But outside experts in clinical trial design worry that the results, instead of leading to a clear picture of whether the medicine is effective, will instead muddy the waters further.
The main concern, they say, stems from the fact that the Gilead trial expected to read out this week, which was conducted among patients with severe disease, lacks a control group — that is, patients who are randomly assigned to receive the best treatment available, but not remdesivir. As designed, the only randomization is the duration of treatment: either five days or 10 days of drug. Without a true control group of patients, many experts say, it will be difficult to determine whether remdesivir is effective.
“The overall study itself has little or no scientific value since all patients are receiving the drug,” said Steven Nissen, the chief academic officer at the Cleveland Clinic and lead investigator of many trials for heart drugs that have been approved by the Food and Drug Administration.
“The study, as designed, is essentially useless and cannot be used by the FDA for consideration of remdesivir for approval to treat coronavirus,” Nissen said.
Peter Bach, director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center, called the situation “frustrating.”
“For them to run the trial in severe but not include a control group, it’s just such a waste,” Bach said.
The predicament is symptomatic of one of the biggest problems in medicine: Collecting data quickly can actually slow things down if studies are not designed in a way that gives clear, definitive answers.