Drug safety assessment in clinical trials: methodological challenges and opportunities

Trials

Volumn 13, Issue , 2012, Pages

  Singh, Sonal ^a   Loke, Yoon K ^b  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF EAST ANGLIA   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

2,4 THIAZOLIDINEDIONE DERIVATIVE; PIOGLITAZONE; ROSIGLITAZONE;

ANALYTICAL ERROR; DRUG EFFICACY; DRUG SAFETY; FOOD AND DRUG ADMINISTRATION; GOLD STANDARD; HUMAN; METHODOLOGY; NONHUMAN; PNEUMONIA; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW;

ADVERSE DRUG REACTION REPORTING SYSTEMS; BIAS (EPIDEMIOLOGY); DATA INTERPRETATION, STATISTICAL; DRUG THERAPY; ENDPOINT DETERMINATION; EVIDENCE-BASED MEDICINE; HUMANS; META-ANALYSIS AS TOPIC; PATIENT SAFETY; PATIENT SELECTION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; REPRODUCIBILITY OF RESULTS; RESEARCH DESIGN; RISK ASSESSMENT; RISK FACTORS; SAMPLE SIZE; TREATMENT OUTCOME;

EID: 84865056356     PISSN: None     EISSN: 17456215     Source Type: Journal    
DOI: 10.1186/1745-6215-13-138     Document Type: Review

References (51)

