Economic and social aspects of pituitary dwarfism treatment with recombinant growth hormone

Rationale. Pituitary dwarfism is an orphan disease requiring pathogenetic treatment. The domestic literature lacks studies devoted to the medical and economic effectiveness of treatment for growth hormone deficiency (GHD) using recombinant growth hormone (rGH) drugs.

Aim. To analyze the cost effectiveness of rGH therapy in GHD children in the Russian Federation under the Program «7 High-Cost Nosologies».

Material and methods. We analyzed data of 50 GHD children living in 4 regions of the Russian Federation and receiving rGH therapy under the Program «7 High-Cost Nosologies». We evaluated the amount of drug consumption and the economic component: the cost of treatment and monitoring for ≥ 6 years. The incremental cost was calculated as the difference between a program providing a GHD child with rGH treatment and monitoring and an alternative program providing a GHD child with financial and social assistance and medical examination.

Results. The median cost of treatment of a single child with rGH at a dose of 0.033 mg/kg/day was 437.5 thousand rubles or 8.12 thousand US dollars for the entire analyzed period (mean, 6.95 years). The median cost of treatment per child per year was 63.6 thousand rubles/year or 1.09 thousand USD/year. Given the cost of treatment and monitoring, the integrated management of one patient cost 68.4 thousand rubles per year (470.7 thousand rubles for 6.95 years), on average, with monitoring accounting for 7.05%. The total cost of all benefits and examinations for one disabled child was 178.97 thousand rubles per year or 1,243.86 thousand rubles for 6.95 years (medical examinations accounted for 1.2%). Upon calculating the cost difference between the program providing a GHD child with treatment and monitoring and the alternative program when a GHD child was not provided with treatment, but received appropriate disability payments and medical examination, the incremental cost amounted to 110.6 thousand rubles per child per year (773.18 thousand rubles for 6.95 years).

Conclusion. The study demonstrates that treatment of GHD children under Program «7 High-Cost Nosologies» is cost-effective for both the patient and society and the state in general. For example, the incremental cost between the two programs is 110.6 thousand rubles for a year or 773.18 thousand rubles for 6.95 years. The study results indicate the importance of thorough analysis of the effects and costs in assessing the effectiveness of medical programs, especially in the case of orphan diseases.

pituitary dwarfism , treatment , monitoring , economic effectiveness , children

1. Федеральный закон от 19.12.2006 №238 «О федеральном бюджете на 2007 год» (в редакции Федерального закона от 17.07.2007 №132-ФЗ «О внесении изменений в федеральный закон «О федеральном бюджете на 2007 год»). — 2007. [Federal Law of Russian Federation on 19.12.2006 No. 238 «On the Federal Budget for 2007» (as amended by Federal Law No. 132-FZ of July 17, 2007 «On Amendments to the Federal Law» On the Federal Budget for 2007»). 2007. (in Russ.)]

2. Приказ Министерства здравоохранения и социального развития Российской Федерации от 04.04.2008 162н «О порядке ведения федерального регистра больных гемофилией, муковисцидозом, гипофизарным нанизмом, болезнью Гоше, злокачественными новообразованиями лимфоидной, кроветворной и родственных им тканей, рассеянным склерозом, лиц после трансплантации органов и (или) тканей» (утратил силу в связи с изданием Приказа Минздрава России от 15.02.2013 № 69н). — (2008). [Order No. 162n of the Ministry of Health and Social Development of the Russian Federation of April 4, 2008, «On the Procedure for Maintaining the Federal Register of Patients with Hemophilia, Cystic Fibrosis, Pituitary Nanizm, Gaucher Disease, Malignant Formation of Lymphoid, Haematopoietic and Related Tissues, Multiple Sclerosis, Persons after Organ Transplantation And (or) tissues «(lost force in connection with the publication of the Order of the Ministry of Health of Russia from 15.02.2013 No. 69n). 2008. (in Russ).]

3. Приказ Министерства здравоохранения и социального развития Российской Федерации от 15 февраля 2013 г. №69н «О мерах реализации постановления Правительства Российской Федерации от 26 апреля 2012 г. № 404 « Об утверждении Правил ведения федерального регистра лиц, больных гемофилией, муковисцидозом, гипофизарным нанизмом, болезнью Гоше, злокачественными новообразованиями лимфоидной, кроветворной и родственных им тканей, рассеянным склерозом, лиц после трансплантации органов и (или) тканей». — 2013. [Order No. 69n of the Ministry of Health and Social Development of the Russian Federation of February 15, 2013, «On Measures for the Implementation of Government Decision No. 404 of April 26, 2012» on approval of the Rules for the management of the federal register of persons affected by hemophilia, cystic fibrosis, pituitary nanism, Gaucher disease, malignant neoplasms of lymphoid, hematopoietic and related tissues, multiple sclerosis, individuals after organ and / or tissue transplantation». 2013. (in Russ).]

