Miami Courant

Imagine you have a loved one hospitalized in intensive care facing death from Covid-19. All approved therapeutics were tried. Time has run out and you have been told to prepare for the worst. Your doctor advises there is one last drug to try, a natural protein that has been safely used in early trials and shown signs of effectiveness in rescuing patients from ventilators when all else has failed. The National Institutes of Health is doing a global study to prove its effectiveness. 

Unfortunately, the Food and Drug Administration will not give Emergency Use Authorization because the medicine showed only a 92 percent chance of being effective, rather than the “magic 95 percent” that is the traditional gold standard for approving drugs. 

I was that patient. I contracted Covid while serving on the front lines of a hospital, caring for my own patients. My story has a happy ending only because my lifelong friend, an ICU doctor, moved heaven and earth to cut through the red tape and obtain the medicine for me with the FDA’s permission. I was especially lucky that the medicine was being tested at a nearby hospital and the pharmaceutical company immediately rushed an emergency dose to my bedside. I was as close to death as one can be, treated not only with a ventilator but also with extracorporeal membrane oxygenation (ECMO), which few survive. By the time I got the medicine and began to recover, the hospital was already preparing me for a lung transplant.

Unfortunately, most Americans are not as fortunate as I was. Each day another 1,400 people die anonymous, lonely deaths in the ICU, isolated from their loved ones when some number could be saved by emergency use medicines.  The nation awaits confirmation of a new FDA Commissioner at a perilous time for public health as Covid continues to ravage the world. 

Congress passed Emergency Use Authorization to empower the FDA to give doctors, patients, and families access to medicines that “may be effective” when all else has failed. For the first year of the pandemic, EUA decisions were overseen by the Commissioner’s office. Promising therapies were given a chance to show their effectiveness, or lack thereof, for the benefit of patients and families. Some early EUA medicines, such as remdesivir and tocilizumab, went on to prove their benefit in definitive trials and became approved drugs, while others failed and were withdrawn. That sort of trial is exactly what Congress and the American people intended in the face of an unrelenting enemy. 

Unfortunately, in the absence of a confirmed FDA Commissioner, EUA decisions have been relegated to the traditional review groups that apply more rigorous measures of safety and effectiveness for permanent new drug approvals. FDA is listening to the voices of statisticians, not to the voices of doctors, patients, and families.

The medicine I received, vasoactive intestinal peptide called Zyesami, was shown in a clinical trial to give patients a substantially higher likelihood of getting off the ventilator to a 92 percent degree of certainty and to double the odds of surviving the ICU to a 99 percent degree of certainty. Of course, the statistic I care about most is that within days of receiving the medicine, my lungs began to function again. Within weeks, I was back home with her.  

Of course, a single experience such as mine cannot drive a drug approval.  However, there are multiple reports in the media of seemingly miraculous recoveries such as mine. The data on Zyesami  were sufficiently promising that Dr. Francis Collins, the director of the NIH, recently identified our medicine as one of a handful still under consideration by NIH in global trials from among more than 600 candidate medicines it initially considered. Dr. Anthony Fauci has similarly briefed the White House and Congress on the medicine, and he has invested substantial taxpayer funds in a global trial to test its effectiveness. As the nation awaits the results of the definitive NIH trial, it seems like common sense that we try medicines that “may be effective” provided they are safe.

Eventually, miraculous new pills may decrease the likelihood that patients will be hospitalized. In the meantime, the medicine that saved me may be the last, best hope for patients in the ICU who have exhausted all approved therapies. Today, physicians, patients, and families are currently denied this last chance, unless they resort to circumventing the FDA through Right to Try laws. Few physicians and hospitals have the courage to do so, even in the face of family demands. Fortunately, I had a doctor who would not take “no” for an answer and a small pharmaceutical company that happened to have enough medicine for me at a nearby clinical trial site.

For more people to have access to lifesaving drugs like this one, we need Congress to restore the original legislative intent of the EUA law.  Restoring EUA decisions to the Commissioner’s Office would allow those decisions to be made by a Congressionally confirmed senior member of the Administration who is willing to take prudent risks on behalf of the American people. It is also important for the commissioner’s door to be open to the voices of patients, families, physicians, and nurses, so that life and death decisions are not calculated simply by the numbers.

I ask the FDA to take measured risk with time-limited approvals and other tools that enable us to gain real world experience with medicines that may be safe and lifesaving. At a time when too many of us will see empty chairs around the holiday table, we must be willing give our loved ones one last chance at life.

– Dr. Elgozy practices internal medicine and pediatrics in Miami Beach, Florida and contracted Covid while serving on the front lines of the pandemic. He has no financial interest in any pharmaceutical company.