A Novel Approach to Compassionate Use Allocation

By Lisa Kearns, Alison S. Bateman-House, Arthur L. Caplan, J. Russell Teagarden

When seriously ill patients cannot enroll in clinical trials and have run out of treatment options, they may ask pharmaceutical manufacturers for “compassionate use” of their drugs in development, knowing that the drugs are experimental and may not help. These are tricky decisions for companies: they may want to help patients but worry that providing experimental agents could jeopardize their drug development programs. They may not have enough of the drug for every patient who requests it and may be unsure whom to give access to.

Drug companies generally make allocation decisions in-house. There are no uniformly accepted best practices to guide such decisions. Even who makes them varies from company to company: it could be a chief medical officer, a patient affairs executive, a regulatory officer, or the CEO. Whoever decides may be subject to pressure for preferential treatment — internally, from executives or other employees who seek a drug for themselves or loved ones, or, externally, from patient advocates, celebrities, politicians, or other well-connected people. Social media, in particular, can be an outsize source of pressure because of its ability to target, often indiscriminately, medical or corporate professionals and paint them as consumed by the drive to make profits and insensitive to the importance of providing possibly lifesaving therapies to patients with no other treatment options.

The Compassionate Use Advisory Committee (CompAC), a collaboration between NYU School of Medicine’s Division of Medical Ethics and Janssen Pharmaceuticals, is a new approach to allocating investigational drugs. CompAC was created as an anonymous body appointed by the Division chair to operate independently of Janssen; by initially shielding its members’ identities, the committee could consider compassionate use requests on their merits, without the threat of being subjected to either internal or external pressure.

CompAC offered a new alternative to traditional methods for scarce resource allocation. Instead of making decisions according to a random lottery system or on a first-come, first-served basis, the committee crafted a set of principles to guide deliberations. The first was that the drug should neither harm a patient nor subject him or her to risks greater than potential benefit (non-maleficence); second, that the drug should have some chance of helping the patient (beneficence); and, third, that the patient be able to achieve appreciable benefits over a sustained period of time. “Lower order” principles — age, whether a patient was solely responsible for dependents or had participated in a clinical trial — were invoked only as tie-breakers when there wasn’t enough drug to satisfy all requests and the chief principles weren’t sufficient to help prioritize requests.

Unlike a random lottery, the CompAC model, by standardizing the information doctors submitted about patients, captured nuances in patients’ health statuses and thus their suitability for the drug. Unlike first-come, first-served, the model doesn’t favor those with access to more in-the-know healthcare providers. Because the principles-based system took into account patients’ medical statuses and likelihood of benefiting from the treatment, the panel considered it to be the fairest way to weigh requests coming in from around the world. A principles-based approach paired with a uniform process by which to evaluate requests differentiates the CompAC model from other ways of dealing with compassionate use requests.

Our paper “Fair, Just, and Compassionate: A Pilot for Making Allocation Decisions for Patients Requesting Experimental Drugs Outside of Clinical Trials” reveals how CompAC was formed and how it built and operated under the ethical framework it developed to guide its work. The importance of fairness in treating like cases alike was a driving feature of the CompAC model and is reflected in both the principles-based framework and the decision to conceal committee members’ identities so they couldn’t be lobbied to give preferential treatment to anyone.

The pilot’s launch was covered extensively in the mainstream press, and earlier papers by CompAC members took a more data-driven or general approach to the formation of the committee. This paper, in contrast, is a behind-the-scenes look at how members were chosen, how they arrived at ethical principles to guide deliberations, and how weekly case reviews were conducted. It conveys the moral distress that plagued members as they deliberated over whom they would and would not recommend to receive a potentially lifesaving drug.

The CompAC pilot showed that a diverse, international panel of doctors, ethicists, and patient advocates can be assembled to make medically sound compassionate use allocation decisions quickly, fairly, and transparently, while sometimes operating under conditions of extreme drug scarcity. It is a model that can be customized to guide decisions for other drugs and other diseases; in fact, it has been scaled up, and there are currently four CompACs advising Janssen on the compassionate use allocation of many of its investigational drugs. The model could also aid allocation decisions for drugs or vaccines during epidemics or humanitarian emergencies.

Paper: Fair, Just, and Compassionate: A Pilot for Making Allocation Decisions for Patients Requesting Experimental Drugs Outside of Clinical Trials

Authors: Arthur L Caplan1, J Russell Teagarden2, Lisa Kearns1, Alison S Bateman-House1, Edith Mitchell3, Thalia Arawi4, Ross Upshur5,6, Ilina Singh7, Joanna Rozynska8, Valerie Cwik9, Sharon L Gardner10

  1. Division of Medical Ethics, NYU School of Medicine, New York City, New York, USA
  2. Brookfield, Connecticut, USA
  3. Department of Medical Oncology, Sidney Kimmel Cancer Center, Jefferson University, Philadelphia, Pennsylvania, USA
  4. Salim El-Hoss Bioethics & Professionalism Program, Faculty of Medicine, American University of Beirut & Medical Center, Beirut, Lebanon
  5. Dalla Lana Faculty of Public Health, University of Toronto, Toronto, Ontario, Canada
  6. Department of Family and Community Medicine, Sinai Health System, Toronto, Ontario, Canada
  7. Department of Psychiatry, Wellcome Centre for Ethics and Humanities, University of Oxford, Oxford, UK
  8. Center for Bioethics & Biolaw, Institute of Philosophy, University of Warsaw, Warsaw, Poland
  9. Muscular Dystrophy Association, Tucson, Arizona, USA
  10. Department of Pediatrics, NYU Langone Health, New York City, New York, USA

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

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