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Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials
  1. Arthur L Caplan1,
  2. J Russell Teagarden2,
  3. Lisa Kearns1,
  4. Alison S Bateman-House1,
  5. Edith Mitchell3,
  6. Thalia Arawi4,
  7. Ross Upshur5,6,
  8. Ilina Singh7,
  9. Joanna Rozynska8,
  10. Valerie Cwik9,
  11. Sharon L Gardner10
    1. 1 Division of Medical Ethics, NYU School of Medicine, New York City, New York, USA
    2. 2 Brookfield, Connecticut, USA
    3. 3 Department of Medical Oncology, Sidney Kimmel Cancer Center, Jefferson University, Philadelphia, Pennsylvania, USA
    4. 4 Salim El-Hoss Bioethics & Professionalism Program, Faculty of Medicine, American University of Beirut & Medical Center, Beirut, Lebanon
    5. 5 Dalla Lana Faculty of Public Health, University of Toronto, Toronto, Ontario, Canada
    6. 6 Department of Family and Community Medicine, Sinai Health System, Toronto, Ontario, Canada
    7. 7 Department of Psychiatry, Wellcome Centre for Ethics and Humanities, University of Oxford, Oxford, UK
    8. 8 Center for Bioethics & Biolaw, Institute of Philosophy, University of Warsaw, Warsaw, Poland
    9. 9 Muscular Dystrophy Association, Tucson, Arizona, USA
    10. 10 Department of Pediatrics, NYU Langone Health, New York City, New York, USA
    1. Correspondence to Dr Arthur L Caplan, Division of Medical Ethics, NYU School of Medicine, New York City, NY 10016, USA ; Arthur.Caplan{at}


    Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or ‘preapproval’, access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients’ best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice.

    • allocation of health care resources
    • research ethics
    • resource allocation
    • ethics
    • public policy
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    • Contributors ALC, JRT, LK and ASBH all contributed to drafting, writing and editing of the manuscript. Other CompAC members provided feedback.

    • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

    • Competing interests Janssen Pharmaceuticals provided administrative costs. First author and NYU-affiliated authors are not paid. Some CompAC members take payment, others do not.

    • Patient consent Not required.

    • Provenance and peer review Commissioned; externally peer reviewed.

    • Collaborators Members of the Compassionate Use Advisory Committee: E Mitchell, T Arawi, R Upshur, I Singh, J Rozynska, V Cwik, S L Gardner.

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