Division of Medical Ethics Compassionate Use Advisory Committees
NYU Langone’s Compassionate Use Advisory Committees are the first independent bodies of their kind to provide guidance on the ethical allocation of drugs in development. We advise Janssen Pharmaceuticals, Inc., part of Johnson & Johnson, on the allocation of investigational medication to patients outside of clinical trials. The committees are led by faculty and staff in the Division of Medical Ethics and include internationally recognized medical experts, bioethicists, and patient representatives.
History of the Compassionate Use Advisory Committees
In May 2015, Janssen 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 transplant, an independent committee was formed, the Compassionate Use Advisory Committee (CompAC) chaired by Arthur L. Caplan, PhD, 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 Alison Bateman-House, PhD, MPH, MA, an assistant professor in the division, served as the committee’s non-paid, non-voting chair and deputy chair, respectively. CompAC had 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 U.S. Food and Drug Administration (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. Dr. Caplan and Dr. 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, president of the Marti Nelson Cancer Foundation. There are also now CompACs for Janssen’s investigational drugs in the therapeutic areas of oncology, infectious diseases and immunology, and neurology/psychiatry.
Janssen provides funding for organizing and staffing the CompACs to NYU Grossman 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, courtesy of Patient Support, Office of the Chief Medical Officer at Johnson & Johnson.
Selected Compassionate Use Media and Resources
Our original CompAC initiative attracted wide interest from medicine, the pharmaceutical industry, mainstream media, and the general public. Following are selected media and resources about CompAC.
MacCracken C. The evolution of compassion: Janssen’s innovative collaboration on behalf of seriously ill patients. Clinical Leader. January 2, 2019.
Caplan AL … Gardner SL. Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials. J Med Ethics. 2018. DOI.
Caplan A … Jansson R. A pilot experiment in responding to individual patient requests for compassionate use of an unapproved drug: The Compassionate Use Advisory Committee (CompAC). Ther Innov Regul Sci. 2018. DOI.
Kearns L, Bateman-House A, and Caplan A. Ensuring justice in access to investigational neurological drugs. Semin Neurol. 2018. DOI.
Kearns L … Teagarden JR. A novel approach to compassionate use allocation. J Med Ethics Blog. June 30, 2018.
May B. Collaboration between pharma and education leads to improved system for compassionate use requests. Medical Bag. September 12, 2018.
Kearns L and Caplan AL. Hard choices for vulnerable patients: Some lessons learned that may apply. Am J Bioeth. 2017. DOI.
McCullough M. Pharma firm pioneers bioethics model to get experimental drug to myeloma patients. Philadelphia Inquirer. April 4, 2017.
Caplan AL and Ray A. The ethical challenges of compassionate use. JAMA. 2016. DOI.
Caplan AL, Bateman-House A, and Waldstreicher J. Compassionate use: A modest proposal. Am Soc Clin Oncol Educational Book. 2016. DOI.
Sutter S. J&J’s compassionate use advisory cmte. may become industry model. Pink Sheet. May 9, 2016.
Usdin S. Piloting compassion: How J&J, NYU pilot made compassionate access decisions fairer, more transparent. BioCentury. February 15, 2016.
Weintraub K. Novel system to get dying patients an experimental cancer drug raises hopes—and thorny questions. STAT News. November 8, 2016.
Johnson & Johnson. Johnson & Johnson announces NYU School of Medicine partnership to evaluate compassionate use of investigational medicines. PR Newswire. May 7, 2015.
Kroll D. Can a pharma-sponsored bioethics board fairly decide who gets experimental drugs? Forbes. May 7, 2015.
Rockoff JD. J&J changes ‘compassionate’ care. The Wall Street Journal. May 7, 2015.
Thomas K. Company creates bioethics panel on trial drugs. The New York Times. May 7, 2015.