Patients with difficult-to-treat illnesses often have limited treatment options, with many patients seeking experimental drugs outside of clinical trials. Compassionate use requests for investigational medications are not unheard of, especially in diseases in which a serious unmet need exists. In an article published in the Journal of Medical Ethics, a multinational team of researchers discussed a pilot for making allocation decisions for patients who ask for investigational drugs outside the clinical trials environment.

A collaboration between Janssen Pharmaceuticals and the New York University School of Medicine, Division of Medical Ethics, aimed to develop a fair and transparent program for managing compassionate use requests. Specifically, the collaboration group sought to develop guidance for daratumumab, an agent used for refractory multiple myeloma that is neither approved nor widely available outside of clinical trials. A set of predefined criteria was established for deciding which compassionate use requests would be sent to the Compassionate Use Advisory Committee (CompAC) for further consideration.

The success of CompAC in helping patients receive daratumumab outside of clinical trials was driven by the multinational makeup of the organization, the varying range of expertise demonstrated by the members, and the direct access the organization had to independent experts in multiple myeloma. Approximately 6 months after the collaboration entity, CompAC, was set in place, daratumumab was approved for use in the United States, resulting in immediate prescribing of the medication by physicians.

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Another key component of CompAC to ensure patient care was the rapid review of requests. Typically, when patients place a compassionate use request to a pharmaceutical company, the review process can be slow, resulting in delayed care. To obtain a fair review process, the organization worked independently from the sponsor, created 1 entry route into the organization, had a double-blinded strategy in place for all requests, and provided a standard form for use in all patient cases. In these statements, there was a patient narrative that allowed patients to communicate what they wanted or needed directly to the committee.

CompAC represents the first major pharmaceutical collaboration with an ethics committee for developing standardized guidelines for passionate use requests. “Patients and physicians are demanding access to promising treatments earlier and earlier, and they are turning more to social and mainstream media to make their cases,” the researchers concluded. “Efforts to find the best ways to expand fair access to experimental treatments for people who have no other options, therefore, must continue.”


Caplan AL, Teagarden JR, Kearns L, et al. Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials [published online July 7, 2018]. J Med Ethics. doi: 10.1136/medethics-2016-103917