Increasing the efficiency of clinical trials of antimicrobials: The scientific basis of substantial evidence of effectiveness of drugs

Clinical Infectious Diseases

Volumn 45, Issue SUPPL. 2, 2007, Pages

  Powers, John H ^a,b,c,d  

^a SCIENCE APPLICATIONS INTERNATIONAL CORPORATION   (United States)

^b UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE   (United States)

^c GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

^d NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIINFECTIVE AGENT;

ACUTE OTITIS MEDIA; CHRONIC BRONCHITIS; CLINICAL TRIAL; CONFERENCE PAPER; DISEASE EXACERBATION; DRUG APPROVAL; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; HUMAN; MEDICAL EXAMINATION; META ANALYSIS; PRIORITY JOURNAL; SINUSITIS; SYSTEMATIC REVIEW;

ANTI-INFECTIVE AGENTS; CLINICAL TRIALS; DRUG AND NARCOTIC CONTROL; HUMANS; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 34548291867     PISSN: 10584838     EISSN: None     Source Type: Journal    
DOI: 10.1086/519253     Document Type: Conference Paper

References (42)

1. Freedman B. Scientific value and validity as ethical requirements for research: a proposed explication. IRB 1987; 9:7-10.
2. US Government Printing Office. Federal Food, Drug, and Cosmetic Act. New drugs. US Code title 21, section 351: chapter V, subchapter A, section 505, 2007. Available at: http://www.fda.gov/opacom/laws/fdcact/fdcact5a.htm.
3. Medical Research Council, Patulin Clinical Trials Committee. Clinical trials of patulin in the common cold. Lancet 1944; ii:373-5.
4. Medical Research Council. Streptomycin treatment of pulmonary tuberculosis. BMJ 1948; ii:769-82.
5. Gower B. Scientific method: an historical and scientific introduction. London: Routledge, 1997.
6. Petitti D. Commentary: hormone replacement therapy and coronary heart disease: four lessons. Int J Epidemiol 2004; 33:461-3.
7. Temple RJ. Development of drug law, regulations, and guidance in the United States. In: Munson PL, Mueller RA, Breese G, eds. Principles of pharmacology: basic concepts and clinical applications, 1st ed. New York: Chapman and Hall, 1995:1643-63.
8. Pharmaceutical Manufacturers Association v Richardson. 318 F Supp 301, 1970.
9. Upjohn Co v Finch. Federal Reporter volume 422, 2nd series, 1970:944, 955.
10. Weinberger v Hynson, Westcott, and Dunning. US Code title 412, section 609, 1973.
11. 'Lectric Law Library. Legal definition of substantial evidence. Available at: http://www.lectlaw.com/def2/s087.htm. Accessed 25 June 2007.
12. United States v Rutherford. US Code title 442, section 544, no 78-605, 1979.
13. US Government Printing Office. Applications for FDA approval to market a new drug: adequate and well-controlled studies. US Code title 21, section 314.126, 2006. Available at: http://a257.g.akamaitech.net/7/257/2422/ 10apr20061500/edocket.access.gpo.gov/cfr_2006/aprqtr/21cfr314.126.htm. Accessed 2 July 2007.
14. Shifting goalposts in antibiotic approval. Lancet Infect Dis 2006; 6:751.
15. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH harmonised tripartite guideline: statistical principles for clinical trials. ICH E-9. Geneva: ICH, 1998. Available at: http://www.ich.org/MediaServer.jser?@_ID= 485&@MODE=GLB. Accessed 2 July 2007.
16. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH harmonised tripartite guideline: choice of control group and related issues in clinical trials. ICH E-10. Geneva: ICH, 2000. Available at: http://www.ich.org/MediaServer. jser?@_ID=486&@_MODE=GLB. Accessed 2 July 2007.
17. US Food and Drug Administration. Center for Drug Evaluation and Research guidances. Available at: http://www.fda.gov/cder/guidance/index.htm. Accessed 2 July 2007.
18. Specific requirements on content and format of labeling for human prescription drugs. US Code title 21, part 201.57, 2006. Available at: http://a257.g.akamaitech.net/7/257/2422/10apr20061500/edocket.access.gpo.gov/ cfr_2006/aprqtr/21cfr201.57.htm. Accessed 2 July 2007.
19. Powers JH. Empirical antifungal therapy in febrile neutropenic patients: caution about composite end points and the perils of P values. Clin Infect Dis 2004; 39:1738-9.
