April 14, 2020
By Naomi Lopez
In what may ultimately be progress in the development of treatments in the fight against COVID-19, Gilead Sciences’ remdesivir may hold promise. In a small observational study, about two-thirds (68 percent) of patients showed clinical improvement with the treatment.
Today, there are no FDA-approved treatments for COVID-19. Remdesivir had been tested in clinical trials for Ebola but was abandoned as other treatments proved to be far more effective. As a “broad spectrum” drug that showed promise in the lab and in animal studies for Middle East respiratory syndrome (MERS) and severe acute respiratory syndrome (SARS), both coronaviruses, later-stage clinical trials for COVID-19 quickly began in China, the U.S., and several other countries.
Beginning in late January, the company began offering the treatment on an emergency basis for hospitalized patients who didn’t qualify for a clinical trial. More than 1,800 patients have been treated outside of clinical trials in multiple countries, but remdesivir is not yet an approved treatment for COVID-19 in any country.
On Friday, initial results of data collected on 53 patients who were treated through “compassionate use” were published in the New England Journal of Medicine. This was an observational study meaning that there was not a randomized control group, less data was collected on the patients than what would normally be collected in a trial, and patients were followed for only 28 days.
There has been much discussion about the importance of only allowing COVID-19 treatments outside of clinical trials if broad data collection is in place. While manufacturers have an incentive to collect some data (to inform future trials, for example), there is no requirement for broad data collection except when there is an adverse event (meaning that a patient has a significant and severe reaction to the treatment). That is because access outside of clinical trials is not a clinical trial; it is intended to help the patient if their doctor recommends the treatment and if the manufacturer is able and willing to make it available.
It is understandable that there is a strong desire for more data both for patient safety and for more certainty around treatments. But the big trade-off is time—making the perfect data the enemy of a treatment delivered in time. At a time when lives hang in the balance, it doesn’t make sense that a desire for more or more perfect data stand in the way of what a doctor thinks is best for their patient.
In fact, the FDA’s own guidance for industry explains why data is less valuable outside of a clinical trial. The guidance states:
“Expanded access INDs and protocols are generally not designed to determine the efficacy of a drug; however, the expanded access regulations do not prohibit the collection of such data. Because expanded access INDs or protocols typically involve uncontrolled exposures (with limited data collection), it is unlikely that an expanded access IND or protocol would yield efficacy information that would be useful to FDA in considering a drug’s effectiveness.”
Sadly, treatment decisions have become politicized. But treatment decisions, whether inside or outside of clinical trials, are deeply personal decisions that belong in the hands of patients and their physicians and the manufacturers who are able and willing to provide them—not in the hands of the president, governors, or what happens to be popular on the internet.
We are in the midst of intense investment, as well rapid medical innovation that holds the promise of treatment and immunization on a shorter timeline, in the fight against this pandemic. And there is a growing realization that today’s FDA, which has responded and adapted quickly in some areas, remains slow to cut the red tape and accelerate the innovations now taking place.
Naomi Lopez is the Director of Healthcare Policy at the Goldwater Institute.