The Use of Real‐World Data to Assess the Impact of Safety‐Related Regulatory Interventions

Abstract

The regulation of medicines seeks to ensure the efficacy, safety, and quality of prescription and non‐prescription medicines. Given that the conditions under which a medicine’s benefits outweigh its risks are complex, it is essential that communications about the safe and effective use of medicines be clear and actionable. Assessing the impact of interventions to improve the safe and effective use of medicines is a developing area, and one in which real‐world data are playing an increasingly important role. Although real‐world data are commonly used to assess the impact of regulatory interventions, there are several areas where their use could be improved. Specific areas for improvement include assessing regulatory interventions across a wider range of medicines, rather than concentrating on a relatively few therapeutic areas; assessing more clinically relevant outcomes rather than relying on measures such as changes in the number of prescriptions, which may not always correlate with the desired impact; assessing the potential unintended or negative consequences of regulatory interventions; applying methods to address potential confounders; assessing long‐term, rather than just short‐term, impacts of an intervention; increasing the use of comparator groups, when feasible; and evaluating the impact of regulatory interventions from multiple dimensions, rather than from a single dimension. Expanded use of real‐world data could inform some of these efforts, although data sources beyond administrative claims data will likely be necessary to achieve all these goals.

Unintended impact of pharmacovigilance regulatory interventions: A systematic review

“…Most publications assessed the impact of DHPCs or other communication strategies to disseminate information about drug safety ( n = 67, 70%) that are meant to remind or update healthcare provider knowledge about the safe use of medicines. These results concur with previous reviews assessing the effectiveness of various pharmacovigilance regulatory interventions 39–42 . However, in many cases, the purpose of DHPC was to inform healthcare professionals about new restrictions, contraindications or other routine measures to minimize risk.…”

“…Three studies assessing possible health outcomes of unintended impact used spontaneous reporting registries 16,24,27 . While health outcomes seem more direct to check the impact on a patient, they might lack the sensitivity to detect possible impact, as discussed by Dal Pan 39 …”