A new toolkit for conducting clinical trials in rare disorders

Journal of Population Therapeutics and Clinical Pharmacology

Volumn 21, Issue 1, 2014, Pages

  Abrahamyan, Lusine ^a   Diamond, Ivan R ^a   Johnson, Sindhu R ^b   Feldman, Brian M ^c  

^a UNIVERSITY OF TORONTO   (Canada)

^b TORONTO WESTERN HOSPITAL   (Canada)

^c HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

Adaptive trial design; Bayesian analysis; Evidence based medicine; NNT; Observational studies; Rare disorders

Indexed keywords

BLOOD CLOTTING FACTOR 8;

ARTICLE; CROSSOVER PROCEDURE; DISEASE COURSE; FOLLOW UP; HUMAN; JUVENILE RHEUMATOID ARTHRITIS; LEUKEMIA; PREVALENCE; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; RISK FACTOR; SAFETY; SURVIVAL; T CELL LYMPHOMA; CLINICAL TRIAL (TOPIC); DRUG MANUFACTURE; EVIDENCE BASED MEDICINE; METHODOLOGY; PROCEDURES; RARE DISEASES;

CLINICAL TRIALS AS TOPIC; EVIDENCE-BASED MEDICINE; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES; RESEARCH DESIGN;

EID: 84895800854     PISSN: 17106222     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (65)

