TY - JOUR
T1 - Is right to try being tried? Using crowdfunding data to better understand usage of nontrial pre-approval access pathways
AU - Snyder, Jeremy
AU - Bateman-House, Alison
AU - Turner, Leigh
N1 - Publisher Copyright:
© 2020 Future Medicine Ltd.. All rights reserved.
PY - 2020/8
Y1 - 2020/8
N2 - Aim: The US FDA 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 campaigns and two referencing both. Twenty one EA campaigns described being approved to receive access to the requested experimental medical product versus one RTT campaign. Conclusion: RTT is associated with poor understanding of nontrial pre-approval access. These campaigns suggest RTT is not offering a practical alternative to EA. Cost remains a significant barrier to these patients.
AB - Aim: The US FDA 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 campaigns and two referencing both. Twenty one EA campaigns described being approved to receive access to the requested experimental medical product versus one RTT campaign. Conclusion: RTT is associated with poor understanding of nontrial pre-approval access. These campaigns suggest RTT is not offering a practical alternative to EA. Cost remains a significant barrier to these patients.
KW - compassionate use
KW - crowdfunding
KW - expanded access
KW - right to try
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U2 - 10.2217/rme-2020-0043
DO - 10.2217/rme-2020-0043
M3 - Article
C2 - 33023369
AN - SCOPUS:85097346314
SN - 1746-0751
VL - 15
SP - 1979
EP - 1985
JO - Regenerative Medicine
JF - Regenerative Medicine
IS - 8
ER -