NYU Langone’s Compassionate Use Advisory Committee was the first independent body of its kind to provide guidance on the ethical allocation of drugs in development. The committee advises Johnson & Johnson (J&J) on the allocation of investigational medical products to patients outside of clinical trials and on other ethics issues related to preapproval access. The committee is led by faculty and staff in the Section of Medical Ethics and includes internationally recognized medical experts, bioethicists, and patient representatives.

History of the Compassionate Use Advisory Committee

In May 2015, Johnson & Johnson contacted the then-Division of Medical Ethics at NYU Grossman School of Medicine to request guidance on developing a framework for reviewing compassionate use (single-patient expanded access) requests that would be transparent, fair, beneficent, evidence-based, and patient-focused. An independent committee, the Compassionate Use Advisory Committee (CompAC), was formed and chaired by Arthur L. Caplan, PhD, founding director of the Division of Medical Ethics. He was approached because of his prior work involving the allocation of cadaver organs for transplant. In a pilot program for one investigational agent in oncology, the committee of clinicians, bioethicists, and patient representatives provided guidance on compassionate use requests.

Dr. Caplan and Alison Bateman-House, PhD, MPH, MA, an assistant professor in the section, served as the committee’s non-paid, non-voting chair and deputy chair, respectively. CompAC had 10 members from many nations. The committee met weekly to consider requests for the drug; three members voted on requests each week. CompAC’s recommendation 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 marketed as Darzalex.

Due to the success of the pilot as determined by an external third party, J&J and NYU Grossman School of Medicine collaborated in additional therapeutic areas, including oncology, infectious diseases and immunology, and neuro-psychiatric conditions. Today CompAC advises J&J on other issues related to expanded access and preapproval access, at the company’s request.

J&J provides funding for organizing and staffing CompAC to NYU Grossman School of Medicine. Because the pilot and continuing collaboration are considered research projects for NYU Grossman School of Medicine, CompAC members are free to write, lecture, and publish about this novel 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, Kearns L, and Caplan A. A pioneering industry-academia collaboration celebrates 10 years. Bioethics Today, May 9, 2025.

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.

Janssen's CompAC information page and video.