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

Jeremy Snyder, Alison Bateman-House, Leigh Turner

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1979-1985
Number of pages7
JournalRegenerative Medicine
Volume15
Issue number8
DOIs
StatePublished - Aug 2020

Keywords

  • compassionate use
  • crowdfunding
  • expanded access
  • right to try

PubMed: MeSH publication types

  • Journal Article

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