Key design considerations on comparative clinical efficacy studies for biosimilars: Adalimumab as an example

RMD Open

Volumn 2, Issue 1, 2016, Pages

  Lai, Zhihong ^a   La Noce, Anna ^b  

^a GLAXOSMITHKLINE   (United States)

^b Scientific Affairs   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84988431098     PISSN: None     EISSN: 20565933     Source Type: Journal    
DOI: 10.1136/rmdopen-2015-000154     Document Type: Review

References (71)

1. Food and Drug Administration. Guidance for industry. Scientific considerations in demonstrating biosimilarity to a reference product. April 2015. http://www.fda.gov/downloads/drugs/guidancecompliance regulatoryinformation/guidances/ucm291128.pdf (accessed 26 Mar. 2015).
2. European Medicines Agency. Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues on similar biological medicinal products. December 2014. http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2015/01/WC500180219.pdf (accessed 26 Mar. 2015).
3. Castañeda-Hernández G, Szekanecz Z, Mysler E, et al. Biopharmaceuticals for rheumatic diseases in Latin America, Europe, Russia, and India: innovators, biosimilars, and intended copies. Joint Bone Spine 2014;81:471-7.
4. Humira drug label approved by the FDA. January 2008. http://www.accessdata.fda.gov/drugsatfda-docs/label/2008/125057s0110lbl.pdf (accessed 26 Mar. 2015).
5. Humira drug label (summary of product characteristics) approved by the EMA. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Product-Information/human/000481/WC500050870.pdf (accessed 14 May 2015).
6. Zydus launches world's first biosimilar of Adalimumab. 9 December 2014. http://zyduscadila.com/wp-content/uploads/2015/05/PressNote09-12-14.pdf (accessed 29 Oct. 2015).
7. Adalimumab similar biologic launched in India. 30 January 2015. http://gabionline.net/Biosimilars/News/Adalimumab-similar-biologiclaunched-in-India (accessed 29 Oct. 2015).
8. European Medicines Agency. Remsima Assessment Report. EMA/CHMP/589317/2013. 2013. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Public-assessment-report/human/002576/WC500151486.pdf (accessed 26 Mar. 2015).
9. Lee H. Is extrapolation of the safety and efficacy data in one indication to another appropriate for biosimilars? AAPS J 2014;16:22-6.
10. Weinblatt ME, Keystone EC, Furst DE, et al. Adalimumab, a fully human anti-tumor necrosis factor alpha monoclonal antibody, for the treatment of rheumatoid arthritis in patients taking concomitant methotrexate: the ARMADA trial. Arthritis Rheum 2003;48:35-45.
11. van de Putte LB, Atkins C, Malaise M, et al. Efficacy and safety of adalimumab as monotherapy in patients with rheumatoid arthritis for whom previous disease modifying antirheumatic drug treatment has failed. Ann Rheum Dis 2004;63:508-16.
12. Keystone EC, Kavanaugh AF, Sharp JT, et al. Radiographic, clinical, and functional outcomes of treatment with adalimumab (a human anti-tumor necrosis factor monoclonal antibody) in patients with active rheumatoid arthritis receiving concomitant methotrexate therapy: a randomized, placebo-controlled, 52-week trial. Arthritis Rheum 2004;50:1400-11.
13. Furst DE, Schiff MH, Fleischmann RM, et al. Adalimumab, a fully human anti tumor necrosis factor-alpha monoclonal antibody, and concomitant standard antirheumatic therapy for the treatment of rheumatoid arthritis: results of STAR (Safety Trial of Adalimumab in Rheumatoid Arthritis). J Rheumatol 2003;30:2563-71.
14. Mease PJ, Gladman DD, Ritchlin CT, et al, Adalimumab Effectiveness in Psoriatic Arthritis Trial Study Group. Adalimumab for the treatment of patients with moderately to severely active psoriatic arthritis: results of a double-blind, randomized, placebo-controlled trial. Arthritis Rheum 2005;52:3279-89.
15. Genovese MC, Mease PJ, Thomson GT, et al, M02-570 Study Group. Safety and efficacy of adalimumab in treatment of patients with psoriatic arthritis who had failed disease modifying antirheumatic drug therapy. J Rheumatol 2007;34:1040-50.
16. van der Heijde D, Kivitz A, Schiff MH, et al, ATLAS Study Group. Efficacy and safety of adalimumab in patients with ankylosing spondylitis: results of a multicenter, randomized, double-blind, placebo-controlled trial. Arthritis Rheum 2006;54:2136-46.
