Клинични ползи и финансова тежест на pasireotidе
Abstract
Цел: Да се извърши клинична и икономическа оценка на pasireotide, прилаган при пациенти с болест на Кушинг и акромегалия от гледна точка на българската здравноосигурителна система.
Материал и методи: Извършен е систематичен преглед в интернет бази данни на проучвания относно ефикасността и безопасността на pasireotide. Приложен е подходът на макроостойностяване за остойностяване на разходите за лекарства, заплащани от Националната здравноосигурителна каса (НЗОК) за периода 2016-2018 г. Разходите са сравнени с помощта на статистическия софтуер MedCalc и екстраполирани за следващите 3 години с прилагане на линейни функции. Приложен е тест на Mann-Whitney за независими извадки за тестване на статистически значими разлики в разходите.
Резултати: Въз основа на доказателствата за ефикасност и безопасност, ръководствата на Европейското ендокринологично дружество посочват pasireotide като втора линия терапия за болестта на Кушинг и акромегалия. Годишните разходи за лечение с pasireotide са статистически значимо по-високи за всяка следваща година спрямо предишната: 310 356 лв. през 2016 г., 419 028.36 лв. през 2017 г. и 502 003.52 лв. през 2018г. Делът на разходите за pasireotide от общите лекарствени разходи, изплатени от НЗОК за амбулаторни грижи за 3-годишния период е между 0,04 и 0,07%.
Изводи: Pasireotide подобрява съответните клинични параметри, но прилагането му е свързано с по-висок риск от хипергликемия, което изисква внимателно мониториране. Свързаните с pasireotide разходи са сравнително високи за българската здравна система и показват тенденция на нарастване за следващия тригодишен период. Вероятно увеличението на годишните разходи е в резултат на увеличаване на посочените пациенти за лечение с pasireotide. Това подчертава значението му като втора линия терапия и за двете групи пациенти.
References
Терапия на синдрома на Кушинг – нови данни. Наука Ендокринология. 2011;1:39-48.
Кратка характеристика на Signifor. www.ema.europa.eu
Novartis Pharmaceuticals. Signifor LAR Prescribing Information. 2014. Available from: http://www.pharma.us.novartis.com/product/pi/pdf/ signifor_lar.pdf. Accessed March 14, 2015.
US Food and Drug Administration. FDA Approves Signifor, a New Orphan Drug for Cushing’s Disease. 2012. Available from: http://www. fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm332351. htm. Accessed July 17, 2015.
Daly AF, Rixhon M, Adam C, et al. High prevalence of pituitary adenomas: a cross-sectional study in the province of Liège, Belgium. J Clin Endocrinol Metab. 2006;91:4769–4775.
Fernandez A, Karavitaki N, Wass JAH. Prevalence of pituitary adenomas: a community-based, cross-sectional study in Banbury (Oxfordshire, UK). Clin Endocrinol (Oxf). 2010;72: 377–382.
Albarel F, Castinetti F, Morange I, et al. Outcome of multimodal therapy in operated acromegalic patients, a study in 115 patients. Clin Endocrinol. 2013;78:263–270.
Mercado M, Espinosa E, Ramirez C. Current status and future directions of the pharmacological therapy of Acromegaly. Minerva Endocrinol. 2016;41(3):351-65.
Cuevas-Ramos D, Fleseriu M. Pasireotide: a novel treatment for patients with acromegaly. Drug Des Devel Ther. 2016;10:227-39.
Holdaway IM, Bolland MJ, Gamble GD. A meta-analysis of the effect of lowering serum levels of GH and IGF-I on mortality in acromegaly. Eur J Endocrinol. 2008;159:89–95.
Reid TJ, Post KD, Bruce JN, et al. Features at diagnosis of 324 patients with acromegaly did not change from 1981 to 2006: acromegaly remains under-recognized and under-diagnosed. Clin Endocrinol (Oxf). 2010; 72:203–208.
Hahl J, Kurki S, Miettinen T, Snicker K. Cost-Effectiveness of Pasireotide Long-Acting in a Treatment of Acromegaly in Finland. Economic Evaluation Based on Finnish Auria Biobank Data on Health Care Resource Utilization. Value Health. 2015;18(7):A609.
Truong HL, Nellesen D, Ludlam WH, Neary MP. Budget impact of pasireotide for the treatment of Cushing’s disease, a rare endocrine disorder associated with considerable comorbidities, Journal of Medical Economics. 2014; 17:4, 288-295.
Peral C, Cordido F, Gimeno-Ballester V, Mir N, Sánchez-Cenizo L, Rubio-Rodríguez D, Rubio-Terrés C. Cost-effectiveness analysis of second-line pharmacological treatment of acromegaly in Spain. Expert Rev Pharmacoecon Outcomes Res. 2019; 6:1-10.
