IMPROVING EFFICIENCY IN REAL-WORLD EVIDENCE SLRS WITHOUT COMPROMISING VALIDITY: IMPLICATIONS FOR BIOPHARMA
Author(s)
Digant Gupta, MBBS, MPH1, Mudasir Khan, MPH1, Smeet Gala, MS1, Monique Martin, PharmD, MSc, MBA2.
1Red Nucleus, Pune, India, 2Red Nucleus, London, United Kingdom.
1Red Nucleus, Pune, India, 2Red Nucleus, London, United Kingdom.
OBJECTIVES: Systematic literature reviews (SLRs) of RWE are critical for informing health technology assessment (HTA), payer engagement, and internal decision-making in biopharmaceutical companies. However, the rapidly growing volume of observational literature creates substantial resource and timeline challenges. This study evaluated whether a “third screening” (prioritisation) approach, applied after full-text screening, can improve efficiency without compromising representativeness of evidence.
METHODS: A previously completed SLR on wet age-related macular degeneration was used. Following full-text screening, 200 observational studies were identified. A third screening was applied using pre-specified criteria (including study design, sample size, follow-up, recency of data collection, confounder adjustment, and geography), such that recently published prospective studies with adequate confounder control and larger sample sizes and follow-up durations from the key geographies of interest were prioritised. Key clinical-demographic characteristics were compared between the prioritised and de-prioritised groups using independent t-tests or Mann-Whitney tests with Bonferroni-adjustment.
RESULTS: Of 200 studies, 37 were prioritised and 163 were de-prioritised. Mean age was 77.7 vs 75.3 years (p=0.04), and mean percentage of males was 43.8% vs 43.2% (p=0.80) in prioritised vs de-prioritised studies, respectively. Disease severity indicators were comparable, including percentage of patients with bilateral involvement (33.9% vs 27.4%; p=0.39), mean ETDRS letter count (54.9 vs 56.7; p=0.64), and mean logMAR score (0.7 vs 0.6; p=0.39). The percentage of patients previously treated was 75.0% vs 60.1% (p=0.12). None of these differences were statistically significant based on Bonferroni-adjusted thresholds (α=0.016), and the observed difference in mean age (2.4 years) was not clinically meaningful.
CONCLUSIONS: These findings demonstrate that third screening or prioritisation can improve the efficiency of RWE SLRs without compromising validity when applied using objective and transparent criteria. Prioritisation can reduce evidence volume while preserving representativeness of the broader evidence base thereby enabling faster development of HTA-ready evidence packages by biopharmaceutical companies.
METHODS: A previously completed SLR on wet age-related macular degeneration was used. Following full-text screening, 200 observational studies were identified. A third screening was applied using pre-specified criteria (including study design, sample size, follow-up, recency of data collection, confounder adjustment, and geography), such that recently published prospective studies with adequate confounder control and larger sample sizes and follow-up durations from the key geographies of interest were prioritised. Key clinical-demographic characteristics were compared between the prioritised and de-prioritised groups using independent t-tests or Mann-Whitney tests with Bonferroni-adjustment.
RESULTS: Of 200 studies, 37 were prioritised and 163 were de-prioritised. Mean age was 77.7 vs 75.3 years (p=0.04), and mean percentage of males was 43.8% vs 43.2% (p=0.80) in prioritised vs de-prioritised studies, respectively. Disease severity indicators were comparable, including percentage of patients with bilateral involvement (33.9% vs 27.4%; p=0.39), mean ETDRS letter count (54.9 vs 56.7; p=0.64), and mean logMAR score (0.7 vs 0.6; p=0.39). The percentage of patients previously treated was 75.0% vs 60.1% (p=0.12). None of these differences were statistically significant based on Bonferroni-adjusted thresholds (α=0.016), and the observed difference in mean age (2.4 years) was not clinically meaningful.
CONCLUSIONS: These findings demonstrate that third screening or prioritisation can improve the efficiency of RWE SLRs without compromising validity when applied using objective and transparent criteria. Prioritisation can reduce evidence volume while preserving representativeness of the broader evidence base thereby enabling faster development of HTA-ready evidence packages by biopharmaceutical companies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
MSR44
Topic
Health Technology Assessment, Methodological & Statistical Research, Real World Data & Information Systems
Disease
No Additional Disease & Conditions/Specialized Treatment Areas