PRM268 - EVALUATION OF REAL-WORLD DATA COLLECTION AND EVIDENCE GENERATION FROM EARLY ACCESS PROGRAM

Abstract

Through early access programs (EAPs) (e.g., compassionate use, name patient programmes), patients with unmet need can receive promising treatments prior to marketing authorisation. EAP data can approximate real-world use, however, limited guidance exists regarding data collection procedures within these programs and acceptability of findings by payers and regulators. We aimed to identify and evaluate EAP data collection guidelines, as well as publications that reported effectiveness and patient reported outcomes (PRO) in EAPs. A targeted literature review was conducted to identify EAP guidelines and original research that disseminated EAP findings. Guidelines were compared and contrasted, and data collection within published EAPs were evaluated and synthesized. EAP data collection guidelines varied by country and European union member states. Safety data collection was consistently required in EAPs, however, guidance on collection of additional outcome measures were limited. The United Kingdom’s Early Access to Medicines Scheme and France’s Temporary Authorisation for Use programs had additional directives that appear to allow supplementary outcome measures to be collected (e.g., efficacy). Identified EAPs (N=10) were mixed with respect to data collection timing and measures included. Some EAPs incorporated data collection into the EAP (N=6), while others implemented data collection via chart review following program completion (N=4). Most data originated from patient charts with two programs also including PRO measures. The majority of studies reported that EAP findings (efficacy and safety) were similar to trial findings and when outcomes differed they offered explanations to validate observed differences. While published EAP guidelines exist, they do not provide clear directives for level of data collection that is acceptable within these programs. Since many EAPs have incorporated data collection, further regulatory guidance is needed to ensure consistency in EAP data collection and reporting, which in turn can improve validity of this research for market access and reimbursement