Clinical trials for therapeutic assessment of antiepileptic drugs in the 21st century: Obstacles and solutions

The Lancet Neurology

Volumn 11, Issue 9, 2012, Pages 827-834

  Friedman, Daniel ^a   French, Jacqueline A ^a  

^a NEW YORK UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTICONVULSIVE AGENT; BRIVARACETAM; CARBAMAZEPINE; CARISBAMATE; ETIRACETAM; FELBAMATE; LAMOTRIGINE; OXCARBAZEPINE; PERAMPANEL; PHENYTOIN; PLACEBO; PREGABALIN; PRIMIDONE; TOPIRAMATE; VALPROIC ACID;

CLINICAL ASSESSMENT; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; EPILEPSY; HUMAN; MONOTHERAPY; NOTE; OUTCOME ASSESSMENT; PATIENT MONITORING; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); TREATMENT DURATION; TREATMENT RESPONSE;

ANTICONVULSANTS; CLINICAL TRIALS AS TOPIC; EPILEPSY; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE);

EID: 84865151333     PISSN: 14744422     EISSN: 14744465     Source Type: Journal    
DOI: 10.1016/S1474-4422(12)70177-1     Document Type: Note

References (54)

1. Loscher W, Schmidt D Modern antiepileptic drug development has failed to deliver: ways out of the current dilemma. Epilepsia 2011, 52:657-678.
2. Brodie MJ, Barry SJ, Bamagous GA, Norrie JD, Kwan P Patterns of treatment response in newly diagnosed epilepsy. Neurology 2012, 78:1548-1554.
3. Gazzola DM, Balcer LJ, French JA Seizure-free outcome in randomized add-on trials of the new antiepileptic drugs. Epilepsia 2007, 48:1303-1307.
4. Jacoby A, Snape D, Baker GA Determinants of quality of life in people with epilepsy. Neurol Clin 2009, 27:843-863.
5. Birbeck GL, Hays RD, Cui X, Vickrey BG Seizure reduction and quality of life improvements in people with epilepsy. Epilepsia 2002, 43:535-538.
6. Jacoby A, Baker GA, Steen N, Potts P, Chadwick DW The clinical course of epilepsy and its psychosocial correlates: findings from a UK community study. Epilepsia 1996, 37:148-161.
7. Hermann BP, Seidenberg M, Dow C, et al. Cognitive prognosis in chronic temporal lobe epilepsy. Ann Neurol 2006, 60:80-87.
8. Devinsky O Sudden, unexpected death in epilepsy. N Engl J Med 2011, 365:1801-1811.
9. Bialer M, White HS Key factors in the discovery and development of new antiepileptic drugs. Nat Rev Drug Discov 2010, 9:68-82.
10. Mohanraj R, Brodie MJ Measuring the efficacy of antiepileptic drugs. Seizure 2003, 12:413-443.
11. Dickson M, Gagnon JP Key factors in the rising cost of new drug discovery and development. Nat Rev Drug Discov 2004, 3:417-429.
12. Faught E, Wilder BJ, Ramsay RE, et al. Topiramate placebo-controlled dose-ranging trial in refractory partial epilepsy using 200-, 400-, and 600-mg daily dosages. Neurology 1996, 46:1684-1690.
13. Faught E, Holmes GL, Rosenfeld WE, et al. Randomized, controlled, dose-ranging trial of carisbamate for partial-onset seizures. Neurology 2008, 71:1586-1593.
14. Glickman SW, McHutchison JG, Peterson ED, et al. Ethical and scientific implications of the globalization of clinical research. N Engl J Med 2009, 360:816-823.
15. Guekht AB, Korczyn AD, Bondareva IB, Gusev EI Placebo responses in randomized trials of antiepileptic drugs. Epilepsy Behav 2010, 17:64-69.
16. Rheims S, Perucca E, Cucherat M, Ryvlin P Factors determining response to antiepileptic drugs in randomized controlled trials: a systematic review and meta-analysis. Epilepsia 2011, 52:219-233.
