[Tecfidera® (delayed-release dimethylfumarate) in the treatment of relapsing-remitting multiple sclerosis]
DOI:
https://doi.org/10.7175/fe.v18i2s.1320Keywords:
Delayed-release dimethylfumarate, Health Technology Assessment, Relapsing-remitting multiple sclerosis, ItalyAbstract
The present health technology assessment (HTA) evaluates the clinical and economic profile of delayed-release dimethylfumarate (DMF, also known as gastro-resistant DMF) in the treatment of relapsing-remitting multiple sclerosis (RRMS) in Italy. Chapter 1 briefly introduces the condition (multiple sclerosis and, more specifically, the relapsing-remitting form) and provides an overall description of the main therapeutic options for physicians, in terms of clinical evidence, regulatory status in Europe and approval status and reimbursement (refunding) criteria in Italy. In the next sections (Chapters 2-5), key-topics regarding RRMS are analysed: epidemiology, clinical burden, quality of life/social impact and the economic implications for healthcare services, patients and society. In Chapter 6, the clinical evidence supporting the use of DMF in RRMS is summarized. Data from phase III randomized clinical trials (DEFINE, CONFIRM), plus the pooled post-hoc analysis of the two studies, were evaluated to assess the level of clinical benefit provided by DMF. Finally, in Chapter 7, a review of the health economic evidence assessing DMF is performed, with a specific focus on Italy. Overall, the methodological quality of registration studies, together with the robustness and consistency of the study results, support the conclusion that DMF is an effective and safe treatment for RRMS. The economic assessment of DMF in Italy, based on cost-effectiveness and budget impact analyses (adopting clinical input data from a mixed treatment comparison and economic input data relative to the Italian healthcare setting), confirm that DMF is a cost-effective and economically sustainable treatment for the Italian National Healthcare Service. These findings are in line with the results of most international publications and the assessments from well-recognized HTA agencies (e.g. NICE/SMC). In summary, both the clinical and economic evidence analyzed in this HTA substantiate DMF as an important therapeutic option for the treatment of RRMS.References
Lin SX, Lisi L, Dello Russo C, et al. The anti-inflammatory effects of dimethyl fumarate in astrocytes involve glutathione and haem oxygenase-1. ASN Neuro 2011; 3; https://doi.org/10.1042/AN20100033
EMA, European Medicine Agency. Tecfidera, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/002601/WC500162069.pdf (ultimo accesso settembre 2016)
WHO Collaborating Centre for Drug Statistics Methodology. New ATC. Disponibile su: http://www.whocc.no/atc/lists_of_new_atc_ddds_and_altera/new_atc/?order_by=0&d=DESC (ultimo accesso settembre 2016)
PHARMASTAR. SM, dal New England conferme per BG-12, nuova terapia orale. 20 Settembre 2012. Disponibile su: http://www.pharmastar.it/?cat=5&id=9045 (ultimo accesso settembre 2016)
Libero. Sm: vicini ad una svolta. 18 Ottobre 2012. Disponibile su: http://www.liberoquotidiano.it/news/salute/1101801/SCLEROSI-MULTIPLA--VICINI-AD-UNA-SVOLTA.html (ultimo accesso settembre 2016)
Parodi B, Rossi S, Morando S, et al. Fumarates modulate microglia activation through a novel HCAR2 signaling pathway and rescue synaptic dysregulation in inflamed CNS. Acta Neuropathol 2015; 130: 279-95; https://doi.org/10.1007/s00401-015-1422-3
Ministero della sanità. ICD-10, classificazione statistica internazionale delle malattie e dei problemi sanitari correlati. Disponibile su: http://en.istat.it/strumenti/definizioni/malattie.pdf (ultimo accesso settembre 2016)
Ministero della salute. IC-9, classificazione statistica internazionale delle malattie – versione Italiana del 1997. Disponibile su: http://www.salute.gov.it/portale/temi/p2_6.jsp?lingua=italiano&id=1278&area=ricoveriOspedalieri&menu=classificazione (ultimo accesso settembre 2016)
SM-e.it. Cos’è la SM. Settembre 2012. Disponibile su: http://www.sclerosimultipla-e.it/capire/cose-sm.shtml (ultimo accesso settembre 2016)
Bainbridge JL, Corboy JR. Multiple sclerosis. In: DiPiro JT, Talbert RL, Yee GC, Matzke GR, Wells BG, Posey LM, editors. Pharmacotherapy: A Pathophysiologic Approach. 7th ed. New York (NY): McGraw-Hill, 2008
Polman CH, Reingold SC, Edan G, et al. Diagnostic criteria for multiple sclerosis: 2005 revisions to the “McDonald Criteria”. Ann Neurol 2005; 58: 840-6; https://doi.org/10.1002/ana.20703
Lublin FD, Reingold SC, Cohen JA, et al. Defining the clinical course of multiple sclerosis: the 2013 revisions. Neurology 2014; 83: 278-86; https://doi.org/10.1212/WNL.0000000000000560
Canal N, Ghezzi A, Zaffaroni M. Sclerosi multipla. Attualità e prospettive. Milano: Elsevier Health Sciences, 2011
Jadidi E, Mohammadi M, Moradi T. High risk of cardiovascular diseases after diagnosis of multiple sclerosis. Mult Scler 2013; 19: 1336-40; https://doi.org/10.1177/1352458513475833
Keegan BM, Noseworthy JH. Multiple sclerosis. Annu Rev Med 2002; 53: 285-302; https://doi.org/10.1146/annurev.med.53.082901.103909
Sclerosi Org - informazione e sostegno per la SM. Sintomi e segni della SM. Disponibile su: http://www.sclerosi.org/sintomi-e-segni-della-sclerosi-multipla.php (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Avonex, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/000102/WC500029425.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Plegridy, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/002827/WC500170302.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Betaferon, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/000081/WC500053225.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Extavia, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/000933/WC500034701.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Rebif, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/000136/WC500048681.pdf (ultimo accesso settembre 2016)