1. Friedman LM, Furberg CD, DeMets DL. Fundamentals of Clinical Trials September 21, 2010, Springer, New York, 4.
2. Ferner RE. Newly licensed drugs. BMJ 1996, 313:1157-1158. 10.1136/bmj.313.7066.1157, 2352471, 8963199.
3. Singh S, Loke YK, Spangler JG, Furberg CD. Risk of serious adverse cardiovascular events with Varenicline- A Systematic Review and Meta-Analysis of Randomized Controlled Trials. CMAJ 2011, 183:1359-1366. 10.1503/cmaj.110218, 3168618, 21727225.
4. Rigotti NA, Pipe AL, Benowitz NL, Arteaga C, Garza D, Tonstad S. Efficacy and safety of varenicline for smoking cessation in patients with cardiovascular disease: a randomized trial. Circulation 2010, 121:221-229. 10.1161/CIRCULATIONAHA.109.869008, 20048210.
5. Harrison-Woolrych M, Maggo S, Tan M, Savage R, Ashton J. Cardiovascular events in patients taking varenicline: a case series from intensive postmarketing surveillance in New Zealand. Drug Saf 2012, 35:33-43. 10.2165/11597690-000000000-00000, 22149418.
6. Singh S, Loke YK, Enright P, Furberg CD. Mortality associated with tiotropium Respimat® in patients with chronic obstructive pulmonary disease - A systematic review and meta-analysis of randomized controlled trials. BMJ 2011, 342:d3215. 10.1136/bmj.d3215, 3114950, 21672999.
7. Singh S, Loke YK, Furberg CD. Inhaled anticholinergics and the risk of major adverse cardiovascular events in patients with chronic obstructive pulmonary disease: A systematic review and meta-analysis. JAMA 2008, 300:1439-1450. 10.1001/jama.300.12.1439, 18812535.
8. Nissen SE, Wolski K. Effect of rosiglitazone on the risk of myocardial infarction and death from cardiovascular causes. N Engl J Med. 2007, 356:2457-2471. 10.1056/NEJMoa072761, 17517853.
9. Singh S, Loke YK, Furberg CD. Long-term risk of cardiovascular events with rosiglitazone - A systematic review and meta-analysis. JAMA 2007, 298:1189-1195. 10.1001/jama.298.10.1189, 17848653.
10. Loke YK, Kwok CS, Singh S. Comparative cardiovascular effects of thiazolidinediones: A systematic review and meta-analysis of observational studies. BMJ 2011, 342:d1309. 10.1136/bmj.d1309, 3230110, 21415101.
11. Loke YK, Singh S, Furberg CD. Long-term use of thiazolidinediones and fractures in type 2 diabetes: Systematic review. CMAJ 2009, 180:32-39. 10.1503/cmaj.080486, 2612065, 19073651.
12. Hammad TA, Pinheiro SP, Neyarapally GA. Secondary use of randomized controlled trials to evaluate drug safety: a review of methodological considerations. Clin Trials 2011, 8:559-570. 10.1177/1740774511419165, 21878445.
13. Scudellari M. Pooled studies can raise misleading alarm bells, regulators say. Nat Med 2010, 16:1051. 10.1038/nm1010-1051, 20930720.
14. Lane PW. Meta-analysis of incidence of rare events. Stat Methods Med Res 2012 Jan 4, Epub ahead of print.
15. Bradburn MJ, Deeks JJ, Berlin JA, et al. Much ado about nothing: a comparison of the performance of meta-analytical methods with rare events. Stat Med 2007, 26:53-77. 10.1002/sim.2528, 16596572.
16. Sweeting MJ, Sutton AJ, Lambert PC. What to add to nothing? Use and avoidance of continuity corrections in meta-analysis of sparse data. Stat Med 2004, 23:1351-1375. 10.1002/sim.1761, 15116347.
17. Food and Drug Administration Amendments Act of 2007. Sep 27 2007, http://www.gpo.gov/fdsys/pkg/PLAW-110publ85/pdf/PLAW-110publ85.pdf Accessed August 17, 2012.
18. Moore TJ, Singh S, Furberg CD. The FDA and new safety warnings. Arch Intern Med 2012, 172:78-80. 10.1001/archinternmed.2011.618, 22232155.
19. Moore TJ, Furberg CD, Glenmullen J, Maltsberger JT, Singh S. Suicidal behavior and depression in smoking cessation treatments. PLoS One 2011, 6:e27016. 10.1371/journal.pone.0027016, 3206890, 22073240.
20. Stone M, Laughren T, Jones ML, Levenson M, Holland PC, Hughes A, Hammad TA, Temple R, Rochester G. Risk of suicidality in clinical trials of antidepressants in adults: analysis of proprietary data submitted to US Food and Drug Administration. BMJ 2009, 339:b2880. 10.1136/bmj.b2880, 2725270, 19671933.
21. Institute of Medicine Report Evaluation of biomarkers and surrogate endpoints in chronic disease. May 12, 2010, Washington:DCNational Academies Press, ISBN-10: 0-309-15129-5. ISBN 10: 0-309-15129-5, Institute of Medicine Report.
22. US Department of Health and Human Services, US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research Guidance for Industry. Diabetes Mellitus. Evaluating Cardiovascular Risk in New Antidiabetic therapies to treat Type 2 Diabetes December 2008, http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071627.pdf Accessed January 30, 2012, US Department of Health and Human Services, US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research.
23. UK Prospective Diabetes Study (UKPDS) Group Effect of intensive blood-glucose control with metformin on complications in overweight patients with type 2 diabetes (UKPDS 34). Lancet 1998, 352:854-865. UK Prospective Diabetes Study (UKPDS) Group.
24. Duckworth W, Abraira C, Moritz T, et al. Glucose control and vascular complications in veterans with type 2 diabetes. N Engl J Med 2009, 360:129-139. 10.1056/NEJMoa0808431, 19092145.
25. The Action to Control Cardiovascular Risk in Diabetes Study Group Effects of intensive glucose lowering in type 2 diabetes. N Engl J Med 2008, 358:2545-2559. The Action to Control Cardiovascular Risk in Diabetes Study Group.
26. The ADVANCE Collaborative Group Intensive blood glucose control and vascular outcomes in patients with type 2 diabetes. N Engl J Med 2008, 358:2560-2572. The ADVANCE Collaborative Group.
