Six hundred severely ill Covid-19 patients have received blood plasma from recovered patients in a study researchers hope sheds light on whether the experimental therapy improves health outcomes, and yields other useful data outside the scientific rigor of a traditional clinical trial, The Wall Street Journal reported.
The patients are participating in a national expanded-access program authorized in early April by the federal Food and Drug Administration. Expanded access, also known as compassionate use, is often sought by patients with life-threatening illnesses for which there are no approved therapies, or who can’t participate in clinical trials, the Journal added.
The utility of data from compassionate-use studies is a source of debate within the medical and scientific community, where the gold standard for determining a new drug’s safety and efficacy has long been the controlled clinical trial.
In those traditional randomized trials, one group of patients gets the experimental drug and a control group gets either the standard therapy or a placebo, the Journal writes.
Critics say it is impossible for compassionate-use studies to show whether a drug is working, because every patient in those studies gets the compound, with no control group for comparison. Opponents also worry that patients could become reluctant to enroll in traditional clinical trials for fear they won’t get the experimental therapy.
Without a control group, though, researchers can’t be certain what is making the difference. Age, gender, weight, underlying health conditions, socioeconomic status and doctors’ own biases all can influence a patient’s outcome.
And in many diseases, including Covid-19, some patients are going to get better on their own. As a result, compassionate use has been viewed as a way to give patients emergency access to experimental therapies rather than a source of reliable data, the Journal adds.