EMC, Medicine Org UK. Copaxone 20mg, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.medicines.org.uk/emc/medicine/17516/spc (ultimo accesso settembre 2016)
EMC, Medicine Org UK. Copaxone 40mg, riassunto delle caratteristiche del prodotto. Disponibile su: https://www.medicines.org.uk/emc/medicine/30795 (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Tysabri, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/000603/WC500044686.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Tysabri, sintesi dell’opinione del CHMP sul cambio dell’autorizzazione al commercio. Disponibile su: http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion/human/000603/WC500207155.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Gilenya, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/002202/WC500104528.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Lemtrada, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/003718/WC500150521.pdf (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Aubagio, riassunto delle caratteristiche del prodotto. Disponibile su: http://www.ema.europa.eu/docs/it_IT/document_library/EPAR_-_Product_Information/human/002514/WC500148682.pdf (ultimo accesso settembre 2016)
Jacobs LD, Cookfair DL, Rudick RA, et al. Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis. The Multiple Sclerosis Collaborative Research Group (MSCRG). Ann Neurol 1996; 39: 285-94; https://doi.org/10.1002/ana.410390304
Derwenskus J. Current disease-modifying treatment of multiple sclerosis. Mt Sinai J Med 2011; 78: 161-75; https://doi.org/10.1002/msj.20239
Jongen PJ, Sindic C, Sanders E, et al. Adverse Events of Interferon Beta-1a: A Prospective Multi-Centre International ICH-GCP-Based CRO-Supported External Validation Study in Daily Practice. PLoS One 2011; 6: e26568; https://doi.org/10.1371/journal.pone.0026568
Calabresi PA, Kieseier BC, Arnold DL, et al.; ADVANCE Study Investigators. Pegylated interferon β-1a for relapsing-remitting multiple sclerosis (ADVANCE): a randomised, phase 3, double-blind study. Lancet Neurol 2014; 13: 657-65; https://doi.org/10.1016/S1474-4422(14)70068-7
Calabresi PA, Kieseier BC, Arnold DL, et al. Clinical efficacy of peginterferon beta-1a in relapsing-remitting multiple sclerosis: 2-year data from the phase 3 ADVANCE study. ECTRIMS Online Library 2014; 64811
Kremenchutzky M, Liu S, Cui Y, et al. Long-Term Safety and Tolerability of Peginterferon Beta-1a: Interim Analysis From ATTAIN, a Phase 3 Extension Study (S4.002). Neurology 2015; 84 Supplement S4.002
IFNB Multiple Sclerosis Study Group. Interferon beta-lb is effective in relapsing-remitting - multiple sclerosis. Neurology 1993; 43: 655-61; https://doi.org/10.1212/WNL.43.4.655
Ebers GC, Rice G, Lesaux J, et al. Randomised double-blind placebo-controlled study of interferon beta-1a in relapsing/remitting multiple sclerosis. PRISMS (Prevention of Relapses and Disability by Interferon beta-1a Subcutaneously in Multiple Sclerosis) Study Group. Lancet 1998; 352: 1498-504; https://doi.org/10.1016/S0140-6736(98)03334-0
Johnson KP, Brooks BR, Cohen JA, et al. Copolymer 1 reduces relapse rate and improves disability in relapsing-remitting multiple sclerosis: results of a phase III multicenter, double-blind placebo-controlled trial. The Copolymer 1 Multiple Sclerosis Study Group. Neurology 1995; 45: 1268-76; https://doi.org/10.1212/WNL.45.7.1268
ÒConnor P, Wolinsky JS, Confavreux C, et al.; TEMSO Trial Group. Randomized trial of oral teriflunomide for relapsing multiple sclerosis. N Engl J Med 2011; 365: 1293-303
Confavreux C, ÒConnor P, Comi G, et al.; TOWER Trial Group.Oral teriflunomide for patients with relapsing multiple sclerosis (TOWER): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol 2014; 13: 247-56; https://doi.org/10.1016/S1474-4422(13)70308-9
Vermersch P, Czlonkowska A, Grimaldi LM, et al.; TENERE Trial Group. Teriflunomide versus subcutaneous interferon beta-1a in patients with relapsing multiple sclerosis: a randomised, controlled phase 3 trial. Mult Scler 2014; 20: 705-16; https://doi.org/10.1177/1352458513507821
Polman CH, ÒConnor PW, Havrdova E, et al.; AFFIRM Investigators. A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med 2006; 354: 899-910; https://doi.org/10.1056/NEJMoa044397
Hutchinson M, Kappos L, Calabresi PA, et al.; AFFIRM and SENTINEL Investigators. The efficacy of natalizumab in patients with relapsing multiple sclerosis: subgroup analyses of AFFIRM and SENTINEL. J Neurol 2009; 256: 405-15; https://doi.org/10.1007/s00415-009-0093-1
Belachew S, Phan-Ba R, Bartholomé E, et al. Natalizumab induces a rapid improvement of disability status and ambulation after failure of previous therapy in relapsing-remitting multiple sclerosis. Eur J Neurol 2011; 18: 240-5; https://doi.org/10.1111/j.1468-1331.2010.03112.x
Putzki N, Kollia K, Woods S, et al. Natalizumab is effective as second line therapy in the treatment of relapsing remitting multiple sclerosis. Eur J Neurol 2009b; 16: 424-6; https://doi.org/10.1111/j.1468-1331.2008.02519.x
Putzki N, Yaldizli O, Mäurer M, et al. Efficacy of natalizumab in second line therapy of relapsing-remitting multiple sclerosis: results from a multi-center study in German speaking countries. Eur J Neurol 2010; 17: 31-7; https://doi.org/10.1111/j.1468-1331.2009.02728.x
Prosperini L, Giannì C, Leonardi L, et al. Escalation to natalizumab or switching among immunomodulators in relapsing multiple sclerosis. Mult Scler 2012; 18: 64-71; https://doi.org/10.1177/1352458511417481
Kappos L, Radue EW, ÒConnor P, et al.; FREEDOMS Study Group. A placebo-controlled trial of oral fingolimod in relapsing multiple sclerosis. N Engl J Med 2010; 362: 387-401; https://doi.org/10.1056/NEJMoa0909494
Cohen JA, Barkhof F, Comi G, et al.; TRANSFORMS Study Group. Oral fingolimod or intramuscular interferon for relapsing multiple sclerosis. N Engl J Med 2010; 362: 402-15; https://doi.org/10.1056/NEJMoa0907839
Sperandeo K, Nogrady L, Moreo K, et al. Managed approaches to multiple sclerosis in special populations. J Manag Care Pharm 2011; 17(9 Suppl C): S1-19
Radue EW, ÒConnor P, Polman CH, et al.; FTY720 Research Evaluating Effects of Daily Oral Therapy in Multiple Sclerosis (FREEDOMS) Study Group. Impact of fingolimod therapy on magnetic resonance imaging outcomes in patients with multiple sclerosis. Arch Neurol 2012; 69: 1259-69; https://doi.org/10.1001/archneurol.2012.1051
Galetta SL, Markowitz C, Lee AG. Immunomodulatory agents for the treatment of relapsing multiple sclerosis: a systematic review. Arch Intern Med 2002; 162: 2161-9; https://doi.org/10.1001/archinte.162.19.2161
Paty DW, Li DKB; MS/MRI Study Group; IFNB MS Study Group. Interferon beta-lb is effective in relapsing-remitting multiple sclerosis. 11. MRI analysis results of a multicenter, randomized, double-blind, placebo-controlled trial. Neurology 1993; 43: 662-7; https://doi.org/10.1212/WNL.43.4.662
Li DK, Paty DW. Magnetic resonance imaging results of the PRISMS trial: a randomized, doubleblind, placebo-controlled study of interferon-beta1a in relapsing-remitting multiple sclerosis.Prevention of Relapses and Disability by Interferon-beta1a Subcutaneously in Multiple Sclerosis. Ann Neurol 1999; 46: 197-206; https://doi.org/10.1002/1531-8249(199908)46:2<197::AID-ANA9>3.0.CO;2-P
Cohen JA, Coles AJ, Arnold DL, et al.; CARE-MS I investigators. Alemtuzumab versus interferon beta 1a as first-line treatment for patients with relapsing-remitting multiple sclerosis: a randomised controlled phase 3 trial. Lancet 2012; 380: 1819-28; https://doi.org/10.1016/S0140-6736(12)61769-3
Coles AJ, Twyman CL, Arnold DL, et al.; CARE-MS II investigators. Alemtuzumab for patients with relapsing multiple sclerosis after disease-modifying therapy: a randomised controlled phase 3 trial. Lancet 2012; 380: 1829-39; https://doi.org/10.1016/S0140-6736(12)61768-1
Coles AJ, Fox E, Vladic A, et al. Alemtuzumab more effective than interferon β-1a at 5-year follow-up of CAMMS223 clinical trial. Neurology 2012; 78: 1069-78; https://doi.org/10.1212/WNL.0b013e31824e8ee7
Gold R, Giovannoni G, Selmaj K, et al.; SELECT study investigators. Daclizumab high-yield process in relapsing-remitting multiple sclerosis (SELECT): a randomised, double-blind, placebo-controlled trial. Lancet 2013; 381: 2167-75; https://doi.org/10.1016/S0140-6736(12)62190-4
Kappos L, Wiendl H, Selmaj K, et al. Daclizumab HYP versus Interferon Beta-1a in Relapsing Multiple Sclerosis. N Engl J Med 2015; 373: 1418-28; https://doi.org/10.1056/NEJMoa1501481
Giovannoni G, Comi G, Cook S, et al.; CLARITY Study Group. A placebo-controlled trial of oral cladribine for relapsing multiple sclerosis. N Engl J Med 2010; 362: 416-26; https://doi.org/10.1056/NEJMoa0902533
Hauser SL, Comi G, Hartung HP, et al. Efficacy and Safety of Ocrelizumab in Relapsing Multiple Sclerosis: Results of the INF beta-1a-Controlled, Double-Blind, Phase III OPERA I and II Studies. ACTRIMS Forum 2016, February 18-20, New Orleans, Louisiana
Montalban X, Hemmer B, Rammohan K, et al. A randomized, Double-Blind, Parallel-Group, Placebo-Controlled Phase III Trial to Evaluate Efficacy and Safety of Ocrelizumab in Primary Progressive Multiple Sclerosis. ACTRIMS Forum 2016, February 18-20, New Orleans, Louisiana
AIFA – Agenzia Italiana del Farmaco. Nota 65. Disponibile su: http://www.agenziafarmaco.gov.it/it/content/nota-65 (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Nota Informativa Importante sui Beta-interferoni. Agosto 2014. Disponibile su: http://www.agenziafarmaco.gov.it/it/content/nota-informativa-importante-sui-beta-interferoni-20082014 (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Nota Informativa Importante su Tysabri. Maggio 2008. Disponibile su: http://www.agenziafarmaco.gov.it/sites/default/files/nii_tysabri220508.pdf (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Nota Informativa Importante su Tysabri. Agosto 2008. Disponibile su: http://www.agenziafarmaco.gov.it/sites/default/files/nii_tysabri.pdf (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Nota Informativa Importante su Tysabri. Febbraio 2010. Disponibile su: http://www.agenziafarmaco.gov.it/sites/default/files/Nota_Informativa_Importante_1.pdf (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Il PRAC aggiorna le raccomandazioni per minimizzare il rischio di PML a seguito dell’utilizzo di Tysabri (natalizumab). Disponibile su: http://www.aifa.gov.it/content/il-prac-aggiorna-le-raccomandazioni-minimizzare-il-rischio-di-pml-seguito-dell%E2%80%99utilizzo-di-t (ultimo accesso settembre 2016)
EMA – European Medicines Agency. EMA confirms recommendations to minimise risk of brain infection PML with Tysabri. Disponibile su: http://www.ema.europa.eu/docs/en_GB/document_library/Referrals_document/Tysabri_20/European_Commission_final_decision/WC500202394.pdf (ultimo accesso settembre 2016)
EMA –European Medicines Agency. Tysabri: Procedural steps taken and scientific information after the authorisation. Disponibile su: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Procedural_steps_taken_and_scientific_information_after_authorisation/human/000603/WC500044692.pdf (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. DETERMINA 13 aprile 2015. Regime di rimborsabilità e prezzo, a seguito di nuove indicazioni terapeutiche, del medicinale per uso umano «Gilenya». (Determina n. 411/2015). Gazzetta n. 96 del 27 Aprile 2015. Disponibile su: http://www.gazzettaufficiale.it/eli/id/2015/04/27/15A03054/sg (ultimo accesso settembre 2016)
