Small sample approach, and statistical and epidemiological aspects

Handbook of Experimental Pharmacology

Volumn 205, Issue , 2011, Pages 181-202

  Offringa, Martin ^a   Van Der Lee, Hanneke ^a  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

Child; Data Monitoring Committees; Interim analysis; Pediatrics; Pharmacokinetics; Phase III trial; Power; Sample size; Sequential design; Triangular test

Indexed keywords

DEXAMETHASONE; INDOMETACIN; LISDEXAMFETAMINE; PROSTAGLANDIN E1;

ADAPTIVE DESIGN; ALTERNATIVE HYPOTHESIS; AREA UNDER THE CURVE; ARTICLE; ATTENTION DEFICIT DISORDER; BLOOD SAMPLING; BOUNDARIES DESIGN; CLINICAL STUDY; CONFIDENCE INTERVAL; DRUG ABSORPTION; DRUG DOSE ESCALATION; DRUG EXCRETION; DRUG HALF LIFE; DRUG METABOLISM; DRUG RESEARCH; EQUIPOISE; FALSE POSITIVE RESULT; GROUP SEQUENTIAL DESIGN; HUMAN; INTERIM ANALYSIS; KIDNEY TUBULE DISORDER; LOWER RESPIRATORY TRACT INFECTION; MAXIMUM PLASMA CONCENTRATION; NULL HYPOTHESIS; PATENT DUCTUS ARTERIOSUS; PATIENT SAFETY; PEDIATRICS; PHASE 3 CLINICAL TRIAL (TOPIC); POPULATION PHARMACOKINETICS; POWER ANALYSIS; PRIORITY JOURNAL; PROFESSIONAL STANDARD; RESEARCH ETHICS; SAMPLE SIZE; STUDY DESIGN; TIME TO MAXIMUM PLASMA CONCENTRATION;

ALGORITHMS; CLINICAL TRIALS AS TOPIC; CLINICAL TRIALS DATA MONITORING COMMITTEES; CLINICAL TRIALS, PHASE III AS TOPIC; EPIDEMIOLOGIC RESEARCH DESIGN; HUMANS; PEDIATRICS; PHARMACOKINETICS; SAMPLE SIZE;

EID: 80054964904     PISSN: 01712004     EISSN: 18650325     Source Type: Book Series    
DOI: 10.1007/978-3-642-20195-0_9     Document Type: Article

References (66)

