Compassionate Use and Pre-Approval Access Working Group Publishes End-of-Year Report

Thursday, December 22 2016

The NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access (CUPA) published an end-of-year report describing their efforts in 2016 to advance knowledge about the ethical issues surrounding pre-approval access, as well as to identify best practices and issues that need further attention.

Among the activities and accomplishments of the past year, CUPA published a major update of its website, began working with patient groups to help demystify pre-approval access and create community-specific resources, and joined with Johnson & Johnson to host a patient advocacy summit on the topic of pre-approval access.

The report also summarizes legislative news of the past year that impacted pre-approval, or “expanded,” access to experimental drugs as well as the landscape for “right to try” laws. CUPA members contributed heavily to the discussion with many publications, interviews, and talks, including a profile in Harper’s that prominently featured Arthur Caplan, PhD, director of the Division of Medical Ethics, and a STAT profile of the Compassionate Use Advisory Committee, or CompAC, a pilot project in which a panel of medical experts, bioethicists, and patient advocates provides guidance on how to fairly and transparently allocate a scarce investigational drug to patients outside of clinical trials.

Read the full report.