EMA – European Medicines Agency. New recommendations to minimise risks of the rare brain infection PML and a type of skin cancer with Gilenya. Disponibile su: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2015/12/news_detail_002447.jsp&mid=WC0b01ac058004d5c1 (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Nota Informativa Importante su Gilenya (fingolimod) (07/01/2016). Disponibile su: http://www.agenziafarmaco.gov.it/it/content/nota-informativa-importante-su-gilenya-fingolimod-07012016 (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Gazzetta n. 82 del 9 Aprile 2015, determina del 23 Marzo 2015. Disponibile su: http://www.agenziafarmaco.gov.it/sites/default/files/Determinazione_Lemtrada.pdf (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Scheda monitoraggio Lemtrada. Disponibile su: http://www.agenziafarmaco.gov.it/it/content/pubblicazione-schede-di-monitoraggio-registro-lemtrada-20042015 (ultimo accesso settembre 2016)
Biogen. Biogen Demonstrates Commitment to Improving Patient Outcomes with Study on Burden of MS and New Clinical Data from Its Leading MS Portfolio. Agosto 2016. Disponibile su: http://media.biogen.com (ultimo accesso settembre 2016)
EMA – European Medicines Agency. Updated recommendations to minimise the risk of the rare brain infection PML with Tecfidera. Disponibil su: http://www.agenziafarmaco.gov.it/sites/default/files/Tecfidera_PHC_EN.pdf (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Nota Informativa Importante su Tecfidera. Novembre 2015. Disponibile su: http://www.aifa.gov.it/sites/default/files/NII_Tecfidera_19.11.2015.pdf (ultimo accesso settembre 2016)
Polman CH, Reingold SC, Banwell B, et al. Diagnostic criteria for multiple sclerosis: 2010 revisions to the McDonald criteria. Ann Neurol 2011; 69: 292-302; https://doi.org/10.1002/ana.22366
AIFA – Agenzia Italiana del Farmaco. DETERMINA 10 giugno 2014. Rinegoziazione del medicinale per uso umano «Tysabri» (natalizumab), ai sensi dell’articolo 8, comma 10, della legge 24 dicembre 1993, n. 537. (Determina n. 589/2014). Gazzetta n. 139 del 18 Giugno 2014. Disponibile su: www.gazzettaufficiale.it/eli/id/2014/06/18/14A04592/sg (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Comunicato relativo alla rimborsabilita’, senza modifica di prezzo del medicinale per uso umano «Tysabry». Gazzetta n. 10 del 14 Gennaio 2016. Disponibile su: http://www.gazzettaufficiale.biz/atti/2016/20160010/16A00137.htm (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Scheda monitoraggio Tysabri. Disponibile su: http://www.agenziafarmaco.gov.it/it/content/pubblicazione-schede-di-monitoraggio-registro-tysabri-15012016 (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Lista farmaci neurologia in legge 648/96. Disponibile su: http://www.agenziafarmaco.gov.it/it/content/legge-64896 (ultimo accesso settembre 2016)
AIFA – Agenzia Italiana del Farmaco. Gazzetta n. 200 del 29 Agosto 2014. Disponibile su: http://www.gazzettaufficiale.it/eli/id/2014/08/29/14A06699/sg (ultimo accesso marzo 2016)
AIFA – Agenzia Italiana del Farmaco. DETERMINA 23 marzo 2015. Riclassificazione del medicinale per uso umano «Lemtrada» ai sensi dell’art. 8, comma 10, della legge 24 dicembre 1993, n. 537. (Determina n. 303/2015). Gazzetta n. 82 del 9 Aprile 2015. Disponibile su: http://www.gazzettaufficiale.it/eli/id/2015/04/09/15A02470/sg (ultimo accesso marzo 2016)
AIFA – Agenzia Italiana del Farmaco. DETERMINA 31 luglio 2014. Regime di rimborsabilità e prezzo del medicinale per uso umano «Aubagio» (teriflunomide). (Determina n. 837/2014). Gazzetta n. 187 del 13 Agosto 2014, determina del 31 Luglio 2014. Disponibile su: http://www.gazzettaufficiale.biz/atti/2014/20140187/14A06412.htm (ultimo accesso settembre 2016)
Multiple Sclerosis International Federation. Atlas of MS database. Disponibile su: http://www.msif.org/wp-content/uploads/2014/09/Atlas-of-MS.pdf (ultimo accesso settembre 2016)
Pugliatti M, Rosati G, Carton H, et al. The epidemiology of multiple sclerosis in Europe. Eur J Neurol 2006; 13: 700-22; https://doi.org/10.1111/j.1468-1331.2006.01342.x
AISM, Associazione Italiana Sclerosi Multipla. Bilancio sociale 2014. Disponibile su: http://bilanciosociale.aism.it/wp-content/uploads/2014/05/AISM-Bilancio-2014.pdf (ultimo accesso settembre 2016)
Pugliatti M, Sotgiu S, Solinas G, et al. Multiple sclerosis epidemiology in Sardinia: evidence for a true increasing risk. Acta Neurol Scand 2001; 103: 20-6; https://doi.org/10.1034/j.1600-0404.2001.00207.x
Granieri E, Casetta I, Govoni V, et al. The increasing incidence and prevalence of MS in a Sardinian province. Neurology 2000; 55: 842-8; https://doi.org/10.1212/WNL.55.6.842
Sironi L , Mamoli A , D’Alessandro G , et al. Epidemiology of Multiple Sclerosis in Valle d’Aosta, Italy. Mult Scler 1997; 3: 283
Ranzato F, Perini P, Tzintzeva E, et al. Increasing frequency of multiple sclerosis in Padova, Italy: a 30 year epidemiological survey. Mult Scler 2003; 9: 387-92; https://doi.org/10.1191/1352458503ms920oa
Granieri E, Malagù S, Casetta I, et al. Multiple sclerosis in Italy. A reappraisal of incidence and prevalence in Ferrara. Arch Neurol 1996; 53: 793-8; https://doi.org/10.1001/archneur.1996.00550080115019
Nicoletti A, Lo Bartolo ML, Lo Fermo S, et al. Prevalence and incidence of multiple sclerosis in Catania, Sicily. Neurology 2001; 56: 62-6; https://doi.org/10.1212/WNL.56.1.62
Totaro R, Marini C, Cialfi A, et al. Prevalence of multiple sclerosis in the L’Aquila district, central Italy. J Neurol Neurosurg Psychiatry 2000; 68: 349-52; https://doi.org/10.1136/jnnp.68.3.349
Iuliano G. Prevalence of multiple sclerosis in Salerno, Italy: preliminary data in a study on general practitioners. Italian Journal of Neurological Sciences 1999; 20 (Suppl. 4): S186
Granieri E, Rosati G, Tola R, et al. The frequency of multiple sclerosis in Mediterranean Europe. An incidence and prevalence study in Barbagia, Sardinia, insular Italy. Acta Neurol Scand 1983; 68: 84-9; https://doi.org/10.1111/j.1600-0404.1983.tb04821.x
Chiao E, Meyer K. Cost effectiveness and budget impact of natalizumab in patients with relapsing multiple sclerosis. Curr Med Res Opin 2009; 25: 1445-54; https://doi.org/10.1185/03007990902876040
