Gillespie SH, Crook AM, McHugh TD, Mendel CM, Meredith SK, Murray SR, et al. Four-month moxifloxacin-based regimens for drug-sensitive tuberculosis. N Engl J Med. 2014;371(17):1577–87.
World Health Organization. Global Tuberculosis Report 2015. Geneva: WHO; 2015.
Boeree MJ, Diacon AH, Dawson R, Narunsky K, du Bois J, Venter A, et al. A dose-ranging trial to optimize the dose of rifampin in the treatment of tuberculosis. Am J Respir Crit Care Med. 2015;191(9):1058–65.
Dooley KE, Obuku EA, Durakovic N, Belitsky V, Mitnick C, Nuermberger EL. World Health Organization group 5 drugs for the treatment of drug-resistant tuberculosis: unclear efficacy or untapped potential? J Infect Dis. 2012;207(9):1352–8.
Dooley KE, Mitnick CD, Ann DeGroote M, Obuku E, Belitsky V, Hamilton CD, et al. Old drugs, new purpose: retooling existing drugs for optimized treatment of resistant tuberculosis. Clin Infect Dis. 2012;55(4):572–81.
Dawson R, Narunsky K, Carman D, Gupte N, Whitelaw A, Efron A, et al. Two-stage activity-safety study of daily rifapentine during intensive phase treatment of pulmonary tuberculosis. Int J Tuberc Lung Dis. 2015;19(7):780–6.
Phillips PPJ, Gillespie SH, Boeree M, Heinrich N, Aarnoutse R, McHugh T, et al. Innovative trial designs are practical solutions for improving the treatment of tuberculosis. J Infect Dis. 2012;205 Suppl 2:S250–7.
Jindani A, Harrison TS, Nunn AJ, Phillips PP, Churchyard GJ, Charalambous S, et al. High-dose rifapentine with moxifloxacin for pulmonary tuberculosis. N Engl J Med. 2014;371(17):1599–608.
Merle CS, Fielding K, Sow OB, Gninafon M, Lo MB, Mthiyane T, et al. A four-month gatifloxacin-containing regimen for treating tuberculosis. N Engl J Med. 2014;371(17):1588–98.
Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. Br Med J. 1948;2(4582):769–82.
Cochrane A. 1931-1971: A critical review, with particular reference to the medical profession. In: Teeling-Smith G, Wells N, editors. Medicines for the Year 2000. London: Office of Health Economics; 1979. p. 1–11.
Fox W, Ellard GA, Mitchison DA. Studies on the treatment of tuberculosis undertaken by the British Medical Research Council tuberculosis units, 1946-1986, with relevant subsequent publications. Int J Tuberc Lung Dis. 1999;3(10 Suppl 2):S231–279.
US FDA Priftin (Rifapentine) Drug Approval Package. [http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21024S5_Priftin.cfm]. Accessed 21 Mar 2016.
Cox E, Laessig K. FDA approval of bedaquiline--the benefit-risk balance for drug-resistant tuberculosis. N Engl J Med. 2014;371(8):689–91.
Ahuja SD, Ashkin D, Avendano M, Banerjee R, Bauer M, Bayona JN, et al. Multidrug resistant pulmonary tuberculosis treatment regimens and patient outcomes: an individual patient data meta-analysis of 9,153 patients. PLoS Med. 2012;9(8):e1001300.
Pocock SJ. Clinical trials: a practical approach. Chichester: Wiley; 1983.
Suntharalingam G, Perry MR, Ward S, Brett SJ, Castello-Cortes A, Brunner MD, et al. Cytokine storm in a phase 1 trial of the anti-CD28 monoclonal antibody TGN1412. N Engl J Med. 2006;355(10):1018–28.
Piantadosi S. Clinical trials: a methodologic perspective. 2nd ed. Hoboken: Wiley-Interscience; 2005.
Diacon AH, Dawson R, Hanekom M, Narunsky K, Maritz SJ, Venter A, et al. Early bactericidal activity and pharmacokinetics of PA-824 in smear-positive tuberculosis patients. Antimicrob Agents Chemother. 2010;54(8):3402–7.
Diacon AH, Dawson R, du Bois J, Narunsky K, Venter A, Donald PR, et al. Phase II dose-ranging trial of the early bactericidal activity of PA-824. Antimicrob Agents Chemother. 2012;56(6):3027–31.
Diacon AH, Dawson R, von Groote-Bidlingmaier F, Symons G, Venter A, Donald PR, et al. 14-day bactericidal activity of PA-824, bedaquiline, pyrazinamide, and moxifloxacin combinations: a randomised trial. Lancet. 2012;380(9846):986–93.
Diacon AH, Dawson R, von Groote-Bidlingmaier F, Symons G, Venter A, Donald PR, et al. Bactericidal activity of pyrazinamide and clofazimine alone and in combinations with pretomanid and bedaquiline. Am J Respir Crit Care Med. 2015;191(8):943–53.
Dawson R, Diacon AH, Everitt D, van Niekerk C, Donald PR, Burger DA, et al. Efficiency and safety of the combination of moxifloxacin, pretomanid (PA-824), and pyrazinamide during the first 8 weeks of antituberculosis treatment: a phase 2b, open-label, partly randomised trial in patients with drug-susceptible or drug-resistant pulmonary tuberculosis. Lancet. 2015;385(9979):1738–47.
