FRAME: Framework for Real-World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness - An Evaluation of Regulatory and Health Technology Assessment Decision Making.

ABSTRACT: Real-World Evidence (RWE) is increasingly used in submissions to regulatory agencies and health technology assessment bodies (HTAbs) to support the efficacy and effectiveness of new medicines and indications. However, there is limited information on the RWE characteristics that impact its role in approval and reimbursement decisions. To investigate these characteristics, we developed FRAME: a Framework for Real-world evidence Assessment to Mitigate Evidence uncertainties for efficacy/effectiveness. We compiled a list of medicinal product indications where RWE supported the efficacy of interventional trials or assessed effectiveness in observational settings. FRAME was applied to a prioritized subset of these submissions to authorities from North America, Europe, and Australia. For each pro

SUBMITTER: Candore G 

PROVIDER: S-EPMC12355015 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature