Economic and social aspects of growth hormone therapy in children and adults with growth hormone deficiency. Literature review

In the Russian Federation growth hormone therapy (GHT) for children with growth hormone deficient children is funded from the state budget. The clinical effectiveness of GHT with human recombinant growth hormone was proven repeatedly in both world wide and Russian practice. Nevertheless, the economic and financial aspects of this therapy are in need of a detailed analysis. Such an analysis could serve as a possible instrument for well-grounded weighed decisions in the health care sector. One such instrument is a cost-effectiveness analysis, which involves the calculation of cost-effectiveness ratios or incremental cost-effectiveness ratios for determining the economic viability of new medical technologies or programs. Moreover, social aspects of GHT are also of great importance. A physician or a health care manager need to be informed about the effect of any therapy, including GHT, on the patient’s quality of life. While there is an overall agreement about the necessity and positive effect of GHT in children, the question remains somewhat disputable in the case of adult patients with growth hormone deficiency. Both world wide and in Russia there is no uniform agreement as to weather treatment is necessary in all cases of adult growth hormone deficiency, or weather treatment should be subsidized and provided for by the state or other financial resources, or weather GHT has indeed such a profound effect on the patient’s quality of life. The present article reviews the state of discussion around these questions to date for both children and adult patients. Available data on the cost-effectiveness analyses of GHT from several countries is highlighted and the effects of GHT on the quality of life are reviewed.

1. Kirk J. Indications For Growth Hormone Therapy In Children. Arch Dis Child. 2012;97(1):63-68. doi: 10.1136/Adc.2010.186205.

2. 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.

3. 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.

4. 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.

5. Phillips C. What Is Cost-Effectiveness? Second Edition. London: Hayward Group Ltd.; 2009 Available From: http://www.medicine.ox.fc.uk|bandolier/painres/download/whatis/Cost-effect.pdf.

6. Phillips C. What Is Qaly? Second Edition. London: Hayward Medical Communications; 2009. Available from: http://www.medicine.ox.fc.uk|bandolier/painres/download/whatis/Cost-effect.pdf.

7. Нагаева Е.В. Гормон Роста - 20 Лет Применения // Проблемы Эндокринологии. — 2011. — Т. 57. — №1. — С. 71-79. [Nagaeva EV. Growth hormone - 20 years of linical practice. Probl Endokrin (Mosc). 2011;57(1):71-79. (In Russ.)]. doi: 10.14341/Probl201157171-79.

8. Al MJ. Cost-Effectiveness Acceptability Curves Revisited. Pharmacoeconomics. 2012;31(2):93-100. doi: 10.1007/S40273-012-0011-8.

9. Persson U, Hjelmgren J. Health services need knowledge of how the public values health. Lakartidningen. 2003;100(43):3436-3437.

10. Capalbo D, Esposito A, Di Mase R, et al. Update on early cardiovascular and metabolic risk factors in children and adolescents affected with growth hormone deficiency. Minerva Endocrinol. 2012;37(4):379-389.

11. Gazzaruso C, Gola M, Karamouzis I, et al. Cardiovascular isk in adult patients with growth hormone (GH) deficiency and following substitution with GH—AN Update. J Clin Endocrinol Metab. 2014;99(1):18-29. doi: 10.1210/Jc.2013-2394.

12. Sanmarti A, Lucas A, Hawkins F, et al. Observational study in adult hypopituitary patients with untreated growth hormone deficiency (ODA Study). Socio-economic impact and health status. Collaborative ODA (Observational GH deficiency in adults) Group. Eur J Endocrinol. 1999;141(5):481-489. doi: 10.1530/Eje.0.1410481.

13. Hakkaart-Van Roijen L, Beckers A, Stevenaert A, Rutten FFH. The burden of illness of hypopituitary adults with growth hormone deficiency. Pharmacoeconomics. 1998;14(4):395-403. doi: 10.2165/00019053-199814040-00006.

14. Ehrnborg C, Hakkaart-Van Roijen L, Jonsson BR, et al. Cost off illness in adult patients with hypopituitarism. Pharmacoeconomics. 2000;17(6):621-628. doi: 10.2165/00019053-200017060-00008.

