How should we decide who gets promising drugs in short supply? In April, the National Institutes of Health announced that an experimental drug, remdesivir, accelerated recovery from COVID-19. Its manufacturer offered to donate 940,00 vials of the antiviral to the U.S. With no end of the epidemic in sight, this donation will likely be insufficient to treat all patients. But who should get the drug, if it cannot be made available to all patients?
The Janssen Pharmaceutical Companies of Johnson & Johnson faced a similar question several years ago. A new drug in development offered promise in treating multiple myeloma, and there were many patients interested in trying the investigational drug: far more than could enroll in the ongoing clinical trials. Janssen was willing to make it available through “compassionate use,” but there was a limited amount available.
As in that experience, we are now in a situation where there is an overwhelming demand for a scarce drug that may offer patients their best chance for medical benefit. What lessons from this previous experience can we use to guide the allocation of limited drugs during the COVID-19 pandemic?
Janssen and the NYU Grossman School of Medicine’s Division of Medical Ethics collaborated to form the Compassionate Use Advisory Committee (CompAC), a group comprised of internationally recognized medical experts, bioethicists, and patient advocates selected and led by the Division of Medical Ethics.
Janssen and the NYU Division of Medical Ethics developed a process whereby any physician could apply for access on behalf of their patient via a portal on the company’s website. Requests for patients eligible to enroll in clinical trials were met with information about suitable trials. In contrast, requests for patients in whom the use of the drug was considered to pose unacceptable safety risks were denied.
Requests on behalf of patients who had not yet tried approved and available multiple myeloma treatments were met with instructions to try those. The remainder–requests for patients with multiple myeloma who had exhausted all approved treatments, did not qualify for a clinical trial, and for whom the use of the drug posed no special medical concerns — were sent to CompAC.
CompAC sought to establish a just and fair process, to incorporate scientific and medical evidence into decisions, and to consider patient perspectives and needs. CompAC considered lottery and first-come, first-served methods for allocating the scarce drug but ultimately rejected these.
Members felt that potentially life or death decisions should be made on patients’ likelihood of benefit from the investigational drug. Yet, CompAC recognized it was impossible to make decisions based solely on medical grounds: the drug was experimental and there was not yet a full understanding of which patients would benefit.
CompAC members thought it important to rely upon the available data to evaluate which applicants seemed most likely to benefit. In addition to the Janssen medical and drug development teams, who were able to answer questions about the applicants and the drug, independent medical advisors were consulted. As clinical trials continued, CompAC was kept updated to make its decisions according to the most recent knowledge about the drug’s safety and efficacy.
CompAC was also concerned about the need to develop an allocation process that was seen as fair. For decades there had been reports that the famous, wealthy, or politically connected had been able to use those advantages to increase their chances of accessing potential new treatments. This benefitted those patients at the cost of disadvantaging others. CompAC sought to avoid possible sources of bias. Applications were redacted to remove the names of the patient and doctor, location, gender, race, and ethnicity. In addition, CompAC utilized age only as a last resort tie-breaker
COVID-19 is not cancer, and a disease that can kill in days is not the same as a slower one. But there are useful lessons from the NYU/Janssen experience. First, allocation decisions must be transparent, and those making these decisions must not be solely clinicians. Some distance from the bedside would be helpful.
As the COVID-19 treatment remdesivir is being distributed via state health departments, they may be the best entities to make allocation decisions concerning it. Deciders must have the most accurate data, to ensure that patients most likely to benefit are prioritized in terms of access.
Recently published data show remdesivir worked better in study participants receiving oxygen support than in those who were sicker, on ventilators, or receiving external cardiac and respiratory support. That information should have been divulged sooner, to guide decisions about who to give that scarce drug. It is morally imperative to continue to gather data to refine our understanding of what interventions work best, in what patients, when given at what point in the disease.
When there is not enough for everyone, medical resources–be those drugs or organs — must be allocated in the fairest, most transparent way possible. We must utilize good data to make those decisions and good communication to explain who is making the decisions and on what grounds.
It is enormously encouraging that a useful treatment has been identified in the short time since the emergence of COVID-19. But the harm of the disease itself now stands to be matched by the harms of poorly planned distribution of treatments. We must not allow that to happen.
Alison Bateman-House is an assistant professor, Division of Medical Ethics, Department of Population Health, NYU Grossman School of Medicine and Chair, CompAC Infectious Disease and CompAC Neuro/Psychology.