Interim Guidance on the use of bedaquiline, a new tuberculosis drug, for the treatment of multidrug-resistant tuberculosis (MDR-TB)

By Masoud Dara, MD Moderator | 13 Jun, 2013

Dear colleagues,

The World Health Organization (WHO) has issued interim guidance< on the use of bedaquiline, a new tuberculosis drug, for the treatment of multidrug-resistant tuberculosis (MDR-TB).

Bedaquiline was granted accelerated approval@@ by the United States Food and Drug Administration in December 2012. There has since been considerable interest in the potential of this drug to treat MDR-TB. However, information about this new drug remains limited and it has only been tested for safety and efficacy in two Phase IIb trials. WHO has therefore issued interim policy guidance.

This interim guidance provides advice on the inclusion of bedaquiline in combination therapies for MDR-TB in accordance with the existing WHO Guidelines@@> for the Programmatic Management of Drug-resistant TB (2011 Update).

The interim guidance lists five conditions that must be in place if bedaquiline is used to treat adults with MDR-TB:

1. Effective treatment and monitoring: Treatment must be closely monitored for effectiveness and safety, using sound treatment and management protocols approved by relevant national authorities.

2. Proper patient inclusion: Special caution is required when bedaquiline is used in people aged 65 and over, and in adults living with HIV. Use in pregnant women and children is not advised.

3. Informed consent: Patients must be fully aware of the potential benefits and harms of the new drug, and give documented informed consent before embarking on treatment.

4. Adherence to WHO recommendations: All principles on which WHO-recommended MDR-TB treatment regimens are based, must be followed, particularly the inclusion of four effective second-line drugs. In line with general principles of TB therapeutics, bedaquiline alone should not be introduced into a regimen in which the companion drugs are failing to show effectiveness.

5. Active pharmacovigilance and management of adverse events: Active pharmacovigilance measures must be in place to ensure early detection and proper management of adverse drug reactions and potential interactions with other drugs.

WHO strongly recommends the acceleration of Phase III trials to generate a more comprehensive evidence base to inform future policy on bedaquiline.

WHO will review, revise, or update the interim guidance as additional information on efficacy and safety become available. The organization is also developing an operational document to facilitate bedaquiline implementation and is working with partners to help ensure rational introduction.



Joanna Keenan Replied at 9:16 AM, 13 Jun 2013

Thanks Masoud for posting the guidelines. Médecins Sans Frontières has issued the below in response:

The World Health Organization has today issued interim guidance on the use of bedaquiline, the first new drug to treat tuberculosis in 50 years, which received accelerated approval by the US Food and Drug Administration (USFDA) on 31 December 2012. Acknowledging the growing crisis of multidrug-resistant TB (MDR-TB) and the urgent need for improved drugs with better efficacy and safety profiles, WHO has taken an unprecedented step to make interim recommendations about a drug based on phase IIb clinical trial data. Given the risk-benefit considerations for many people living with MDR-TB, MSF applauds this strong move to accelerate improved treatment to those who need it most.

The scale of the DR-TB epidemic is huge, with 310,000 new cases notified in 2011. But globally, only 19% of people thought to be infected are receiving some kind of treatment. It is hoped that bedaquiline – which has shown in trials to be potentially very effective against Mycobacterium tuberculosis, the bacteria that causes TB – could become a powerful tool in much-needed treatment regimens that are significantly shorter, more effective and less toxic than current regimens. Treatment today for DR-TB involves a two-year course of up to 20 pills per day and eight months of daily injections, which cause patients excruciating side effects and ultimately cures only one in two.

In 2012, Médecins Sans Frontières (MSF) treated 31,000 people with TB in 36 countries, 1,780 of whom had drug-resistant forms of the disease. MSF responds to the release of the WHO guidelines below:

“The new WHO guidelines on bedaquiline use are welcomed, and are very timely given the drug’s recent approval by the USFDA 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.

As one of the biggest NGO providers of DR-TB treatment, MSF sees people with increasingly drug-resistant forms of the disease, and without the development of new drugs that could, in combination with other drugs, result in shorter, more effective and less toxic regimens, DR-TB will continue to grow as a global emergency.

Drug companies and researchers have a big role to play in developing better new treatment regimens that can be scaled-up and avert a crisis. But that can only happen if companies collaborate and researchers accelerate studies of new drug combinations.

Securing affordability of these medicines will also be critical.”

- Dr Jennifer Cohn, Medical Coordinator, Médecins Sans Frontières Access Campaign

Kind regards


Joanna Keenan
Press Officer
Médecins Sans Frontières - Access Campaign
P: +41 22 849 87 45
M: +41 79 203 13 02
E: joanna.keenan[at]

Masoud Dara, MD Moderator Replied at 9:30 AM, 13 Jun 2013

Thank you Joanna,
MSF has been in the forefront of TB and MDR-TB prevention, control and care in many settings and your efforts are highly acknowledged.