Expanding the Scope: Utilizing Early Access/Compassionate Use Pathways to Generate Market-Specific Real-World Evidence Across LATAM Prior to Launch

OBJECTIVES: Early access/ Compassionate Use programs (EAPs/CUPs) allow for patients to access therapies prior to marketing authorization. These can be leveraged by manufacturers to generate RWE for the product prior to its launch and pose an opportunity for market-specific data generation especially in countries that otherwise may not be included in the pivotal clinical trial, such as with LATAM countries. This research evaluates EAPs available in major LATAM countries to determine whether they provide opportunities for RWE collection pre-launch.

METHODS: Publicly available information was screened to identify EAPs in Argentina, Brazil, Chile, Colombia, and Mexico using their respective websites to extract key information.

RESULTS: Four countries offer EAPs, with Argentina, Brazil, and Chile offering two pathways (Colombia offers 1, Mexico none). Inclusion criteria are broadly aligned across markets, allowing access to individuals or patient groups with severe, debilitating conditions with no effective therapeutic alternatives. Applications for EAPs can be made by either manufacturers or physicians depending on the respective pathway. In 3/4 (Argentina, Brazil, Chile) countries that offer EAPs, manufacturers must provide their treatments at no cost. In 1/4 (Colombia), reimbursement may be provided in select cases, but decision criteria are unclear. In the markets that offer EAPs, RWE-generated data can be leveraged to supplement both regulatory and reimbursement evidence dossier submissions.

CONCLUSIONS: Four out of five major LATAM markets offer EAPs, providing manufacturers the opportunity to collect market-specific data to support their regulatory and reimbursement submissions. The lack of reimbursement incentives may pose a barrier, but this can be offset by the opportunity to collect data from countries that may be excluded from pivotal trials.