1. Institute of Medicine. Rare disease and orphan products: Accelerating research and development. Washington, D.C.: The National Academies Press. 2010;420.
2. Gustason W. Reasoning from evidence. Inductive logic. New York: Macmillan College Publishing Company. 1994;318.
3. Morabia APCA. Louis and the birth of clinical epidemiology. J Clin Epidemiol 1996;49(12):1327-33.
4. Wikipedia contributors. Evidence-based medicine: Wikipedia, The Free Encyclopedia.; 2011 [updated 27 April 2011 08:08 UTC; cited 2011 12 May]. Available from: http://en.wikipedia.org/w/index.php?title=Evidence-based_medicine&oldid=426171031.
5. Sackett DL, Rosenberg WM, Gray JA, Haynes RB, Richardson WS. Evidence based medicine: what it is and what it isn't. BMJ 1996;312(7023):71-2.
6. Savitz DA. Interpreting Epidemiologic Evidence. Strategies for study design and analysis. Oxford: Oxford University Press, Inc.; 2003.
7. Feinstein AR. Clinical Epidemiology. The Architecture of clinical research. Philadelphia: W. B. Saunders Company; 1985.
8. Bosco JL, Silliman RA, Thwin SS, et al. A most stubborn bias: no adjustment method fully resolves confounding by indication in observational studies. J Clin Epidemiol. 2010;63(1):64-74.
9. Kaplan W, Laing R. Priority medicines for Europe and the World. World Health Organization, Department of Essential Drugs and Medicines Policy. 2004. Available: http://mednet3.who.int/prioritymeds/repo?rt/final18october.pdf. Accessed 23 April, 2011.
10. Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 2005;98(11):829-36.
11. European Organisation for Rare Diseases (EURORDIS). Rare diseases: understanding this public health priority. 2005. Available: http://www.eurordis.org/IMG/pdf/princeps_document-EN.pdf. Accessed 23 April, 2011.
12. Gerss JWO, Kopcke W. Clinical trials and rare diseases. Adv Exp Med Biol 2010;686:173-90.
13. Behera M, Kumar A, Soares HP, Sokol L, Djulbegovic B. Evidence-based medicine for rare diseases: implications for data interpretation and clinical trial design. Cancer Control 2007;14(2):160-6.
14. Gallin JI, Alling DW, Malech HL, et al. Itraconazole to prevent fungal infections in chronic granulomatous disease. N Engl J Med 2003;348(24):2416-22.
15. Wilcken B. Rare diseases and the assessment of intervention: what sorts of clinical trials can we use? J Inherit Metab Dis 2001;24(2):291-8.
16. Abrahamyan L, Johnson SR, Beyene J, Shah PS, Feldman BM. Quality of randomized clinical trials in juvenile idiopathic arthritis. Rheumatology (Oxford) 2008;47(5):640-5.
17. Laupacis A, Sackett DL, Roberts RS. An assessment of clinically useful measures of the consequences of treatment. N Engl J Med 1988;318(26):1728-33.
18. Halpern SD, Karlawish JH, Casarett D, Berlin JA, Townsend RR, Asch DA. Hypertensive patients' willingness to participate in placebo-controlled trials: implications for recruitment efficiency. Am Heart J 2003;146(6):985-92.
19. Halpern SD, Ubel PA, Berlin JA, Townsend RR, Asch DA. Physicians' preferences for active-controlled versus placebo-controlled trials of new antihypertensive drugs. J Gen Intern Med 2002;17(9):689-95.
20. Makuch RW, Johnson MF. Dilemmas in the use of active control groups in clinical research. IRB 1989;11(1):1-5.
21. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357(6):535-44.
22. The World Medical Association Declaration of Helsinki. Ethical Principles for Medical Research Involving Human Subjects. 59th WMA General Assembly. Seoul: 2008 October. Report No.
23. Zong Z. Should post-trial provision of beneficial experimental interventions be mandatory in developing countries? J Med Ethics 2008;34(3):188-92.
24. Grady C. The challenge of assuring continued post-trial access to beneficial treatment. Yale J Health Policy Law Ethics 2005;5(1):425-35.
25. Senn S. The AB/BA crossover: past, present and future? Stat Methods Med Res 1994;3(4):303-24.
26. Elbourne DR, Altman DG, Higgins JP, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. Int J Epidemiol 2002;31(1):140-9.
27. Campbell H, Surry SA, Royle EM. A review of randomised controlled trials published in Archives of Disease in Childhood from 1982-96. Arch Dis Child 1998;79(2):192-7.
28. Reed JF, 3rd. Analysis of two-treatment, two-period crossover trials in emergency medicine. Ann Emerg Med 2004;43(1):54-8.
29. Straube S, Derry S, McQuay HJ, Moore RA. Enriched enrollment: definition and effects of enrichment and dose in trials of pregabalin and gabapentin in neuropathic pain. A systematic review. Br J Clin Pharmacol 2008;66(2):266-75.
30. Katz N. Enriched enrollment randomized withdrawal trial designs of analgesics: focus on methodology. Clin J Pain 2009;25(9):797-807.
31. Lovell DJ, Giannini EH, Reiff A, et al. Etanercept in children with polyarticular juvenile rheumatoid arthritis. Pediatric Rheumatology Collaborative Study Group. The New England journal of medicine 2000;342(11):763-9.
32. Feldman B, Wang E, Willan A, Szalai JP. The randomized placebo-phase design for clinical trials. J Clin Epidemiol 2001;54(6):550-7.
33. Abrahamyan L, Li CS, Beyene J, Willan AR, Feldman BM. Survival distributions impact the power of randomized placebo-phase design and parallel groups randomized clinical trials. J Clin Epidemiol 2010;64(3):286-92.
34. Shook S. Randomized placebo-phase design: evaluation, interim monitoring and analysis. Diss: University of Pittsburgh; 2010.
