HIV prevention trial design in an era of effective pre-exposure prophylaxis

References

• Truvada [package insert]. Foster City, CA: Gilead Sciences, Inc; 2013.
   Google Scholar
• US Food and Drug Administration. FDA approves first drug for reducing the risk of sexually acquired HIV infection [press release]. https://aidsinfo.nih.gov/news/1254/fda-approves-first-drug-for-reducing-the-risk-of-sexually-acquired-hiv-infection. Accessed September 22, 2017.
   Google Scholar
• Medicines Control Council (South Africa). Medicines Control Council approves fixed-dose combination of tenofovir disoproxyl fumarate and emtricitabine for pre-exposure prophylaxis of HIV [press release]. http://www.mccza.com/documents/2e4b3a5310.11_Media_release_ARV_FDC_PrEP_Nov15_v1.pdf. Accessed March 15, 2017.
   Google Scholar
• European Commission grants marketing authorization for Gilead’s once-daily Truvada® for reducing the risk of sexually acquired HIV-1 [press release]. https://www.gilead.com/news/press-releases/2016/8/european-commission-grants-marketing-authorization-for-gileads-oncedaily-truvada-for-reducing-the-risk-of-sexually-acquired-hiv1. Accessed March 15, 2017.
   Google Scholar
• World Health Organization. Guideline on when to start antiretroviral therapy and on pre-exposure prophylaxis for HIV. http://www.who.int/hiv/pub/guidelines/earlyrelease-arv/en/. Accessed March 15, 2017.
   Google Scholar
• Grant RM, Lama JR, Anderson PL, et al. Preexposure chemoprophylaxis for HIV prevention in men who have sex with men. N Engl J Med. 2010;363(27):2587–2599.10.1056/NEJMoa1011205
   PubMed Web of Science ®Google Scholar
• McCormack S, Dunn DT, Desai M, et al. Pre-exposure prophylaxis to prevent the acquisition of HIV-1 infection (PROUD): effectiveness results from the pilot phase of a pragmatic open-label randomised trial. Lancet. 2016;387(10013):53–60.10.1016/S0140-6736(15)00056-2
   PubMed Web of Science ®Google Scholar
• Molina JM, Capitant C, Spire B, et al. On-demand preexposure prophylaxis in men at high risk for HIV-1 infection. N Engl J Med. 2015;373(23):2237–2246.10.1056/NEJMoa1506273
   PubMed Web of Science ®Google Scholar
• Volk JE, Marcus JL, Phengrasamy T, et al. No new HIV infections with increasing use of HIV preexposure prophylaxis in a clinical practice setting. Clin Infect Dis. 2015;61(10):1601–1603.10.1093/cid/civ778
   PubMed Web of Science ®Google Scholar
• Baeten JM, Donnell D, Ndase P, et al. Antiretroviral prophylaxis for HIV prevention in heterosexual men and women. N Engl J Med. 2012;367(5):399–410.10.1056/NEJMoa1108524
   PubMed Web of Science ®Google Scholar
• Marrazzo JM, Ramjee G, Richardson BA, et al. Tenofovir-based preexposure prophylaxis for HIV infection among African women. N Engl J Med. 2015;372(6):509–518.10.1056/NEJMoa1402269
   PubMed Web of Science ®Google Scholar
• Van Damme L, Corneli A, Ahmed K, et al. Preexposure prophylaxis for HIV infection among African women. N Engl J Med. 2012;367(5):411–422.10.1056/NEJMoa1202614
   PubMed Web of Science ®Google Scholar
• Thigpen MC, Kebaabetswe PM, Paxton LA, et al. Antiretroviral preexposure prophylaxis for heterosexual HIV transmission in Botswana. N Engl J Med. 2012;367(5):423–434.10.1056/NEJMoa1110711
   PubMed Web of Science ®Google Scholar
• Choopanya K, Martin M, Suntharasamai P, et al. Antiretroviral prophylaxis for HIV infection in injecting drug users in Bangkok, Thailand (the Bangkok Tenofovir Study): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2013;381(9883):2083–2090.10.1016/S0140-6736(13)61127-7
   PubMed Web of Science ®Google Scholar
• Patterson KB, Prince HA, Kraft E, et al. Penetration of tenofovir and emtricitabine in mucosal tissues: implications for prevention of HIV-1 transmission. Sci Transl Med. 2011;3(112):112re114.
   Web of Science ®Google Scholar
• Hanscom B, Janes HE, Guarino PD, et al. Brief report: preventing HIV-1 infection in women using oral preexposure prophylaxis: a meta-analysis of current evidence. J Acquir Immune Defic Syndr. 2016;73(5):606–608.10.1097/QAI.0000000000001160
   PubMed Web of Science ®Google Scholar
• Baeten JM, Palanee-Phillips T, Brown ER, et al. Use of a vaginal ring containing dapivirine for HIV-1 prevention in women. N Engl J Med. 2016;375(22):2121–2132.10.1056/NEJMoa1506110
   PubMed Web of Science ®Google Scholar
• Nel A, Saidi K, Bekker L-G, et al. Abstract presented at: Conference on Retroviruses and Opportunistic Infections 2016; February 22–25, 2016; Boston, MA.