27. Singh S, Amin AV, Loke YK. Long-term use of inhaled corticosteroids and the risk of pneumonia in chronic obstructive pulmonary disease: a meta-analysis. Arch Intern Med 2009, 169(3):219-229. 10.1001/archinternmed.2008.550, 19204211.
28. Singh S, Loke YK. Risk of pneumonia associated with long-term use of inhaled corticosteroids in chronic obstructive pulmonary disease: a critical review and update. Curr Opin Pulm Med 2010, 16(2):118-122. 10.1097/MCP.0b013e328334c085, 2908947, 19926996.
29. Singh S, Loke YK, Furberg CD. Long-term use of thiazolidinediones and the associated risk of pneumonia or lower respiratory tract infection: systematic review and meta-analysis. Thorax 2011, 66:383-388. 10.1136/thx.2010.152777, 21325145.
30. Kwok CS, Loke YK. Effects of proton pump inhibitors on platelet function in patients receiving clopidogrel: a systematic review. Drug Saf 2012, 35:127-139. 10.2165/11594900-000000000-00000, 22204719.
31. Singh S: Loke YK, Enright P: Furberg CD. Thorax 2012 Jul 4, Pro-arrhythmic and pro-ischaemic effects of inhaled anticholinergic medications, [Epub ahead of print], Singh S: Loke YK, Enright P: Furberg CD. Thorax.
32. Tashkin DP, Celli B, Senn S, Burkhart D, Kesten S, Menjoge S, Decramer M. UPLIFT Study Investigators. A 4-year trial of tiotropium in chronic obstructive pulmonary disease. N Engl J Med 2008, 359:1543-1554. 10.1056/NEJMoa0805800, 18836213.
33. Derry S, Loke YK. Risk of gastrointestinal hemorrhage with long term use of aspirin: meta-analysis. BMJ 2000, 321:1183-1187. 10.1136/bmj.321.7270.1183, 27521, 11073508.
34. Loke YK, Derry S, Aronson JK. A comparison of three different sources of data in assessing the frequencies of adverse reactions to amiodarone. Br J Clin Pharmacol 2004, 57:616-621. 10.1111/j.0306-5251.2003.02055.x, 1884501, 15089815.
35. Loke YK, Golder S, Vanderbroucke JP. Comprehensive evaluation of adverse effects of drugs: importance of appropriate study selection and data source. Therapeutic Advances in Drug Safety. 2011, 2:59-68.
36. Golder S, Loke YK, Bland M. Meta-analyses of adverse effects data derived from randomized controlled trials as compared to observational studies: methodological overview. PLoS Med 2011, 8:e1001026. 10.1371/journal.pmed.1001026, 3086872, 21559325.
37. Woodcock J, Behrman RE, Dal Pan GJ. Role of postmarketing surveillance in contemporary medicine. Annu Rev Med 2011, 62:1-10. 10.1146/annurev-med-060309-164311, 20809798.
38. Home PD, Pocock SJ, Beck-Nielsen H, et al. RECORD Study Team. Rosiglitazone evaluated for cardiovascular outcomes in oral agent combination therapy for type 2 diabetes (RECORD): a multicentre, randomised, open-label trial. Lancet 2009, 373:2125-2135. 10.1016/S0140-6736(09)60953-3, 19501900.
39. Treadwell JR, Uhl S, Tipton K, Shamliyan T, Viswanathan M, Berkman ND, Sun X, Coleman CI, Elshaug AG, Singh S, Wang SY, Ramakrishnan R. Assessing equivalence and noninferiority. J Clin Epidemiol 2012 Jun 23, Epub ahead of print.
40. Dormandy JA, Charbonnel B, Eckland DJ, Erdmann E, Massi-Benedetti M, Moules IK, et al. PROactive investigators. Secondary prevention of macrovascular events in patients with type 2 diabetes in the PROactive Study (PROspective pioglitAzone Clinical Trial In macroVascular Events): a randomized controlled trial. Lancet 2005, 366:1279-1289. 10.1016/S0140-6736(05)67528-9, 16214598.
41. Hillaire-Buys D, Faillie JL, Montastruc JL. Pioglitazone and bladder cancer. Lancet 2011, 378:1543-1544.
42. Granger CB, Vogel V, Cummings SR, Held P, Fiedorek F, Lawrence M, et al. Do we need to adjudicate major clinical events?. Clin Trials 2008, 5:56-60. 10.1177/1740774507087972, 18283081.
43. Vedula SS, Bero L, Scherer RW, Dickersin K. Outcome reporting in industry-sponsored trials of gabapentin for off-label use. N Engl J Med 2009, 361:1963-1971. 10.1056/NEJMsa0906126, 19907043.
44. Wang AT, McCoy CP, Murad MH, Montori VM. Association between industry affiliation and position on cardiovascular risk with rosiglitazone: cross sectional systematic review. BMJ 2010, 340:c1344. 10.1136/bmj.c1344, 2841746, 20299696.
45. Mills EJ, Wu P, Chong G, Ghement I, Singh S, et al. Efficacy and safety of statin treatment for cardiovascular disease: a network meta-analysis of 170 255 patients from 76 randomized trials. Q J Med 2011, 104:109-124.
46. Thorlund K. What is the power to demonstrate that varenicline use for tobacco cessation is not associated with an increase in cardiovascular serious adverse events? http://www.bmj.com/content/344/bmj.e2856/rr/587196 Accessed July 17, 2012.
47. Sherman RB, Woodcock J, Norden J, Grandinetti C, Temple RJ. New FDA regulation to improve safety reporting in clinical trials. N Engl J Med 2011, 365:3-5. 10.1056/NEJMp1103464, 21651388.
48. Zarin DA, Tse T, Williams RJ, Califf RM, Ide NC. The ClinicalTrials.gov results database-update and key issues. N Engl J Med 2011, 364:852-860. 10.1056/NEJMsa1012065, 3066456, 21366476.
49. Abernethy DR, Woodcock J, Lesko LJ. Clin Pharmacol Ther. 2011, 89:793-797. 10.1038/clpt.2011.55, 21490594.
50. Li T, Puhan MA, Vedula SS, Singh S, et al. Network meta-analysis-highly attractive but more methodological research is needed. BMC Medicine 2011, 9:79. 10.1186/1741-7015-9-79, 3159133, 21707969.
51. European Medicines Agency Benefit Risk Methodology http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/document_listing/document_listing_000314.jsp&mid=WC0b01ac0580223ed6 Accessed July 17, 2012, European Medicines Agency.