ASSESSMENT OF GROWTH HORMONE USE IN SHORT CHILDREN

这一建议的重点是使用生物合成的人类生长激素 (GH)简而言之，孩子们。 缺乏内源性生长激素的儿童 以正常的速度生长，补充缺乏的激素可以 预防严重的侏儒症。 生物合成GH，由重组DNA衍生 技术并获得FDA批准（1985年），最初的目标是使用 GH缺乏症的儿童。 然而，目前GH的使用是 扩大，因为1）定义GH缺乏症的标准已经成为 有争议的，2）证据表明，生长激素增加生长在许多 非生长激素缺乏的矮小儿童，3）没有普遍接受的 使用GH的指南。 由于缺乏明确的指导方针， GH的潜在候选人数量范围从少于20，000，如果 经典GH缺乏症的严格标准适用于超过1，900，000 如果适应症自由化， 身高低于百分之五的儿童。 鉴于这种 不确定性，GH的使用不仅会受到其数据的影响， 医疗结果（即对矮小儿童身高的影响）， 它的价值的看法，医生，家庭的短 儿童和第三方付款人。 人们对这些地区知之甚少。 因此，该项目的具体目标是确定 GH治疗的候选人，父母对短期的态度， 身高和他们对身高增加治疗的偏好， 医生关于身材矮小和生长激素的意见和做法 治疗，以及第三方支付者对GH治疗的政策。 这将通过多种数据收集方法来实现- 包括利用现有数据来源估计 GH的潜在美国候选人，详细的面试和偏好 对矮小儿童父母的分析，一项全国医生调查， 以及对第三方付款人的全国性研究。 这些研究将决定 GH使用模式在当前情况下，并将导致一个模型 以预测在不同情况下对生长激素的未来需求。 通过这项研究的结果，我们将能够预测 成本、保险范围、疗效数据变化的后果，以及 生长激素的管理途径对未来的生长激素利用模式。 这些结果对于制定合理的政策指导方针是必要的 使用GH。 此外，开发的方法-很可能是 广泛适用于其他情况，其中a）治疗可以 超出最初的目标人群，B）区别 疾病治疗和美容治疗之间的关系变得模糊，c）- 治疗的实施受消费者偏好的影响， 以及通过医生实践。

This proposal focuses on the use of biosynthetic human growth hormone (GH) in short children. Children who are deficient in endogenous GH fail to grow at a normal rate, and replacement of the deficient hormone may prevent severe dwarfism. Biosynthetic GH, derived by recombinant DNA technology and approved by the FDA (1985), was initially targeted for use in children with GH deficiency. However, the current use of GH is broadening because 1) the criteria for defining GH deficiency have become controversial, 2) evidence suggests that GH increases growth in many non-GH deficient short children, and 3) there are no generally accepted guidelines for the use of GH. In the absence of clear guidelines, the potential number of candidates for GH ranges from fewer than 20,000 if strict criteria for classical GH deficiency are applied to over 1,900,000 with a cost of $27 billion if indications are liberalized to include children who fall below the-fifth percentile for height. Given this uncertainty, the use of GH will be influenced not only by data on its medical outcome (i.e. its effect on height of short children) but also by the perceptions of its value among physicians, families of short children, and third party payers. Little is known about these areas. The specific aims of this project are, therefore, to determine the number of candidates for GH therapy, the attitudes of parents towards short stature and their preferences regarding height augmentation therapy, the opinions and practices of physicians regarding short stature and GH treatment, and the policies of thirdparty payers towards GH therapy. This will be accomplished by multiple methods of data collection - including the use of existing data sources to estimate the number of potential U.S. candidates for GH, detailed interviews and preference analysis with parents of short children, a national survey of physicians, and a national study of third party payers. These studies will determine patterns of GH use under current circumstances, and Will lead to a model for projecting the future demand for GH under different circumstances. Through the results of this study, we will be able to project the consequences of changes in cost, insurance coverage, efficacy data, and route of administration for GH on future patterns of GH utilization. These results are needed for the generation of rational policy guidelines for the use of GH. In addition, the approach developed-is likely to be widely applicable to other situations in which a) treatments may be expanded beyond the initially targeted population, b) the distinction between disease therapy and cosmetic therapy becomes blurred, and c) the- implementation of a treatment is influenced by consumer preference as well as by physician practice.