Designing RWE that drives access

By Prof. Dr Thomas Wilke, expert trainer of the Generating RWE for Optimising Market/Patient Access course.

Why one type of study is not enough

Real-world evidence (RWE) has become a critical component of market access, pricing, and clinical decision-making. Yet there is still a common misconception about how RWE is actually generated and used.

Many assume that a single, well-designed RWE study can answer all relevant questions. In reality, that is rarely the case.

Individual RWE studies are often built to answer one specific research question, focusing on a clearly defined primary outcome. However, decision-makers rarely rely on a single outcome when evaluating a therapy.

What matters is not just the study, but the overall evidence strategy.
 

One study, one question

Like clinical trials, RWE studies are designed around a primary objective.

This means:

• A cost study focuses on healthcare resource use and cost
• An effectiveness study focuses on clinical outcomes
• An adherence study focuses on persistence and compliance

Trying to answer too many questions within a single study often leads to:

• methodological weaknesses
• unclear results
• reduced credibility with regulators and HTA bodies

Well-designed RWE studies are focused, precise, and transparent in what they aim to demonstrate.
 

But decision-makers don’t think in single outcomes

While individual studies are narrow by design, stakeholders are not.

Different decision-makers require different types of evidence:

• Regulators focus on benefit-risk
• HTA bodies assess comparative effectiveness and cost-effectiveness
• Payers look for real-world value and budget impact

No single outcome, and no single study, can satisfy all of these perspectives.
 

The full spectrum of RWE outcomes

Across RWE studies, a broad range of outcomes can be generated, including:

• Epidemiology (incidence, prevalence)
• Treatment patterns
• Adherence and persistence
• Healthcare resource use and costs
• Soft clinical outcomes (e.g. biomarkers, surrogate endpoints)
• Hard clinical outcomes (e.g. survival, major events)
• Patient-reported outcomes (quality of life, symptoms)

Each of these answers a different type of question.
 

From single studies to evidence strategy

The real value of RWE emerges when these different outcomes are generated across a coordinated set of studies.

For example:

• A treatment pattern study shows how patients are currently managed
• A comparative effectiveness study demonstrates clinical benefit
• A cost study quantifies economic impact
• A patient-reported outcome study captures patient relevance

Individually, each study provides a partial answer. Together, they create a coherent and persuasive evidence base.
 

Why this matters for market access

Market access decisions are multi-dimensional.

To achieve reimbursement and adoption, companies must demonstrate:

• clinical value
• economic value
• relevance to real-world populations
• impact on patients’ lives

RWE plays a role across the entire product lifecycle, from early development to post-launch optimisation. This makes it essential to move beyond a “study-by-study” mindset.
 

Common mistake: letting data drive the question

A frequent pitfall in RWE is starting with available data rather than a clear strategic objective. 

This often results in:

• studies that are easy to execute but hard to use
• evidence that does not align with stakeholder needs
• missed opportunities to support key decisions

Instead, the process should be reversed:

1. Define the decision to support
2. Identify the evidence required
3. Design the appropriate RWE studies
    

The takeaway ✔️

RWE is not about running a single study; it is about building a structured evidence strategy where:

• each study has a clear purpose
• each outcome answers a specific question
• all studies together support a broader value story

Companies that approach RWE this way are far better positioned to:

• meet regulatory and HTA expectations
• support pricing and reimbursement
• demonstrate real-world value