Operational Insights

  1. Studies of the human response to M. tuberculosis require a multidisciplinary approach given the complexities of the host-pathogen interaction, clinical research in resource limited settings and the long-term human and financial resources required for clinical TB research.
  2. Specimen collection and well-designed and focused sub-studies increase the cost up front but greatly enhance the value of expensive clinical trials and observational/epidemiologic studies of M. tuberculosis infection and disease (in particular when the primary endpoints of the clinical trial are negative).
  3. Collaboration across institutions and investigators from TB endemic and non-endemic areas is essential for TB clinical research.
  4. TB differs from region to region in terms of relative importance of HIV infection, drug resistance (MDR/XDR), role of private vs. public TB care, use of preventive treatment for latent M. tuberculosis infection, access to anti-retroviral therapy, and BCG vaccination; these issues affect the clinical research questions that one can address, issues one needs to confront in study design, and barriers to the successful implementation of a research study.
  5. Excellent baseline clinical phenotypes and follow-up are essential for clinical TB research.
  6. Solid epidemiological study design provides an optimal framework for genetic and immunologic studies of M. tuberculosis natural history and disease.
  7. The time to complete clinical trials of new anti-TB drugs using current endpoints and paradigms is a major obstacle to improving treatment. Validated biomarkers of clinical endpoints are needed.  These can be initially evaluated in small cohorts but ultimately must be validated in large clinical trials based on failure/recurrence endpoints.