4. Lipwo Lipworth WL, Ho K, Kerridge IH, Day RO. Drug policy at the margins: the case of growth hormone replacement for adults with severe growth hormone deficiency. Med J Aust. 2012;197(4):204-205.

5. Christensen T, Fidler C, Bentley A, Djurhuus C. The cost-effectiveness of somatropin treatment for short children born small for gestational age (SGA) and children with growth hormone deficiency (GHD) in Sweden. J Med Econ. 2010;13(1):168-178. doi: 10.3111/13696991003652248.

6. Christensen T, Buckland A, Bentley A et al. Cost-effectiveness of somatropin for the treatment of short children born small for gestational age. Clin Ther. 2010;32(6):1068-1082. doi: 10.1016/j.clinthera.2010.05.012.

7. Phillips C. What is cost-effectiveness? Second edition. London: Hayward Group Ltd.; 2009. Available from:http://www.medicine.ox.ac.uk/bandolier/painres/download/whatis/Cost-effect.pdf.

8. Brook DCG, Kelnar CJH. Controversy: Which children should receive growth hormone treatment. Arch Dis Child. 2000;83(2):176-178. doi: 10.1136/adc.83.2.176.

9. Bryant J, Cave C, Mihaylova B et al. Clinical effectiveness and cost-effectiveness of growth hormone in children: a systematic review and economic evaluation. Health Technol Assess. 2002;6(18):1-168.

10. Tanaka T, Hasegawa T, Ozono K et al. Effect of growth hormone treatment on quality of life in Japanese children with growth hormone deficiency: an analysis from a prospective observational study. Clin Pediatr Endocrinol. 2014;23(3):83-92. doi: 10.1297/cpe.23.83.

11. Blum WF, Deal C, Zimmermann AG et al. Development of additional pituitary hormone deficiencies in pediatric patients originally diagnosed with idiopathic isolated GH deficiency. Eur J Endocrinol. 2014;170(1):13-21. doi: 10.1530/EJE-13-0643.

12. Child CJ, Blum WF, Deal C et al. Development of additional pituitary hormone deficiencies in pediatric patients originally diagnosed with isolated growth hormone deficiency due to organic causes. Eur J Endocrinol. 2016;174(5):669-679. doi: 10.1530/EJE-15-1203.

13. Федеральные клинические рекомендации (протоколы) по ведению детей с эндокринными заболеваниями. / Под ред. Дедова И.И., Петерковой В.А. — Москва: Практика; 2014. — 442 с. [Dedov II, Peterkova VA, editors. Russian clinical guidelines (protocols) for the management of children with endocrine diseases. Moscow: Praktika; 2014. 442 p.]

14. Growth Hormone Research Society. Consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency in childhood and adolescence: summary statement of the GH Research Society. GH Research Society. J Clin Endocrinol Metab. 2000;85(11):3990-3993. doi: 10.1210/jcem.85.11.6984.

15. Geffner M, Lundberg M, Koltowska-Haggstrom M et al. Changes in height, weight, and body mass index in children with craniopharyngioma after three years of growth hormone therapy: analysis of KIGS (Pfizer International Growth Database). J Clin Endocrinol Metab. 2004;89(11):5435-5440. doi: 10.1210/jc.2004-0667.

16. Cutfield W, Lindberg A, Albertsson Wikland K et al. Final height in idiopathic growth hormone deficiency: the KIGS experience. KIGS International Board. Acta Paediatr Suppl. 1999;88(428):72-75.

17. Воронцова М.В. Заболеваемость гипофизарным нанизмом в Российской Федерации по данным официальной медицинской статистики и Регистра пациентов с гипофизарным нанизмом // Проблемы эндокринологии. — 2016. — Т. 62. — №4. — С. 18-26. [Vorontsova MV. Incidence and prevalence of growth hormone deficiency in the russian federation according to the official medical statistics data and the growth hormone deficiency patients registry. Problems of Endocrinology. 2016;62(4):18-26. (in Russ)] doi: 10.14341/probl201662418-26.