-
1Academic Journal
Συγγραφείς: V. A. Merkulov, R. I. Yagudina, V. G. Serpik, В. А. Меркулов, Р. И. Ягудина, В. Г. Серпик
Συνεισφορές: The study reported in this publication was carried out as part of publicly funded research project No. 056-00026-24-00 and was supported by the Scientific Centre for Expert Evaluation of Medicinal Products (R&D reporting No. 124022200093-9), Работа выполнена в рамках государственного задания ФГБУ «НЦЭСМП» Минздрава России № 056-0002624-00 на проведение прикладных научных исследований (номер государственного учета НИР 124022200093-9)
Πηγή: Regulatory Research and Medicine Evaluation; Том 14, № 1 (2024); 14-28 ; Регуляторные исследования и экспертиза лекарственных средств; Том 14, № 1 (2024); 14-28 ; 3034-3453 ; 3034-3062
Θεματικοί όροι: РНК-терапия, cost of medicinal products, value of medicinal products, pipeline, Alzheimer’s disease, gene therapy, cell therapy, RNA therapy, стоимость лекарственных препаратов, ценность лекарственных препаратов, пайплайн, болезнь Альцгеймера, генная терапия, клеточная терапия
Περιγραφή αρχείου: application/pdf
Relation: https://www.vedomostincesmp.ru/jour/article/view/579/1301; https://www.vedomostincesmp.ru/jour/article/downloadSuppFile/579/497; Vignali V, Hines PA, Cruz AG, Ziętek B, Herold R. Health horizons: future trends and technologies from the European Medicines Agency’s horizon scanning collaborations. Front Med (Lausanne). 2022;9:1064003. https://doi.org/10.3389/fmed.2022.1064003; Adedeji WA. The treasure called antibiotics. Ann Ib Postgrad Med. 2016;14(2):56–7. PMID: 28337088; Buxbaum JD, Chernew ME, Fendrick AM, Cutler DM. Contributions of public health, pharmaceuticals, and other medical care to US life expectancy changes, 1990–2015. Health Aff (Millwood). 2020;39(9):1546–56. https://doi.org/10.1377/hlthaff.2020.00284; Lichtenberg FR. The effect of pharmaceutical innovation on longevity: evidence from the U.S. and 26 high-income countries. Econ Hum Biol. 2022;46:101124. https://doi.org/10.1016/j.ehb.2022.101124; Lichtenberg FR. The impact of new (orphan) drug approvals on premature mortality from rare diseases in the United States and France, 1999–2007. Eur J Health Econ. 2013;14(1):41–56. https://doi.org/10.1007/s10198-011-0349-4; MacEwan JP, Dennen S, Kee R, Ali F, Shafrin J, Batt K. Changes in mortality associated with cancer drug approvals in the United States from 2000 to 2016. J Med Econ. 2020;23(12):1558–69. https://doi.org/10.1080/13696998.2020.1834403; Manns MP, Maasoumy B. Breakthroughs in hepatitis C research: from discovery to cure. Nat Rev Gastroenterol Hepatol. 2022;19(8):533–50. https://doi.org/10.1038/s41575-022-00608-8; Campollo O, Amaya G, McCormick PA. Milestones in the discovery of hepatitis C. World J Gastroenterol. 2022;28(37):5395–402. https://doi.org/10.3748/wjg.v28.i37.5395; Krupa D, Czech M, Chudzyńska E, Koń B, Kostera-Pruszczyk A. Real world evidence on the effectiveness of nusinersen within the national program to treat spinal muscular atrophy in Poland. Healthcare. 2023;11(10):1515. https://doi.org/10.3390/healthcare11101515; Berglund A, Berkö S, Lampa E, Sejersen T. Survival in patients diagnosed with SMA at less than 24 months of age in a population-based setting before, during and after introduction of nusinersen therapy. Experience from Sweden. Eur J Paediatr Neurol. 2022;40:57–60. https://doi.org/10.1016/j.ejpn.2022.07.005; Beakes-Read G, Neisser M, Frey P, Guarducci M. Analysis of FDA’s accelerated approval program performance December 1992 — December 2021. Ther Innov Regul Sci. 2022;56(5):698–703. https://doi.org/10.1007/s43441-022-00430-z; Lakdawalla DN, Doshi JA, Garrison LP Jr, Phelps CE, Basu A, Danzon PM. Defining elements of value in health care — A health economics approach: An ISPOR Special Task Force Report. Value Health. 2018;21(2):131–9. https://doi.org/10.1016/j.jval.2017.12.007; Franken M, Stolk E, Scharringhausen T, de Boer A, Koopmanschap M. A comparative study of the role of disease severity in drug reimbursement decision making in four European countries. Health Policy. 