Cost-effectiveness of adding bedaquiline to drug regimens for the treatment of multidrug-resistant tuberculosis in the UK

PLoS ONE

Volumn 10, Issue 3, 2015, Pages

  Wolfson, Lara J ^a   Walker, Anna ^b   Hettle, Robert ^b   Lu, Xiaoyan ^a   Kambili, Chrispin ^c   Murungi, Andrew ^d   Knerer, Gerhart ^d  

^a JANSSEN RESEARCH FOUNDATION   (Belgium)

^b HERON Commercialization A PAREXEL Company   (United Kingdom)

^c JANSSEN RESEARCH AND DEVELOPMENT   (United States)

^d Janssen Cilag   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BEDAQUILINE; TUBERCULOSTATIC AGENT; QUINOLINE DERIVATIVE;

ADD ON THERAPY; ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; COST BENEFIT ANALYSIS; COST CONTROL; COST EFFECTIVENESS ANALYSIS; DRUG COST; DRUG EFFICACY; HEALTH CARE PERSONNEL; HIDDEN MARKOV MODEL; HOSPITALIZATION; HUMAN; MULTIDRUG RESISTANT TUBERCULOSIS; NATIONAL HEALTH SERVICE; PATIENT CARE; PHASE 2 CLINICAL TRIAL; PROBABILITY; QUALITY ADJUSTED LIFE YEAR; RANDOMIZED CONTROLLED TRIAL; SENSITIVITY ANALYSIS; UNITED KINGDOM; ECONOMICS; MARKOV CHAIN; TREATMENT OUTCOME; TUBERCULOSIS, MULTIDRUG-RESISTANT; UNCERTAINTY;

ANTITUBERCULAR AGENTS; COST-BENEFIT ANALYSIS; DIARYLQUINOLINES; HUMANS; MARKOV CHAINS; QUALITY-ADJUSTED LIFE YEARS; TREATMENT OUTCOME; TUBERCULOSIS, MULTIDRUG-RESISTANT; UNCERTAINTY; UNITED KINGDOM;

EID: 84926389223     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0120763     Document Type: Article

References (56)

