Background: Off-label medicines use is a common and sometimes necessary practice in many populations, with important clinical, ethical and financial consequences, including potential unintended harm or lack of effectiveness. There has not been globally recognised guidance to inform off-label therapeutic decision-making by prescribers and other stakeholders, with highly variable approaches internationally. An ISPE-funded, multinational, multidisciplinary expert group conducted a scoping review to summarize the existing literature on available guidances and developed consensus recommendations to address identified gaps.

Objectives: This workshop aims to: 1) Provide a global overview and critical analysis of key off-label treatment considerations, 2) Introduce consensus recommendations for optimizing off-label therapeutic decision-making and driving relevant research concurrently; 3) Stimulate interactive discussion with workshop participants to identify barriers, facilitators, and next steps to effective implementation of the recommendations. Target audience: those involved in medicines evaluation and therapeutic decision-making, including prescribers, regulators, policy makers, industry, academic researchers, patient advocates, and Health Technology Assessment representatives. All stakeholders interested in exploring opportunities for generating more timely and meaningful RWE to inform therapeutic decision-making would also benefit from attending.

Description: This workshop consists of the following: 1) Overview of the workshop (5 minutes); 2) Critical analysis of off-label medicines use, including reasons for off-label prescribing and potential risks and benefits of off-label use (10 minutes); 3) Introduction of consensus recommendations for optimizing off-label therapeutic decision-making and generating meaningful RWE to better inform benefit: risk assessments for off-label treatment (20 minutes); 4) Interactive, small-group discussions about illustrative case examples from various disciplines (e.g., intensive care, mental health, rheumatology) to discuss the potential applications, facilitators of, and barriers to implementing the recommendations (30 minutes); 5) Large-group recap and panel discussion of key points from small groups, with solicitation of perspectives from diverse stakeholders and concrete suggestions for implementation (25 minutes). This workshop features organizers with diverse clinical and methodological expertise, work experience (academia, clinical practice, pharmaceutical industry, medicines policy), and geographic perspectives (Australia, Canada, Europe, USA). * This workshop is based on a manuscript (DOI:10.1002/pds.5640) endorsed by the International Society for Pharmacoepidemiology (ISPE)