Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials

Abstract

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.

Bioethics in the COVID-19 Pandemic Research: Challenges and Strategies

“…a) Governments and institutions should work together with drug companies to design and implement compassionate use programs and prevent unethical conducts. An example is to follow the practices of the "Compassionate Use Advisory Committee (CompAC)" sponsored by Pharma companies but organized by an academic institution 22…”

Ethical decision-making in biopharmaceutical research and development: applying values using the TRIP & TIPP model

“…Finally, a further limitation is the likelihood of bias resulting from the decision-making team being company employees, with no independent actors. In fact, our model has been specifically developed for internal decision making because of the need to generate applicable solutions to the specific biopharmaceutical context, that is, R&D. Although it does not foresee systematic recourse to external (bioethics) expert advice or panels, 39 it does not preclude asking for or including such knowledge as appropriate. In some cases, specific questions may require input from independent stakeholders, for example, patient and/or ethical advisory groups.…”

Ethics codes and use of new and innovative drugs

“…This results from the fact that over the last decade or so the importance and prevalence of experimental treatments in different medical specialties has substantially grown . Growing interest in novel therapies results from a variety of reasons including the existence of serious unmet medical needs, high activity of patient advocacy groups and the availability of data about emerging treatments in the Internet . Moreover, therapeutic use of unproven treatments is associated with a number of tough ethical dilemmas ; therefore, relevant guidance contained in ethics codes would be very helpful for doctors of various specialties.…”