for underrepresented patient subgroups and uncover insights that controlled studies may have missed. This enables proactive identification and management of safety signals, supporting regulatory decision-making in the post-marketing phase and building long-term confidence in a therapy’s benefit-risk profile.

Industry collaborations illustrate this impact. For example, partnerships between pharmaceutical companies, local governments, and health authorities, such as Takeda’s work with ministries of health on public vaccination programs, leverage RWE to enhance pharmacovigilance and ensure innovations meet the needs of communities effectively.

In these programs, near‑real‑time analyses of adverse event trends, stratified by age, comorbidities, and geography, can guide tailored communication to providers and patients and support rapid refinement of risk‑management plans. Importantly, the same infrastructure that supports rigorous safety monitoring can be used to generate effective data in everyday practice, informing decisions about booster strategies, targeted outreach, and resource allocation.

These untapped insights, when integrated appropriately within clinical development, represent an enormous opportunity to accelerate access and better serve patient needs.

Shaping a clear value narrative

For payers and health systems operating under increasing budget constraints, the fundamental question extends beyond clinical efficacy to sustainable utilization: Will this treatment deliver meaningful outcomes for our specific patient population within our budget? RWE transforms how we answer this critical question, providing the comprehensive evidence needed to support compelling, well-defined value propositions that resonate with healthcare providers and payers.

At the clinical level, RWE brings clarity to many questions physicians grapple with daily: Which patients will benefit most from this treatment? How does it perform in complex populations with multiple comorbidities? What adherence patterns can we expect in everyday practice? These insights enable more informed, personalized treatment decisions, helping clinicians match the right therapy to the right patient at the right time.

Navigating challenges and embracing opportunities

Generating RWE at scale presents real challenges that our industry must address. Data privacy remains paramount, with regulatory frameworks varying significantly across regions. Database fragmentation can complicate pooling data across sources and geographies, and standardization challenges persist as stakeholders have different objectives and requirements. Aligning methodologies to meet these diverging needs while maintaining scientific rigor requires ongoing effort.

While these challenges aren't insurmountable, cross-sector collaboration is essential. Partnerships with academic institutions, technology companies, and healthcare systems can pool expertise and resources to overcome fragmentation and build robust data infrastructure. Artificial intelligence and other digital tools also offer significant potential for analyzing complex datasets and distilling actionable insights. However, they must be deployed thoughtfully with appropriate validation and human oversight.

Ultimately, for RWE to support access and reimbursement decisions, it must be trusted. We need to ask the right scientific questions, use the appropriate methodology, and clearly communicate the output.

The path forward

Today, our methods for evidence generation stand at an inflection point. We need trials that truly reflect the patients who need our medicines. We need evidence that answers the questions payers and clinicians actually ask. We need data that bridges the gap between regulatory approval and clinical practice. In these ways, RWE has evolved from a supplementary tool to a strategic imperative.

With this vision, we can create a healthcare landscape where evidence-driven insights lead to more timely and broad access to groundbreaking innovations. Where every patient, regardless of their circumstances, can benefit from therapies that were designed with them in mind and proven to work in their world.

Awny Farajallah, MD, is Chief Medical Officer and Head of Global Medical at Takeda