15. Brook DCG. Controversy: which children should receive growth hormone treatment. Archives of Disease in Childhood. 2000;83(2):176-178. doi: 10.1136/Adc.83.2.176.

16. 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 Technology Assessment. 2002;6(18):1-168. doi: 10.3310/Hta6180.

17. Bolin K, Sandin R, Koltowska-Haggstrom M, et al. The Cost-effectiveness of growth hormone replacement therapy (Genotropin(R) in hypopituitary adults in Sweden. Cost Eff Resour Alloc. 2013;11(1):24. doi: 10.1186/1478-7547-11-24.

18. Bansback N, Brazier J, Chilcott J, et al. Clinical and cost effectiveness of recombinant human growth hormone (Somatropin) in adults. Report prepared on behalf of the National Institute for clinical excellence (NICE), 10 April 2002. School of health and related research (ScHARR), University of Sheffield. 2002.

19. Корчагин В.П. Финансовое Обеспечение Здравоохранения. М.: Российский Фонд «Здоровье Человека»; 1997. [Korchagin Vp. Finansovoe Obespechenie Zdravoohranenija. Moscow: Rossijskij Fond “Zdorov’e Cheloveka”; 1997. (In Russ).].

20. Geisler A, Lass N, Reinsch N, et al. Quality of life in children and adolescents with growth hormone deficiency: association with growth hormone treatment. Horm Res Paediatr. 2012;78(2):94-99. doi: 10.1159/000341151.

21. Ravens-Sieberer U, Bullinger M. Assessing health-related quality of life in chronically ill children with the German kindl: first psychometric and content analytical results. Qual Life Res. 1998;7(5):399-407. doi: 10.1023/A:1008853819715.

22. Winkler J, Stolzenberg H. Social class index in the Federal health survey. Gesundheitswesen. 1999;61 Spec No:S178-S183.

23. 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.

24. Oswiecimska JM, Roczniak W, Romanowicz D, et al. Quality of life in transition phase in adolescents and young adults with severe and partial growth hormone deficiency. Neuro Endocrinol Lett. 2014;35(8):676-683.

25. Mckenna SP, Doward LC, Alonso J, et al. The Qol-AGHDA: an instrument for the assessment of quality of life in adults with growth hormone deficiency. Qual Life Res. 1999;8(4):373-383. doi: 10.1023/A:1008987922774.

26. Mchorney CA, Ware JEJr, Lu JF, Sherbourne CD. The MOS 36-item Short-Form Health Survey (SF-36): III. Tests of data quality, scaling assumptions, and reliability across diverse patient groups. Med Care. 1994;32(1):40-66. doi: 10.1097/00005650-199401000-00004.

27. Andrews G, Slade T. Interpreting scores on the Kessler Psychological Distress Scale (K10). Aust N Z J Public Health. 2001;25(6):494-497. doi: 10.1111/J.1467-842x.2001.Tb00310.X.

28. Rosen R, Brown C, Heiman J, et al. The female sexual function Index (FSFI): a multidimensional self-report instrument for the assessment of female sexual function. J Sex Marital Ther. 2000;26(2):191-208. doi: 10.1080/009262300278597.

29. Abdo CH. The male sexual quotient: a brief, self-administered questionnaire to assess male sexual satisfaction. J Sex Med. 2007;4(2):382-389. doi: 10.1111/j.1743-6109.2006.00414.x.

30. Kao KT, Stargatt R, Zacharin M. Adult quality of life and Psychosocial Outcomes Of Childhood Onset Hypopituitarism. Horm Res Paediatr. 2015;84(2):94-101. doi: 10.1159/000430863.

31. Quitmann JH, Rohenkohl AC, Kammerer U, et al. Quality of life of young adults after a growth hormone therapy with childhood onset. Dtsch Med Wochenschr. 2014;139(46):2335-2338. doi: 10.1055/S-0034-1387314.

32. Radcliffe DJ, Pliskin JS, Silvers JB, Cuttler L. Growth hormone therapy and quality of life in adults and children. Pharmacoeconomics. 2004;22(8):499-524. doi: 10.2165/00019053-200422080-00003.