IMS Health. Ricerca IMFO – Periodo Maggio 2013-Aprile 2014. ©IMS Health S.p.A. Tutti i diritti riservati
AISM – Associazione Italiana SM. Riabilitazione cognitiva: nuovo approccio terapeutico per i difetti di attenzione nella SM. Disponibile su: http://www.aism.it/index.aspx?codpage=2012_12_fism_cerasa (ultimo accesso settembre 2016)
Ruet A, Deloire M, Charré-Morin J, et al. Cognitive impairment differs between primary progressive and relapsing-remitting MS. Neurology 2013; 80: 1501-8; https://doi.org/10.1212/WNL.0b013e31828cf82f
PHARMSTAR. Conferme dall’ECTRIMS per natalizumab nelle forme recidivanti di SM. Ottobre 2012. Disponibile su: http://www.pharmastar.it/popup/stampa.html?idlink=9242 (ultimo accesso settembre 2016)
Zamboni P, Bertolotto A, Boldrini P, et al. Efficacy and safety of venous angioplasty of the extracranial veins for multiple sclerosis. Brave dreams study (brain venous drainage exploited against multiple sclerosis): study protocol for a randomized controlled trial. Trials 2012; 13: 183; https://doi.org/10.1186/1745-6215-13-183
Business Wire, a berkshire hathaway company. Il New England Journal of Medicine ha pubblicato dati cruciali che dimostrano l’efficacia e la sicurezza del farmaco BG-12 (dimetilfumarato) per la sclerosi multipla somministrato per via orale. Settembre 2012. Disponibile su: http://www.businesswire.com/news/home/20120919007019/it/ (ultimo accesso settembre 2016)
World Health Organization (1958). The first ten years of the World Health Organization. Geneva: World Health Organization
Rothwell PM, McDowellZ, Wong CK, et al. Doctors and patients don’t agree: cross-sectional study of patients and doctors perceptions and assessments of disability in multiple sclerosis. BMJ 1997; 314: 1580-3; https://doi.org/10.1136/bmj.314.7094.1580
Solari A. Role of health-related quality of life measures in the routine care of people with multiple sclerosis. Health Qual Life Outcomes 2005; 3: 16; https://doi.org/10.1186/1477-7525-3-16
Bandari DS, Vollmer TL, Bhupendra OK, et al. Assessing quality of life in patients with multiple sclerosis. Int J MS Care 2010; 12: 34-41; https://doi.org/10.7224/1537-2073-12.1.34
Mitchell AJ, Benito-León J, González JM, et al. Quality of life and its assessment in multiple sclerosis: integrating physical and psychological components of wellbeing. Lancet Neurol 2005; 4: 556-66; https://doi.org/10.1016/S1474-4422(05)70166-6
Acquadro C, Lafortune L, Mear I. Quality of life in multiple sclerosis: translation in French Canadian of the MSQOL-54. Health Qual Life Outcomes 2003; 1: 70; https://doi.org/10.1186/1477-7525-1-70
Ajmerich M, Guillamon I, Perkal H, et al. Spanish adaptation of the disease-specific questionnaire MSQOL-54 in multiple sclerosis patients. Neurologia 2006; 21: 181-7
El Alaoui Taoussi K, Ait Ben Haddou E, Benomar A, et al. Quality of life and multiple sclerosis: Arabic language translation and transcultural adaptation of “MSQOL-54”. Rev Neurol 2012; 168: 444-9; https://doi.org/10.1016/j.neurol.2011.10.011
Fuvesi J, Bencsik K, Benedek K, et al. Cross-cultural adaptation and validation of the ‘Multiple Sclerosis Quality of Life Instrument’ in Hungarian. Mult Scler 2008; 14: 391-8; https://doi.org/10.1177/1352458507082724
Ghaem H, Borhani Haghighi A, et al. Validity and reliability of the Persian version of the multiple sclerosis quality of life questionnaire. Neurol India 2007; 55: 369-75; https://doi.org/10.4103/0028-3886.33316
Idiman E, Uzunel F, Ozakbas S, et al. Cross-cultural adaptation and validation of multiple sclerosis quality of life questionnaire (MSQOL-54) in a Turkish multiple sclerosis sample. J Neurol Sci 2006; 240: 77-80; https://doi.org/10.1016/j.jns.2005.09.009
Yamamoto T, Ogata K, Katagishi M, et al. Validation of the Japanese-translated version Multiple Sclerosis Quality of Life-54 instrument. Rinsho Shinkeigaku 2004; 44; 417-21
Pekmezovic T, Tepavcevic K, Kostic J, et al. Validation and cross-cultural adaptation of the disease-specific questionnaire MSQOL-54 in Serbian multiple sclerosis patients sample. Qual Life Res 2007; 16: 1383-7; https://doi.org/10.1007/s11136-007-9234-0
Solari A, Filippini G, Mendozzi L, et al. Validation of Italian multiple sclerosis quality of life 54 questionnaire. J Neurol Neurosurg Psychiatry 1999; 67: 158-62; https://doi.org/10.1136/jnnp.67.2.158
Vernay D, Gerbaud L, Biolay S, et al. Quality of life and multiple sclerosis: Validation of the French version of the self-questionnaire (SEP-59). Rev Neur 2000; 156: 247-63
Baumstarck-Barrau K, Pelletier J, Simeoni MC, et al. French validation of the multiple sclerosis International quality of life questionnaire. Rev Neurol 2011; 167: 511-21; https://doi.org/10.1016/j.neurol.2010.10.008
Beiske AG, Baumstarck K, Nilsen RM, et al. Validation of the multiple sclerosis International quality of life (MusiQol) questionnaire in Norwegian patients. Acta Neurol Scand 2012; 12: 171-9; https://doi.org/10.1111/j.1600-0404.2011.01518.x
Flachenecker P, Vogel U, Simeoni MC, et al. MusiQol: International questionnaire investigating quality of life in multiple sclerosis: valdation results of the German sub-population in an international comparison. Nevertenarzt 2011; 82: 1281-9; https://doi.org/10.1007/s00115-011-3276-9
Fernandez O, Fernandez V, Baumstarck-Barrau K, et al.; MusiQoL study group of Spain. Validation of the Spanish version of the multiple sclerosis International quality of life (MusiQol) questionnaire. BMC Neurol 2011; 11: 127; https://doi.org/10.1186/1471-2377-11-127
Jamroz-Winiewska A, SStelmasiak Z, Bartosik-Psujek H. Validation analysis of the Polish version of the multiple sclerosis International quality of life questionnaire (MusiQol). Neurol Neurochir Pol 2011; 45: 235-44; https://doi.org/10.1016/s0028-3843(14)60076-1
Thumboo J, Seah A, Tan CT, et al. Asian adaptation and validation of an English version of the multiple sclerosis International quality of life questionnaire (MusiQol). Ann Acad Med Singapore 2011; 40: 67-73