1. WHO. Tuberculosis Fact Sheet Number 104. October 2014. Available: http://www.who.int/mediacentre/factsheets/fs104/en/index.html. Accessed 2015 Feb 12.
2. HPA. Tuberculosis in the UK: 2013 report. January 2014. Available: https://www.gov.uk/government/uploads/system/uploads/attachment-data/file/325632/TB-in-the-UK.pdf. Accessed 2015 Feb 12.
3. ECDC. Tuberculosis surveillance and monitoring in Europe. 2013. Available: http://www.ecdc.europa.eu/en/publications/Publications/Tuberculosis-surveillance-monitoring-2013.pdf. Accessed 2015 Feb 12.
4. British Thoracic Society. Chemotherapy and management of tuberculosis in the United Kingdom: recommendations 1998. Joint Tuberculosis Committee of the British Thoracic Society. Thorax. 1998; 53: 536-548. PMID: 9797751
5. Curry International TB Center. CITC (Curry International Tuberculosis Center) and TBCB (TB control branch) of California department, Drug-resistant tuberculosis: a survival guide for clinicians. 2008.
6. US CDC. Multidrug-Resistant Tuberculosis (MDR TB) and Extensively-Drug Resistant (XDR) TB. 2007.
7. WHO. Global Tuberculosis Report 2013. 2013. Available: http://apps.who.int/iris/bitstream/10665/91355/1/9789241564656-eng.pdf. Accessed 2015 Feb 12.
8. Brigden G, Nyang'wa BT, du CP, Varaine F, Hughes J, Rich M, et al. Principles for designing future regimens for multidrug-resistant tuberculosis. Bull World Health Organ. 2014; 92: 68-74. doi: 10.2471/BLT.13.122028 PMID: 24391302
9. WHO. Stop TB Partnership. The Global Plan to stop TB 2011-2015. Transforming the fight towards elimination of tuberculosis. 2011. Available: http://www.stoptb.org/assets/documents/global/plan/tb-globalplantostoptb2011-2015.pdf. Accessed 2015 Feb 12.
10. FDA. FDA News Release: Approval of bedaquiline as part of a combination therapy to treat adults with multi-drug resistant pulmonary tuberculosis. 31 December 2012.
11. European Medicines Agency. EMA/CHMP/771324/2013 - EMA Committee for Medicinal Products for Human Use (CHMP), Summary of opinion (initial authorisation) - Sirturo (bedaquiline). 19 December 2013.
12. Janssen. Sirturo: highlights of US Prescribing Information. 2013. Available: http://www.accessdata.fda.gov/drugsatfda-docs/label/2012/204384s000lbl.pdf. Accessed 12 February 2015.
13. FDA. Anti-Infective Drugs Advisory Committee Meeting: TMC207 (Bedaquiline). December 2012. Available: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/Anti-InfectiveDrugsAdvisoryCommittee/UCM329260.pdf. Accessed 2014 Sep 1.
14. WHO. The use of bedaquiline in the treatment of multidrug-resistant tuberculosis. Interim policy guidance. 2013. Available: http://apps.who.int/iris/bitstream/10665/84879/1/9789241505482-eng.pdf. Accessed 2015 Feb 12.
15. 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: 723-732. doi: 10.1056/NEJMoa1313865 PMID: 25140958
16. WHO, Vassell A. Cost-effectiveness of introducing bedaquiline in MDR-TB regimens - an exploratory analysis. 26 January 2013. Available: http://who.int/tb/challenges/mdr/CEA-bdqreport-final.pdf. Accessed 2015 Feb 12.
17. White VL, Moore-Gillon J. Resource implications of patients with multidrug resistant tuberculosis. Thorax. 2000; 55: 962-963. PMID: 11050268
18. Diel R, Vandeputte J, De Vries G, Stillo J, Wanlin M, Nienhaus A. Costs of tuberculosis disease in the EU - a systematic analysis and cost calculation. Eur Respir J. 2013.
19. EMA. Orphan designation. 2014. Available: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general-content-000029.jsp. Accessed 2015 Feb 15.
20. European Commission. Community register of medicinal products for human use: bedaquiline product information. 2015. Available: http://ec.europa.eu/health/documents/community-register/html/h901.htm. Accessed 2015 Feb 15.
21. Rawlins MD, Culyer AJ. National Institute for Clinical Excellence and its value judgments. BMJ. 2004; 329: 224-227. PMID: 15271836
22. Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007; 23: 36-42. PMID: 17234015
23. Siebert U, Alagoz O, Bayoumi AM, Jahn B, Owens DK, Cohen DJ, et al. State-transition modeling: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force - 3. Value Health. 2012; 15: 812-820. doi: 10.1016/j.jval.2012.06.014 PMID: 22999130
24. NICE. Measuring effectiveness and cost effectiveness: the QALY. April 2010. Available: http://www.nice.org.uk/newsroom/features/measuringeffectivenessandcosteffectivenesstheqaly.jsp. Accessed 2014 Sep 1.
25. Murray CJL. Quantifying the burden of disease: the technical basis for disability-adjusted life years. Bulletin of the World Health Organization. 1994; 72: 429-445. PMID: 8062401
26. NICE. Methods for the development of NICE public health guidance (second edition). 2009. London, UK.
27. WHO. Guidelines for the programmatic management of drug-resistant tuberculosis. 2008. Available: http://whqlibdoc.who.int/publications/2008/9789241547581-eng.pdf. Accessed 2015 Feb 12.
28. WHO. Definitions and reporting framework for tuberculosis - 2013 revision. Available: http://apps.who.int/iris/bitstream/10665/79199/1/9789241505345-eng.pdf. Accessed 2015 Feb 12.
29. Franke MF, Appleton SC, Bayona J, Arteaga F, Palacios E, Llaro K, et al. Risk factors and mortality associated with default from multidrug-resistant tuberculosis treatment. Clin Infect Dis. 2008; 46: 1844-1851. doi: 10.1086/588292 PMID: 18462099
30. Liu CH, Li L, Chen Z, Wang Q, Hu YL, Zhu B, et al. Characteristics and treatment outcomes of patients with MDR and XDR tuberculosis in a TB referral hospital in Beijing: a 13-year experience. PLoS One. 2011; 6: e19399. doi: 10.1371/journal.pone.0019399 PMID: 21559362
31. Tiemersma EW, van der Werf MJ, Borgdorff MW, Williams BG, Nagelkerke NJ. Natural history of tuberculosis: duration and fatality of untreated pulmonary tuberculosis in HIV negative patients: a systematic review. PLoS One. 2011; 6: e17601. doi: 10.1371/journal.pone.0017601 PMID: 21483732
32. Lunn D, Thomas A, Best N, Spiegelhalter D. WinBUGS - A Bayesian modelling framework: Concepts, structure, and extensibility. Statistics and Computing. 2000; 10: 325-337.
33. Department of Health. British National Formulary. 2013. Available: http://www.bnf.org/bnf/index.htm.Accessed 2014 Sep 1.
34. Department of Health. NHS Reference Costs 2011-2012. 2012. Available: https://www.gov.uk/government/uploads/system/uploads/attachment-data/file/213060/2011-12-reference-costs-publication.pdf. Accessed 2015 Feb 12.
35. NICE. CG117: Tuberculosis: Clinical diagnosis and management of tuberculosis, and measures for its prevention and control. 2011. Available: http://www.nice.org.uk/guidance/cg117/resources/cg117-tuberculosis-full-guideline3. Accessed 2015 Feb 12.
36. Jit M, Stagg HR, Aldridge RW, White PJ, Abubakar I. Dedicated outreach service for hard to reach patients with tuberculosis in London: observational study and economic evaluation. BMJ. 2011; 343: d5376. doi: 10.1136/bmj.d5376 PMID: 22067473
37. Kind P, Hardman G, Macran S. UK population norms for EQ-5D. November 1999. Available: https://www.york.ac.uk/media/che/documents/papers/discussionpapers/CHE%20Discussion%20Paper%20172.pdf. Accessed 2015 Feb 12.
38. Salomon JA, Vos T, Hogan DR, Gagnon M, Naghavi M, Mokdad A, et al. Common values in assessing health outcomes from disease and injury: disability weights measurement study for the Global Burden of Disease Study 2010. Lancet. 2012; 380: 2129-2143. doi: 10.1016/S0140-6736(12)61680-8 PMID: 23245605
39. ONS. Office for National Statistics Life Tables 2009-2011 - England and Wales. 2013. Available: http://www.ons.gov.uk/ons/rel/lifetables/interim-life-tables/2009-2011/rft-england-and-wales.xls. Accessed 2014 Sep 1.
