Optimizing RWE Design and Conduct by Leveraging Computable Operational Definitions Indication Libraries: Development, Verification, and Value
Author(s)
Kamauu J1, Buck M2, Thompson D3, Shields A4, Somerday M4, Kamauu AG5, Parker CG6, DuVall S7, Kamauu A8
1Navidence LLC, Draper, UT, USA, 2Navidence, Cedar Hills, UT, USA, 3Rubidoux Research LLC, Manchester, MA, USA, 4Navidence LLC, Chapel Hill, NC, USA, 5Navidence LLC, Salt Lake City, UT, USA, 6Navidence LLC, Sandy, UT, USA, 7VA Informatics and Computing Infrastructure, VA Salt Lake City Health Care System, Salt Lake City, UT, USA, 8Navidence LLC, Bountiful, UT, USA
Presentation Documents
OBJECTIVES: Real-world research (RWR) involves the interpretation of study elements into conceptual and operational definitions, including up-to-date code lists. Computable operational definitions (CODefs) have been shown to maximize comparability and consistency across data sources in global RWR programs. Thoughtful CODef selection is important because it has a direct impact on study results as indicated by FDA Guidance [Dec 2023]. This research aims to support the optimization of real-world evidence (RWE) design and conduct by allowing researchers to select CODefs from pre-curated indication-specific libraries.
METHODS: Five indications were selected from different therapeutic areas: asthma (respiratory), heart failure (cardiovascular), immunocompromised conditions, lung cancer (oncology), and type 2 diabetes mellitus (metabolics).
Literature and reputable references were used to guide the creation of these indication-specific libraries of CODefs to support RWR study design, data source fit-for-purpose qualification, and protocol development. Where available, published validation metrics for CODefs were captured to provide context. CODefs for clinical and research concepts relevant to each of the selected indications were organized into distinct libraries to help with determining the most appropriate CODef for each study design element when designing a RWR study.RESULTS: Each indication library includes a comprehensive set of the most relevant CODefs (with code lists) for RWR studies. The heart failure library (smallest) contains 86 CODefs covering 45 concepts (avg 1.9 CODefs/concept), and the immunocompromised conditions library (largest) contains 252 CODefs covering 74 concepts (avg 3.4 CODefs/concept). The 5 libraries ranged from 22 to 241 code lists each (>35,000 codes) using 14 international code systems (e.g., ICD-10-WHO, CPT, ATC, READ2). To-date 15 RWE studies have been modeled using CODefs from these libraries.
CONCLUSIONS: Pre-curated indication-specific libraries of CODefs provide significant value to RWR. Researchers can select the most appropriate CODef from a library, saving time while ensuring consistency within studies and across the industry.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
RWD172
Topic
Real World Data & Information Systems
Topic Subcategory
Data Protection, Integrity, & Quality Assurance, Reproducibility & Replicability
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Diabetes/Endocrine/Metabolic Disorders (including obesity), Oncology, Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory), Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-on