1. (2004) PEST 4.4 operating manual. The University of Reading, Reading
2. Anderson TW (1960) A modification of the sequential probability ratio test to reduce the sample size. Ann Math Stat 31:165-197
3. Armitage P (1958) Sequential methods in clinical trials. Am J Public Health 48:1395-1402
4. Armitage P (1975) Sequential medical trials. Blackwell, Oxford
5. Bassler D, Briel M, Montori VM, Lane M, Glasziou P, Zhou Q, Heels-Ansdell D, Walter SD, Guyatt GH, Flynn DN, Elamin MB, Murad MH, Abu Elnour NO, Lampropulos JF, Sood A, Mullan RJ, Erwin PJ, Bankhead CR, Perera R, Ruiz CC, You JJ, Mulla SM, Kaur J, Nerenberg KA, Schunemann H, Cook DJ, Lutz K, Ribic CM, Vale N, Malaga G, Akl EA, Ferreira- Gonzalez I, Alonso-Coello P, Urrutia G, Kunz R, Bucher HC, Nordmann AJ, Raatz H, da Silva SA, Tuche F, Strahm B, Djulbegovic B, Adhikari NK, Mills EJ, Gwadry-Sridhar F, Kirpalani H, Soares HP, Karanicolas PJ, Burns KE, Vandvik PO, Coto-Yglesias F, Chrispim PP, Ramsay T (2010) Stopping randomized trials early for benefit and estimation of treatment effects: Systematic review and meta-regression analysis. JAMA 303:1180-1187
6. Bauer P, Kieser M (1999) Combining different phases in the development of medical treatments within a single trial. Stat Med 18:1833-1848 (Pubitemid 29364054)
7. Bauer P, Kohne K (1994) Evaluation of experiments with adaptive interim analyses. Biometrics 50:1029-1041
8. Bellissant E, Duhamel JF, Guillot M, Pariente-Khayat A, Olive G, Pons G (1997) The triangular test to assess the efficacy of metoclopramide in gastroesophageal reflux. Clin Pharmacol Ther 61:377-384 (Pubitemid 27145439)
9. Boellner SW, Stark JG, Krishnan S, Zhang Y (2010) Pharmacokinetics of lisdexamfetamine dimesylate and its active metabolite, d-amphetamine, with increasing oral doses of lisdexamfetamine dimesylate in children with attention-deficit/hyperactivity disorder: A single-dose, randomized, open-label, crossover study. Clin Ther 32:252-264
10. Bretz F, Koenig F, Brannath W, Glimm E, Posch M (2009) Adaptive designs for confirmatory clinical trials. Stat Med 28:1181-1217
11. CHMP (2006a) Guideline on clinical trials in small populations. EMA, London
12. CHMP (2006b) Reflection paper on methodological issues in confirmatory clinical trials with flexible design and analysis plan. EMA, London
13. Coad DS, Ivanova A (2005) The use of the triangular test with response-adaptive treatment allocation. Stat Med 24:1483-1493 (Pubitemid 40716348)
14. Cytel Software Corporation (1992) EaSt: A software package for the design and interim monitoring of group sequential clinical trials. Cytel Software Corporation, Cambridge, MA
15. De Cock RF, Piana C, Krekels EH, Danhof M, Allegaert K, Knibbe CA (2011) The role of population PK-PD modelling in paediatric clinical research. Eur J Clin Pharmacol. 67 Suppl 1:5-16
16. Edwards SJ, Lilford RJ, Braunholtz DA, Jackson JC, Hewison J, Thornton J (1998) Ethical issues in the design and conduct of randomised controlled trials. Health Technol Assess 2:1-132
17. Ellenberg SS, Fleming TR, DeMets DL (2002) Data Monitoring Committees in Clinical Trials. Chichester, John Wiley Sons, Ltd
18. Emerson SS, Fleming TR (1989) Symmetric group sequential test designs. Biometrics 45: 905-923 (Pubitemid 19249330)
19. Fernandes RM, van der Lee JH, Offringa M (2009) A systematic review of the reporting of Data Monitoring Committees' roles, interim analysis and early termination in pediatric clinical trials. BMC Pediatr 9:77
20. Filler G, Browne R, Seikaly MG (2003) Glomerular filtration rate as a putative 'surrogate endpoint' for renal transplant clinical trials in children. Pediatr Transplant 7:18-24 (Pubitemid 36314185)
21. Freiman JA, Chalmers TC, Smith H Jr, Kuebler RR (1978) The importance of beta, the type II error and sample size in the design and interpretation of the randomized control trial. Survey of 71 "negative" trials. N Engl J Med 299:690-694 (Pubitemid 9053296)
22. Glasziou P, Chalmers I, Rawlins M, McCulloch P (2007) When are randomised trials unnecessary? Picking signal from noise. BMJ 334:349-351 (Pubitemid 46328045)
23. Grant AM, Altman DG, Babiker AB, Campbell MK, Clemens FJ, Darbyshire JH, Elbourne DR, McLeer SK, Parmar MK, Pocock SJ, Spiegelhalter DJ, Sydes MR, Walker AE, Wallace SA (2005) Issues in data monitoring and interim analysis of trials. Health Technol Assess 9:1-238
24. Haybittle JL (1971) Repeated assessment of results in clinical trials of cancer treatment. Br J Radiol 44:793-797
25. Hirtz DG, Gilbert PR, Terrill CM, Buckman SY (2006) Clinical trials in children - how are they implemented? Pediatr Neurol 34:436-438 (Pubitemid 43831577)
26. Hommel G (2001) Adaptive modifications of hypotheses after an interim analysis. Biometrical J 43:581-589 (Pubitemid 33588682)
27. Jennison C, Turnbull BW (2003) Mid-course sample size modification in clinical trials based on the observed treatment effect. Stat Med 22:971-993 (Pubitemid 36322098)
28. Kelly PJ, Stallard N, Todd S (2005) An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several. J Biopharm Stat 15:641-658 (Pubitemid 40967305)
29. Kieser M, Bauer P, Lehmacher W (1999) Inference on multiple endpoints in clinical trials with adaptive interim analyses. Biometrical J 41:261-277
30. Klassen TP, Hartling L, Hamm M, van der Lee JH, Ursum J, OffringaM(2009) StaR Child Health: An initiative for RCTs in children. Lancet 374:1310-1312
31. Knottnerus JA, Bouter LM (2001) The ethics of sample size: Two-sided testing and one-sided thinking. J Clin Epidemiol 54:109-110 (Pubitemid 32112367)
32. Lan KKG, DeMets DL (1983) Discrete sequential boundaries for clinical trials. Biometrika 70:659-663
