Anything to Stay Alive: The Challenges of a Campaign for an Experimental Drug

Developing World Bioethics

Volumn 16, Issue 1, 2016, Pages 45-54

  Geffen, Nathan ^a  

^a UNIVERSITY OF CAPE TOWN   (South Africa)

Author keywords

Developing world bioethics; Ethics; HIV AIDS; Informed consent; MSF; South Africa; Treatment

Indexed keywords

BEDAQUILINE; NEW DRUG; QUINOLINE DERIVATIVE; TUBERCULOSTATIC AGENT;

CLINICAL TRIAL (TOPIC); CONSUMER ADVOCACY; DRUG APPROVAL; DRUG EFFECTS; ETHICS; FOOD AND DRUG ADMINISTRATION; HUMAN; MULTIDRUG RESISTANT TUBERCULOSIS; MYCOBACTERIUM TUBERCULOSIS; PATIENT RIGHT; PROCEDURES; SOUTH AFRICA; UNITED STATES;

ANTITUBERCULAR AGENTS; CLINICAL TRIALS AS TOPIC; CONSUMER ADVOCACY; DIARYLQUINOLINES; DRUG APPROVAL; DRUGS, INVESTIGATIONAL; HUMANS; MYCOBACTERIUM TUBERCULOSIS; PATIENT RIGHTS; SOUTH AFRICA; TUBERCULOSIS, MULTIDRUG-RESISTANT; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84959550280     PISSN: 14718731     EISSN: 14718847     Source Type: Journal    
DOI: 10.1111/dewb.12084     Document Type: Article

References (61)