Triatafyllou N, Triatafyllou A, Tsivgoulis G. Validity and reliability of the Greek version of the multiple sclerosis International quality of life questionnaire. J Clin Neurol 2009; 5: 173-7; https://doi.org/10.3988/jcn.2009.5.4.173
Akbiyik D, Sumbuloglu V, Guney Z, et al. The validity and test-retest reliability of the Leeds Multiple Sclerosis Quality of Life scale in Turkish patients. J Rehab Res 2009; 32: 162-6; https://doi.org/10.1097/MRR.0b013e328329820f
Patti F, Russo P, Pappalardo A, et al.; FAMS study group. Predictors of quality of life among patients with multiple sclerosis: An Italian cross-sectional study. J Neurol Sci 2007; 252: 121-9; https://doi.org/10.1016/j.jns.2006.10.017
Sørensen J, Bay J, Damsgaard T, et al. Validation of the Danish version of Functional Assessment of Multiple Sclerosis: a quality of life instrument. Mult Scler Int 2011; 2011: 121530; https://doi.org/10.1155/2011/121530
Mendes MF, Balsimelli S, Stangelhaus G, et al. Validation of the functional assessment of multiple sclerosis quality of life instrument in a Portuguese language. Arq Neuropssiquatr 2004; 62: 108-13; https://doi.org/10.1590/S0004-282X2004000100019
Solari A. Quality of life reporting in multiple sclerosis clinical trials: enough quality? Mult Scler 2012; 18: 1668-9; https://doi.org/10.1177/1352458512447595
Wild D, Grove A, Martin M, et al.; ISPOR Task Force for Translation and Cultural Adaptation. Principles of Good Practice for the translation and cultural adaptation process of patient-reported outcome (PRO) measures: report of the ISPOR task force for the translation and cultural adaptation. Value Health 2005; 8: 94-104; https://doi.org/10.1111/j.1524-4733.2005.04054.x
Vickrey BG, Hays RD, Harooni R, et al. A health-related quality of life measure for multiple sclerosis. Qual Life Res 1995; 4: 187-206; https://doi.org/10.1007/BF02260859
Simeoni M, Auquier P, Fernandez O, et al.; MusiQol study group. Validation of the multiple sclerosis International quality of life questionnaire. Mult Scler 2008; 14: 219-30; https://doi.org/10.1177/1352458507080733
Cella DF, Dineen K, Arnason B, et al. Validation of the functional assessment in multiple sclerosis quality of life instrument. Neurology 1996; 47: 129-39; https://doi.org/10.1212/WNL.47.1.129
Gold SM, Heesen C, Schulz H, et al. Disease specific quality of life instruments in multiple sclerosis: validation of the Hamburg Quality of Life Questionnaire in Multiple Sclerosis (HAQAMS). Mult Scler 2001; 7: 119-30; https://doi.org/10.1191/135245801678227649
Ford HL, Gerry E, Tennant A, et al. Developing a disease-specific quality of life measure for people with multiple sclerosis. Clin Rehab 2001; 15: 247-58; https://doi.org/10.1191/026921501673658108
Rotstein Z, Barak Y, Noy S, et al. Quality of life in multiple sclerosis: development and validation of the ‘Rays’ scale and comparison with the SF-36. Int Qual Health Care 2000; 12: 511-7; https://doi.org/10.1093/intqhc/12.6.511
Hobart J, Lamping, D, Fitzpatrick R, et al. The Multiple Sclerosis Impact Scale (MSIS-29: a new patient-based outcome measure. Brain 2001; 124: 962-73; https://doi.org/10.1093/brain/124.5.962
Ruta DA, Garratt AM, Leng M, et al. A new approach to the measurement of quality of life. The Patient-Generated Index. Med Care 1994; 32: 1109-26; https://doi.org/10.1097/00005650-199411000-00004
ÒBoyle CA, Browne J, Hickey A, et al. Manual for the SEIQoL-DW. Dublin: Department of Psychology, Royal College of Surgeons in Ireland, 1995
Kuspinar A, Mayo NE. Do generic utility measures capture what is important to the quality of life of people with multiple sclerosis? Health Qual Life Outcomes 2013; 11:71; https://doi.org/10.1186/1477-7525-11-71
Veronese S, Gallo G, Valle A, et al. Specialist palliative care improve the quality of life in advanced neurodegenerative disorders: NE-PAL, a pilot randomised controlled study. BMJ Support Palliat Care 2015; https://doi.org/10.1136/bmjspcare-2014-000788
Patel KK, Veenstra DL, Patrick DL. A review of selected patient-generated outcome measures and their application in clinical trials. Value Health 2003;6: 595-603; https://doi.org/10.1046/j.1524-4733.2003.65236.x
Detmar SB, Muller MJ, Schornagel JH, et al. Health-related quality-of-life assessments and patient-physician communication: a randomized controlled trial. JAMA 2002; 288: 3027-34; https://doi.org/10.1001/jama.288.23.3027
Ritvo PG, Fischer JS, Miller DM, et al. Multiple Sclerosis Quality of Life Inventory: A user’s manual. New York, NY: National Multiple Sclerosis Society, 1997
Kuspinar A, Rodriguez AM, Mayo NE. The effects of clinical interventions on health-related quality of life in multiple sclerosis: a meta-analysis. Mult Scler 2012; 18: 1686-704; https://doi.org/10.1177/1352458512445201
Giordano A, Pucci E, Naldi P, et al. Responsiveness of patient reported out come measures in multiple sclerosis relapses: the REMS study. J Neurol Neurosurg Psychiatry 2009; 80: 1023-8; https://doi.org/10.1136/jnnp.2008.171181
Moore F, Vickrey B, Fortin K, Lee L. Two multiple sclerosis quality-of-life measures: comparison in a national sample. Can J Neurol Sci 2015; 42: 55-63; https://doi.org/10.1017/cjn.2014.128
Schwartz CE, Bode R, Repucci N, et al. The clinical significance of adaptation to changing health: a meta-analysis of response shift. Quality of Life Research 2006; 15: 1533-50; https://doi.org/10.1007/s11136-006-0025-9
Kind P. Values and valuation in the measurement of HRQoL. In Assessing quality of life in clinical trials. 2nd edition. Edited by Fayers P, Hays D. New York: Oxford University Press Inc., 2005
Feeny D. Preference-based measures: utility and quality-adjusted life years. In Assessing quality of life in clinical trials. 2nd edition. Edited by Fayers P, Hays D. New York: Oxford University Press Inc., 2005
