Prospective Benefit-Risk Monitoring Of New Drugs For Rapid Assessment Of Net Favorability In Electronic Healthcare Data

Abstract

A11at market entry of each drug of interest and using a sequential propensity score matched cohort design. We applied four BRA metrics: number needed to treat and number needed to harm (NNT|NNH); incremental net benefit (INB) with maximum acceptable risk [MAR], INB with relative-value adjusted life years [RVALYs], and INB with quality-adjusted life years [QALYs]. We determined whether and when the bootstrapped 99% confidence interval (CI) for each metric excluded zero, indicating net favorability of one drug over the other. Results: For rofecoxib, all four metrics yielded a negative value, suggesting net favorability of ns-NSAIDs over rofecoxib, and the 99% CI for all but the NNT|NNH excluded the null during follow-up. For prasugrel, only the 99% CI for INB-QALY excluded the null, but trends in values over time were similar across the four metrics, suggesting overall net favorability of prasugrel versus clopidogrel. The 99% CI for INB-RVALY and INB-QALY excluded the null in the denosumab example, suggesting net favorability of denosumab over bisphosphonates. ConClusions: Prospective benefit-risk monitoring can be used to determine net favorability of a new drug in electronic healthcare data. In three examples, existing BRA metrics produced qualitatively similar results but differed with respect to alert generation. INB-QALY produced the most conclusive findings across the three examples.

Approaches to enabling rapid evaluation of innovations in health and social care: a scoping review of evidence from high-income countries

“…We included 162 studies in the review (table 1); these were methods papers, primary evaluations or evaluations of implementation undertaken in high-income countries; one was a systematic review 9 which included eight of the primary studies included here. [26][27][28][29][30][31][32] Ninety of the included studies were identified from the update search in September 2022. Most studies were undertaken in primary care, community settings or in specific non-hospital-based services (often described as 'clinics'), reflecting the health and related care focus of the review.…”

“… 58 129 138 145–151 153 154 173 181 197–209 Studies which involved developing, acquiring, sharing or using existing data sets to enable modelling of effectiveness were also included. 29 129 173 200 203 206–210 Seven studies were undertaken in the context of monitoring of uptake and safety profiles of vaccinations even prior to COVID-19. For study details see online supplemental appendix 2 tables 2 and 3 .…”

Rapid Evaluations of Innovations: A Scoping Review

“…These ten studies involved using technology to automate or otherwise try to improve rapidity of one or more of data collection, collation, or analysis (44,86,105,(109)(110)(111)(112)(113)(114)(115). Studies which involved acquiring or using existing data sets to enable modelling of effectiveness were also included (111,114,119). Seven of these studies were undertaken in the context of monitoring of uptake and safety pro les of vaccinations.…”