2015;119(2):195–202. https://doi.org/10.1016/j.healthpol.2014.10.007; Khunti K, Seidu S, Kunutsor S, Davies M. Association between adherence to pharmacotherapy and outcomes in type 2 diabetes: a meta-analysis. Diabetes Care. 2017;40(11):1588–96. https://doi.org/10.2337/dc16-1925; van Boven JF, Chavannes NH, van der Molen T, Rutten-van Mölken MP, Postma MJ, Vegter S. Clinical and economic impact of non-adherence in COPD: a systematic review. Respir Med. 2014;108(1):103–13. https://doi.org/10.1016/j.rmed.2013.08.044; Abegaz TM, Shehab A, Gebreyohannes EA, Bhagavathula AS, Elnour AA. Nonadherence to antihypertensive drugs: a systematic review and meta-analysis. Medicine (Baltimore). 2017;96(4):e5641. https://doi.org/10.1097/md.0000000000005641; Ruppar TM, Cooper PS, Mehr DR, Delgado JM, Dunbar-Jacob JM. Medication adherence interventions improve heart failure mortality and readmission rates: systematic review and meta-analysis of controlled trials. J Am Heart Assoc. 2016;5(6):e002606. https://doi.org/10.1161/jaha.115.002606; Du L, Cheng Z, Zhang Y, Li Y, Mei D. The impact of medication adherence on clinical outcomes of coronary artery disease: a meta-analysis. Eur J Prev Cardiol. 2017;24(9):962–70. https://doi.org/10.1177/2047487317695628; Lai H, Li R, Li Z, Zhang B, Li C, Song C, et al. Modelling the impact of treatment adherence on the transmission of HIV drug resistance. J Antimicrob Chemother. 2023;78(8):1934–43. https://doi.org/10.1093/jac/dkad186; Zozaya N, Alcalá B, Galindo J. The offset effect of pharmaceutical innovation: a review study. Glob Reg Health Technol Assess. 2019;2019(5):228424031987510. https://doi.org/10.1177/2284240319875108; Тихомирова АВ, Ягудина РИ. Фармакоэкономический анализ прямых медицинских затрат при лечении метастатического колоректального рака режимами XELOX или FOLFOX4 в сочетании с бевацизумабом или без него в качестве терапии первой линии. ФАРМАКОЭКОНОМИКА. Современная фармакоэкономика и фармакоэпидемиология. 2010;3(2):22–7. EDN: MUDQUX; Ягудина РИ, Куликов АЮ, Комаров ИА. Анализ «затраты–эффективность» лечения пациентов, которым за последние 6 месяцев был поставлен диагноз хронический миелолейкоз в хронической фазе, лекарственными средствами группы ингибиторов тирозинкиназы–нилотиниба в сравнении с иматинибом. ФАРМАКОЭКОНОМИКА. Современная фармакоэкономика и фармакоэпидемиология. 2013;6(2):42–7. EDN: RNKORX; Куликов АЮ, Нгуен T. Фармакоэкономический анализ одногодичной адъювантной терапии трастузумабом при НЕR2-положительном раке молочной железы ранней стадии. ФАРМАКОЭКОНОМИКА. Современная фармакоэкономика и фармакоэпидемиология. 2010;3(4):28–34. EDN: NXZQIJ; Серпик ВГ. Фармакоэкономическая оценка терапии редких заболеваний на примере лечения первичного миелофиброза препаратом руксолитиниб. Фармакоэкономика: теория и практика. 2015;3(2):20–3. https://doi.org/10.30809/phe.2.2015.10; DiMasi JA, Grabowski HG, Hansen RW. Innovation in the pharmaceutical industry: new estimates of R&D costs. J Health Econ. 2016;47:20–33. https://doi.org/10.1016/j.jhealeco.2016.01.012; Schlander M, Hernandez-Villafuerte K, Cheng CY, Mestre-Fernandiz J, Baumann M. How much does it cost to research and develop a new drug? A systematic review and assessment. Pharmacoeconomics. 2021;39(11):1243–69. https://doi.org/10.1007/s40273-021-01065-y; Moon S, Mariat S, Kamae I, Pedersen HB. Defining the concept of fair pricing for medicines. BMJ. 2020;368:l4726 https://doi.org/10.1136/bmj.l4726; Angelis A, Polyakov R, Wouters OJ, Torreele E, McKee M. High drug prices are not justified by industry’s spending on research and development. BMJ. 2023;380:e071710. https://doi.org/10.1136/bmj-2022-071710; Chan K, Sepassi A, Saunders IM, Goodman A, Watanabe JH. Effects of financial toxicity on prescription drug use and mental well-being in cancer patients. Explor Res Clin Soc Pharm. 2022;6:100136. https://doi.org/10.1016/j.rcsop.2022.100136; Hussaini SMQ, Gupta A, Dusetzina SB. Financial toxicity of cancer treatment. JAMA Oncol. 2022;8(5):788. https://doi.org/10.1001/jamaoncol.2021.7987; Jung YL, Hwang J, Yoo HS. Disease burden metrics and the innovations of leading pharmaceutical companies: a global and regional comparative study. Global Health. 