ASSESSMENT OF GROWTH HORMONE USE IN SHORT CHILDREN

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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.