17. Hanauer SB, Sandborn WJ, Rutgeerts P, et al. Human anti-tumor necrosis factor monoclonal antibody (adalimumab) in Crohn's disease: the CLASSIC-I trial. Gastroenterology 2006;130:323-33; quiz 591.
18. Sandborn WJ, Rutgeerts P, Enns R, et al. Adalimumab induction therapy for Crohn disease previously treated with infliximab. Ann Intern Med 2007;146:829-38.
19. Colombel JF, Sandborn WJ, Rutgeerts P, et al. Adalimumab for maintenance of clinical response and remission in patients with Crohn's disease: the CHARM trial. Gastroenterology 2007;132:52-65.
20. Reinisch W, Sandborn WJ, Hommes DW, et al. Adalimumab for induction of clinical remission in moderately to severely active ulcerative colitis: results of a randomised controlled trial. Gut 2011;60:780-7.
21. Sandborn WJ, van Assche G, Reinisch W, et al. Adalimumab induces and maintains clinical remission in patients with moderate-to-severe ulcerative colitis. Gastroenterology 2012;142:257-65.e1-3.
22. Menter A, Tyring SK, Gordon K, et al. Adalimumab therapy for moderate to severe psoriasis: a randomized, controlled phase III trial. J Am Acad Dermatol 2008;58:106-15.
23. Saurat JH, Stingl G, Dubertret L, et al. CHAMPION Study Investigators. Efficacy and safety results from the randomized controlled comparative study of adalimumab vs. methotrexate vs. placebo in patients with psoriasis (CHAMPION). Br J Dermatol 2008;158:558-66.
24. Bartelds GM, Krieckaert CL, Nurmohamed MT, et al. Development of antidrug antibodies against adalimumab and association with disease activity and treatment failure during long-term follow-up. JAMA 2011;305:1460-8.
25. Krieckaert CL, Nurmohamed MT, Wolbink GJ. Methotrexate reduces immunogenicity in adalimumab treated rheumatoid arthritis patients in a dose dependent manner. Ann Rheum Dis 2012;71:1914-15.
26. ClinicalTrials.gov. Comparison of CHS-0214 to Enbrel (Etanercept) in patients with chronic plaque psoriasis (PsO). 26 June 2015. https://www.clinicaltrials.gov/ct2/show/NCT02134210 (accessed 10 Apr. 2015).
27. ClinicalTrials. Gov. Study to demonstrate equivalent efficacy and to compare safety of biosimilar Etanercept (GP2015) and Enbrel (EGALITY). 24 June 2015. https://www.clinicaltrials.gov/ct2/show/NCT01891864 (accessed 10 Apr. 2015).
28. Health Canada, Office of Regulatory Affairs, Biologics and Genetic Therapies Directorate. Inflectra. 16 June 2015. http://www.hc-sc.gc. ca/dhp-mps/prodpharma/sbd-smd/drug-med/sbd-smd-2014-inflectra-159493-eng.php2 (accessed 10 Jul. 2015).
29. Australian approval for infliximab biosimilar. 21 August 2015. http://www.gabionline.net/Biosimilars/General/Australian-approval-forinfliximab-biosimilar (accessed 30 Oct. 2015).
30. Danese S, Gomollon F. ECCO position statement: the use of biosimilar medicines in the treatment of inflammatory bowel disease (IBD). J Crohns Colitis 2013;7:586-9.
31. Rompas S, Goss T, Amanuel S, et al. Demonstrating value for biosimilars: a conceptual framework. Am Health Drug Benefits 2015;8:129-39.
32. Bissonnette R, Bolduc C, Poulin Y, et al. Efficacy and safety of adalimumab in patients with plaque psoriasis who have shown an unsatisfactory response to etanercept. J Am Acad Dermatol 2010;63:228-34.
33. Strober BE, Poulin Y, Kerdel FA, et al. Switching to adalimumab for psoriasis patients with a suboptimal response to etanercept, methotrexate, or phototherapy: efficacy and safety results from an open-label study. J Am Acad Dermatol 2011;64:671-81.
34. Bombardieri S, Ruiz AA, Fardellone P, et al. Effectiveness of adalimumab for rheumatoid arthritis in patients with a history of TNF-antagonist therapy in clinical practice. Rheumatology (Oxford) 2007;46:1191-9.
35. Nikas SN, Voulgari PV, Alamanos Y, et al. Efficacy and safety of switching from infliximab to adalimumab: a comparative controlled study. Ann Rheum Dis 2006;65:257-60.
36. Remy A, Avouac J, Gossec L, et al. Clinical relevance of switching to a second tumour necrosis factor-alpha inhibitor after discontinuation of a first tumour necrosis factor-alpha inhibitor in rheumatoid arthritis: a systematic literature review and meta-analysis. Clin Exp Rheumatol 2011;29:96-103.
