Online Program

Does "Compassionate Use" of Investigational Treatments Prioritize Individual Rescue over Public Welfare?

Tuesday, November 3, 2015

Alison Bateman-House, PhD, MPH, MA, Division of Medical Ethics, Department of Population Health, New York University Langone Medical Center, New, NY
Occassionally, when a sick or dying individual has no treatment options left, she may seek access to an investigational therapy that has not received approval for use on patients.  Such provision of investigational therapies to patients outside of clinical trials is called, by the FDA, "expanded access," but they are commonly referred to as "compassionate use."  Compassionate use cases in the traditional (and, more recently, social) media typically receive support from the American public, which views the patient as needing rescue and the investigational treatment as being of potential benefit to the patient.  When companies decline compassionate use requests for whatever the reason, the decision is typically portrayed as the company prioritizing profits over individual lives; however, this framing of the situation conceals the fact that the patient seeking compassionate use access is not the sole patient who will be impacted by this decision.  There is a population of patients awaiting each new therapy, and if use of the investigational therapy in a compassionate use context results in death or other adverse outcome, the development of that potential therapy will likely be slowed and possibly halted.  Thus, while individual compassionate use cases are not considered research, they do impact drug development.  What is viewed as a case involving a single patient has implications for a "hidden" population of other patients, as well as  - potentially - the fortunes of an entire class of innovative treatments, such as gene therapy.  Making the investigational treatment available to a patient in the name of compassion thus has implications for the health of a larger number of individuals and, indeed, for the population as a whole.  This typically unacknowledged truth about compassionate use needs to be figured into individual compassionate use request decisions.

Learning Areas:

Ethics, professional and legal requirements
Protection of the public in relation to communicable diseases including prevention or control
Provision of health care to the public
Public health or related public policy

Learning Objectives:
Analyze the interests at stake in "compassionate use" requests. Explain how individual compassionate use requests may impact public health.

Keyword(s): Clinical Trials, Public Policy

Presenting author's disclosure statement:

Qualified on the content I am responsible for because: I serve as the non-voting, nonpaid deputy chairperson of the Compassionate Use Advisory Committee, an external, expert panel of internationally recognized medical experts, bioethicists, and patient representatives formed by NYU School of Medicine, which advises the Janssen division of Johnson&Johnson about requests for compassionate use of some of its investigational medicines. I am also the deputy director of the NYULMC Working Group on Compassionate Use & Pre-Approval Access.
Any relevant financial relationships? No

I agree to comply with the American Public Health Association Conflict of Interest and Commercial Support Guidelines, and to disclose to the participants any off-label or experimental uses of a commercial product or service discussed in my presentation.