Leveraging Real-World Evidence Through Primary Research

In an industry where clinical trials have traditionally been the gold standard for evidence generation, pharmaceutical organizations are increasingly recognizing the value of real-world evidence (RWE). As regulatory bodies, payers, and healthcare providers demand more data on the potential of therapies to perform outside tightly controlled clinical settings, RWE has become a critical component of pharma strategy. While claims databases, electronic health records (EHRs), and registries provide a wealth of retrospective data, primary research offers a complementary and often necessary approach to capturing nuanced insights that secondary data sources simply cannot provide.

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The Growing Importance of Real-World Evidence

Real-world evidence is reshaping how pharmaceutical organizations approach everything from drug development to commercialization. RWE can provide insight into how treatments perform in diverse patient populations, under varied conditions, and over extended periods. Relying solely on retrospective data sources has limitations, primary research can play a critical role in filling gaps.

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Limitations of Secondary Data Sources

Claims databases, EHRs, and registries for RWE offer extensive patient records but come with significant challenges:

• Data Gaps & Incompleteness: Claims data do not capture patient-reported outcomes, reasons for treatment discontinuation, or the full picture of disease burden.
• Lagging Indicators: Retrospective analyses often rely on older data that may not reflect the latest treatment trends.
• Lack of Context: While secondary data can show what is happening, it often fails to explain why specific patterns emerge in prescribing, adherence, or disease progression.

To bridge these gaps, pharmaceutical organizations can integrate primary research methodologies to collect real-time insights directly from healthcare providers, patients, and payers.

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The Value of Primary Research in Real-World Evidence

Primary research offers a proactive approach to evidence generation by capturing perspectives that secondary data sources cannot. Through primary research, pharmaceutical organizations can:

1. Capture Patient and Physician Perspectives

Clinical trials provide structured efficacy data, but they do not always reflect how patients experience treatments in their daily lives. Primary research allows organizations to collect patient-reported outcomes, adherence challenges, and quality-of-life measures that are often missing from claims data. Similarly, physician interviews and surveys can shed light on real-world prescribing behaviors, treatment hesitations, and the impact of clinical guidelines on decision making.

2. Understand Unmet Needs and Treatment Gaps

Real-world data can indicate suboptimal outcomes, but primary research helps uncover why these gaps exist. Understanding the root cause of observed trends enables pharmaceutical organizations to address barriers and improve patient outcomes.

3. Inform Post-Marketing and Lifecycle Management Strategies

The role of RWE extends beyond regulatory approval. As new competitors enter the market and treatment guidelines evolve, pharmaceutical organizations must continuously adapt their strategies. Primary research enables ongoing data collection from key stakeholders to refine messaging, optimize market positioning, and identify opportunities for label expansion or new indications.

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How to Collect Real World Data Utilizing Primary Research Techniques

Compared to traditional marketing research which often focuses on brand perception, messaging, effectiveness, or competitive dynamics, primary research in the RWE setting is fundamentally different in both its purpose and methodology. While some familiar research tactics may still apply, the emphasis shifts toward understanding real-world experiences, uncovering barriers to treatment, and contextualizing retrospective data.

Selecting the right method of data collection is critical to moving beyond simply identifying “what is happening” to exploring “why” it is happening and how the healthcare ecosystem can improve outcomes. Depending on the objectives at hand, different primary research techniques should be leveraged:

Qualitative In-depth Interviews (IDIs)

How they work:

• 1-on-1 interviews conducted with patients, caregivers, HCPs, payers or other stakeholders to explore treatment experiences, decision-making rationales, and adherence challenges.
• An open-ended, conversational format allows for deeper insights into personal experiences that secondary data sources do not capture.

When to use them:

• Understanding why certain prescribing patterns, adherence challenges, or treatment preferences exist in the real world.
• Exploring perceptions of treatment burden or side effect management among patients.
• To capture decision-making factors when selecting treatments or switching therapies.

Online Surveys

How they work:

• Structured questionnaires are deployed to relevant participants to gather insights on items such as treatment experiences, preferences, and outcomes providing data that can be projected to the larger population.
• Surveys can be cross-sectional (single point in time) or longitudinal (tracking respondents over time) to assess trends in real-world treatment and use.

