NYU Langone Medical Center Working Group on Compassionate Use and Pre-Approval Access
Patients facing death or serious disease who have exhausted all FDA-approved treatment options sometimes seek pre-approval, or “compassionate use,” access to experimental interventions outside of clinical trials. Access of this type is dependent on the willingness of the company that is developing the medical product to make it available, according to preliminary studies conducted by researchers and bioethicists at NYU Langone Medical Center (NYULMC). However, many pharmaceutical and life sciences companies either do not have policies governing pre-approval access, or, if they have policies, they are not made publicly available. As a result, patients and their advocates who request pre-approval access to experimental medical products frequently feel confused and frustrated, and they may perceive companies as being unresponsive.
Of particular concern has been the escalating use of social media by patients and their representatives to pressure companies into providing unapproved products — drugs, biologics, devices, or vaccines — on the grounds of compassionate use. This trend can lead to inequitable distribution of unapproved treatments: those most capable of exploiting social relationships (online or off-line) and connections are more likely to gain access.
“Use of social media or mainstream media pressure to achieve access favors the savvy and the well-connected over the poor. Equity, always at risk in our health care system, is again being flouted,” said Working Group member Nancy Dubler, LLB, an adjunct professor at the Division of Medical Ethics at NYULMC, the consultant for ethics for the New York City Health and Hospitals Corporation, professor emerita of bioethics at the Albert Einstein College of Medicine, and the founding director of the Montefiore Medical Center Division of Bioethics.
The unfairness in how unapproved interventions can be distributed was dramatically demonstrated in early 2014, when scarce supplies of an experimental Ebola drug were given to American and European relief workers rather than to West Africans affected by the ongoing Ebola epidemic in that region.
“This is an enormously important, albeit inadequately studied, problem in research ethics,” said Arthur Caplan, PhD, director of the NYULMC Division of Medical Ethics and a founding member of the interdisciplinary NYU Langone Medical Center Working Group on Compassionate Use and Pre-Approval Access. “The need to provide access and a chance of rescue for those in desperate straits must be thoughtfully balanced against the reality that cure with an unapproved intervention is a long shot, that not everyone can mount a media campaign, and that society is best served when drugs and vaccines are carefully tested.”
Also among the Working Group’s preliminary findings:
- Pharmaceutical and life science companies have no legal or regulatory obligation to provide access to unapproved treatments on the grounds of compassionate use. Even so-called “right to try” laws leave decisions about patient access to unapproved interventions to industry. Some companies allow access under the guidance of well-thought-out policies, some decline to allow access, some grant access but have no public guidelines, and some alter their practices midstream as a result of public pressure. This lack of uniform policy is confusing to those seeking unapproved treatments.
- Contrary to widespread perception, the FDA is not an obstacle to those seeking pre-approval access. In fact, the agency approves more than 99 percent of the pre-approval access requests it receives.
- The human impulse to help patients facing insurmountable odds motivates the general public’s support for both compassionate use and right-to-try laws. However, increasing access to unapproved therapies may prove detrimental to longstanding and proven regulatory requirements for clinical trials, delaying or even preventing promising therapies from reaching the market.
- Speeding up development of new therapies may result in the approval of unsafe treatments. Likewise, efforts to facilitate the use of unapproved treatments in seriously or terminally ill patients may actually harm those patients by causing more suffering or earlier death. Such adverse outcomes can, fairly or unfairly, tarnish trust in the new treatment.
The Working Group also found that questions about compassionate access arise in both developed and less-developed nations, for both infectious and chronic diseases and terminal and non-terminal conditions, and in both children and adults. It concludes that the issue of access to unapproved treatments demands domestic and global responses.
The Working Group conducts stakeholder interviews and surveys and examines relevant case studies from the United States and other nations as it strives to understand the issue of pre-approval access. As it reviews all facets of this complex issue, the group is authoring and disseminating recommendations for improving equitable access to unapproved treatments.
In spring 2015, the Compassionate Use Advisory Committee (CompAC) was launched as a pilot program to advise Johnson & Johnson about how to fairly manage requests for a scarce investigational cancer drug that the company was developing. Working Group members Drs. Arthur Caplan and Alison Bateman-House serve, respectively, as chair and deputy chair of CompAC. The investigational drug was approved by the FDA in late 2015, and CompAC is now working to publish its findings from the pilot project, which remains ongoing, as the drug has not yet been approved by regulators in other countries.