TAC: Unconscionable delay of TB drug trial
Janssen must run a Phase III trial of bedaquiline urgently; More investment in TB drugs and diagnostics urgently needed.
The current standard of care for multi-drug resistant tuberculosis (MDR-TB) consists of poorly tested drugs with horrible side-effects. We are desperately in need of new properly tested TB drugs.
Bedaquiline, made by the pharmaceutical company Janssen (a subsidiary of Johnson & Johnson), is a promising drug for treating drug-resistant TB. Two phase II trials have shown compelling evidence that the drug is effective at destroying TB bacteria in the body, however there are safety concerns. In one trial more deaths occurred in the bedaquiline arm than the control.* This was a statistically significant result. It is unknown if the higher mortality is related to the drug and many researchers believe the result might be an unfortunate fluke, but we cannot know until it is further tested in a phase III trial. Usually, drug approval requires phase III trials.
In December 2012 the United States Food and Drug Administration gave accelerated approval to bedaquiline on condition that a phase III trial be conducted and reported on by 2022. We welcome the requirement for a phase III trial. However, the 2022 deadline is far too late and shows little awareness for the urgency to test new TB drugs and determine their safety. Also, very few people in the US will need bedaquiline. TB is not an epidemic in the US.
Following the FDA approval, Janssen has shown no urgency to conduct a phase III trial. This could be because of the lack of profit Janssen expects from TB drugs as well as the possibility of a phase III trial confirming safety concerns. Activists across the world have to put pressure on Janssen to begin a phase III trial urgently.
We recently contacted South Africa’s Medicines Control Council (MCC) to inquire about the status of Janssen’s application to register Bedaquiline in South Africa. We were told that the MCC’s review of the application is ongoing. Given the lack of data from a phase III trial and the worrying safety data, a decision by the MCC to reject the application would not be unreasonable. TAC supports bedaquiline being offered to patients with drug-resistant TB under compassionate use (as is currently done in South Africa), so long as patients are properly informed of the drug's risks and benefits.
## The fundamental problem: TB is an underfunded neglected disease
A recent report published by the Treatment Action Group in the United States (http://www.treatmentactiongroup.org/tb/press/2013/double-blow-private-se...) found that annual investment in TB research has declined, both in the public and private sectors. This is unconscionable when you consider that according to the World Health Organisation (WHO), "Tuberculosis (TB) is second only to HIV/AIDS as the greatest killer worldwide due to a single infectious agent. In 2012, 8.6 million people fell ill with TB and 1.3 million died from TB." (http://www.who.int/mediacentre/factsheets/fs104/en/)
A worldwide activist movement is desperately needed to put pressure on governments and the pharmaceutical industry to increase investment in better TB diagnostics and newer, better TB drugs.
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Footnote:
* Two out of 81 patients on the control arm died. Nine out of 79 patients on the bedaquiline arm died. Source: FDA bedaquiline label: http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Label_ApprovalHistory#labelinfo
Source: Treatment Action Campaign
