PHARMACOVIGILANCE-BASED DRUG REPURPOSING: A SYSTEMATIC LITERATURE REVIEW...
Author(s)
SWETA ROY, PhD1, BHOLANATH CHOWDHURY, MPH2, Gurpreet Kaur, Other3, Jatin Sharma4, Mohammed Zuber, PharmD5, Anoop Kumar, PhD2, Manik Chhabra, PhD6.
1Pharmacology, Delhi Pharmaceutical Sciences and Research University (DPSRU), New Delhi, India, 2Delhi Pharmaceutical Sciences and Research University (DPSRU), New Delhi, India, 3Delhi Pharmaceutical Sciences & Research University (DPSRU), New Delhi, India, 4University College Of Medical Sciences, Delhi, India, 5University of Georgia, Athens, GA, USA, 6Department of Epidemiology and Biostatistics, Western University, London, ON, Canada.
1Pharmacology, Delhi Pharmaceutical Sciences and Research University (DPSRU), New Delhi, India, 2Delhi Pharmaceutical Sciences and Research University (DPSRU), New Delhi, India, 3Delhi Pharmaceutical Sciences & Research University (DPSRU), New Delhi, India, 4University College Of Medical Sciences, Delhi, India, 5University of Georgia, Athens, GA, USA, 6Department of Epidemiology and Biostatistics, Western University, London, ON, Canada.
OBJECTIVES: Objective: To systematically review PV-based drug repurposing, including data sources, methodological approaches, and validation strategies
METHODS: Methods: MEDLINE, Embase, and Web of Science were searched from inception to December 2025 to include observational studies using PV data for drug repurposing. Two reviewers independently performed tile abstract screening, full-text review, data extraction, and quality assessment. The review was conducted in accordance with PRISMA and PRISMA-S guidelines and registered with PROSPERO (CRD420261285424).
RESULTS: Results: Of 14,188 retrieved citations, 16 unique observational studies met the inclusion criteria. Studies were conducted predominantly in Japan (31.3%; 5/16), China (18.8%; 3/16), France and India (12.5% each). The FDA adverse event reporting system (FAERS) was the most commonly used database (81.3%; 13/16), followed by VigiBase (25%; 4/16), administrative claims databases (18.7%; 3/16), and the Japanese adverse drug event report (JADER) (6.25%; 1/16). The most frequent therapeutic targets were neurodegenerative and psychiatric disorders, and autoimmune and inflammatory conditions (25%; 4/16 each). Disproportionality analysis was the primary signal detection method, with reporting odds ratio (ROR) used in most of the studies (87.5%; 14/16), followed by information components (ICs) (37.5%; 6/16). The empirical Bayes geometric mean (EBGM) was used in two studies (12.5%; 2/16), the proportional reporting ratio (PRR) and empirical Bayes regression adjusted mean (ERAM) in one study each (6.3%; 1/16), the normalised discounted cumulative gain (NDCG) in one study (6.3%; 1/16), and sequence symmetry analysis (SSA) methods in nearly one fifth of studies (18.8%; 3/16). Internal validation was reported in 68.8% of the studies, and mechanistic validation in 62.5%, predominantly through in silico approaches. The RORs were the most frequently used metric.
CONCLUSIONS: Conclusion: PV-based drug repurposing is an emerging hypothesis strategy leveraging real-world safety data. Substantial methodological heterogeneity and the absence of external validation highlight the need for standardised framework and robust translational validation approaches in this field.
METHODS: Methods: MEDLINE, Embase, and Web of Science were searched from inception to December 2025 to include observational studies using PV data for drug repurposing. Two reviewers independently performed tile abstract screening, full-text review, data extraction, and quality assessment. The review was conducted in accordance with PRISMA and PRISMA-S guidelines and registered with PROSPERO (CRD420261285424).
RESULTS: Results: Of 14,188 retrieved citations, 16 unique observational studies met the inclusion criteria. Studies were conducted predominantly in Japan (31.3%; 5/16), China (18.8%; 3/16), France and India (12.5% each). The FDA adverse event reporting system (FAERS) was the most commonly used database (81.3%; 13/16), followed by VigiBase (25%; 4/16), administrative claims databases (18.7%; 3/16), and the Japanese adverse drug event report (JADER) (6.25%; 1/16). The most frequent therapeutic targets were neurodegenerative and psychiatric disorders, and autoimmune and inflammatory conditions (25%; 4/16 each). Disproportionality analysis was the primary signal detection method, with reporting odds ratio (ROR) used in most of the studies (87.5%; 14/16), followed by information components (ICs) (37.5%; 6/16). The empirical Bayes geometric mean (EBGM) was used in two studies (12.5%; 2/16), the proportional reporting ratio (PRR) and empirical Bayes regression adjusted mean (ERAM) in one study each (6.3%; 1/16), the normalised discounted cumulative gain (NDCG) in one study (6.3%; 1/16), and sequence symmetry analysis (SSA) methods in nearly one fifth of studies (18.8%; 3/16). Internal validation was reported in 68.8% of the studies, and mechanistic validation in 62.5%, predominantly through in silico approaches. The RORs were the most frequently used metric.
CONCLUSIONS: Conclusion: PV-based drug repurposing is an emerging hypothesis strategy leveraging real-world safety data. Substantial methodological heterogeneity and the absence of external validation highlight the need for standardised framework and robust translational validation approaches in this field.
Conference/Value in Health Info
2026-09, ISPOR Asia Pacific 2026, Bangkok, Thailand
Value in Health, Volume 55, Issue S1
Code
MSR10
Topic
Methodological & Statistical Research
Disease
No Additional Disease & Conditions/Specialized Treatment Areas