37. Feuchtenberger M, Kleinert S, Scharbatke EC, et al. The impact of prior biologic therapy on adalimumab response in patients with rheumatoid arthritis. Clin Exp Rheumatol 2015;33:321-9.
38. Smolen JS, Kay J, Doyle MK, et al, GO-AFTER study investigators. Golimumab in patients with active rheumatoid arthritis after treatment with tumour necrosis factor alpha inhibitors (GO-AFTER study): a multicentre, randomised, double-blind, placebo-controlled, phase III trial. Lancet 2009;374:210-21.
39. Rendas-Baum R, Wallenstein GV, Koncz T, et al. Evaluating the efficacy of sequential biologic therapies for rheumatoid arthritis patients with an inadequate response to tumor necrosis factor-alpha inhibitors. Arthritis Res Ther 2011;13:R25.
40. Kleinert S, Tony HP, Krause A, et al. Impact of patient and disease characteristics on therapeutic success during adalimumab treatment of patients with rheumatoid arthritis: data from a German noninterventional observational study. Rheumatol Int 2012;32:2759-67.
41. Sokka T, Kautiainen H, Pincus T, et al. Disparities in rheumatoid arthritis disease activity according to gross domestic product in 25 countries in the QUEST-RA database. Ann Rheum Dis 2009;68:1666-72.
42. Putrik P, Ramiro S, Kvien TK, et al. Inequities in access to biologic and synthetic DMARDs across 46 European countries. Ann Rheum Dis 2014;73:198-206.
43. Amgen. Amgen announces positive top-line results from phase 3 study evaluating the efficacy and safety of biosimilar candidate ABP 501 compared with adalimumab in patients with moderate-to-severe plaque psoriasis. 8 October 2014. http://www.amgen. com/media/media-pr-detail.jsp?releaseID=1975377 (accessed 10 Jul. 2015).
44. Breedveld FC, Weisman MH, Kavanaugh AF, et al. The PREMIER study: a multicenter, randomized, double-blind clinical trial of combination therapy with adalimumab plus methotrexate versus methotrexate alone or adalimumab alone in patients with early, aggressive rheumatoid arthritis who had not had previous methotrexate treatment. Arthritis Rheum 2006;54:26-37.
45. Burmester GR, Kivitz AJ, Kupper H, et al. Efficacy and safety of ascending methotrexate dose in combination with adalimumab: the randomised CONCERTO trial. Ann Rheum Dis 2015;74:1037-44.
46. Manders SH, Kievit W, Adang E, et al. Effectiveness of TNF inhibitor treatment with various methotrexate doses in patients with rheumatoid arthritis: results from clinical practice. Ann Rheum Dis 2015;74:e24.
47. Smolen JS, Aletaha D, Bijlsma JW, et al. Treating rheumatoid arthritis to target: recommendations of an international task force. Ann Rheum Dis 2010;69:631-7.
48. Genovese MC, Kavanaugh A, Weinblatt ME, et al. An oral Syk kinase inhibitor in the treatment of rheumatoid arthritis: a three-month randomized, placebo-controlled, phase II study in patients with active rheumatoid arthritis that did not respond to biologic agents. Arthritis Rheum 2011;63:337-45.
49. Crowson CS, Rahman MU, Matteson EL. Which measure of inflammation to use? A comparison of erythrocyte sedimentation rate and C-reactive protein measurements from randomized clinical trials of golimumab in rheumatoid arthritis. J Rheumatol 2009;36:1606-10.
50. Karsh J, Keystone EC, Haraoui B, et al. Canadian recommendations for clinical trials of pharmacologic interventions in rheumatoid arthritis: inclusion criteria and study design. J Rheumatol 2011;38:2095-104.
51. Kay J, Morgacheva O, Messing SP, et al. Clinical disease activity and acute phase reactant levels are discordant among patients with active rheumatoid arthritis: acute phase reactant levels contribute separately to predicting outcome at one year. Arthritis Res Ther 2014;16:R40.
52. Menter A, Gordon KB, Leonardi CL, et al. Efficacy and safety of adalimumab across subgroups of patients with moderate to severe psoriasis. J Am Acad Dermatol 2010;63:448-56.
53. Carrascosa JM, Rocamora V, Fernandez-Torres RM, et al. Obesity and psoriasis: inflammatory nature of obesity, relationship between psoriasis and obesity, and therapeutic implications. Actas Dermosifiliogr 2014;105:31-44.
