Is right to try being tried? Using crowdfunding data to better understand usage of nontrial pre-approval access pathways.

Abstract

Aim: The USFDA has two nontrial pre-approval access pathways: expanded access (EA) and right to try (RTT). Reports of successful RTT use are scarce, and the FDA has not yet published RTT usage data, yet proponents tout its utility. In the face of this discrepancy and a lack of transparency of usage statistics, our aim is to add to the limited understanding of RTT usage.Materials &m ethods: We searched crowdfunding campaigns referencing 'expanded access', 'right to try'or 'compassionate use'since 2018. Results: We identified 26 EA campaigns, 29 RTT campaignsand tworeferencing both. Twenty oneEA campaigns described being approved to receive access to the requested experimental medical product versus oneRTT campaign. Conclusion: RTT is associated with poor understanding of nontrial pre-approval access. These campaigns suggestRTT is not offering a practical alternative to EA. Cost remains a significant barrier to these patients.