Final phase trials of drug pending; World Health Organization issues guidelines on interim use
Despite the lack of a Phase III clinical trial report of bedaquiline, the newly discovered drug for treatment of multidrug-resistant tuberculosis (MDR-TB), the World Health Organization (WHO) on Thursday issued an interim policy guidance notification on the use of the drug. Bedaquiline was discovered after a fifty year lull in development of drugs against TB – a period during which drug resistance of the disease made treatment difficult.
| Conditions for use of bedaquiline
- Treatment to be closely monitored for effectiveness and safety, using protocols approved by relevant national authorities
- Proper patient inclusion; caution when used in elderly, HIV infected. Not to be administered to pregnant women and children
- Patients to be made aware of potential benefits and harsm; to give documented informed consent
- Adherence to WHO recommendations, particularly inclusion of four effective second-line drugs
- Active pharmacovigilance to detect and manage adverse drug reactions
WHO estimates that up to half a million new cases of multidrug-resistant tuberculosis (MDR-TB) occur worldwide each year. Current treatment regimens for MDR-TB present many challenges, including treatment that lasts 20 months or more, requiring daily administration of drugs that are more toxic, less effective, and far more expensive than those used to treat drug-susceptible TB. Globally, less than half of all patients who start MDR-TB therapy are treated successfully.
Lack of complete data
Bedaquiline was approved in the US last December and has a novel mechanism of action. However, many countries have been avoiding its use due to absence of data showing its efficiency and efficacy against TB. The drug has undergone only two Phase IIb trials for safety and efficacy. It is keeping these facts in mind that WHO is issuing interim policy guidance.
The guide provides advice on the inclusion of bedaquiline in combination therapy against MDR-TB in accordance with existing WHO Guidelines for the programmatic management of drug-resistant TB. The interim guidance lists five conditions that must be in place if bedaquiline is used to treat adults with MDR-TB (see box). WHO also strongly recommends the acceleration of Phase III trials of bedaquiline to generate a more comprehensive evidence base to inform future policy on use of the drug.
One of few drugs effective
Médecins Sans Frontières (MSF), an international non profit that provides TB treatment to people around the world, approved of the move. “The new WHO guidelines on bedaquiline use are welcomed, and are very timely given the drug’s recent approval by the USFDA (US Food and Drug Administration) and the urgent need to scale-up treatment of drug-resistant TB. Regulated, controlled use of bedaquiline is essential in ensuring we don’t burn one of the very few drugs available that could effectively treat drug-resistant TB,” said MSF in a press release.
Experts believe that bedaquiline could become a powerful tool in much-needed treatment regimens that are significantly shorter, more effective and less toxic than current regimens.
Global tuberculosis report 2012
Guidelines for the programmatic management of drug-resistant tuberculosis
Lives saved by tuberculosis control and prospects for achieving the 2015 global target for reducing tuberculosis mortality
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