2020;16:80. https://doi.org/10.1186/s12992-020-00610-2; The Lancet Respiratory Medicine. Where are the innovations in tuberculosis drug discovery? Lancet Respir Med. 2017;5(11):835. https://doi.org/10.1016/S2213-2600(17)30376-4; Abdelsayed M, Kort EJ, Jovinge S, Mercola M. Repurposing drugs to treat cardiovascular disease in the era of precision medicine. Nat Rev Cardiol. 2022;19(11):751–64. https://doi.org/10.1038/s41569-022-00717-6; Van Norman GA. Overcoming the declining trends in innovation and investment in cardiovascular therapeutics: beyond EROOM’s law. JACC Basic Transl Sci. 2017;2(5):613–25. https://doi.org/10.1016/j.jacbts.2017.09.002; Klug DM, Idiris FIM, Blaskovich MAT, von Delft F, Dowson CG, Kirchhelle C, et al. There is no market for new antibiotics: this allows an open approach to research and development. Wellcome Open Res. 2021;6:146. https://doi.org/10.12688/wellcomeopenres.16847.1; Miller KL, Fermaglich LJ, Maynard J. Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases. Orphanet J Rare Dis. 2021;16(1):265. https://doi.org/10.1186/s13023-021-01901-6; Wellman-Labadie O, Zhou Y. The US Orphan Drug Act: rare disease research stimulator or commercial opportunity? Health Policy. 2010;95(2–3):216–28. https://doi.org/10.1016/j.healthpol.2009.12.001; Årdal C, Lacotte Y, Ploy MC. Financing pull mechanisms for antibiotic-related innovation: opportunities for Europe. Clin Infect Dis. 2020;71(8):1994–9. https://doi.org/10.1093/cid/ciaa153; Проценко МВ, Серпик ВГ. Обзор международной классификации редких заболеваний. Фармакоэкономика: теория и практика. 2021;9(2):18–20. https://doi.org/10.30809/phe.2.2021.3; Ding C, Wu Y, Chen X, Chen Y, Wu Z, Lin Z, et al. Global, regional, and national burden and attributable risk factors of neurological disorders: the Global Burden of Disease study 1990–2019. Front Public Health. 2022;10:952161. https://doi.org/10.3389/fpubh.2022.952161; Cummings J, Zhou Y, Lee G, Zhong K, Fonseca J, Cheng F. Alzheimer’s disease drug development pipeline: 2023. Alzheimers Dement (NY). 2023;9(2):e12385. Erratum: Alzheimers Dement (NY). 2023;9(2):e12407. https://doi.org/10.1002/trc2.12385; Бачурин СО. Препараты для лечения болезни Альцгеймера по данным клинических испытаний и основные тенденции в подходах к поиску новых лекарственных средств. Журнал неврологии и психиатрии им. С.С. Корсакова. 2016;116(8):77–87. https://doi.org/10.17116/jnevro20161168177-87; Rabaneda-Bueno R, Mena-Montes B, Torres-Castro S, Torres-Carrillo N, Torres-Carrillo NM. Advances in genetics and epigenetic alterations in Alzheimer’s disease: a notion for therapeutic treatment. Genes (Basel). 2021;12(12):1959. https://doi.org/10.3390/genes12121959; Sasso JM, Ambrose BJB, Tenchov R, Datta RS, Basel MT, DeLong RK, Zhou QA. The progress and promise of RNA medicine — an arsenal of targeted treatments. J Med Chem. 2022;65(10):6975–7015. https://doi.org/10.1021/acs.jmedchem.2c00024; Yu AM, Choi YH, Tu MJ. RNA drugs and RNA targets for small molecules: principles, progress, and challenges. Pharmacol Rev. 2020;72(4):862–98. https://doi.org/10.1124/pr.120.019554; Boada C, Sukhovershin R, Pettigrew R, Cooke JP. RNA therapeutics for cardiovascular disease. Curr Opin Cardiol. 2021;36(3):256–63. https://doi.org/10.1097/hco.0000000000000850; Biernacki MA, Brault M, Bleakley M. T-cell receptor-based immunotherapy for hematologic malignancies. Cancer J. 2019;25(3):179–90. https://doi.org/10.1097/ppo.0000000000000378; Helsen CW, Hammill JA, Lau VWC, Mwawasi KA, Afsahi A, Bezverbnaya K, et al. The chimeric TAC receptor co-opts the T cell receptor yielding robust anti-tumor activity without toxicity. Nat Commun. 2018;9(1):3049. https://doi.org/10.1038/s41467-018-05395-y; Chen Y, Yu Z, Tan X, Jiang H, Xu Z, Fang Y, et al. CAR-macrophage: a new immunotherapy candidate against solid tumors. Biomed Pharmacother. 2021;139:111605. https://doi.org/10.1016/j.biopha.2021.111605; Wrona E, Borowiec M, Potemski P. CAR-NK cells in the treatment of solid tumors. Int J Mol Sci. 2021;22(11):5899. https://doi.org/10.3390/ijms22115899; https://www.vedomostincesmp.ru/jour/article/view/579