MSF Access Campaign Drug-Resistant Tuberculosis Research and Development Case Study May 2020

Two novel drugs, bedaquiline and delamanid, have recently become available to treat drug resistant tuberculosis (DR-TB) after many decades of little innovation in the field of DR-TB treatment. Despite evidence of improved efficacy and reduced toxicity of multi-drug regimens including the two agents, access to bedaquiline and delamanid has been very limited in many settings with a high burden of DR-TB and consistently poor treatment outcomes. Aside from regulatory, logistic, and cost barriers at country level, uptake of the novel agents was complicated by gaps in knowledge for optimal use in clinical practice after initial market approval. The main incentives of the current pharmaceutical research and development paradigm are structured around obtaining regulatory approval, which in turn requires efficacy and safety data generated by clinical trials. Recently completed and ongoing clinical trials did not answer critical questions of how to provide shorter, less toxic treatment DR-TB treatment regimens containing bedaquiline and delamanid and improve patient outcomes. Voluntary generation of evidence that is not part of this process – yet essential from a clinical or policy perspective – has been left to non-sponsor partners and researchers, often without collaborative efforts to improve post-regulatory approval access to life saving drugs. Additionally, these efforts are currently not recognized in the value chain of the research and development process, and there are no incentives to make this critical research happen in a coordinated way.