Faculty Bibliography

BACKGROUND:Janssen Research & Development, LLC, part of the Janssen pharmaceutical companies of Johnson & Johnson, and NYU School of Medicine partnered to establish the Compassionate Use Advisory Committee (CompAC) to evaluate the use of an independent, external, expert committee in ensuring transparent, fair, beneficent, evidence-based, and patient-focused compassionate access to investigational medicines, a public health challenge that has been an ongoing issue for over 3 decades. METHODS:To this end, NYU School of Medicine was responsible for the formation, member selection, and operation of CompAC, consisting of physicians, ethicists, and patient advocates, under Johnson & Johnson's sponsorship. RESULTS:A pilot was successfully run using CompAC to provide recommendations on compassionate use access to a Johnson & Johnson oncology investigational asset called daratumumab. CONCLUSION/CONCLUSIONS:This innovative model provides a framework that can be emulated by the industry globally.

BACKGROUND:Patients who are seriously ill and have run out of available treatment options may seek access to investigational agents that have not yet been fully vetted by regulatory agencies for safety and efficacy and approved for use in human subjects. Over time, a variety of terms have evolved internationally to denote mechanisms for providing access to such unapproved investigational agents. The lack of consistency in terminology used to describe this process is confusing at best and, at worst, possibly even detrimental to patients. METHODS:To highlight variation around the globe in terminology denoting pre-approval access to investigational agents, we conducted extensive Internet searches to locate specific legislation, guidance, or policy documents describing access mechanisms in numerous countries. We created a table of results intended to convey a sampling of international terminological diversity. RESULTS:The profusion of terms used internationally to indicate pre-approval access to investigational agents is evident. We recommend a shift toward the use of "pre-approval access" as an umbrella term encompassing all forms of access to unapproved agents. We also recommend use of the phrases "individual/named patient regulatory routes for pre-approval access" and "group/cohort regulatory routes for pre-approval access" to differentiate between pre-approval access programs designed for single patients, versus those designed for groups of patients. CONCLUSIONS:There is a pressing need to revisit and better align pre-approval access terminology at the international level. Adopting the umbrella term "pre-approval access" may be a useful strategy for initiating and promoting harmonization of terms to reduce potential confusion by patients and health care decision makers regarding experimental treatment options.

As of late November 2016, 32 states had adopted right to try laws. These laws are intended to allow terminally ill patients pre-approval, or "compassionate," access to drugs, devices, or biologics that are in development and have not yet been approved by the United States Food and Drug Administration (FDA). While the laws' intentions and their impact have been examined, little has been written about variations among the state laws. An examination of the specific provisions in and differences among the 32 statutes, and who stands to gain from them, suggests that the benefits of right to try laws are largely rhetorical. So, although the laws have been heralded as pro-patient, they ought to be understood as merely masquerading as patient-centric legislation. We call for a re-examination and amendment or, ideally, repeal of these laws in order to prevent the very real risk of patient harm caused by both some of the laws' provisions and patients' confusion arising from these misleading statutes.

Institutional review boards (IRBs) are one of the bodies charged with prospectively reviewing compassionate use, the hopefully therapeutic use of an unapproved drug in a seriously ill or dying patient who has no other treatment options. However, there are ethical issues in assigning this role to a body whose primary purpose is to review research proposals. The role of IRBs with regard to compassionate use must be examined and potentially revised.

Patient access to experimental drugs outside of clinical trials is called compassionate use or expanded access. Compassionate use/expanded access presents a powerful ethical dilemma in that it involves competing claims that both have moral weight: specifically, an individual patient's very understandable desire to try to extend his or her life versus the orderly and efficient functioning of a drug development and clinical trial system that benefits much larger numbers of patients. Patient advocates, the FDA, pharmaceutical trade groups, and state and national legislators in the US are all currently weighing in on patient access to experimental drugs, and new guidelines and rules are being introduced. In this editorial, we discuss the impulse to rescue individual patients facing dire diseases and underscore the ethical questions that such rescue efforts raise.

How ought public officials address policy choices that entail trade-offs between desirable public health goods? Increasing cycling improves public health both by promoting physical activity and by decreasing vehicle use, thus reducing vehicular emissions. Proponents of bicycle helmets argue that, used properly, they protect individual cyclists; however, there is concern that mandating helmet use may result in a decrease in cycling. In 2012, New York City Mayor Michael Bloomberg opposed a bicycle helmet mandate, concerned that it would have a negative impact on the city's cycling rate, which he had sought to increase. The mayor did not explain his rationale, leaving constituents unsure why he opposed the proposal. This case study underscores the challenge of creating public policy in the context of competing public health goods.