CompAC: The Compassionate Use Advisory Committees

In May 2015, Janssen, the pharmaceutical companies of Johnson & Johnson, contacted the Division of Medical Ethics at NYU to request a method for reviewing compassionate use requests that would be transparent, fair, beneficent, evidence-based and patient focused. Based on prior work involving allocation of cadaver organs for transplantation, an independent committee was formed, the Compassionate Use Advisory Committee (CompAC) chaired by Dr. Arthur Caplan, founding director of the Division of Medical Ethics. In a pilot program for one investigational agent in oncology, the committee of internationally recognized medical experts, bioethicists, and patient representatives provided guidance on compassionate use requests.

Dr. Caplan and Dr. Alison Bateman-House, an assistant professor in the Division, served as the committee’s non-paid, non-voting chair and deputy chair, respectively. CompAC has 10 members from many nations—physicians, ethicists, and patient representatives. The committee met weekly to consider requests for the drug; three members voted on requests each week. CompAC’s decision on each case was conveyed to Janssen, which maintained the authority to grant or deny requests.

The drug was approved by the FDA in November 2015 and is now marketed as Darzalex®. 

Due to the success of the pilot as determined by an external third-party, Janssen and NYU are collaborating to continue piloting in additional therapeutic areas. Drs. Caplan and Bateman-House have established other CompACs to provide guidance on allocating investigational drugs that Janssen is developing. For individual compassionate use requests that Janssen receives for drugs outside the purview of these CompACs, NYU has formed a committee called Pre-Com to evaluate and provide guidance about these requests. Its members are Dr. Caplan, Dr. Bateman-House, Dr. Ross Upshur of the University of Toronto Joint Centre for Bioethics, and Dr. Robert Erwin, the President of the Marti Nelson Cancer Foundation. There are also now CompACs for Janssen's investigational drugs in the therapeutic areas of oncology, infectious disease, and neurology/psychiatry. 

Janssen provides funding for organizing and staffing the CompACs to the NYU School of Medicine. Janssen agreed to permit CompAC members to write, lecture, and publish about this novel pilot experiment in translational bioethics.

An infographic on single patient expanded access is available here, courtesy of Patient Support, Office of the Chief Medical Officer at Johnson & Johnson.

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