Carlqvist P, Wilen-Koort A. A cost-effectiveness analysis of pasireotide long-acting compared to continued use of a first-line somatostatin antagonist for the treatment of acromegaly in Sweden. Value in Health. 2016; 19(7):A591.
Nieman LK, Biller B, Findling JW, et al. Treatment of Cushing's Syndrome: An Endocrine Society Clinical Practice Guideline.The Journal of Clinical Endocrinology & Metabolism. 2015; 100(8): 2807–2831.
Melmed S, Bronstein M, Chanson P, et al. A Consensus Statement on acromegaly therapeutic outcomes. Nature Reviews Endocrinology. 2018;14: 552–561. https://www.nature.com/articles/s41574-018-0058-5
Silverstein JM. Hyperglycemia induced by pasireotide in patients with Cushing's disease or acromegaly. Pituitary. 2016; 19(5): 536-43.
Muhammad A, van der Lely AJ, Delhanty PJD, et al. Efficacy and Safety of Switching to Pasireotide in Patients With Acromegaly Controlled With Pegvisomant and First-Generation Somatostatin Analogues (PAPE Study). J Clin Endocrinol Metab. 2018;103(2): 586-595.
Muhammad A, Coopmans EC, Delhanty PJD, et al. Efficacy and Safety of switching to Pasireotide in Acromegaly Patients controlled with Pegvisomant and Somatostatin Analogues: PAPE extension study. Eur J Endocrinol. 2018;179(5): 269-277.
Shimon I, Adnan Z, Gorshtein A, et al. Efficacy and safety of long-acting pasireotide in patients with somatostatin-resistant acromegaly: a multicenter study. Endocrine. 2018; 62(2), 448-455.
Ciresi A, Radellini S, Guarnotta V, et al. Efficacy of combined treatment with pasireotide, pegvisomant and cabergoline in an acromegalic patient resistant to other treatments: a case report. BMC Endocr Disord. 2018;18(1):2.
Tahara S, Murakami M, Kaneko T, et al. Efficacy and safety of long-acting pasireotide in Japanese patients with acromegaly or pituitary gigantism: results from a multicenter, open-label, randomized, phase 2 study. Endocr J. 2017; 64(7): 735-747.
Fleseriu M, Rusch E2, Geer EB3; ACCESS Study Investigators. Safety and tolerability of pasireotide long-acting release in acromegaly-results from the acromegaly, open-label, multicenter, safety monitoring program for treating patients who have a need to receive medical therapy (ACCESS) study. Endocrine. 2017; 5(1): 247-255.
Bronstein MD, Fleseriu M, Neggers S, et al. Switching patients with acromegaly from octreotide to pasireotide improves biochemical control: crossover extension to a randomized, double-blind, Phase III study. BMC Endocr Disord. 2016; 16:16.
Gadelha MR, Bronstein MD, Brue T, et al. Pasireotide versus continued treatment with octreotide or lanreotide in patients with inadequately controlled acromegaly (PAOLA): a randomised, phase 3 trial. Lancet Diabetes Endocrinol. 2014; 2(11): 875-84.
Sheppard M, Bronstein MD, Freda P, et al. Pasireotide LAR maintains inhibition of GH and IGF-1 in patients with acromegaly for up to 25 months: results from the blinded extension phase of a randomized, double-blind, multicenter, Phase III study. Pituitary. 2015;18(3): 385-94.
Petersenn S, Farrall AJ, De Block C, et al. Long-term efficacy and safety of subcutaneous pasireotide in acromegaly: results from an open-ended, multicenter, Phase II extension study. Pituitary. 2014; 17(2):132-40.
Petersenn S, Schopohl J, Barkan A et al. Pasireotide (SOM230) demonstrates efficacy and safety in patients with acromegaly: a randomized, multicenter, phase II trial. J Clin Endocrinol Metab. 2010; 95(6): 2781-9.
Lacroix A, Gu F, Gallardo W, et al. Efficacy and safety of once-monthly pasireotide in Cushing's disease: a 12 month clinical trial. Lancet Diabetes Endocrinol. 2018; 6(1):17-26.
Schopohl J, Gu F, Rubens R, et al. Pasireotide can induce sustained decreases in urinary cortisol and provide clinical benefit in patients with Cushing's disease: results from an open-ended, open-label extension trial. Pituitary. 2015;18(5): 604-12.
Colao A, Petersenn S, Newell-Price J, et al. A 12-month phase 3 study of pasireotide in Cushing's disease. N Engl J Med. 2012; 366(10): 914-24.
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