33. Lehmacher W, Wassmer G (1999) Adaptive sample size calculations in group sequential trials. Biometrics 55:1286-1290
34. McKinney RE Jr, Johnson GM, Stanley K, Yong FH, Keller A, O'Donnell KJ, Brouwers P, Mitchell WG, Yogev R, Wara DW, Wiznia A, Mofenson L, McNamara J, Spector SA (1998) A randomized study of combined zidovudine-lamivudine versus didanosine monotherapy in children with symptomatic therapy-naive HIV-1 infection. The Pediatric AIDS Clinical Trials Group Protocol 300 Study Team. J Pediatr 133:500-508 (Pubitemid 28465375)
35. Moye LA, Tita AT (2002) Hypothesis testing complexity in the name of ethics: Response to commentary. J Clin Epidemiol 55:209-211
36. Nyman Y, Von HK, Palm C, Eksborg S, Lonnqvist PA (2006) Etomidate-Lipuro is associated with considerably less injection pain in children compared with propofol with added lidocaine. Br J Anaesth 97:536-539 (Pubitemid 44419969)
37. O'Brien PC, Fleming TR (1979) A multiple testing procedure for clinical trials. Biometrics 35:549-556 (Pubitemid 10242750)
38. Ogungbenro K, Aarons L (2008) How many subjects are necessary for population pharmacokinetic experiments? Confidence interval approach. Eur J Clin Pharmacol 64:705-713
39. Pampallona S, Tsiatis AA (1994) Group sequential designs for one-sided and two-sided hypothesis testing with provision for early stopping in favor of the null hypothesis. J Stat Plan Infer 42:19-35
40. Peto R, Baigent C (1998) Trials: The next 50 years. Large scale randomised evidence of moderate benefits. BMJ 317:1170-1171
41. Pocock SJ (1977) Group sequential methods in the design and analysis of clinical trials. Biometrika 64:191-199 (Pubitemid 8145484)
42. Rosenberger WF, Huc F (2004) Maximizing power and minimizing treatment failures in clinical trials. Clin Trials 1:141-147
43. Sackett DL, Cook DJ (1993) Can we learn anything from small trials? Ann NY Acad Sci 703:25-31
44. Sammons HM (2011) Avoiding clinical trials in children. Arch Dis Child 96:291-292
45. Schulz KF, Grimes DA (2005) Sample size calculations in randomised trials: Mandatory and mystical. Lancet 365:1348-1353
46. Sebille V, Bellissant E (2000) Comparison of four sequential methods allowing for early stopping of comparative clinical trials. Clin Sci (Lond) 98:569-578 (Pubitemid 30261006)
47. Sebille V, Bellissant E (2003) Sequential methods and group sequential designs for comparative clinical trials. Fundam Clin Pharmacol 17:505-516 (Pubitemid 37215076)
48. Simes RJ (1995) Prospective meta-analysis of cholesterol-lowering studies: The Prospective Pravastatin Pooling (PPP) Project and the Cholesterol Treatment Trialists (CTT) Collaboration. Am J Cardiol 76:122C-126C
49. Smith GC, Pell JP (2003) Parachute use to prevent death and major trauma related to gravitational challenge: Systematic review of randomised controlled trials. BMJ 327:1459-1461 (Pubitemid 38045824)
50. Streiner DL, Norman GR (2008) Health Measurement Scales: A practical guide to their development and use. Oxford University Press, Oxford
51. Tarnow-Mordi WO, Healy MJ (1999) Distinguishing between "no evidence of effect" and "evidence of no effect" in randomised controlled trials and other comparisons. Arch Dis Child 80:210-211
52. Twisk JWR (2003) Applied longitudinal data analysis for epidemiology; a practical guide. Cambridge University Press,Cambridgevan
53. der Lee JH, Wesseling J, Tanck MW, Offringa M (2008) Efficient ways exist to obtain the optimal sample size in clinical trials in rare diseases. J Clin Epidemiol 61:324-330
54. van der Lee JH, Tanck MW, Wesseling J, Offringa M (2009) Pitfalls in the design and analysis of paediatric clinical trials: A case of a 'failed' multi-centre study, and potential solutions. Acta Paediatr 98:385-391
55. van der Lee JH, Wesseling J, Tanck MW, Offringa M (2010) Sequential design with boundaries approach in pediatric intervention research reduces sample size. J Clin Epidemiol 63:19-27
56. van Houwelingen HC (1999) On "Bayesian monitoring⋯". J Clin Epidemiol 52:713-714
57. van Woensel JB, van Aalderen WM, de Weerd W, Jansen NJ, van Gestel JP, Markhorst DG, van Vught AJ, Bos AP, Kimpen JL (2003) Dexamethasone for treatment of patients mechanically ventilated for lower respiratory tract infection caused by respiratory syncytial virus. Thorax 58:383-387 (Pubitemid 36560939)
58. Vickers AJ (2003) Underpowering in randomized trials reporting a sample size calculation. J Clin Epidemiol 56:717-720 (Pubitemid 37040806)
59. Vickers AJ, Altman DG (2001) Statistics notes: Analysing controlled trials with baseline and follow up measurements. BMJ 323:1123-1124 (Pubitemid 33063362)
60. von Goedecke A, Brimacombe J, Hormann C, Jeske HC, Kleinsasser A, Keller C (2005) Pressure support ventilation versus continuous positive airway pressure ventilation with the ProSeal laryngeal mask airway: A randomized crossover study of anesthetized pediatric patients. Anesth Analg 100:357-360 (Pubitemid 40170523)
61. Wald A (1947) Sequential analysis. Wiley, New York
62. Wassmer G (2000) Basic concepts of group sequential and adaptive group sequential test procedures. Stat Pap 41:253-279
63. Whitehead J (1997) The design and analysis of sequential clinical trials. Wiley, Chichester
64. Whitehead J, Jones DR (1979) The analysis of sequential clinical trials. Biometrika 66: 443-452 (Pubitemid 10218392)
65. Whitehead J, Stratton I (1983) Group sequential clincial trials with triangular continuation regions. Biometrics 39:227-236
66. Woods JR, Williams JG, Tavel M (1989) The two-period crossover design in medical research. Ann Intern Med 110:560-566 (Pubitemid 19097323)