54. European Medicines Agency. Guideline on similar biological medicinal products containing monoclonal antibodies-non-clinical and clinical issues. EMA/CHMP/BMWP/403543/2010. 30 May 2012. http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2012/06/WC500128686.pdf (accessed 10 Jul. 2015).
55. FDA. Biosimilars: Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009. April 2015. Guidance for Industry. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm444661.pdf (accessed 30 Oct. 2015).
56. ClinicalTrials.gov. Efficacy and safety study of ABP 501 compared to adalimumab in subjects with moderate to severe rheumatoid arthritis (RA). 14 May 2015. https://clinicaltrials.gov/ct2/show/NCT01970475 (accessed 10 Jul. 2015).
57. ClinicalTrials.gov. Long-term safety and efficacy of ABP 501 in subjects with moderate to severe rheumatoid arthritis. 14 January 2015. https://clinicaltrials.gov/ct2/show/NCT02114931 (accessed 10 Jul. 2015).
58. ® in rheumatoid arthritis patients (ARABESC). 5 June 2015. https://clinicaltrials.gov/ct2/show/NCT02260791 (accessed 10 Jul. 2015).
59. ClinicalTrials.gov. Study to demonstrate equivalent efficacy and to compare safety of biosimilar adalimumab (GP2017) and Humira (ADACCESS). 25 February 2015. https://clinicaltrials.gov/ct2/show/NCT02016105 (accessed 10 Jul. 2015).
60. EU Clinical Trials Register. A phase 3 randomized, double-blind study assessing the efficacy and safety of PF-06410293 and adalimumab in combination with methotrexate in subjects with moderately to severely active rheumatoid arthritis who have had an inadequate response to methotrexate. https://www.clinicaltrialsregister.eu/ctr-search/search?query=2014-000352-29 (accessed 10 Jul. 2015).
61. ®) in adults with moderate to severe plaque psoriasis. 19 May 2015. https://clinicaltrials.gov/ct2/show/NCT01970488 (accessed 10 Jul. 2015).
62. Kay J, Smolen JS. Biosimilars to treat inflammatory arthritis: the challenge of proving identity. Ann Rheum Dis 2013;72:1589-93.
63. ICH Harmonised Tripartite Guideline: Statistical Principles for Clinical Trials E9. 5 Feb. 1997. http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E9/Step4/E9-Guideline. pdf (accessed 30 Oct. 2015).
64. ICH Harmonised Tripartite Guideline: Choice of Control Group and Related Issues in Clinical Trials E10. 20 July 2000. http://www.ich.org/fileadmin/Public-Web-Site/ICH-Products/Guidelines/Efficacy/E10/Step4/E10-Guideline.pdf (accessed 30 Oct. 2015).
65. Maini R, St Clair EW, Breedveld F, et al. Infliximab (chimeric anti-tumour necrosis factor alpha monoclonal antibody) versus placebo in rheumatoid arthritis patients receiving concomitant methotrexate: a randomised phase III trial. ATTRACT Study Group. Lancet 1999;354:1932-9.
66. Choe JY, Prodanovic N, Niebrzydowski J, et al. A randomised, double-blind, phase III study comparing SB2, an infliximab biosimilar, to the infliximab reference product Remicade in patients with moderate to severe rheumatoid arthritis despite methotrexate therapy. Ann Rheum Dis Published Online First 28 Aug. 2015. doi:10.1136/annrheumdis-2015-207764
67. Emery P, Vencovský J, Sylwestrzak A, et al. A phase III randomised, double-blind, parallel-group study comparing SB4 with etanercept reference product in patients with active rheumatoid arthritis despite methotrexate therapy. Ann Rheum Dis Published Online First 6 Jul. 2015. doi:10.1136/annrheumdis-2015-207588.
68. Jani RH, Gupta R, Bhatia G, et al. A prospective, randomized, double-blind, multicentre, parallel-group, active controlled study to compare efficacy and safety of biosimilar adalimumab (Exemptia; ZRC-3197) and adalimumab (Humira) in patients with rheumatoid arthritis. Int J Rheum Dis 2015. [Epub ahead of print 14 Jul. 2015]. doi:10.1111/1756-185X.12711
69. Kay J, Chopra A, Chandrashekara S, et al. OP0012A Phase 3, randomized, double-blind, active comparator study of the efficacy and safety of Bow015, a biosimilar infliximab, in patients with active rheumatoid arthritis on stable methotrexate doses. Ann Rheum Dis 2014;73(Suppl 2):64.
70. Bui LA, Taylor C. Developing clinical trials for biosimilars. Semin Oncol 2014;41(Suppl 1):S15-25.
71. Combest A, Wang S, Healey B, et al. Alternative statistical strategies for biosimilar drug development. GaBI J 2014;3:13-20.