20. Powers JH. Interpreting the results of clinical trials on antimicrobial agents. In: Mandell GL, Bennett JE, Dolin R, eds. Principles and practice of infectious diseases. 6th ed. Philadelphia: Elsevier Churchill Livingstone, 2005:619-28.
21. Senn S. Design and interpretation of clinical trials. In: Senn S, ed. Statistical issues in drug development. Chichester, England: John Wiley and Sons, 1997:27-42.
22. Pocock SJ. The pros and cons of noninferiority trials. Fundam Clin Pharmacol 2003; 17:483-90.
23. US Food and Drug Administration. Clinical trial design in studies of acute bacterial sinusitis. Anti-Infective Drugs Advisory Committee Meeting, 2003. Available at: http://www.fda.gov/ohrms/dockets/ac/03/transcripts/ 3997T2.pdf. Accessed 2 July 2007.
24. US Food and Drug Administration. Antimicrobial Resistance & Drug Development FDA/IDSA/ISAP Working Group presentations and transcripts, 2004. Available at: http://www.fda.gov/cder/drug/antimicrobial/ FDA_IDSA_ISAP_Presentations.htm. Accessed 2 July 2007.
25. US Food and Drug Administration. Transcript of the IDSA/PhRma/FDA Working Group Meeting, November 19-20, 2002. Available at: http://www.fda.gov/ cder/present/idsaphrma/default.htm. Accessed 2 July 2007.
26. US Food and Drug Administration. Transcripts of the Anti-Infective Drugs Advisory Committee: gemifloxacin NDA 21-158/S006. 2006. Available at: http://www.fda.gov/ohrms/dockets/ac/cder06.html#AntiInfective. Accessed 2 July 2007.
27. US Food and Drug Administration. Transcripts of the Joint Meeting of the Anti-Infective Drugs and Drug Safety and Risk Management Advisory Committees: telithromycin NDA 21-144. 2006. Available at: http://www.fda.gov/ohrms/ dockets/ac/cder06.html#AntiInfective.
28. Almyroudis NG, Kontoyiannis DP, Sepkowitz KA. Issues related to the design and interpretation of clinical trials for salvage therapy for invasive mold infections. Clin Infect Dis 2006; 43:1449-55.
29. Powers JH. Salvage therapy trials in invasive fungal disease: challenges and opportunities. Clin Infect Dis 2006; 43:1456-60.
30. Hill AB. The environment and disease: association or causation? Proc R Soc Med 1965; 58:295-300.
31. Johann-Liang R, Zalkikar J, Powers JH. Correlation between bacteriologic and clinical endpoints in trials of acute otitis media. Pediatr Infect Dis J 2003; 22:936-7.
32. Powers JH. Microbiologic surrogate end points in clinical trials of infectious diseases: example of acute otitis media trials. Pharmacotherapy 2005; 25(12 Pt 2):109S-23S.
33. Fleming TR, DeMets DL. Surrogate end points in clinical trials: are we being misled? Ann Intern Med 1996; 125:605-13.
34. Snapinn SM. Noninferiority trials. Curr Control Trials Cardiovasc Med 2000; 1:19-21.
35. Hanley JA, Lippman-Hand A. If nothing goes wrong, is everything all right? Interpreting zero numerators. JAMA 1983; 249:1743-5.
36. Powers JH. Antimicrobial drug development - the past, the present, and the future. Clin Microbiol Infect 2004; 10(Suppl 4):23-31.
37. Hong L, Levy SM, Warren JJ, Dawson DV, Bergus GR, Wefel JS. Association of amoxicillin use during early childhood with developmental tooth enamel defects. Arch Pediatr Adolesc Med 2005; 159:943-8.
38. Powers JH, Ross DB, Lin D, et al. Linezolid and vancomycin for methicillin-resistant Staphylococcus aureus nosocomial pneumonia: the subtleties of subgroup analyses. Chest 2004; 126:314-6.
39. Bailar JC III. The promise and problems of meta-analysis. N Engl J Med 1997; 337:559-61.
40. Rovers MM, Glasziou P, Appelman CL, et al. Antibiotics for acute otitis media: a meta-analysis with individual patient data. Lancet 2006; 368:1429-35.
41. Saint S, Bent S, Vittinghoff E, Grady D. Antibiotics in chronic obstructive pulmonary disease exacerbations: a meta-analysis. JAMA 1995; 273:957-60.
42. LeLorier J, Gregoire G, Benhaddad A, Lapierre J, Derderian F. Discrepancies between meta-analyses and subsequent large randomized, controlled trials. N Engl J Med 1997; 337:536-42.