17. Sperling MR, Greenspan A, Cramer JA, et al. Carisbamate as adjunctive treatment of partial onset seizures in adults in two randomized, placebo-controlled trials. Epilepsia 2010, 51:333-343.
18. Biton V, Werhahn K, Johnson M, et al. Brivaracetam as adjunctive treatment of refractory partial-onset seizures in adults: results from two randomized, double-blind, placebo-controlled trials (accessed July 12, 2012). http://www.aesnet.org/go/publications/aes-abstracts/abstract-search?mode=display&sy=2009&sb=all&startrow=871&id=9599.
19. Bridge JA, Birmaher B, Iyengar S, Barbe RP, Brent DA Placebo response in randomized controlled trials of antidepressants for pediatric major depressive disorder. Am J Psychiatry 2009, 166:42-49.
20. Niklson I, Edrich P, Verdru P Identifying baseline characteristics of placebo responders versus nonresponders in randomized double-blind trials of refractory partial-onset seizures. Epileptic Disord 2006, 8:37-44.
21. Katz R Why are epilepsy trials failing? Interview with Russell Katz. Clin Invest 2011, 1:1487-1489.
22. Chung S, Sperling MR, Biton V, et al. Lacosamide as adjunctive therapy for partial-onset seizures: a randomized controlled trial. Epilepsia 2010, 51:958-967.
23. Benbadis S, Elger C, Hebert D, Isojarvi J Efficacy of adjunctive lacosamide in patients with partial-onset seizures and prior surgical intervention for epilepsy (accessed July 12, 2012). http://www.aesnet.org/go/publications/aes-abstracts/abstract-search/mode/display/st/lacosamide/sy/all/sb/All/page/7/id/9942.
24. French JA, Ben-Menachem E, Brodie MJ, et al. Global phase 3 trial of perampanel, a selective, noncompetitive AMPA receptor antagonist, as adjunctive therapy in patients with refractory partial-onset seizures. Presented at the 2011 American Academy of Neurology Annual Meeting; Honolulu, Hawaii; April 9-16, 2011. Abstr LBS.002.
25. Walsh BT, Seidman SN, Sysko R, Gould M Placebo response in studies of major depression: variable, substantial, and growing. JAMA 2002, 287:1840-1847.
26. Callaghan BC, Anand K, Hesdorffer D, Hauser WA, French JA Likelihood of seizure remission in an adult population with refractory epilepsy. Ann Neurol 2007, 62:382-389.
27. Choi H, Heiman GA, Munger Clary H, Etienne M, Resor SR, Hauser WA Seizure remission in adults with long-standing intractable epilepsy: an extended follow-up. Epilepsy Res 2011, 93:115-119.
28. Hesdorffer DC, Tomson T, Benn E, et al. Combined analysis of risk factors for SUDEP. Epilepsia 2011, 52:1150-1159.
29. Ryvlin P, Cucherat M, Rheims S Risk of sudden unexpected death in epilepsy in patients given adjunctive antiepileptic treatment for refractory seizures: a meta-analysis of placebo-controlled randomised trials. Lancet Neurol 2011, 10:961-968.
30. Janszky J, Kovacs N, Gyimesi C, Fogarasi A, Doczi T, Wiebe S Epilepsy surgery, antiepileptic drug trials, and the role of evidence. Epilepsia 2010, 51:1004-1009.
31. Bourgeois B, Leppik IE, Sackellares JC, et al. Felbamate: a double-blind controlled trial in patients undergoing presurgical evaluation of partial seizures. Neurology 1993, 43:693-696.
32. French JA, Wang S, Warnock B, Temkin N Historical control monotherapy design in the treatment of epilepsy. Epilepsia 2010, 51:1936-1943.
33. Haut SR, Lipton RB, LeValley AJ, Hall CB, Shinnar S Identifying seizure clusters in patients with epilepsy. Neurology 2005, 65:1313-1315.
