Case 1:Bedaquiline (Sirturo®), Johnson & Johnson
Access to this new, on-patent MDR-TB medicine is tightly controlled through national TB programmes and donations, coupled with tight stewardship controls. These measures have a good chance of preventing drug-resistance.
- First MDR-TB treatment to reach market in 40 years
- New mechanism of action (ATP Synthase Inhibitor)
- Only available for pulmonary MDR-TB with long-course regimens and shorter regimens
- Restrictive access conditions require pharmacovigilance infrastructure, clinical monitoring
- Access provided through national programmes and donations via the USAID Bedaquiline Donation Programme
- Equity-based inter-country tiered pricing approach in place
- Registered in 23 countries where access to medicine is likely limited
- Supports education for healthcare professionals on AMR, including on pharmacovigilance and TB-specific workshops
- No sales teams deployed
- Surveillance programme running (DREAM)
Tuberculosis (TB) poses a critical threat from AMR. In 2012, the U.S. Food & Drug Administration (FDA) gave fasttrack accelerated approval, based on Phase II clinical studies, to the first innovative treatment for pulmonary multi-drug resistant tuberculosis (MDR-TB) and extensively drug-resistant tuberculosis (XDR-TB) in 40 years: Johnson & Johnson’s bedaquiline (Sirturo®).
Due to its accelerated approval, the medicine was not tested for long-term effectiveness and safety in larger populations. To further test the medicine and also to prevent acquired resistance from emerging, WHO issued a strict interim guideline on its use, monitoring and pharmacovigilance. Countries with weaker health systems may be less likely to meet some of these criteria.
When filing bedaquiline for registration, Johnson & Johnson prioritised countries with high TB burdens. Russia was second to receive market approval, following the USA. Approval was granted in South Africa, the Philippines and Peru in 2014, the same year as in the European Union. These are countries with some of the highest MDR-TB burdens.
Today, bedaquiline is available via five routes: traditional reimbursement by national authorities, purchasing via the Global Drug Facility (GDF), equitable tiered pricing, institutional purchasing by international NGOs, and through a tightly controlled donation programme managed by USAID. In countries where bedaquiline is not registered, access is possible with a WHO import waiver. Across the various access channels, 35,000 cumulative treatments have been delivered, mostly to India, Russia and South Africa. The donation programme was developed in 2015 with USAID to increase access in over 100 low- and middle-income countries. The company committed to donating 30,000 treatment courses in four years. According to Johnson & Johnson, 103 countries now have access, including 29 of the 30 countries with high MDR-TB burdens. The programme also includes specialist education on the use of bedaquiline and on activities to minimise risk, as well as technical support for establishing active pharmacovigilance. These capacity building activities are supported by USAID, national and local partners.
Addressing the cost barrier
To address affordability of bedaquiline, Johnson & Johnson has an equitable pricing strategy that sets prices according to a country’s ability to pay. Its pricing tiers are based on criteria such as World Bank income classifications, GDP/GNI per capita and MDR-TB burden. Countries that do not meet the criteria or WHO’s Guidelines, may receive additional technical support via other stakeholders, including the WHO and USAID.
Johnson & Johnson supports activities to raise awareness of TB and MDR-TB among healthcare professionals (HCPs) and achieve optimal clinical outcomes with bedaquiline. These include awarding unrestricted educational grants through the International Union Against TB for the design and delivery of MDR-TB HCP educational programmes in Ghana, Lesotho, Peru, South Africa, the Democratic Republic of Congo, and Rwanda. With USAID, Johnson & Johnson runs workshops on national pharmacovigilance programmes for staff of national TB programmes and national regulators in several low- and middle-income countries.
The company only provides bedaquiline under restricted access conditions – with no promotional activities or sales agents for the medicine, removing perverse incentives to oversell. Johnson & Johnson also supports efforts to track and predict the emergence of resistance to bedaquiline. Its DREAM surveillance programme (Drug Resistance Emergence Assessment in MDR-TB) runs in collaboration with National Tuberculosis Programmes and WHO’s Supranational Reference Laboratories.
The Benchmark has assessed access and stewardship practices for 18 companies in relation to their antibiotics. The measures put in place by Johnson & Johnson for bedaquiline represent the most comprehensive of those specifically linked to a single product.
For a full list of references, please see the 2018 AMR Benchmark report.