Spandonaro F, Cappa M, Castello R, Chiarelli F, Ghigo E, Mancusi L
CREA Sanità (Consortium for Applied Economic Research in Health), University of Rome Tor Vergata, Via Columbia 2, 00133, Rome, Italy,
J Endocrinol Invest. 2014 Oct;37(10):979-90. doi: 10.1007/s40618-014-0138-x. Epub 2014 Aug 8.
Growth hormone (GH) consumption is the object of a particular attention by regulatory bodies, due to its financial impact; nevertheless, GH treatment has been demonstrated to be cost-effective and is, therefore, usually reimbursed by public health service systems. In Italy, significant differences in GH consumption between regions have been recorded. Different appropriateness in real practice is a possible explanation, but the proportion of drug wasted due to different combinations of therapeutic regimes and types of devices used in drug administration is a complementary explanation. Aim of the study is, therefore, to determine how much of the variability in GH consumption is actually due to differences in clinical practice, and how much to waste.
A model was settled to estimate the population with indication for GH administration, separately for children, transition subjects and adults, based on both the scientific evidence available and directly collected clinical evaluations. A systematic literature search was conducted using Cochrane Library (HTA and NHSEE) databases, Medline via Ovid, Econlit via Ovid, Embase.
The model applied to the Italian population showed that there was apparently unexplainable over-prescription and potential under-prescription in various regions, ranging from 20 to 40 % less than the estimated theoretical consumption to over 200 %. Wastage, at level of single device, could amount to as much as 15 % of the consumption, demonstrating that price per mg is not in general a good proxy of the cost per mg of therapy. Our estimates of the wastage shows a significant potential gap in the model assessment of the HTA bodies, as far as they do not explicitly take into account the issue of wastage and, consequently, the actual variability in local clinical practice.
由于生长激素（GH）的使用成本较高，监管机构对其使用情况格外关注；然而，生长激素治疗已被证明具有成本效益，因此通常由公共卫生服务系统报销。在意大利，各地区生长激素的使用量存在显著差异。实际使用中的不同适宜性可能是一个解释，但由于治疗方案和药物给药所用器械类型的不同组合导致的药物浪费也是一个补充解释。因此，本研究的目的是确定生长激素使用量的差异中有多少实际上是由于临床实践的差异，又有多少是由于浪费。
基于现有科学证据和直接收集的临床评估结果，建立了一个模型，分别针对儿童、过渡人群和成人，估算有生长激素给药指征的人群数量。使用Cochrane图书馆（卫生技术评估和非健康相关卫生经济评估）数据库、通过Ovid检索的Medline、通过Ovid检索的Econlit、Embase进行了系统的文献检索。
应用于意大利人群的模型显示，各地区存在明显无法解释的过度处方和潜在的处方不足情况，比估计的理论使用量少20%至40%，甚至超过200%。在单个器械层面，浪费量可能高达使用量的15%，这表明每毫克的价格通常不能很好地代表每毫克治疗成本。我们对浪费情况的估计表明，卫生技术评估机构的模型评估中存在显著的潜在差距，因为它们没有明确考虑浪费问题，因此也没有考虑当地临床实践中的实际差异。
