Alison Bateman-House, PhD, MPH, MA, and colleagues at the New York University School of Medicine Working Group on Compassionate Use and Pre-Approval Access (CUPA) recently issued a statement1 that cited the following unresolved issues on the federal Right to Try (RTT) law.
• How many companies or healthcare institutions will permit use of the RTT pathway?
• Who will oversee the implementation of the law (write rules/guidance) and compliance with it?
• Although the statute references a federal regulation that stipulates that sponsors can charge only direct costs for investigational products, what will this restriction mean in practice? How will companies interpret it? How will it be enforced, and by whom?
• Who will pay for drugs obtained via RTT and the costs of administering them? What about related medical, travel, childcare, and other expenses?
• Are companies’ RTT policies subject to the 21st Century Cures Act’s transparency provisions that require companies with investigational products in Phase II or later to make their expanded access policies publicly available?
• In the absence of federally mandated FDA and IRB oversight, will healthcare institutions or companies require in-house or other third-party oversight of nontrial use of investigational drugs?
1. Statement on the Enactment of a Federal “Right to Try” Law from the NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access (CUPA). June 25, 2018. Available at: https://bit.ly/2m5E4oQ.