Advancing clinical trial design in pulmonary hypertension

Pulmonary Circulation

Volumn 3, Issue 1, 2013, Pages 217-225

  Grieve, Andy P ^a   Chow, Shien Chung ^b   Curram, John ^c   Dawe, Stephen ^d   Harnisch, Lutz O ^e   Henig, Noreen R ^f   Hung, Hsien Ming J ^g   Ivy, D Dunbar ^h   Kawut, Steven M ⁱ   Rahbar, Mohammad H ^j   Xiao, Shen ^g   Wilkins, Martin R ^k  

^a ICON PLC   (United Kingdom)

^b DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c Bayer plc UK   (United Kingdom)

^d Novartis Pharmaceuticals UK Limited   (United Kingdom)

^e PFIZER GLOBAL RESEARCH AND DEVELOPMENT   (United Kingdom)

^f GILEAD SCIENCES   (United States)

^g CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^h UNIVERSITY OF COLORADO   (United States)

ⁱ UNIVERSITY OF PENNSYLVANIA   (United States)

^j UNIVERSITY OF TEXAS SCHOOL OF PUBLIC HEALTH   (United States)

^k IMPERIAL COLLEGE LONDON   (United Kingdom)

more..

Author keywords

Adaptive designs; Bayesian modeling; Clinical trial design; Group sequential designs; Modeling and simulation; Population enrichment; Pulmonary arterial hypertension

Indexed keywords

EID: 84885859637     PISSN: 20458932     EISSN: 20458940     Source Type: Journal    
DOI: 10.4103/2045-8932.109933     Document Type: Article

References (43)

