Patient benefits from access to innovative drug that still awaits U.S. approval
A note from the field in this week’s Morbidity and Mortality Weekly Report describes the case of a dangerously ill man in the United States. Weak, coughing, and losing weight, with pain in his chest, he was tested for tuberculosis, which it turned out he had, and put on the first-line of treatment for the disease.
Three family members also were screened and tested for the disease, and his wife, testing positive for infection, started four months of preventive treatment.The extent of the danger, to him, and potentially to those exposed to him, began to become clear when he came down with a rash, when further testing revealed that his illness was resistant not only to the first line of treatment, but confirmed that it was resistant to five more medicines used to treat drug-resistant tuberculosis.
With what doctors now knew was extensively drug-resistant tuberculosis — XDR TB — the two newest approved treatments, and the only new treatments developed during this century, were seen as critical to improving his chances. One of them, bedaquiline was approved by the U.S. Food and Drug Administration in 2012. The other, delamanid, however, stalled by limited studies, had not been. Approved by the European Medicines Agency, though, it has been used, and shown safe and effective in countries with some of the highest tuberculosis burdens on earth.
With limited options his doctors applied for approval to use delamanid under a compassionate use program — an option opened by tuberculosis treatment advocates who had sought widened access to medicines found to be safe and effective. The FDA granted the approval in a day. Access to the drug however, the report notes, took four weeks.
It took just another four weeks, once the requirements had been met, the medicine shipped, and the patient received it, for his condition to improve. Within a couple of months, signs indicated he was on his way to recovery. He still faces at least another year of treatment, but is continuing to improve, and with no adverse effects. The report notes that the access to the drug added to his options, and it encourages more physicians to pursue its use.
It would be better still to have the drug already approved in the United States, noted Erica Lessem of the Treatment Action Group, which has long campaigned for expanded access to delamanid and other effective medicines.
“We need all of the tools possible to combat drug-resistant tuberculosis,” she said. “Delamanid is one of the safest drugs for treating multidrug-resistant TB, with more evidence available to support its efficacy than many other drugs.”
It is a treatment that can be used for children, she added. “Having delamanid more routinely available for treating drug-resistant tuberculosis in the United States and around the world, is critical.”