When to use them:

• To measure patient-reported outcomes (PROs), treatment satisfaction, or quality-of-life metrics at scale.
• When looking to develop quantifiable data or to validate trends observed in claims or EHR data.
• To identify variations in treatment perceptions across different specialties, geographies, or across social determinants of health (SDoH).

Ethnographic Research

How it works:

• Digitally documenting patient treatment experiences in real time using mobile apps, video diaries, or structured forums to track items such as symptoms, medication usage, and lifestyle impact.
• In-person ethnographic techniques continue to be a viable method for data collection and involve specifically trained researchers observing patient-HCP interactions and patients at home in a real-world setting.

When to use it:

• When there is a need to capture real-time, unfiltered patient experiences beyond structure surveys or interviews.
• Understanding daily disease burden, the impact on activities of daily living (ADLs) and how a treatment may fit into a patient’s lifestyle.
• Identifying barriers to adherence that may not be reported in traditional clinical settings.

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Hybrid Quantitative and Qualitative Approaches

How they work:

• Combines techniques within one respondent interaction or across phases of research to provide both rigor and depth.
• For example, combining structured surveys with follow-up qualitative interviews can lead to both statistical findings and contextual understanding from the same study.
• Initial survey findings can then inform deeper qualitative exploration (or vice versa).

When to use them:

• When both statistical validation and contextual depth are needed to interpret findings or tell the intended story.
• Exploring complex patient pathways or decision journeys where survey responses alone often lack the necessary nuance to be impactful.
• Understanding regional or specialty-specific treatment variations by validating insights across different provider types.

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Best Practices for Integrating Primary Research into RWE Strategies

To maximize the impact of primary research within an RWE framework, the following best practices should be considered:

• Combine Quantitative and Qualitative Approaches when Feasible: Online surveys can provide broad insights, while qualitative methods uncover deeper contextual factors influencing treatment decisions.
• Leverage Mixed Stakeholder Inputs: Engaging both HCPs and patients ensures a 360-degree view of treatment dynamics.
• Incorporate Longitudinal Studies: Tracking patients and providers over time reveals evolving perceptions and adherence patterns that cross-sectional data cannot capture.

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Ensuring Rigor in Sample Design & Collection

Credibility of any RWE study depends on the rigor of data collection and sampling design. A robust and appropriately stratified sample frame is critical to ensure that findings are valid, reproducible, and actionable. Ensuring a representative sample and minimizing bias are critical for meaningful insights.

When collecting data with the intent to publish, Institutional Review Board (IRB) approval or exemption can enhance the credibility of findings and increase acceptance among academic audiences. A well designed, and when appropriate, IRB approved study is more publishable in peer-reviewed journals than those that are not.

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Get Your Timing Right (and Your Objectives)

The ideal timing for integrating RWE and primary research into your strategy depends on your product’s development stage and the specific gaps that need to be addressed.

• Early Development - Pre-launch – This is the time to start thinking about identifying unmet needs and the current treatment gaps that can inform clinical trial design. In addition, understanding real-world treatment dynamics that might not be captured in controlled clinical trial settings and potential barriers to uptake (e.g., patient burden, hesitation) can help to build your medical strategy and get ahead of any challenging launch dynamics.
• Post Approval & Launch Preparation – Real-world patient experience data that strengthens your value proposition can support payer negotiations. Real-world effectiveness and experiences adds color to your messaging and can expand the educational materials within your armamentarium.
• Post-Launch & Lifecycle Management – As competitors theoretically enter your market, staying ahead on impactful data that can move the needle is critical. RWE provides a significantly more cost and time effective approach to generating these data than supplemental trials. It can also act as a springboard in identifying opportunities for label expansion or novel indications as lifecycle management becomes a priority.

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Bringing it all Together

Real-world evidence is no longer optional, it is a strategic imperative for pharmaceutical organizations seeking to demonstrate value, secure market access, and optimize patient outcomes. While secondary data sources provide valuable insights, they are inherently limited in scope and depth. By integrating primary research into your RWE strategy, pharmaceutical organizations can gain a deeper understanding of patient experiences and HCP decision-making. In an era where data-driven decision-making is critical, those who harness the full power of RWE are positioned for the best long-term success.

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