Indirect treatment comparison of the efficacy of patisiran and inotersen for hereditary transthyretin-mediated amyloidosis with polyneuropathy

References

• Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care. 2017;23(suppl 7):S107–S112.
   PubMed Web of Science ®Google Scholar
• Vita G, Vita GL, Stancanelli C, et al. Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 1: peripheral neuropathies. Neurol Sci. 2019;40(4):661–669.
   PubMed Web of Science ®Google Scholar
• Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31. PubMed PMID: 23425518; PubMed Central PMCID: PMCPMC3584981.
   PubMed Web of Science ®Google Scholar
• Hawkins PN, Ando Y, Dispenzeri A, et al. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015 Nov 17;47(8):625–638. .
   PubMed Web of Science ®Google Scholar
• Gonzalez-Duarte A. Autonomic involvement in hereditary transthyretin amyloidosis (hATTR amyloidosis). Clin Auton Res. 2019;29(2):245–251.
   PubMed Web of Science ®Google Scholar
• Planté-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol. 2011;10(12):1086–1097.
   PubMed Web of Science ®Google Scholar
• Parman Y, Adams D, Obici L, et al. Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe: where are we now? A European network approach to defining the epidemiology and management patterns for TTR-FAP. Curr Opin Neurol. 2016;29(Suppl 1):S3.
   PubMed Web of Science ®Google Scholar
• Yamamoto S, Wilczek HE, Nowak G, et al. Liver transplantation for familial amyloidotic polyneuropathy (FAP): a single-center experience over 16 years. Am J Transplant. 2007;7(11):2597–2604. PubMed PMID: 17868062.
   PubMed Web of Science ®Google Scholar
• Coutinho P, Martins da Silva A, Lopes Lima J, et al. Forty years of experience with type I amyloid neuropathy. Review of 483 cases. Amyloid and Amyloidosis; 1980, 88–98.
   Google Scholar
• Berk JL, Lin H, Agarwal S, et al. Impact of hereditary transthyretin-mediated amyloidosis on daily living and work productivity: baseline results from APOLLO. 16th International Symposium on Amyloidosis; 2018 Mar 26–29; Kumamoto, Japan.
   Google Scholar
• Yarlas A, Gertz MA, Dasgupta NR, et al. Burden of hereditary transthyretin amyloidosis on quality of life. Muscle Nerve. 2019;60(2):169–175.
   PubMed Web of Science ®Google Scholar
• European Medicines Agency (EMA). Vyndaqel (tafamidis) 20 mg soft capsules: summary of product characteristics. 2013 [cited 2019 Jul 17]. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/vyndaqel
   Google Scholar
• United States Food and Drug Administration (FDA). Highlights of prescribing information: tegsedi (inotersen). 2018 [cited 2018 Oct 8]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/211172lbl.pdf
   Google Scholar
• United States Food and Drug Administration (FDA). Highlights of prescribing information: onpattro (patisiran). 2018 [cited 2018 Oct 8]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210922s000lbl.pdf
   Google Scholar
• Health Canada. Product monograph and patient information: tegsedi (Inotersen). 2018 [cited 2018 Nov 14]. Available from: https://pdf.hres.ca/dpd_pm/00047651.PDF
   Google Scholar
• Health Canada. Product monograph including patient medication information, Onpattro (patisiran). [cited 2020 Apr 9]. Available from: https://pdf.hres.ca/dpd_pm/00052462.PDF
   Google Scholar
• PTC Therapeutics. PTC Therapeutics provides corporate update and highlights pipeline progress at 2020 J.P. Morgan Healthcare conference. [cited 2020 Apr 9]. Available from: http://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-provides-corporate-update-and-highlights
   Google Scholar
• Alnylam Pharmaceuticals. Alnylam announces approval in Brazil of ONPATTRO® for the treatment of hereditary ATTR amyloidosis with polyneuropathy. 2020 [cited 2020 Apr 9]. Available from: https://investors.alnylam.com/press-release?id=24606
   Google Scholar
• European Medicines Agency (EMA). Tegsedi (inotersen): summary of product characteristics. 2018 [cited 2018 Sept 27]. Available from: https://www.ema.europa.eu/en/documents/product-information/tegsedi-epar-product-information_en.pdf
   Google Scholar
• European Medicines Agency (EMA). Onpattro (patisiran): summary of product characteristics. 2018 [cited 2019 May 5]. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/onpattro
   Google Scholar
• Planté-Bordeneuve V, Lin H, Gollob J, et al. An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. Expert Opin Pharmacother. 2019;20(4):473–481.
