MSF trial finds better treatment for people with TB

NEW YORK/LONDON, DECEMBER 21, 2022—A new all-oral, six-month treatment regimen is safer and more effective at treating multidrug-resistant tuberculosis (MDR-TB) than the current options, according to results of a Doctors Without Borders/Médecins Sans Frontières (MSF) study published in the New England Journal of Medicine today. These findings come from MSF’s TB PRACTECAL, the first-ever multi-country, randomized, controlled clinical trial to report on the efficacy and safety of a six-month, all-oral TB treatment regimen.

“We’re delighted that the trial results have been published in the New England Journal of Medicine after a stringent peer review process,” said Dr. Bern-Thomas Nyang’wa, MSF medical director and chief investigator of the clinical trial. “This publication will provide deeper evidence to policymakers and treatment providers deciding to use the TB PRACTECAL regimen. Until relatively recently, there were no new treatments for TB introduced for over 50 years. Why? Because the disease doesn’t affect the people who have the resources to deal with it. This trial was an attempt by MSF to fill that gap. Now it is essential that the new treatment is made available to everyone who needs it.”

Launched in 2017, TB PRACTECAL tested three combinations of new treatments against the locally accepted standard of care in seven sites across Belarus, South Africa, and Uzbekistan. Of these three combinations, a six-month regimen of bedaquiline, pretomanid, linezolid, and moxifloxacin (BPaLM) proved most effective and safe. The trial ended enrollment in March 2021, with 552 patients overall. Now, five countries, including Belarus, Uzbekistan, and Tajikistan, have begun implementing the shorter regimens with MSF's help, resulting in almost 400 patients starting treatment. Eight more countries are set to implement it in 2023.

“We began the TB PRACTECAL clinical trial nine years ago because something had to be done,” Dr. Nyang’wa said. “Patients were telling us that the previous regimens were lengthy, ineffective, and grueling—and that the side effects were worse than the disease itself. They also weren’t very effective; just one in two people were cured. The new regimen, BPaLM, has an 89 percent cure rate, is safer, shorter, and more tolerable with many fewer pills.”

MSF’s phase II/III clinical trial found that the new shorter BPaLM treatment regimen was very effective as 89 percent of patients in the BPaLM group were cured, compared to 52 percent in the group that received the standard regimen. There were also fewer recorded side effects in the BPaLM group than among those who received the standard regimen.

“When I learned that I had TB, I just couldn’t believe it was true," said Abdirakhman, a 24-year-old mathematics student in Uzbekistan who had to pause his studies when he was diagnosed. "[Being diagnosed with drug-resistant TB (DR-TB)] was devastating. I chose to join the TB PRACTECAL clinical trial and was randomly selected to receive the short treatment course of six months compared to the standard two years or more. There were difficult periods, but it was still better than two years of treatment. Now I have finished my course of treatment, and I am back at university."

While this new regimen provides hope for the 500,000 people who fall sick each year with DR-TB, the current lowest global price provided to the Global Drug Facility (GDF) for a six-month treatment course of BPaLM is around $600, still above the $500 ceiling price called for by MSF. One of the newer TB drugs, bedaquiline—developed by Johnson & Johnson with substantial government and philanthropic support—is priced at $270 for six months as the lowest global price. This is despite the fact that researchers have estimated that bedaquiline could be produced and sold at a profit for less than $102 for six months. In fact, all three combinations studied in TB PRACTECAL are likely to reduce treatment costs compared to the current standard of care.

“Rolling out the shorter, safer, and more effective BPaLM treatment regimen trialed in TB PRACTECAL could transform the lives of people with TB, but only if the drugs in this regimen are affordable,” said Christophe Perrin, TB advocacy pharmacist with MSF’s Access Campaign. “As significant public funds helped to pay for the development of bedaquiline, we’re calling on Johnson & Johnson to bring down the price of this drug so that a complete DR-TB treatment course is no more than $500 per person. Too many lives have been lost due to this killer disease. People with TB deserve urgent access to shorter, safer, and affordable treatments.”

The publication of these results follows last week’s release of new World Health Organization guidelines for TB treatment that recommend the use of BPaLM.

MSF is one of the largest non-governmental providers of TB treatment worldwide. In 2021, 17,221 people in MSF’s care were started on TB treatment, including 2,309 people with DR-TB. MSF is working closely with national TB programs, ministries of health, and other key stakeholders to ensure that this new regimen is available to people with DR-TB as soon as possible.