Brazier J, Ratcliffe J, Salomon JA, et al. Measuring and valuing health benefits for economic evaluation. New York: Oxford University Press Inc., 2007
Torrance GW, Barr RD, Zhang Y, et al. Multiattribute utility function for a comprehensive health status classification system. Health Utilities Index Mark 2. Med Care 1996; 34: 702-22; https://doi.org/10.1097/00005650-199607000-00004
Horsman J, Furlong W, Feeny D, et al. The Health Utilities Index (HUI): concepts, measurement properties and applications. Health Qual Life Outcomes 2003; 1: 54; https://doi.org/10.1186/1477-7525-1-54
Feeny D, Furlong W, Torrance GW, et al. Multi-attribute and single-attribute utility functions for the health utilities index mark 3 system. Med Care 2002; 40: 113-28; https://doi.org/10.1097/00005650-200202000-00006
Gudex C. The descriptive system of the EuroQOL instrument. In EQ-5D concepts and methods: a developmental history. Edited by Kind P, Brooks R, Rabin R. Dordrecht: Springer, 2005; https://doi.org/10.1007/1-4020-3712-0_2
Brazier J, Usherwood T, Harper R, et al. Deriving a preference-based single index from the UK SF-36 Health Survey. J Clin Epidemiol 1998; 51: 1115-28; https://doi.org/10.1016/S0895-4356(98)00103-6
Ponzio M, Gerzeli S, Brichetto G, et al. Economic impact of multiple sclerosis in Italy: focus on rehabilitation costs. Neurol Sci 2015; 36: 227-34; https://doi.org/10.1007/s10072-014-1925-z
Acquadro C, Berzon R, Dubois D, et al.; PRO Harmonization Group. Incorporating the patient’s perspective into drug development and communication: an ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001. Value Health 2003; 6: 522-31; https://doi.org/10.1046/j.1524-4733.2003.65309.x
Brundage M, Bass B, Davidson J, et al. Patterns of reporting health-related quality of life outcomes in randomised clinical trials: implications for clinicians and quality of life researchers. Qual Life Res 2011; 20: 653-64; https://doi.org/10.1007/s11136-010-9793-3
EMA – European Medicines Agency. Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQoL) measures in the evaluation of medicinal products. European Medicines Agency. Committee for medicinal products for human use (CHMP), 2005. Disponibile su: www.ispor.org/workpaper/emea-hrql-guidance.pdf (ultimo accesso settembre 2016)
Xu Z, Zhang F, Sun F, et al. Dimethyl fumarate for multiple sclerosis. Cochrane Database of Systematic Reviews 2015; 4: CD011076; https://doi.org/10.1002/14651858.cd011076.pub2
Calvert M, Brundage M, Jacobsen PB, et al. The CONSORT Patient-Reported Outcome (PRO) extension: implications for clinical trials and practice. Health Qual Life Outcomes 2013; 11: 184; https://doi.org/10.1186/1477-7525-11-184
Kingwell E, Marriott JJ, Jetté N, et al. Incidence and prevalence of multiple sclerosis in Europe: a systematic review. BMC Neurol 2013; 13: 128; https://doi.org/10.1186/1471-2377-13-128
Quotidiano Sanità. Sclerosi Multipla. Esplode il costo sociale della malattia: 2,5 miliardi l’anno. Giugno 2012. Disponibile su: http://www.quotidianosanita.it/scienza-e-farmaci/articolo.php?articolo_id=9517 (ultimo accesso settembre 2016)
Kobelt G, Berg J, Atherly D, et al. Costs and quality of life in multiple sclerosis: a cross-sectional study in the United States. Neurology 2006; 66: 1696-702; https://doi.org/10.1212/01.wnl.0000218309.01322.5c
Kobelt G, Berg J, Lindgren P, et al. Costs and quality of life of patients with multiple sclerosis in Europe. J Neurol Neurosurg Psychiatry 2006; 77: 918-26; https://doi.org/10.1136/jnnp.2006.090365
Kobelt G, Berg J, Lindgren P, et al. Costs and quality of life of multiple sclerosis in Italy. Eur J Health Econ 2006; 7:S45-S54; https://doi.org/10.1007/s10198-006-0365-y
Berto P, Amato MP, Bellantonio P, et al. The direct cost of patients with multiple sclerosis: a survey from Italian MS centres. Neurol Sci 2011; 32: 1035-41; https://doi.org/10.1007/s10072-011-0578-4
Karampampa K, Gustavsson A, Miltenburger C, et al. Treatment experience, burden and unmet needs (TRIBUNE) in MS study: results from Italy. Mult Scler 2012; 18(2 Suppl): 29-34; https://doi.org/10.1177/1352458512441566c
AIS – Agenzia Informazione Sanità. Sclerosi multipla. AISM: ha costo sociale di 400 mila euro. Disponibile su: http://allegati.aism.it/manager/trackdoc.asp?file=/manager/UploadFile/2/sn09_terapie.2009520_14112.pdf&open=1 (ultimo accesso settembre 2016)
Gold R, Kappos L, Arnold DL, et al.; DEFINE Study Investigators. Placebo-controlled phase 3 study of oral BG-12 for relapsing multiple sclerosis. N Engl J Med 2012; 367: 1098-107; https://doi.org/10.1056/NEJMoa1114287
Fox RJ, Miller DH, Phillips JT, et al.; CONFIRM Study Investigators. Placebo-controlled phase 3 study of oral BG-12 or glatiramer in multiple sclerosis. N Engl J Med 2012; 367: 1087-97; https://doi.org/10.1056/NEJMoa1206328
Selmaj K, Gold R, Kappos L, et al. Safety and tolerability of BG-12 in the phase 3 DEFINE trial in patients with relapsing-remitting multiple sclerosis. Poster presented at the 5th Joint Triennial Congress of the European and Americas Committees for Treatment and Research in Multiple Sclerosis (ECTRIMS and ACTRIMS); October 19-22, 2011. Amsterdam, The Netherlands (Poster 994)
Hutchinson M, Fox RJ, Miller DH, et al. Clinical efficacy of BG-12 (dimethyl fumarate) in patients with relapsing–remitting multiple sclerosis: subgroup analyses of the CONFIRM study. J Neurol 2013; 260: 2286-96; https://doi.org/10.1007/s00415-013-6968-1
Phillips JT, Selmaj K, Gold R, et al. Clinical Significance of Gastrointestinal and Flushing Events in Patients with Multiple Sclerosis Treated with Delayed-Release Dimethyl Fumarate. Int J MS Care 2015; 17: 236-243; https://doi.org/10.7224/1537-2073.2014-069
Phillips JT, Hutchinson M, Fox R, et al. Managing flushing and gastrointestinal events associated with delayed-release dimethyl fumarate: Experiences of an international panel. Mult Scler Relat Disord 2014; 3: 513-9; https://doi.org/10.1016/j.msard.2014.03.003
