Some design issues in phase 2B vs phase 3 prevention trials for testing efficacy of products or concepts

Statistics in Medicine

Volumn 29, Issue 10, 2010, Pages 1061-1071

  Gilbert, Peter B ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Clinical trial; Decision analysis; HIV vaccine; Intermediate sized efficacy trial; Licensure; Microbicide; Phase 2b vs phase 3

Indexed keywords

HUMAN IMMUNODEFICIENCY VIRUS VACCINE;

ARTICLE; DECISION MAKING; DRUG EFFICACY; FALSE NEGATIVE RESULT; FALSE POSITIVE RESULT; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PRACTICE GUIDELINE; PREVENTION STUDY; SENSITIVITY ANALYSIS;

AIDS VACCINES; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; DECISION SUPPORT TECHNIQUES; HIV INFECTIONS; HUMANS; VACCINES;

EID: 77951619664     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3676     Document Type: Article

References (17)

1. Fleming TR, Richardson BA. Some design issues in trials of microbicides for the prevention of HIV infection. The Journal of Infectious Diseases 2004; 190:666-674. DOI: 10.1086/422603. (Pubitemid 39049994)
2. Moore JP, Ho DD. HIV-1 neutralization: the consequences of viral adaptation to growth on transformed T cells. AIDS 1995; 9(Suppl A):S117-S136.
3. Flynn NM, Forthal DN, Harro CD, Judson FN, Mayer KH, Para MF, The rgp120 HIV Vaccine Study Group. Placebo-controlled phase 3 trial of recombinant glycoprotein 120 vaccine to prevent HIV-1 infection. The Journal of Infectious Diseases 2005; 191:654-665. DOI: 10.1086/428404.
4. Pitisuttithum P, Gilbert PB, Gurwith M, Heyward W, Martin M, van Griensven F, Hu D, Tappero JW, Choopanya K, The Bangkok Vaccine Evaluation Group. Randomized, double-blind, placebo-controlled efficacy trial of a bivalent recombinant glycoprotein 120 HIV-1 vaccine among injection drug users in Bangkok, Thailand. The Journal of Infectious Diseases 2006; 194:1661-1671. DOI: 10.1086/508748. (Pubitemid 44901996)
5. Buchbinder SP, Mehrotra DV, Duerr A, Fitzgerald DW, Mogg R, Li D, Gilbert PB, Lama JR, Marmor M, del Rio C, McElrath MJ, Casimiro DR, Gottesdiener KM, Chodakewitz JA, Corey L, Robertson MN, Step Study Protocol Team. Efficacy assessment of a cell-mediated immunity HIV-1 vaccine (the Step Study): a double-blind, randomised, placebo-controlled, test-of-concept trial. Lancet 2008; 372:1881-1893. DOI: 10.1016/S0140-6736(08)61591-61593
6. Anderson RM, Garnett GP. Low-efficacy HIV vaccines: potential for community-based intervention programs. Lancet 1996; 348:1010-1013. DOI: 10.1016/S0140-6736(96)07100-7110
7. Self SG. Issues in the design of HIV vaccine efficacy trials. In Accelerating AIDS Vaccine Development: Challenges and Opportunities, Kahn P, Gust I, Koff W (eds). Horizon Scientific Press: Norfolk, U.K., 2006.
8. Rida W, Fast P, Hoff R, Fleming TR. Intermediate-size trials for the evaluation of an HIV vaccine candidate: a workshop summary. Journal of the Acquired Immune Deficiency Syndrome and Human Retrovirology 1997; 16:195-203. (Pubitemid 27519933)
9. Karim SA. Safety and effectiveness of vaginal microbicides BufferGel and 0.5% PRO 20005 Gel for the prevention of HIV infection in women: results of the HPTN 035 trial. In Program and Abstracts of the 16th Conference on Retroviruses and Opportunistic Infections, Montreal, 2009; Abstract no. 48LB.
10. Fleming TR, Harrington DP. Counting Processes and Survival Analysis. Wiley: New York, 1991.
11. Berry DA. Statistics: A Bayesian Perspective. Duxbury Press: Belmont, CA, 1996.
12. Girón FJ. Applied Decision Analysis. Springer: New York, 1998.
13. Petitti DB. Meta-analysis, Decision Analysis, and Cost-Effectiveness Analysis: Methods for Quantitative Synthesis in Medicine. Oxford University Press: London, 2000.
14. Pascal B. The Pensées (Translation by Krailsheimer AJ). Penguin Books: London, 1966; (1670).
15. Hacking I. The Emergence of Probability. Cambridge University Press: London, 1975.
16. Mehrotra DV, Li X, Gilbert PB. A comparison of eight methods for the dual-endpoint evaluation of efficacy in a proof-of-concept HIV vaccine trial. Biometrics 2006; 62:893-900. DOI: 10.1111/j.1541-0420.2005.00516.x. (Pubitemid 44410648)
17. Excler J-L, Rida W, Priddy F, Fast P, Koff W. A strategy for accelerating the development of preventive AIDS vaccines. AIDS 2007; 21:2259-2263. DOI: 10.1097/QAD.0b013e3282eee70c. (Pubitemid 350287451)