1. Rothwell PM. External validity of randomised controlled trials: “To whom do the results of this trial apply?”. Lancet 2005;365:82-93.
2. Schwartz D, Lellouch J. Explanatory and pragmatic attitudes in therapeutic trials. J Chronic Dis 1967;20:637-48.
3. Hoeper MM, Oudiz RJ, Peacock A, Tapson VF, Howarth SG, Frost AE, et al. Endpoints and clinical trial designs in pulmonary arterial hypertension. J Am Coll Cardiol 2004;43:48S-55.
4. Gomberg-Maitland M. Traditional and alternative designs for pulmonary arterial hypertension trials. Proc Am Thorac Soc 2008;5:610-6.
5. McLaughlin VV, Badesch DB, Delcroix M, Fleming TR, Gaine SP, Galie N, et al. End points and clinical trial designs in pulmonary arterial hypertension. J Am Coll Cardiol 2009;54:S97-107.
6. Peacock AJ, Naeije R, Galie N, Rubin L. End-points and clinical trial designs in pulmonary arterial hypertension: Have we made progress? Eur Respir J 2009;34:231-42.
7. Halpern S, Doyle R, Kawut SM. The Ethics of Randomized Clinical Trials in Pulmonary Arterial Hypertension. Proc Am Thorac Soc 2008;5:631-5.
8. Kawut SM, Bagiella E, Lederer DJ, Shimbo D, Horn EM, Roberts KE, et al. Randomized clinical trial of aspirin and simvastatin for pulmonary arterial hypertension: ASA-STAT. Circulation 2011;123:2985-93.
9. Rubenfire M, McLaughlin VV, Allen RP, Elliott G, Park MH, Wade M, et al. Transition from IV epoprostenol to subcutaneous treprotinil in pulmonary arterial hypertension: A controlled trial. Chest 2007;132:757-63.
10. European Medicines Agency, 2009. Reflection Paper on Methodological Issues in Confirmatory Clinical Trials Planned With an Adaptive Design [Internet]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003616.pdf. [Last accessed 2012 Oct 01].
11. Food and Drug Administration, 2010. Adaptive Design Clinical Trials for Drugs and Biologics (Draft Guidance) [Internet]. Available from: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm201790.pdf. [Last accessed on 2012 Oct 01].
12. Miller R, Ewy W, Corrigan BW, Ouellet D, Hermann D, Kowalski KG, et al. How modeling and simulation have enhanced decision making in new drug development. J Pharmacokinet Pharmacodyn 2005;32:185-97.
13. Lalonde RL, Kowalski KG, Hutmacher MM, Ewy W, Nichols PA, Milligan PA, et al. Model-based drug development. Clin Pharmacol Ther 2007;82:21-32.
14. Pocock SJ. The combination of randomized and historical controls in clinical trials. J Chronic Dis 1976;29:175-88.
15. Spiegelhalter DJ, Abrams KR, Myles JP. Bayesian Approaches to Clinical Trials and Health-Care Evaluation. Chichester: John Wiley and Sons; 2003.
16. Neuenschwander B, Capkun-Niggli G, BransonB, Spiegelhalter DJ. Summarizing historical information on controls in clinical trials. Clin Trials 2010;7:5-18.
17. O’Quigley J, Pepe M, Fisher L. Continual reassessment method: A practical design for phase 1 clinical trials in cancer. Biometrics 1990;46:33-48.
18. Zohar S, Resche-Rigon M, Chevret S. Using the continual reassessment method to estimate the minimum effective dose in phase II dose-finding studies: A case study. Clin Trials 2011; published online August 8th 2011.
19. Babb J, Rogatko A, Zacks S. Cancer Phase I clinical trials: Efficient dose escalation with overdose control. Stat Med 2012;17:1103-20.
20. Neuenschwander B, Branson M, Gsponer G. Critical aspects of the Bayesian approach to Phase I cancer trials. Stat Med 2008;27:2420-39.
21. Berry DA, Müller P, Grieve AP, Smith MK, Parke T, Blazek R, et al. Adaptive Bayesian designs for dose-ranging trials. In: Carlin B, Carriquiry A, Gatsonis C, Gelman A, Kass RE, Verdinelli I, et al. editors. Case Studies in Bayesian Statistics V, New York: Springer Verlag; 2002. p. 99-181.
22. Grieve AP, Krams M. ASTIN: A Bayesian adaptive dose-response trial in acute stroke. Clin Trials 2005;2:340-51.
23. Berry SM, Spinelli W, Littman GS, Liang JZ, Fardipour P, Berry DA, et al. A Bayesian dose-finding trial with adaptive dose expansion to flexibly assess efficacy and safety of an investigational drug. Clin Trials 2010;7:121-35.
24. Sheiner LB. Learning versus confirming in clinical drug development. Clin Pharmacol Ther 1997;61:275-91.
25. Mehta CR, Pocock SJ. Adaptive increase in sample size when interim results are promising: A practical guide with examples. Stat Med 2012;30:3267-84.
26. Pocock SJ. Group sequential methods in the design and analysis of clinical trials Biometrika 1977;64:191-9.
27. O’Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1977;35:549-56.
28. Galbraith S, Marschner I. Interim analysis of continuous long-term endpoints in clinical trials with longitudinal outcomes. Stat Med 2003;22:1787-805.
29. Marschner IC, Becker SL. Interim monitoring of clinical trials based on long-term binary endpoints. Stat Med 2001;20:177-92.
30. Sooriyarachchi MR, Whitehead J, Whitehead A, Bolland K. The sequential analysis of binary responses: A score test for the case of three time points. Stat Med 2005;25:2196-214.
31. Hampson LV, Jennison C. Group sequential tests for delayed responses. J R Stat Soc Series B 2013;75:1-37.
32. Bauer P, Köhne K. Evaluation of experiments with adaptive interim analyses. Biometrics 1994;50:1029-41.
33. Gallo P, Anderson K, Chuang-Stein C, Dragalin V, Gaydos B, Krams M, et al. Viewpoints on the FDA draft adaptive designs guidance from the pharma working group. J Biopharm Stat 2010;20:1115-24.
34. Posch M, König F, Branson M, Brannath W, Dunger-Baldauf C, Bauer P. Testing and estimating in flexible group sequential designs with adaptive treatment selection. Stat Med 2005;24:3697-714.
35. Biomarkers Definitions Working Group. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther 2001;69:89-95.
36. Temple R. Special study designs: Early escape, enrichment, studies in non-responders. Commun Stat Theory Methods 1994;23:499-531.
37. Temple R. Enrichment designs: Efficiency in development of cancer treatments. J Clin Ocnol 2005;23:4838-9.
38. National Academy of Sciences. Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease. Washington DC: The National Academies Press; 2011.
39. Brannath W, Zube E, Branson M, Bretz F, Gallo P, Posch M, et al. Confirmatory adaptive designs with bayesian decision tools for a targeted therapy in oncology. Stat Med 2009;28:1445-63.
40. Wang SJ, Hung HMJ, O’Neill RT. Adaptive patient enrichment designs in therapeutic trials. Biom J 2009;51:358-74.
41. Wang SJ, O’Neill R, Hung J. Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharm Stat 2007; 6:227-44.
42. Barker AD, Sigman CC, Kelloff GJ, Hylton NM, Berry DA, Esserman LJ. I-SPY 2: An adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther 2009;86:97-100.
43. European Medicines Agency, 2012. Concept paper on extrapolation of efficacy and safety in medicine development. EMA/129698/2012 [Internet] (available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2012/06/WC500129285.pdf) [Last accessed on 2012 Oct 01].