   PubMed Web of Science ®Google Scholar
• Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):11–21.
   PubMed Web of Science ®Google Scholar
• Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):22–31.
   PubMed Web of Science ®Google Scholar
• Jansen JP, Trikalinos T, Cappelleri JC, et al. Indirect treatment comparison/network meta-analysis study questionnaire to assess relevance and credibility to inform health care decision making: an ISPOR-AMCP-NPC good practice task force report. Value Health. 2014 Mar 1;17(2):157–173. .
   PubMed Web of Science ®Google Scholar
• Jansen JP, Fleurence R, Devine B, et al. Interpreting indirect treatment comparisons and network meta-analysis for health-care decision making: report of the ISPOR task force on indirect treatment comparisons good research practices: part 1. Value Health. 2011;14(4):417–428.
   PubMed Web of Science ®Google Scholar
• European Medicines Agency (EMA). Public assessment report for Tegsedi (Inotersen). 2018 [cited 2018 Sept 28]. Available from: https://www.ema.europa.eu/documents/assessment-report/tegsedi-epar-public-assessment-report_en.pdf
   Google Scholar
• Dyck PJ, Kincaid JC, Dyck PJB, et al. Assessing mNIS+ 7Ionis and international neurologists’ proficiency in a familial amyloidotic polyneuropathy trial. Muscle Nerve. 2017;56(5):901–911.
   PubMed Web of Science ®Google Scholar
• Vinik EJ, Vinik AI, Paulson JF, et al. Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2014 Jun 1;19(2):104–114. .
   PubMed Web of Science ®Google Scholar
• Suanprasert N, Berk J, Benson M, et al. Retrospective study of a TTR FAP cohort to modify NIS+ 7 for therapeutic trials. J Neurol Sci. 2014;344(1–2):121–128.
   PubMed Web of Science ®Google Scholar
• Signorovitch JE, Sikirica V, Erder MH, et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health. 2012;15(6):940–947.
   PubMed Web of Science ®Google Scholar
• Signorovitch JE, Wu EQ, Andrew PY, et al. Comparative effectiveness without head-to-head trials. Pharmacoeconomics. 2010;28(10):935–945.
   PubMed Web of Science ®Google Scholar
• European Medicines Agency (EMA). ICH harmonised guideline E9(R1). Estimands and sensitivity analysis in clinical trials. Step 2b, version dated August 30, 2017. 2017 [cited 2020 Apr 3]. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/draft-ich-e9-r1-addendum-estimands-sensitivity-analysis-clinical-trials-guideline-statistical_en.pdf
   Google Scholar
• Bucher HC, Guyatt GH, Griffith LE, et al. The results of direct and indirect treatment comparisons in meta-analysis of randomized controlled trials. J Clin Epidemiol. 1997;50(6):683–691.
   PubMed Web of Science ®Google Scholar
• Song F, Altman DG, Glenny A-M, et al. Validity of indirect comparison for estimating efficacy of competing interventions: empirical evidence from published meta-analyses. Br Med J. 2003;326(7387):472.
   PubMed Web of Science ®Google Scholar
• Adams D, Suhr OB, Dyck PJ, et al. Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol. 2017;17(1):181.
   PubMed Web of Science ®Google Scholar
• Carvalho A, Rocha A, Lobato L. Liver transplantation in transthyretin amyloidosis: issues and challenges. Liver Transplant. 2015;21(3):282–292.
   PubMed Web of Science ®Google Scholar
• Phillippo DM, Ades AE, Dias S, et al. Methods for population-adjusted indirect comparisons in health technology appraisal. Med Decis Making. 2018;38(2):200–211.
   PubMed Web of Science ®Google Scholar