   Google Scholar
• Institute of Medicine. Methodological Challenges in Biomedical HIV Prevention Trials. Washington, DC: The National Academies Press; 2008.
   Google Scholar
• Donnell D, Hughes JP, Wang L, Chen YQ, Fleming TR. Study design considerations for evaluating efficacy of systemic preexposure prophylaxis interventions. J Acquir Immune Defic Syndr. 2013;63(suppl 2):S130–S134.10.1097/QAI.0b013e3182986fac
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Human immunodeficiency virus-1 infection: developing antiretroviral drugs for treatment: guidance for industry, revision 1. Silver Spring, MD: US Dept of Health and Human Services; US Food and Drug Administration; Center for Drug Evaluation and Research; 2015.
   Google Scholar
• Hernandez AV, Pasupuleti V, Deshpande A, Thota P, Collins JA, Vidal JE. Deficient reporting and interpretation of non-inferiority randomized clinical trials in HIV patients: a systematic review. PlS One. 2013;8(5):e63272.10.1371/journal.pone.0063272
   PubMed Web of Science ®Google Scholar
• Spilker B. Guide to Clinical Trials. New York, NY: Raven Press; 1991.
   Google Scholar
• Fleming TR, Richardson BA. Some design issues in trials of microbicides for the prevention of HIV infection. J Infect Dis. 2004;190(4):666–674.10.1086/jid.2004.190.issue-4
   PubMed Web of Science ®Google Scholar
• Padian NS. Evidence‐based prevention: increasing the efficiency of HIV intervention trials. J Infect Dis. 2004;190(4):663–665.10.1086/jid.2004.190.issue-4
   PubMed Web of Science ®Google Scholar
• Stein ZA, Susser MW. Control groups in microbicide trials: in defense of orthodoxy. J Infect Dis. 2005;191(8):1377–1378.10.1086/jid.2005.191.issue-8
   PubMed Web of Science ®Google Scholar
• Skoler S, Govender S, Altini L, et al. Risks in the use of an unblinded‐control group. J Infect Dis. 2005;191(8):1378–1379.10.1086/jid.2005.191.issue-8
   PubMed Web of Science ®Google Scholar
• Fleming TR. Reply to Skoler et al. and to Stein and Susser. J Infect Dis. 2005;191(8):1379–1380.10.1086/jid.2005.191.issue-8
   Google Scholar
• Padian NS. Reply to Stein and Susser. J Infect Dis. 2005;191(8):1380–1381.10.1086/jid.2005.191.issue-8
   Google Scholar
• Richardson BA, Kelly C, Ramjee G, et al. Appropriateness of hydroxyethylcellulose gel as a placebo control in vaginal microbicide trials: a comparison of the two control arms of HPTN 035. J Acquir Immune Defic Syndr. 2013;63(1):120–125.10.1097/QAI.0b013e31828607c5
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration. Guidance for industry: adaptive design clinical trials for drugs and biologics. https://www.fda.gov/downloads/drugs/guidances/ucm201790.pdf. Accessed March 15, 2017.
   Google Scholar
• Nason M, Follmann D. Design and analysis of crossover trials for absorbing binary endpoints. Biometrics. 2010;66(3):958–965.10.1111/j.1541-0420.2009.01358.x
   PubMed Web of Science ®Google Scholar
• Auvert B, Sitta R, Zarca K, Mahiane SG, Pretorius C, Lissouba P. The effect of heterogeneity on HIV prevention trials. Clin Trials. 2011;8(2):144–154.10.1177/1740774511398923
   PubMed Web of Science ®Google Scholar
• Makubate B, Senn S. Planning and analysis of cross-over trials in infertility. Stat Med. 2010;29(30):3203–3210.10.1002/sim.3981
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003616.pdf. Accessed March 15, 2017.
   Google Scholar
• Baeten JM, Heffron R, Kidoguchi L, et al. Integrated delivery of antiretroviral treatment and pre-exposure prophylaxis to HIV-1-serodiscordant couples: a prospective implementation study in Kenya and Uganda. PLoS Med. 2016;13(8):e1002099.10.1371/journal.pmed.1002099
   PubMed Web of Science ®Google Scholar
• Dunn DT, Glidden DV. Statistical issues in trials of preexposure prophylaxis. Curr Opin HIV AIDS. 2016;11(1):116–121.10.1097/COH.0000000000000218
   PubMed Web of Science ®Google Scholar