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“Compassionate Use” and Other Means of Accessing Unproven and Unapproved Treatments Could Impact Long-Term Medical Benefits

Researchers at NYU Langone Medical Center Analyze Who Gets Access and How, Looks to Propose a More Equitable System for Treatment Dissemination

Patients facing death or irreversible disease progression – most of whom have exhausted all approved treatment options -- sometimes seek access to unapproved and unproven interventions.  This type of access, often referred to as “compassionate use,” is  unregulated by federal authorities, subject to corporate pharmaceutical policies that change mid-stream, and could potentially adversely affect  clinical care in the future, according to preliminary studies conducted by researchers and bioethicists at NYU Langone Medical Center (NYULMC).

Of particular concern, the researchers point out, is the escalating use of social media by patients and their representatives to pressure decision-makers into providing unapproved drugs, devices, or vaccines on the grounds of compassionate use.  This shift, they say, highlights inequitable distribution of unapproved treatments: Those most capable of exploiting their social relationships (online or off-line) are more likely to gain access to unapproved treatments -- and the possibility of medical benefit.

This has been demonstrated  recently with scarce supplies of an experimental Ebola treatment being given first to American and European relief workers rather than to the West Africans impacted 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 the founding member of an interdisciplinary group working on guidelines for more equitable access of compassionate use.  “The need to provide access and a chance at rescue for those in desperate straits must be thoughtfully balanced against the reality that cure is a long shot and society is best served when drugs and vaccines are carefully tested.”

The researchers explored the primary factors contributing to the current inequities in compassionate use and similar protocols by analyzing what  is available on the subject in published literature and on the internet.  In addition, they scoured through resources on the subject provided by other experts in bioethics.   “While we readily admit that this was an imperfect model for conclusive findings, it was more information, analyzed more systematically, than anything else done previously,” Dr. Caplan added. “A more systematic survey is planned when more funding is made available.”

  Among their preliminary findings: 

  • Biotechnology companies have no legal or regulatory obligation to provide access to unapproved treatments on the grounds of compassionate use.  Some companies allow access under the guidance of well thought out policies; some companies decline to allow access; some companies grant access but have no set guidelines; and some companies change 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 U.S. Food and Drug Administration (FDA) is not an obstacle to those seeking compassionate use.  In fact, the FDA almost always defers to the company that is developing the unapproved treatment to decide whether to grant compassionate use access.
  • Contrary to widespread perception, the U.S. Food and Drug Administration (FDA) is not an obstacle to those seeking compassionate use.  In fact, the FDA almost always defers to the company that is developing the unapproved treatment to decide whether to grant compassionate use access.
  • The “human impulse” to help patients facing insurmountable odds motivates both the general public’s support for compassionate use and so-called "right to try” laws to help gain access to unapproved treatments.  However, increasing access to unapproved therapies may prove detrimental in the long run to longstanding and effective research and clinical trial systems through which interventions are proven effective and safe, and given regulatory approval.
  • Efforts to speed up the development of new therapies and to shorten the time it takes for a treatment to move from clinical testing to clinical use may result in unsafe treatments being approved.  Likewise, efforts to facilitate the use of unapproved treatments in terminally ill patients or others who need immediate treatment may result in those individuals being at increased risk of harm.  Furthermore, if very sick patients get worse after treatment with an unapproved intervention, that intervention may be (fairly or unfairly) tarnished by the adverse outcomes.

Researchers also point out that questions about who should receive access to unapproved treatments arise in both developed and less-developed nations.  These treatment requests most often concern both infectious and chronic diseases, and both children and adults.  

The researchers conclude that the issue of access to unapproved treatments demands both an analysis of what is happening domestically and globally, and a global response that is driven not by innuendo and outside pressures, but by solid data.

“Use of social media pressure to achieve goals of early access favor the savvy and the well-connected over the poor.  Equity, always at risk in our health care system, will again be flaunted.”said working group member Nancy Dubler, LLB, faculty affiliate of the Division of Medical Ethics at NYULMC, consultant in Bioethics for the New York City Health and Hospitals Corporation and founding director of the Montefiore Medical Center Division of Bioethics .

To accurately depict the current application of compassionate use, the researchers are continuing their work by conducting stakeholder interviews and surveys; and by examining relevant case studies from the United States and other nations.  After reviewing all facets of this vexing issue, the group plans to author and disseminate clear recommendations for improving equitable access to unapproved treatments. 

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