eal-world evidence (RWE) has taken center stage in the biopharma landscape, offering insights into drug applications and therapeutic impacts in real-world settings. As the field evolves, understanding the present use of RWE and anticipating its future trajectory is key. At DIA’s recent RWE Conference, stakeholders and experts from the pharmaceutical industry, regulatory bodies, and academia convened. Various questions surrounding RWE were broached, addressing concerns about data quality and access, compliance, and the application of emerging technologies.

5 Key RWE Trends

RWE provides the pharmaceutical industry insights beyond those of typical clinical trials, elucidating drug safety, effectiveness, and potential novel applications. Regulatory bodies are beginning to adapt their processes based on RWE, especially when traditional trials are not the most practical or pose ethical challenges. For instance, in the case of rare diseases where conducting large-scale clinical trials is logistically challenging and time-consuming, regulatory agencies like the FDA have started accepting real-world data to evaluate the safety and efficacy of orphan drugs, expediting the approval process.

Nonprofits are also starting to gravitate towards using RWE to understand patient experiences, guide policy, and shape interventions. One notable example is the Friends of Cancer Research Real-World Evidence portfolio that was developed to establish methodology for using real-world data (RWD) to showcase benefit to patients. One outcome of this project was the development of a white paper, Establishing a Framework to Evaluate Real-World Endpoints.

Moreover, health policy decision-makers leverage RWE amid rising healthcare costs and the need for personalized medicine by influencing treatment guidelines and reimbursement frameworks. An example of this can be seen in the adoption of RWE by the National Institute for Health and Care Excellence (NICE) in the UK, which integrates RWD to make informed decisions about drug pricing, access, and treatment recommendations, ensuring that patients receive the most effective and cost-efficient care.

It is crystal clear: RWE goes beyond just collecting data; it’s about transmuting information into actionable healthcare strategies with transformative potential. As its significance and wider acceptance grows, the imperative for increased collaboration becomes even more critical, ensuring that all stakeholders coalesce their efforts as the RWE landscape continues to advance.

2. Growing Importance of Data and Methodology:
The increasing significance of RWE in healthcare is largely due to the data it’s built upon and the methodology utilized for analysis. As RWE becomes a robust tool for stakeholders, the underlying data and the methods used to interpret it grow in importance.

Ensuring data quality is the cornerstone of credible RWE. High-quality data (which is accessible, interpretable, relevant, and accurate) is fundamental to deriving insights that are both meaningful and actionable. Incomplete or inconsistent data can lead to erroneous conclusions, potentially affecting patient care, policy decisions, and industry practices. As the healthcare landscape becomes more data-driven, the need for rigorous data validation, cleansing, and standardization processes becomes further elucidated.

Furthermore, the methodology used in analyzing this data is equally paramount. Given the diversity of potential use cases for RWE, pinpointing the appropriate analytical methods is critical. Different scenarios may require different analytical techniques, and using an unsuitable method could skew results or offer insights that are not contextually relevant. For instance, while some RWE studies might benefit from advanced machine learning (ML) algorithms to discern patterns, others might be better served by more traditional statistical analyses. As RWE continues to gain ground in the healthcare sector, the emphasis on data quality and rigorous, context-appropriate methodology is key.

3. Landmark Legislation: The 21st Century Cures Act
Signed into law in December 2016, the 21st Century Cures Act was designed to expedite the development and approval of new medical therapies. It introduced reforms in the clinical trial process, bolstered biomedical research, and pushed precision medicine forward. A significant element of the act was its definition of RWE as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials.”

This required FDA to devise a comprehensive framework and guidelines for incorporating RWE in regulatory decision-making, a strategic approach to standardize RWE use and thereby ensure its credibility and reliability. The framework clarifies that RWE is clinical evidence derived from analyzing RWD applications and potential impacts of medical products. In contrast, RWD is routine patient health data garnered from various sources. When making regulatory decisions, the FDA evaluates RWD’s reliability and relevance, as well as the methods used to produce RWE from this data. This act has encouraged the FDA to explore how RWE might support the approval of new uses of already approved drugs or meet the needs of post-approval studies or both.

Traditionally, randomized controlled trials (RCTs) are the gold standard for procuring evidence to support medical product approvals. However, the act acknowledges the significance of RWE, sourced from diverse outlets like patient registries and electronic health records (EHRs), and emphasizes its integration into the drug development process. RWE not only offers insights into the real-world safety and efficacy of treatments, but also streamlines certain processes, reducing the overhead associated with conventional trials. This can lead to faster patient access to treatments and decreased drug development costs, particularly when considering the time-consuming and resource-intensive nature of traditional RCTs.

In simpler terms:

• RWD is the raw data collected from real-world settings.
• RWE is the clinical evidence or insights derived from analyzing those/that raw data.

4. Global Expansion and Integration of RWE
RWE is not just a buzzword in one region or country; its relevance is increasingly recognized on a global scale. Key organizations that regulate prescription drugs such as the FDA and EMA have been at the forefront of this movement.

Representatives from these regulatory bodies have strengthened efforts in weaving RWE into their decision-making protocols. Such integration is seen as a pivotal step to ensure that regulatory processes benefit from a wider array of data sources, not only traditional clinical trials. RWE, being derived from real-world scenarios, provides insights that can help make informed, patient-centric decisions.

However, the globalization of RWE comes with its own set of challenges, one of the most common being data access harmonization across geographic regions. Healthcare systems, data collection protocols, and patient experiences can vary widely from one country to another, or even within regions of the same country. Therefore, to truly realize the potential of RWE on a global scale, there is a pressing need to standardize how data is accessed and interpreted across these diverse geographical regions. Such harmonization would ensure that insights derived from one region are applicable and valuable to another, to create a more collaborative and integrated global healthcare landscape.

Regulatory agencies across the globe are not only integrating RWE into their processes but also emphasizing the critical importance of harmonizing data access worldwide. This focus on integration and harmonization underscores the potential of RWE to revolutionize healthcare on a global scale.

5. RWE Roadmap
The concept of an RWE roadmap seeks to construct a strategic framework for the integration and utilization of RWE throughout the lifecycle of medical products and interventions. At its core, the roadmap acknowledges the multifaceted challenges and vast opportunities that RWE presents.

While building an RWE roadmap, standardizing data collection poses challenges due to the real-world diversity of different data sources. Combined with the distinctiveness of emerging markets—characterized by varied healthcare infrastructures, patient demographics, and disease prevalence—this diversity can lead to heterogeneity in data. Moreover, when compounded by challenges in quality, consistency, and integration of EHRs, patient-generated health data, and other disparate data sources, overcoming these obstacles is a daunting yet essential task for ensuring the robustness and reliability of RWE.

However, the roadmap also underscores the immense potential of RWE, especially when recognized early in clinical development. Harnessing RWE from the get-go can offer insights that traditional clinical trials might miss, potentially accelerating product development and regulatory approvals.

Conclusion
Discussions held at #RWE2023 play a vital role in updating stakeholders on RWE advancements, collaborations, and global initiatives. The interdisciplinary collaboration, seen in the pharmaceutical, regulatory, and nonprofit sectors, reflected a shared commitment to harnessing real-world data for transformative insights. Moreover, an emphasis on data quality and methodology underscores the importance of rigorous practices in ensuring meaningful and actionable outcomes. As global efforts intensify, the RWE discussions signal a collective push toward a standardized, collaborative, and impactful future for RWE in shaping healthcare.