1. C.J.L. Murray, K.F. Ortblad, C. Guinovart, S.S. Lim, T.M. Wolock, D.A. Roberts, etal. Global, regional, and national incidence and mortality for HIV, tuberculosis, and malaria during 1990-2013: a systematic analysis for the Global Burden of Disease Study 2013. Lancet. 2014 Sep 13; 384(9947): 1005-1070.
2. Statistics South Africa. Mortality and causes of death in South Africa, 2012: Findings from death notification. Pretoria: Statistics South Africa; 2014 Sep.
3. K. Weyer . Case study: South Africa. Bull World Health Organ. 2007 May; 85(5): 325-420.
4. N. Massyn, C. Day, N. Peer, A. Padarath, P. Barron & R. English . District Health Barometer 2013/14. Health Systems Trust; 2014. Available at: http://www.hst.org.za/publications/district-health-barometer-201314 [Accessed 14 April 2015].
5. World Health Organization. Guidelines for treatment of tuberculosis. World Health Organization; 2010. Available at: http://www.who.int/tb/publications/2010/9789241547833/en/ [Accessed 14 April 2015]; Department of Health. National Tuberculosis Management Guidelines 2014. Department of Health; 2014. Available at: http://www.sahivsoc.org/upload/documents/NTCP_Adult_TB%20Guidelines%2027.5.2014.pdf [Accessed 14 April 2015].
6. Centers for Disease Control and Prevention (CDC). Emergence of Mycobacterium tuberculosis with extensive resistance to second-line drugs-worldwide, 2000-2004. MMWR Morb Mortal Wkly Rep. 2006 Mar 24; 55(11): 301-305.
7. G.B. Migliori, R. Centis, L. D'Ambrosio, A. Spanevello, E. Borroni, D.M. Cirillo, etal. Totally Drug-Resistant and Extremely Drug-Resistant Tuberculosis: The Same Disease? Clin Infect Dis. 2012 Jan 5; 54(9): 1379-1380; A. Matteelli, A. Roggi & A.C. Carvalho . Extensively drug-resistant tuberculosis: epidemiology and management. Clin Epidemiol. 2014; 6: 111-118.
8. N. Ndjeka, F. Conradie, J. Hughes, S. Moyo, N. Bantubani & H. Ferreira . Access to Bedaquiline and interim outcomes for patients with DR-TB within the National Bedaquiline Clinical Access Programme in South Africa. 4th South African TB Conference; 2014; Durban.
9. World Health Organization, op. cit., p. 62.
10. Ndjeka etal. op. cit.; World Health Organization, op. cit., p. 58.
11. T. Törün, G. Güngör, I˙. Özmen, Y. Bölükbaşi, E. Maden, B. Biçakçi, etal. Side effects associated with the treatment of multidrug-resistant tuberculosis. Int J Tuberc Lung Dis. 2005 Dec 1; 9(12): 1373-1377; D. Knoetze . You see yourself vanishing and you think: I'm going to die. Available at: http://groundup.org.za/article/the_need_for_medicines_for_drug_resistant_tb_2475 [Accessed 14 April 2015].
12. Medprax. Private Sector Single Exit Medicine Prices. 2014. Available at: http://www.tac.org.za/community/node/2021 [Accessed 14 April 2015].
13. World Health Organization. Drug-resistant tuberculosis now at record levels. WHO. 2010. Available at: http://www.who.int/mediacentre/news/releases/2010/drug_resistant_tb_20100318/en/ [Accessed 14 April 2015].
14. O.R. McCarthy . The key to the sanatoria. J R Soc Med. 2001 Jan 8; 94(8): 413-417.
15. A. Zumla, P. Nahid & S.T. Cole . Advances in the development of new tuberculosis drugs and treatment regimens. Nat Rev Drug Discov. 2013 Apr 30; 12(5): 388-404.
16. O.R. McCarthy . op. cit.; J.P. Cegielski, D.P. Chin, M.A. Espinal, T.R. Frieden, R.R. Cruz, E.A. Talbot, etal. The global tuberculosis situation: Progress and problems in the 20th century, prospects for the 21st century. Infect Dis Clin North Am. 2002 Mar; 16(1): 1-58
17. W.F. Paolo & J.D. Nosanchuk . Tuberculosis in New York city: recent lessons and a look ahead. Lancet Infect Dis. 2004 May; 4(5): 287-293
18. T. Navin, S. McNabb & J.T. Crawford . The Continued Threat of Tuberculosis. Emerg Infect Dis. 2002 Nov; 8(11): 1187
19. R. Mahajan . Bedaquiline: First FDA-approved tuberculosis drug in 40 years. Int J Appl Basic Med Res. 2013; 3(1): 1-2.
20. L.A. Gillum, C. Gouveia, E.R. Dorsey, M. Pletcher, C.D. Mathers, C.E. McCulloch, etal. NIH Disease Funding Levels and Burden of Disease. PLoS ONE. 2011 Feb 24; 6(2). Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3044706/ [Accessed 14 April 2015]; M. Frick . 2014 Report on Tuberculosis Research Funding Trends, 2005-2013. Treatment Action Group; 2014. Available at: http://www.treatmentactiongroup.org/tbrd2014 [Accessed 14 April 2015].
21. NIH. NIH Categorical Spending. 2014 Feb. Available at: http://report.nih.gov/categorical_spending.aspx [Accessed 14 April 2015].
22. C. Harper . Tuberculosis, a neglected opportunity? Nat Med. 2007 Mar; 13(3): 309-312.
23. J. Surowiecki . The Economics of Ebola Drugs. The New Yorker. 2014. Available at: http://www.newyorker.com/magazine/2014/08/25/ebolanomics [Accessed 14 April 2015].
24. D. Chang Blanc & P. Nunn . Incentives and disincentives for new anti-tuberculosis drug development. World Health Organization; 1999. Available at: http://www.who.int/tdr/publications/documents/incentives-disincentives-anti-tuberculosis.pdf [Accessed 14 April 2015].
