In complex conditions such as Alzheimer's Disease, where the majority of clinical information resides in unstructured data, finding real-world data can be difficult. Key data elements like functional and cognitive assessment scores, safety events, and progression often go untapped because they are not readily available in structured formats—but are critical for studies. Here, we evaluate types of RW data sources—examining various concepts such as representativeness, time to data access, and breadth of clinical data available—to highlight strengths and limitations and provide a framework for selecting data sources suitable for AD research.

Recent studies have underscored the growing importance of RWD in the regulatory landscape, particularly in areas like oncology, hematology and rare diseases where traditional randomized controlled trials (RCTs) are not feasible due to an absence of a set standard of care or too few patients to achieve enrollment goals. In this white paper, we evaluate whether RWE submitted for regulatory decision-making was accepted by the FDA, aim to elucidate the critique provide by the agency, and provided insights into how variations in evidentiary standards influence the acceptance of RWE.

The contribution of real-world evidence (RWE) to regulatory submissions as supportive or substantial evidence has increased in the pre-marketing authorization space over the last decade. An extensive series of guidance documents have been put forth by the United States (US) Food and Administration (FDA) on the use of real-world data (RWD) and RWE in regulatory submissions, with several guidance themes emerging. In this white paper, we discuss key takeaways from a series of FDA guidance and review selected case studies of RWD/RWE to highlight best practices and learnings that sponsors can implement when planning and managing their regulatory submissions.

Recent FDA guidance emphasizes that real-world data (RWD) / real-world evidence (RWE) used in regulatory submissions must be submitted electronically and adhere to the approved data standards for such submissions. Not adhering to these guidelines on data standards may have implications on the regulatory submission and review process. Therefore, aligning RWD/RWE with the FDA’s requirements is crucial to facilitate a streamlined regulatory review for submissions containing RWD/RWE.

This White Paper aims to: 

• Summarize relevant FDA guidance documents
• Describe currently accepted data standards
• Summarize key elements of the data submission
• Discuss key challenges that companies may encounter for RWD