35. Abrahamyan L, Beyene J, Feng J, et al. Response times follow lognormal or gamma distribution in arthritis patients. J Clin Epidemiol 2010;63(12):1363-9.
36. Goodman SN. Toward evidence-based medical statistics. 1: The P value fallacy. Ann Intern Med 1999;130(12):995-1004.
37. Sterne JA, Davey Smith G. Sifting the evidence-what's wrong with significance tests? BMJ 2001;322(7280):226-31.
38. Lilford RJ, Thornton JG, Braunholtz D. Clinical trials and rare diseases: a way out of a conundrum. BMJ 1995;311(7020):1621-5.
39. Diamond GA, Kaul S. Prior convictions: Bayesian approaches to the analysis and interpretation of clinical mega trials. J Am Coll Cardiol 2004;43(11):1929-39.
40. Spiegelhalter DJ, Myles JP, Jones DR, Abrams KR. Bayesian methods in health technology assessment: a review. Health Technol Assess 2000;4(38):1-130.
41. Johnson SR, Tomlinson GA, Hawker GA, Granton JT, Feldman BM. Methods to elicit beliefs for Bayesian priors: a systematic review. J Clin Epidemiol 2010;63(4):355-69.
42. Howard G, Coffey CS, Cutter GR. Is Bayesian analysis ready for use in phase III randomized clinical trials? Beware the sound of the sirens. Stroke 2005;36(7):1622-3.
43. Goodman SN. Toward evidence-based medical statistics. 2: The Bayes factor. Ann Intern Med 1999;130(12):1005-13.
44. Johnson SR, Feldman BM, Pope JE, Tomlinson GA. Shifting our thinking about uncommon disease trials: the case of methotrexate in scleroderma. J Rheumatol 2009;36(2):323-9.
45. Menzies D, Popa J, Hanley JA, Rand T, Milton DK. Effect of ultraviolet germicidal lights installed in office ventilation systems on workers' health and wellbeing: double-blind multiple crossover trial. Lancet 2003;362(9398):1785-91.
46. Gilron I, Booher SL, Rowan JS, Max MB. Topiramate in trigeminal neuralgia: a randomized, placebo-controlled multiple crossover pilot study. Clin Neuropharmacol 2001;24(2):109-12.
47. Zucker D, Schmid C, McIntosh M, D'Agostino R, Selker H, Lau J. Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol 1997;50(4):401-10.
48. Guyatt G, Sackett D, Adachi J, et al. A clinician's guide for conducting randomized trials in individual patients. CMAJ 1988;139(6):497-503.
49. Guyatt GH, Heyting A, Jaeschke R, Keller J, Adachi JD, Roberts RS. N of 1 randomized trials for investigating new drugs. Control Clin Trials 1990;11(2):88-100.
50. Nathan PC, Tomlinson G, Dupuis LL, et al. A pilot study of ondansetron plus metopimazine vs. ondansetron monotherapy in children receiving highly emetogenic chemotherapy: a Bayesian randomized serial N-of-1 trials design. Support Care Cancer 2006;14(3):268-76.
51. Huber AM, Tomlinson GA, Koren G, Feldman BM. Amitriptyline to relieve pain in juvenile idiopathic arthritis: a pilot study using Bayesian meta-analysis of multiple N-of-1 clinical trials. J Rheumatol 2007;34(5):1125-32.
52. Sung L, Tomlinson GA, Greenberg ML, et al. Serial controlled N-of-1 trials of topical vitamin E as prophylaxis for chemotherapy-induced oral mucositis in paediatric patients. Eur J Cancer 2007;43(8):1269-75.
53. Zucker DR, Ruthazer R, Schmid CH. Individual (N-of-1) trials can be combined to give population comparative treatment effect estimates: methodologic considerations. J Clin Epidemiol 2010;63(12):1312-23.
54. Rubin DB. Estimating causal effects from large data sets using propensity scores. Ann Intern Med 1997;127(8 Pt 2):757-63.
55. Rosenbaum PR, Rubin DB. The central role of the propensity score in observational studies for causal effects. Biometrika 1983;70(1):41-55.
56. D'Agostino RB. Tutorial in biostatistics. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group. Statist Med 1998;17:2265-81.
57. Rosenbaum PR, Rubin DB. Reducing bias in observational studies using subclassification on the propensity score. JAMA 1984;79(387):516-24.
58. Austin PC. Balance diagnostics for comparing the distribution of baseline covariates between treatment groups in propensity-score matched samples. Statist Med 2009;28:3083-107.
59. Johnson SR, Granton JT, Tomlinson GA, et al. Warfarin in scleroderma-associated and idiopathic pulmonary arterial hypertension. A Bayesian approach to evaluating treatment in uncommon disease. Submitted 2011.
60. Condliffe R, Kiely DG, Peacock AJ, et al. Connective tissue disease-associated pulmonary arterial hypertension in the modern treatment era. Am J Respir Crit Care Med 2009;179(2):151-7.
61. Koh ET, Lee P, Gladman DD, Abu-Shakra M. Pulmonary hypertension in systemic sclerosis: an analysis of 17 patients. Br J Rheumatol 1996;35(10):989-93.
62. Seshadri R, Feldman BM, Ilowite N, Cawkwell G, Pachman LM. The role of aggressive corticosteroid therapy in patients with juvenile dermatomyositis: a propensity score analysis. Arthritis Rheum 2008;59(7):989-95.
63. Robins JM, Hernan MA, Brumback B. Marginal structural models and causal inference in epidemiology. Epidemiology 2000;11(5):550-60.
64. Stukel TA, Fisher ES, Wennberg DE, Alter DA, Gottlieb DJ, Vermeulen MJ. Analysis of observational studies in the presence of treatment selection bias: effects of invasive cardiac management on AMI survival using propensity score and instrumental variable methods. JAMA 2007;297(3):278-85.
65. Pullenayegum EM, Lam C, Manlhiot C, Feldman BM. Fitting marginal structural models: estimating covariate-treatment associations in the reweighted data set can guide model fitting. J Clin Epidemiol 2008;61(9):875-81