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.