25. N. Geffen . Meeting with Tibotec regarding compassionate care access to TMC207. 2009. [Email]
26. Stop TB Partnership, Treatment Action Group. Agenda for Addressing Critical Challenges in TB/HIV Research and Program Implementation. 2009.
27. Treatment Action Campaign, Treatment Action Group, HIV i-Base, Global TB Community Advisory Board, Southern African HIV Clinicians Society, Medecins Sans Frontieres. The case for pre-approval access to bedaquiline. 2012. Available at: http://www.tbonline.info/posts/2012/11/24/case-pre-approval-access-bedaquiline/ [Accessed 14 April 2015].
28. South African Government. Medicines and Related Substances Control Act, 1965 (Act No. 101 of 1965). 1965. Available at: http://www.acts.co.za/medicines-and-related-substances-control-act-1965/ [Accessed 14 April 2015].
29. Global TB CAB. Open letter to Otsuka: continued concerns about delamanid's accessibility. http://www.tbonline.info/posts/2014/3/19/open-letter-otsuka-continued-concerns-about-delama/ [Accessed 2 May 2015].
30. World Health Organization. Guidelines for the programmatic management of drug-resistant tuberculosis. World Health Organization; 2011. Available at: http://whqlibdoc.who.int/publications/2011/9789241501583_eng.pdf [Accessed 14 April 2015].
31. A. Jindani, T.S. Harrison, A.J. Nunn, P.P.J. Phillips, G.J. Churchyard, S. Charalambous, etal. High-dose rifapentine with moxifloxacin for pulmonary tuberculosis. N Engl J Med. 2014 Oct 23; 371(17): 1599-1608.
32. A.H. Diacon, A. Pym, M. Grobusch, R. Patientia, R. Rustomjee, L. Page-Shipp, etal. The Diarylquinoline TMC207 for Multidrug-Resistant Tuberculosis. N Engl J Med. 2009 Jun 4; 360(23): 2397-2405.
33. V. Amorosa & P. Tebas . Is It Time to Rethink the Expanded-Access Programs for HIV Infection? J Infect Dis. 2007 Jan 10; 196(7): 974-977; T. Creagh-Kirk, P. Doi, E. Andrews, S. Nusinoff-Lehrman, H. Tilson, D. Hoth, etal. Survival experience among patients with AIDS receiving zidovudine. Follow-up of patients in a compassionate plea program. JAMA. 1988 Nov 25; 260(20): 3009-3015.
34. L. Highleyman . Preveon expanded access program broadened. BETA Bull Exp Treat AIDS Publ San Franc AIDS Found. 1998 Jul; 3; Clinical and Research Information on Drug-Induced Liver Injury. Adefovir. Adefovir. Available at: http://livertox.nih.gov/Adefovir.htm [Accessed 14 April 2015].
35. DECRANE LFB c/o J-CSA, GUILLEMONT JEG johnson&jonhson pharma, F.C. Odds, H.J.J. Poignet, G.J.F.E. Van, M.G. Venet, etal. Quinoline derivatives and their use as mycobacterial inhibitors. EP1527050 A1, 2005. Available at: http://www.google.com/patents/EP1527050A1 [Accessed 14 April 2015].
36. H. Azijn, I. Tirry, J. Vingerhoets, M.-P. de Béthune, G. Kraus, K. Boven, etal. TMC278, a Next-Generation Nonnucleoside Reverse Transcriptase Inhibitor (NNRTI), Active against Wild-Type and NNRTI-Resistant HIV-1. Antimicrob Agents Chemother. 2010 Jan 2; 54(2): 718-727;
37. P. Stoffels . 4-[[4-[[4-(2-cyanoethenyl)-2, 6-dimethylphenyl]-amino]-2-pyrimidinyl]-amino]-benzonitrile with nucleoside and/or nucleotide reverse transcriptase inhibitors; prevention of HIV transmission; pharmacokinetic profile allowing once daily dosing. US20080200435 A1, 2008. Available at: http://www.google.com/patents/US20080200435 [Accessed 14 April 2015].
38. Treatment Action Campaign. Open letters by AIDS and TB organisations calling for access to a new TB drug and a new TB diagnostic. 2010. Available at: http://www.tac.org.za/community/node/2962 [Accessed 14 April 2015]; G. Van Cutsem . Open letter to the Medicines Control Council of South Africa regarding compassionate use of bedaquiline for drug-resistant tuberculosis in South Africa. 2012. Available at: http://www.tbonline.info/posts/print/open-letter-medicines-control-council-south-africa/ [Accessed 14 April 2015]; Treatment Action Campaign. TAC puts pressure on Janssen finalize development of TB drug bedaquili. 2013. Available at: http://www.thepharmaletter.com/article/tac-puts-pressure-on-janssen-finalize-development-of-tb-drug-bedaquiline [Accessed 14 April 2015].
39. Stop TB Partnership. FAQs regarding procurement of Bedaquiline (BDQ) through the Global Drug Facility (GDF). 2014. Available at: http://www.stoptb.org/assets/documents/gdf/drugsupply/FAQonprocurementofbedaquilinethroughGDFFINALVERSION300414.pdf [Accessed 14 April 2015].
40. A. Thom . MCC says no to TB drug access. 2012. Available at: http://www.health-e.org.za/2012/09/27/mcc-says-no-to-tb-drug-access/ [Accessed 14 April 2015].
41. A. Barton . FDA panel finds new type of TB drug effective, raising hopes for treatment of drug-resistant strains. Science Speaks: HIV & TB News. Available at: http://sciencespeaksblog.org [Accessed 14 April 2015]; L.L. Gonzalez . Doctor turns patient after contracting XDR-TB. Available at: http://www.health-e.org.za/2014/03/24/doctor-turns-patient-contracting-xdr-tb/ [Accessed 14 April 2015].