Biogen Idec. Statistical analysis plan (SAP) for the analyses of pooled data in the BG-12 Integrated Summary of Efficacy (ISE). Date of SAP: 5 October 2011
Gold R, Giovannoni G, Phillips JT, et al. Sustained Effect of Delayed-Release Dimethyl Fumarate in Newly Diagnosed Patients with Relapsing-Remitting Multiple Sclerosis: 6-Year Interim Results From an Extension of the DEFINE and CONFIRM Studies. Neurol Ther 2016; 5: 45-57; https://doi.org/10.1007/s40120-016-0042-8
Gold R, Arnold DL, Bar-Or A, et al. Long-term effects of delayed-release dimethyl fumarate in multiple sclerosis: Interim analysis of ENDORSE, a randomized extension study. Mult Scler 2016; pii: 1352458516649037
Linker RA, Haghikia A. Dimethyl fumarate in multiple sclerosis: latest developments, evidence and place in therapy. Ther Adv Chronic Dis 2016; 7: 198-207; https://doi.org/10.1177/2040622316653307
Furneri G, Santoni L, Marchesi C, et al. Cost-effectiveness analysis of delayed-release dimethyl-fumarate in the treatment of relapsing-remitting multiple sclerosis in Italy. Farmeconomia. Health economics and therapeutic pathways 2016; 17: 67-80; http://dx.doi.org/10.7175/fe.v17i2.1251
Furneri G, Santoni L, Marchetti C. Budget impact analysis of delayed-release dimethyl-fumarate in the treatment of relapsing-remitting multiple sclerosis in Italy. Farmeconomia. Health economics and therapeutic pathways 2016; 17: 29-39; http://dx.doi.org/10.7175/fe.v17i1.1237
Hutchinson M, Fox RJ, Havrdova E, et al. Efficacy and safety of BG-12 (dimethyl fumarate) and other disease-modifying therapies for the treatment of relapsing-remitting multiple sclerosis: a systematic review and mixed treatment comparison. Curr Med Res Opin 2014; 30: 613-27; https://doi.org/10.1185/03007995.2013.863755
Zhang X, Hay JW, Niu X. Cost effectiveness of fingolimod, teriflunomide, dimethyl fumarate and intramuscular interferon-β1a in relapsing-remitting multiple sclerosis. CNS Drugs 2015; 29: 71-81; https://doi.org/10.1007/s40263-014-0207-x
Gasche D, Restovic G, Casado V, et al. Cost-Effectiveness Analysis of Delayed-Release Dimethyl Fumarate for The Treatment of Relapsing Remitting Multiple Sclerosis In Spain. Value Health 2015; 18: A755; https://doi.org/10.1016/j.jval.2015.09.2931
Granfeldt D, Björstad Å, Öhrman S, et al. The Cost Effectiveness of Delayed-Release Dimethyl Fumarate Versus Interferon Beta-1b In A Swedish Setting. Value Health 2015; 18: A757; https://doi.org/10.1016/j.jval.2015.09.2467
Miguel LS, de Sá J, Pinheiro B, et al. Cost-Utility Analysis of Delayed-Release Dimethyl Fumerate For The Treatment of Relapsing-Remitting Multiple Sclerosis In Portugal. Value Health 2015; 18: A758; https://doi.org/10.1016/j.jval.2015.09.2469
Olsen J, Wiren A. Cost-Effectiveness of Dimethyl Fumarate Treatment for Relapsing-Remitting Multiple Sclerosis From A Danish Perspective. Value Health 2015; 18: A758; https://doi.org/10.1016/j.jval.2015.09.2469
Soini EJ, Joutseno J, Sumelahti M. Cost-Effectiveness of First-Line Disease-Modifying Therapies (Dmts) For Relapsing-Remitting Ms (Rrms). Value Health 2015; 18: A756; https://doi.org/10.1016/j.jval.2015.09.2459
NICE – National Institute for Health and Care Excellence. Dimethyl fumarate for treating relapsing-remitting multiple sclerosis - NICE technology appraisal guidance [TA320]. Disponibile su: https://www.nice.org.uk/guidance/ta320 (ultimo accesso settembre 2016)
SMC – Scottish Medicine Consortium. Technology Assessment - Dimethyl fumarate (Tecfidera). Disponibile su: https://www.scottishmedicines.org.uk/SMC_Advice/Advice/886_13_dimethyl_fumarate_Tecfidera/dimethyl_fumarate_Tecfidera (ultimo accesso settembre 2016)
HAS – Haute Autoritè de Santè. Transparency Committee Opinion. Tecidera, 7 May 2014. Disponibile su: http://www.has-sante.fr/portail/jcms/c_1743058/fr/tecfidera-dimethyl-fumarate-traitement-de-fond-de-la-sclerose-en-plaques (ultimo accesso settembre 2016)
QWiG Reports – Commission No. A14-14. Dimethyl fumarate –Benefit assessment according to §35a Social Code Book V1. Disponibile su: https://www.iqwig.de/en/projects-results/projects/drug-assessment/a14-14-dimethyl-fumarate-benefit-assessment-according-to-35a-social-code-book-v-dossier-assessment.6130.html (ultimo accesso settembre 2016)
The Pharmaceutical Benefits Scheme of Australia. Application for: Dimethyl Fumarate, capsules, 120 mg and 240 mg, Tecfidera® - July 2013. Disponibile su: http://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/psd/2013-07/dimethyl-fumarate (ultimo accesso settembre 2016)
Canadian Agency for Drugs and Technologies in Health. Drug Expert Committee - Final recommendation for Tecfidera -February 2013. Disponibile su: https://www.cadth.ca/dimethyl-fumarate-6 (ultimo accesso settembre 2016)
Published
How to Cite
Issue
Section
License
Authors who publish with this journal agree to the following terms:
- Authors retain copyright and grant the journal right of first publication with the work simultaneously licensed under a Creative Commons Attribution-NonCommercial 4.0 License that allows others to share the work with an acknowledgement of the work's authorship and initial publication in this journal.
- Authors are able to enter into separate, additional contractual arrangements for the non-exclusive distribution of the journal's published version of the work (e.g., post it to an institutional repository or publish it in a book), with an acknowledgement of its initial publication in this journal. The Publication Agreement can be downloaded here, and should be signed by the Authors and sent to the Publisher when the article has been accepted for publication in this journal.
- Authors are permitted and encouraged to post their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work (see The Effect of Open Access).
- Authors are permitted to post their work online after publication (the article must link to publisher version, in html format)