34. Baulac M, Leon T, O'Brien TJ, Whalen E, Barrett J A comparison of pregabalin, lamotrigine, and placebo as adjunctive therapy in patients with refractory partial-onset seizures. Epilepsy Res 2010, 91:10-19.
35. St Louis EK, Rosenfeld WE, Bramley T Antiepileptic drug monotherapy: the initial approach in epilepsy management. Curr Neuropharmacol 2009, 7:77-82.
36. Brodie MJ, Whitehead J Active control comparisons: the ideal trial design. Epilepsy Res 2006, 68:69-73.
37. Perucca E Innovative monotherapy trial designs for the assessment of antiepileptic drugs: a critical appraisal. Eur J Clin Pharmacol 1998, 54:1-5.
38. Mattson RH, Cramer JA, Collins JF, et al. Comparison of carbamazepine, phenobarbital, phenytoin, and primidone in partial and secondarily generalized tonic-clonic seizures. N Engl J Med 1985, 313:145-151.
39. Beydoun A, Milling CJ Active-control comparative equivalency monotherapy trials in epilepsy: are they scientifically valid?. Epilepsy Behav 2001, 2:187-192.
40. Kwan P, Brodie MJ, Kalviainen R, Yurkewicz L, Weaver J, Knapp LE Efficacy and safety of pregabalin versus lamotrigine in patients with newly diagnosed partial seizures: a phase 3, double-blind, randomised, parallel-group trial. Lancet Neurol 2011, 10:881-890.
41. Enck P, Klosterhalfen S, Weimer K, Horing B, Zipfel S The placebo response in clinical trials: more questions than answers. Philos Trans R Soc Lond B Biol Sci 2011, 366:1889-1895.
42. Pledger GW, Kramer LD Clinical trials of investigational antiepileptic drugs: monotherapy designs. Epilepsia 1991, 32:716-721.
43. Bien CG, Elger CE Monotherapy trials in antiepileptic drugs: are modified " presurgical studies" a way out of the dilemma?. Epilepsy Res 2001, 44:1-5.
44. Sachdeo RC, Reife RA, Lim P, Pledger G Topiramate monotherapy for partial onset seizures. Epilepsia 1997, 38:294-300.
45. Sachdeo R, Kramer LD, Rosenberg A, Sachdeo S Felbamate monotherapy: controlled trial in patients with partial onset seizures. Ann Neurol 1992, 32:386-392.
46. Gilliam F, Vazquez B, Sackellares JC, et al. An active-control trial of lamotrigine monotherapy for partial seizures. Neurology 1998, 51:1018-1025.
47. Katz R FDA update. Epilepsy Res 2006, 68:85-94.
48. French JA, Temkin NR, Shneker BF, Hammer AE, Caldwell PT, Messenheimer JA Lamotrigine XR conversion to monotherapy: first study using a historical control group. Neurotherapeutics 2012, 9:176-184.
49. Perucca E Designing clinical trials to assess antiepileptic drugs as monotherapy: difficulties and solutions. CNS Drugs 2008, 22:917-938.
50. Perucca E When clinical trials make history: demonstrating efficacy of new antiepileptic drugs as monotherapy. Epilepsia 2010, 51:1933-1935.
51. Kim LG, Johnson TL, Marson AG, Chadwick DW Prediction of risk of seizure recurrence after a single seizure and early epilepsy: further results from the MESS trial. Lancet Neurol 2006, 5:317-322.
52. Banerjee PN, Filippi D, Allen Hauser W The descriptive epidemiology of epilepsy-a review. Epilepsy Res 2009, 85:31-45.
53. DrugsFDA: FDA approved drug products-Requip XL US Food and Drug Administration, (accessed June 3, 2012). http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Set_Current_Drug&ApplNo=022008&DrugName=REQUIP%20XL&ActiveIngred=ROPINIROLE%20HYDROCHLORIDE&SponsorApplicant=SMITHKLINE%20BEECHAM&ProductMktStatus=1&goto=Search.DrugDetails.
54. Baulac M Historical data in the design and interpretation of trials with newly diagnosed patients. Epilepsy Res 2006, 68:77-81.