42. N. Geffen . Debunking Delusions: The Inside Story of the Treatment Action Campaign. 1 edition. Auckland Park, South Africa: Jacana Media; 2010. p. 63.
43. UNAIDS. Access to antiretroviral therapy in Africa: Status report on progress towards the 2015 targets. UNAIDS; 2013. Available at: http://www.unaids.org/sites/default/files/media_asset/20131219_AccessARTAfricaStatusReportProgresstowards2015Targets_en_0.pdf [Accessed 14 April 2015]; D. Bradshaw, R. Dorrington & R. Laubscher . Rapid Mortality Surveillance Report 2014. Burden of Disease Research Unit, Medical Research Council; 2014 Feb. Available at: http://www.mrc.ac.za/bod/RapidMortality2011.pdf [Accessed 14 April 2015].
44. B. Goldacre . Bad Pharma: How Drug Companies Mislead Doctors and Harm Patients. Reprint edition. New York: Faber & Faber; 2013. p. 140.
45. Janssen Pharmaceuticals. TMC207 (bedaquiline) Treatment of Patients with MDR-TB. Janssen Pharmaceuticals; 2012. Available at: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/Anti-InfectiveDrugsAdvisoryCommittee/UCM329260.pdf [Accessed 14 April 2015]; A.H. Diacon, A. Pym, M.P. Grobusch, J.M. de los Rios, E. Gotuzzo, I. Vasilyeva, etal. Multidrug-resistant tuberculosis and culture conversion with bedaquiline. N Engl J Med. 2014 Aug 21; 371(8): 723-732.
46. Diacon etal. op. cit.
47. M.E. Tucker . FDA Approves Bedaquiline for Resistant TB Treatment. 2012. Available at: http://www.medscape.com/viewarticle/776901 [Accessed 14 April 2015].
48. Food and Drug Administration. FDA approves first drug to treat multi-drug resistant tuberculosis. 2012. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm333695.htm [Accessed 14 April 2015].
49. E. Cox & K. Laessig . FDA Approval of Bedaquiline - The Benefit-Risk Balance for Drug-Resistant Tuberculosis. N Engl J Med. 2014 Aug 20; 371(8): 689-691.
50. E.M. Cox . Accelerated Approval. 2012. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2012/204384Orig1s000ltr.pdf [Accessed 14 April 2015].
51. Public Citizen. Public Citizen Urges FDA to Reject Accelerated Approval for Possibly Dangerous Anti-TB Drug Bedaquiline. 2012. Available at: http://www.citizen.org/pressroom/pressroomredirect.cfm?ID=3786 [Accessed 14 April 2015].
52. AIDS Treatment Activists Coalition, Community Research Advisors Group, European AIDS Treatment Group, Global TB Community Advisory Board. Concerns regarding STREAM stage II trial design for bedaquiline approval. 2014. Available at: http://www.tbonline.info/media/uploads/documents/letter_to_regulators_re_stream_design_from_community_%281%29_%282%29.pdf [Accessed 14 April 2015]; M. Frick . Fool's Errand: The Sloppy Science of the MDR-TB STREAM Trial. Available at: http://www.treatmentactiongroup.org/tagline/2014/spring/fool%E2%80%99s-errand-sloppy-science-mdr-tb-stream-trial [Accessed 14 April 2015].
53. K. Dheda & A. Goolam-Mahomed . Evaluating a new treatment regimen for patients with multidrug-resistant TB (MDR-TB) - a prospective open-label randomised controlled trial. 2014.
54. R. Prasad . Clinical trials on child-friendly bedaquiline MDR-TB drug for children initiated. 2013. Available at: http://www.thehindu.com/sci-tech/health/medicine-and-research/clinical-trials-on-childfriendly-bedaquiline-mdrtb-drug-for-children-initiated/article5459156.ece [Accessed 14 April 2015].
55. Food and Drug Administration. Bedaquiline package label. Food and Drug Administration; 2012. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/204384s000lbl.pdf [Accessed 14 April 2015].
56. World Health Organization. WHO interim guidance on the use of bedaquiline to treat MDR-TB. WHO. 2013. Available at: http://www.who.int/mediacentre/news/notes/2013/bedaquiline_mdr_tb_20130613/en/ [Accessed 14 April 2015].
57. European Medicines Agency. Sirturo: EPAR summary for the public. European Medicines Agency; 2014. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002614/human_med_001730.jsp&mid=WC0b01ac058001d124 [Accessed 14 April 2015].
58. L. Garrido . SIRTURO® (bedaquiline), A Novel Treatment for Multidrug-Resistant Tuberculosis, to be Made Available to Patients in South Africa. 2014.
59. Treatment Action Group and others. Open letter: Reducing the price of bedaquiline. 2014. Available at: http://www.tbonline.info/media/uploads/documents/bedaquiline_pricing_letter_2014_sept_10_compressed_.pdf [Accessed 14 April 2015].
60. J.J. Darrow, A. Sarpatwari, J. Avorn & A.S. Kesselheim . Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs. N Engl J Med. 2015 Jan 15; 372(3): 279-286. p. 283.
61. S. Okie . Access before Approval - A Right to Take Experimental Drugs? N Engl J Med. 2006 Aug 3; 355(5): 437-440. p. 437.