Recently developed drugs for the treatment of drug-resistant TB: a research and development case study
In an article published in the journal BMJ Global Health, authors from the Médecins Sans Frontières (MSF) Access Campaign analyse the development and approval of two newer drugs for treating drug-resistant tuberculosis (DR-TB), bedaquiline and delamanid, in the current pharmaceutical research and development (R&D) paradigm, and the failure of the regulatory approval process to define the drugs’ role in multidrug regimens to improve treatment outcomes.
Despite evidence of improved efficacy and reduced toxicity of treatment regimens using bedaquiline and delamanid, access to these two drugs has been 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 R&D 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 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 lifesaving drugs. Additionally, these efforts are currently not recognised in the value chain of the R&D process, and there are no incentives to make this critical research happen in a coordinated way.
The authors recommend inclusion of public health-driven expertise early in the regulatory process; establishment of a legal framework within the regulatory process that would acknowledge societal contributions; and organised, longitudinal inputs from multiple stakeholders into the regulatory process.
Source: Médecins Sans Frontières
