Standardized Assessment Tool Designed to Assist in Evaluation of RWE on Drug Effectiveness and Safety

PURPOSE: Non-randomized RWE studies are subject to multiple sources of bias. The complexity of data, study design, and analytical methods makes evaluation of such studies challenging. Building consensus on the evaluation of quality and validity of RWE studies is critical if RWE is to play an important role in decision-making. As part of the International Society for Pharmacoepidemiology’s (ISPE) effort to improve the quality of RWE evaluation, an ISPE-led group developed an assessment tool to support HTA and payer decision-makers in evaluating RWE studies of medication safety and effectiveness. The tool was further refined in collaboration with HTA agencies. This session aims to introduce the ISPOR community to the tool and solicit feedback to ensure the tool addresses the needs of HTA agencies and the broader ISPOR community.

DESCRIPTION: The discussion leader will introduce the need for a standardized assessment tool in evaluating real-world comparative effectiveness and safety studies for bias (5 mins). Dr. Girman will introduce the assessment tool and briefly discuss study relevance and data quality domain (5 mins). Dr. Bykov will walk the audience through bias-related domains (10 mins). Dr. Franklin will present analytic methods domain (10 min). Using a published RWE study as an example, the discussants will lead the attendees through real-time RWE study evaluation using the tool (20 mins). The discussants will then take questions and feedback from the audience (10 mins). While the tool was developed with HTA agencies in mind, it can be used in any other setting where RWE evaluation is needed. This interactive workshop will be valuable to HTA representatives, payers, industry analysts preparing RWE submissions to HTAs, and any ISPOR members who need to critically evaluate RWE on drug safety and effectiveness.