Dorman SE, Goldberg S, Stout JE, Muzanyi G, Johnson JL, Weiner M, et al. Substitution of rifapentine for rifampin during intensive phase treatment of pulmonary tuberculosis: study 29 of the tuberculosis trials consortium. J Infect Dis. 2012;206(7):1030–40.
Mitchison DA. The action of antituberculosis drugs in short-course chemotherapy. Tubercle. 1985;66(3):219–25.
Prideaux B, Via LE, Zimmerman MD, Eum S, Sarathy J, O'Brien P, Chen C, Kaya F, Weiner DM, Chen PY, et al. The association between sterilizing activity and drug distribution into tuberculosis lesions. Nat Med. 2015;21(10):1223–7.
Wallis RS, Nacy C. Early bactericidal activity of new drug regimens for tuberculosis. Lancet. 2013;381(9861):111–2.
Rustomjee R, Diacon AH, Allen J, Venter A, Reddy C, Patientia RF, et al. Early bactericidal activity and pharmacokinetics of the diarylquinoline TMC207 in treatment of pulmonary tuberculosis. Antimicrob Agents Chemother. 2008;52(8):2831–5.
Diacon AH, Dawson R, Von Groote-Bidlingmaier F, Symons G, Venter A, Donald PR, et al. Randomized dose-ranging study of the 14-day early bactericidal activity of bedaquiline (TMC207) in patients with sputum microscopy smear-positive pulmonary tuberculosis. Antimicrob Agents Chemother. 2013;57(5):2199–203.
Diacon AH, Pym A, Grobusch MP, de los Rios JM, Gotuzzo E, Vasilyeva I, et al. Multidrug-resistant tuberculosis and culture conversion with bedaquiline. N Engl J Med. 2014;371(8):723–32.
Tang S, Yao L, Hao X, Liu Y, Zeng L, Liu G, et al. Clofazimine for the treatment of multidrug-resistant tuberculosis: prospective, multicenter, randomized controlled study in China. Clin Infect Dis. 2015;60(9):1361–7.
Phillips PP, Mendel CM, Burger DA, Crook A, Nunn AJ, Dawson R, et al. Limited role of culture conversion for decision-making in individual patient care and for advancing novel regimens to confirmatory clinical trials. BMC Med. 2016;14(1):19.
Phillips PPJ, Fielding K, Nunn AJ. An evaluation of culture results during treatment for tuberculosis as surrogate endpoints for treatment failure and relapse. PLoS One. 2013;8(5):e63840.
Wallis RS, Wang C, Doherty TM, Onyebujoh P, Vahedi M, Laang H, et al. Biomarkers for tuberculosis disease activity, cure, and relapse. Lancet Infect Dis. 2010;10(2):68–9.
Phillips PP, Davies GR, Mitchison DA. Biomarkers for tuberculosis disease activity, cure, and relapse. [Correspondence]. Lancet Infect Dis. 2010;10(2):69–70. author reply 70-61.
Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol. 2014;32(1):40–51.
Treatment Action Group, Frick M. 2015 report on tuberculosis research funding trends, 2005–2014: a decade of data. 2015. http://www.treatmentactiongroup.org/tbrd2015. Accessed 21 Mar 2016.
Boeree M, Hoelscher M; The PanACEA consortium. High-dose rifampin, SQ109 and moxifloxacin for treating tb: The PanACEA MAMS-TB Trial. In: 2015 Conference on retroviruses and opportunistic infection (CROI), 23–26 February 2015. Seattle, Washington: CROI Foundation; 2015.
Nunn AJ, Phillips PPJ, Mitchison DA. Timing of relapse in short-course chemotherapy trials for tuberculosis [Short communication]. Int J Tuberc Lung Dis. 2010;14:241–242(242).
Dorman SE, Savic RM, Goldberg S, Stout JE, Schluger N, Muzanyi G, et al. Daily rifapentine for treatment of pulmonary tuberculosis. A randomized, dose-ranging trial. Am J Respir Crit Care Med. 2015;191(3):333–43.
Machin D. Sample size tables for clinical studies. 3rd ed. Wiley-Blackwell: Chichester; 2008.
Berry SM. Bayesian adaptive methods for clinical trials. Boca Raton: CRC Press; 2011.
Wallis RS, Peppard T, Hermann D. Month 2 culture status and treatment duration as predictors of recurrence in pulmonary tuberculosis: model validation and update. PLoS One. 2015;10(4):e0125403.
Parker CC, Sydes MR, Mason MD, Clarke NW, Aebersold D, de Bono JS, et al. Prostate radiotherapy for men with metastatic disease: a new comparison in the STAMPEDE trial. Clin Oncol (R Coll Radiol). 2013;25(5):318–20.
O'Hagan A, Stevens JW, Campbell MJ. Assurance in clinical trial design. Pharm Stat. 2005;4(3):187–201.
PUSH, PULL, POOL: Accelerating innovation and access to medicines for tuberculosis. [http://www.msfaccess.org/3Ps-project]. Accessed 21 Mar 2016.
Ventz S, Trippa L. Bayesian designs and the control of frequentist characteristics: a practical solution. Biometrics. 2014;71(1):218–26.