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Compassionate Use: Fix What Is Broken

arthurcaplanby Kelly McBride Folkers and Arthur L. Caplan, Division of Medical Ethics, NYU School of Medicine


Over the past decade, cancer research and treatment has advanced well beyond what scientists thought possible. The rhetoric around cancer certainly has changed, and breaking its fearful grip on patients and their loved ones seems more within reach than ever. Vice President Joe Biden’s chairmanship of the cancer “moonshot” project is just one example of our society’s hopefulness that we can, once and for all, beat cancer.


But, hope still can seem elusive in the real world of cancer. Patients spend days in the confines of a hospital, receiving uncomfortable infusions that make them sick, being poked and prodded with huge needles, and anxiously waiting for scan results in waiting rooms. Treatments fail, many times inexplicably. Cancer patients who participate in clinical trials, which provide a great service for advancing medical research, don’t always benefit. For those whose disease continues to evade the best and brightest doctors and researchers, the rhetoric of cure is just that. Hope can be easily lost.


Compassionate use is one way to provide some hope to patients who have tried every available treatment option or clinical trial to no avail. Pharmaceutical companies currently investigating medical products that have not yet received Food and Drug Adminstration (FDA) approval can choose to allow patients to try an experimental treatment. Patients and their physicians interested in trying an experimental drug must first ask the manufacturer if the company is willing to provide the drug on a compassionate use basis. Then, the FDA must approve an individual patient’s request to try an experimental treatment outside of a clinical trial followed by approval by a research ethics committee or institutional review board (IRB).


Some patient advocates claim that the current compassionate use system does not do enough for patients, citing the bureaucratic hurdles that prevent their doctors from getting them potentially life-saving medications. In response to these concerns, the FDA recently streamlined the process for applying for compassionate use, consolidating the required paperwork into a two-page document that takes approximately 45 minutes for a physician to complete. The agency approves over 99% of requests for compassionate use that it receives within 30 days, though most requests are approved much faster.


The main issue with compassionate use is that, contrary to what many say, it is drug manufacturers, not the FDA, who are ultimately responsible for determining who gets access to their products. When a drug is being investigated in a clinical trial, it is not yet mass produced, meaning that the supply may be scarce. Adverse events can occur before a drug has been fully and completely understood, causing some companies to be hesitant to accept compassionate use requests for fear that their product will get a bad reputation on Wall Street or among angel investors.


What makes this process even more frustrating is the lack of transparency about compassionate use policies. A recent study showed that a mere 19% of pharmaceutical companies they studied in the United States make their compassionate use policies publicly available. Patients often feel responsible for researching their treatment options, especially if they are receiving care in a medical center that has little experience with compassionate use requests. Without publicly available compassionate use policies, without some kind of legal regulation that requires good tools to let doctors and patients find what is out there, patients are left wondering why some people are granted compassionate use and others aren’t. For all they know, the system is not fair.


Hospitals also struggle with how to handle compassionate use requests. Before a patient who has successfully gotten access to an experimental drug can start using it, a hospital’s IRB must sign off, as they must whenever any patient is taking an experimental medication. There is no consistency with how IRBs handle these requests, and patients are often confused about why their doctors need to ask permission from yet another party, besides a company and then the FDA.


Compassionate use has a significant set of hurdles which understandably frustrate terminally ill cancer patients and their families and friends. The FDA’s ultimate approval of medications is necessary to ensure that what reaches the market is safe and effective thereby insuring the needs of the many are protected.


But asking an individual patient to weigh the needs of the many while fighting to survive seems too big of a burden to bear. It is the responsibility of the pharmaceutical industry, the player ultimately responsible for deciding who gets compassionate use of a drug, to make its system fair. Some are already making great strides, like Johnson & Johnson’s use of the NYU School of Medicine’s Compassionate Use Advisory Committee (CompAC), an independent panel of physicians, bioethicists, and patient advocates who provide guidance on how to transparently and fairly allocate investigational drugs to patients outside of a clinical trial. CompAC’s findings are publicly available. Hopefully other companies will create their own committees.


Compassionate use is not just about having the liberty to ask for an experimental drug; it is a matter of respect for patients. It’s a matter of responding to patient requests in a timely manner making company policies publicly available and insuring they are accessible to all. Compassionate use, despite the great uncertainty of whether an experimental drug will work at all, provides some hope that patients can fight cancer while understanding that clinical research is the key to ultimately winning the battle.



arthurcaplanDrs. William F and Virginia Connolly Mitty Professor and founding head of the Division of Bioethics at New York University Langone Medical Center in New York City.  He is currently a member of the University of Pennsylvania’s External Advisory Committee for its Orphan Disease Center and a member of the Ethics and Ebola Working Group of the World Health Organization. Dr. Caplan also serves as the Chairperson of the Compassionate Use Advisory Committee (CompAC), an independent group of internationally recognized medical experts, bioethicists and patient representatives which advises Janssen/J&J about requests for compassionate use of some of its investigational medicines.





Kelly McBride Folkers is a graduate student in bioethics at NYU and a researcher at the NYU School of Medicine’s Division of Medical Ethics.




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One Response to Compassionate Use: Fix What Is Broken

  1. Amanda says:

    Thank you for sharing more insight with regard to the accessibility of experimental drugs on compassionate use grounds.

    No words offer any justice to the memory of my 14 year old daughter as she lay dying of DIPG. Trapped inside a failing body, yet fully aware of everything around her, begging for access drugs to drugs on compassionate grounds because she did not want to die! Demanding her human right to at least try a particular drug which had shown effiacy in pre-clinical trial studies and was awaiting FDA approval. A drug that is manufactured here in Australia where we live. A drug that was recommended by a leading research Professor 6 months before my child died. A drug that my child was fully aware of. A drug that would have been offered to my daughter if she was an adult with skin cancer.

    We had authorisation from TGA and the hospital (I believe) and a doctor willing to assume responsibility for administering the drug; we even had a protocol (albeit not authorised at the time by the FDA) – Despite all this – access was denied. I offered to purchase the drug at whatever cost – again access was denied.

    I know this particular drug was nearing the end of an adult phase 3 trial.. I can only assume the correlation, although this statement appears fitting
    “….hesitant to accept compassionate use requests for fear that their product will get a bad reputation on Wall Street or among angel investors… ”

    My child’s human rights were denied as she tried everything possible to sustain her own life, grasp hold of that last glimmer of hope. She was frightened, and ‘She Knew’ her life ‘as a child’ was not deemed important. My 14 year old daughter died with full knowledge that her life came down to cost. In many ways I wish she had not been as articulate.. sadly her advocacy role well equipped her with the dire state of affairs in paediatric oncology.

    I truly welcome improvements around disclosure, transparency and accessibility to experimental drugs on compassionate grounds.

    Thank you

    Amanda, Forever Erin’s mum

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