40. Health Protection Agency. Tuberculosis in the UK: Annual report on tuberculosis surveillance in the UK, 2013. Available: https://www.gov.uk/government/uploads/system/uploads/attachment-data/file/325632/TB-in-the-UK.pdf. Accessed 2015 Feb 12.
41. Kurbatova E, Smith S, Cegielski J. Evaluation of sputum culture conversion as a surrogate marker of treatment outcome in patients with multidrug-resistant tuberculosis. 2013. CDC, Atlanta, GA, United States.
42. Guglielmetti L, Le DD, Jachym M, Henry B, Martin D, Caumes E, et al. Compassionate Use of Bedaquiline for the Treatment of Multidrug-Resistant and Extensively Drug-Resistant Tuberculosis: Interim Analysis of a French Cohort. Clin Infect Dis. 2014; ciu786 [pii];10.1093/cid/ciu786.
43. Ndjeka N, Conradie F, Hughes J, Schnipel K, Cox H, Bantubani N, et al. Safe and effective bedaquiline treatment of drug-resistant tuberculosis (DR-TB) within the National Bedaquiline Clinical Access Programme in South Africa. S Afr Med J. 2014; 104(3):164-166 PMID: 24897814
44. Day M, Middlemiss A, Thorpe J, Okereke E. What really happens to tuberculosis patients classified as lost to follow-up in West Yorkshire? Euro Surveill. 2012; 17.
45. Guo N, Marra F, Marra CA. Measuring health-related quality of life in tuberculosis: a systematic review. Health Qual Life Outcomes. 2009; 7:14. doi: 10.1186/1477-7525-7-14 PMID: 19224645
46. Van Deun A, Maug AK, Salim MA, Das PK, Sarker MR, Daru P, et al. Short, highly effective, and inexpensive standardized treatment of multidrug-resistant tuberculosis. Am J Respir Crit Care Med. 2010; 182: 684-692. doi: 10.1164/rccm.201001-0077OC PMID: 20442432
47. Blower SM, Chou T. Modeling the emergence of the 'hot zones': tuberculosis and the amplification dynamics of drug resistance. Nat Med. 2004; 10: 1111-1116. PMID: 15378053
48. Murphy BM, Singer BH, Anderson S, Kirschner D. Comparing epidemic tuberculosis in demographically distinct heterogeneous populations. Math Biosci. 2002; 180: 161-185. PMID: 12387922
49. Denholm JT, Leslie DE, Jenkin GA, Darby J, Johnson PD, Graham SM, et al. Long-term follow-up of contacts exposed to multidrug-resistant tuberculosis in Victoria, Australia, 1995-2010. Int J Tuberc Lung Dis. 2012; 16: 1320-1325. doi: 10.5588/ijtld.12.0092 PMID: 22863690
50. Grandjean L, Crossa A, Gilman RH, Herrera C, Bonilla C, Jave O, et al. Tuberculosis in household contacts of multidrug-resistant tuberculosis patients. Int J Tuberc Lung Dis. 2011; 15:1164-9. doi: 10.5588/ijtld.11.0030 PMID: 21943839
51. Resch SC, Salomon JA, Murray M, Weinstein MC. Cost-effectiveness of treating multidrug-resistant tuberculosis. PLoS Med. 2006; 3: e241. PMID: 16796403
52. Singla N, Singla R, Jain G, Habib L, Behera D. Tuberculosis among household contacts of multidrug-resistant tuberculosis patients in Delhi, India. Int J Tuberc Lung Dis. 2011; 15:1326-1330. doi: 10.5588/ijtld.10.0564 PMID: 22283889
53. Neely F, Maguire H, Le BF, Davies A, Gelb D, Yates S. High rate of transmission among contacts in large London outbreak of isoniazid mono-resistant tuberculosis. J Public Health (Oxf). 2010; 32: 44-51. doi: 10.1093/pubmed/fdp056 PMID: 19542269
54. Luciani F, Sisson SA, Jiang H, Francis AR, Tanaka MM. The epidemiological fitness cost of drug resistance in Mycobacterium tuberculosis. Proc Natl Acad Sci U S A. 2009; 106: 14711-14715. doi: 10.1073/pnas.0902437106 PMID: 19706556
55. Cohen T, Murray M. Modeling epidemics of multidrug-resistant M. tuberculosis of heterogeneous fitness. Nat Med. 2004; 10: 1117-1121. PMID: 15378056
56. Liao CM, Cheng YH, Lin YJ, Hsieh NH, Huang TL, Chio CP, et al. A probabilistic transmission and population dynamic model to assess tuberculosis infection risk. Risk Anal. 2012; 32: 1420-1432. doi: 10.1111/j.1539-6924.2011.01750.x PMID: 22211354