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Objectives: Overall growth hormone (GH) use depends on decisions to both initiate treatment and continue treatment. The determinants of both are unclear. We studied how physicians decided to begin GH in idiopathic short stature and how, after an initial course of treatment, they decided to continue, intensify (increase the dose), or terminate treatment., Methods: We used a national census study of 727 pediatric endocrinologists involving a structured questionnaires with a factorial experimental design. Main outcome measures were GH recommendations for previously untreated children and those children who were treated with GH for 1 year., Results: The response rate was 90%. In previously untreated children, recommendations to initiate GH were consistent with guidelines and also influenced by family preferences and physician attitudes (P<.001). In children treated with GH, recommendations on whether to continue GH were influenced by the growth response to therapy (P<.01) but were divided regarding course of action. With identical growth responses to treatment, physician decisions diverged (intensify versus discontinue GH) and were driven by independent, nonphysiologic, and contextual factors (eg, physician attitudes, family preferences, and GH-initiation recommendation; each P<.001). Together, attitudinal and contextual factors exerted more influence on continuation decisions than did the growth response to therapy., Conclusions: Physician decisions to initiate GH are largely consistent with evidence-based medicine. However, decisions about continuing GH vary and are strongly influenced by factors other than response to treatment. With a potential market of 500 000 US children and costs exceeding $10 billion per year, changes in GH use may depend on potentially modifiable physician attitudes and family preferences as much as physiologic evidence.

GH treatment for short children is representative of many frontline issues in health care policy. In this paper, we highlight key policy issues exemplified by GH, focusing on pharmaceutical innovation, insurance coverage and pricing, and physician decisions, and we discuss their implications for endocrinology and GH use.

Background: Candidates for specialty drugs, the fastest growing and costliest pharmaceuticals, typically originate with primary care referrals. However, little is known about what drives such referrals-especially for large populations such as short, otherwise normal children (idiopathic short stature). Recent expanded approval of growth hormone (GH) makes more than 585,000 US children eligible for such treatment, potentially costing over $11 billion/y., Methods: To quantify the relative impact of patient physiological indicators, physician characteristics, and consumer preferences on referrals to endocrinologists (and potential access to GH) for short children, a national study of 1268 randomly selected US pediatricians was conducted, based on a full factorial experimental design in a structured survey., Results: While patient indicators (height, growth pattern) influenced referrals (P < 0.001), consumer drivers (family concern) and physician attitudes had almost as great an impact-especially for children with less severe growth impairment (P < 0.001). Physician belief that short stature impairs emotional well-being and physician characteristics (female, older, shorter, beliefs about drug company information) increased referrals (P < 0.03-0.001)-independent of growth parameters., Conclusions: Referral recommendations that create the pool of candidates for the specialty drug GH are heavily swayed by physician characteristics and consumer preferences, particularly in the absence of compelling physiological evidence. This makes most of children with short stature strikingly susceptible to nonphysiological influences on referrals that render them candidates for this specialty drug. Only 1 additional referral per US pediatrician would likely increase GH costs by over $100 million/y.

This paper examines the role of parents' attitudes and preferences regarding growth hormone therapy for childhood short stature. Four main questions are addressed. First, what are the demographic characteristics of families seeking medical advice for their child's short stature? Second, what are parents' attitudes towards short stature? Third, what are parents' treatment preferences (i.e. what characteristics of growth treatments are important to parents)? Finally, how do the attitudes of parents affect physician decision making? Several studies are reviewed and data are presented to answer these questions.

Objectives: This study examines two questions that relate to patients' role in medical decision making: (1) Do patients utilize multiple attributes in evaluating different treatment options?, and (2) Do patient treatment preferences evidence heterogeneity and disparate patterns? Although research has examined these questions by using either individual- or aggregate-level approaches, the authors demonstrate an intermediate level approach (ie, relating to patient subgroups)., Methods: The authors utilize growth augmentation therapy (GAT) as a context for analyzing these questions because GAT reflects a class of nonemergency treatments that (1) are based on genetic technology, (2) aim to improve the quality (rather than quantity) of life, and (3) offer useful insights for the patient's role in medical decision making. Using conjoint analysis, a methodology especially suited for the study of patient-consumer preferences but largely unexplored in the medical field, data were obtained from 154 parents for their decision to pursue GAT for their child., Results: In all, six attributes were utilized to study GAT, including risk of long-term side effects (1:10,000 or 1:100,000), certainty of effect (50% or 100% of cases), amount of effect (1-2 inches or 4-5 inches in adult height), out-of-pocket cost ($100, $2,000, or $10,000/year) and child's attitude (likes or not likes therapy). An experimental design using conjoint analysis procedures revealed five preference patterns that reflect clear disparities in the importance that parents attach to the different attributes of growth therapy. These preference patterns are (1) child-focused (23%), (2) risk-conscious (36%), (3) balanced (23%), (4) cost-conscious (14%), and (5) ease-of-use (4%) oriented. Additional tests provided evidence for the validity of these preference patterns. Finally, this preference heterogeneity related systematically to parental characteristics (eg, demographic, psychologic)., Conclusions: The study results offer additional insights into medical decision making with the consumer as the focal point and extend previous work that has tended to emphasize either an individual- or aggregate-based analysis. Implications for researchers and health care delivery in general and growth hormone management in particular are provided.

Context: There is concern in both the medical community and the general public about mechanisms of medical decision making and the interplay of physician and insurer decisions in determining access to care., Objective: To examine the medical process influencing access to growth hormone (GH) therapy for childhood short stature by comparing coverage policies of US insurers with the treatment recommendations of US physicians., Design and Participants: Independent national representative surveys were mailed to insurers (private, Blue Cross/Blue Shield, health maintenance organizations, programs for Children with Special Health Care Needs, and Medicaid programs, n=113), primary care physicians (n=1504), and pediatric endocrinologists (n=534) with response rates of 75%, 60%, and 81%, respectively. Each survey included identical case scenarios. Primary care physicians were asked decisions about referrals to pediatric endocrinologists. Endocrinologists were asked GH treatment recommendations. Insurers were asked coverage decisions for GH therapy., Main Outcome Measures: Insurer coverage decisions for GH in specific case scenarios were compared with the recommendations of primary care physicians and pediatric endocrinologists., Results: Physician recommendations and insurance coverage decisions differed strikingly. For example, while 96% of pediatric endocrinologists recommended GH therapy for children with Turner syndrome, insurer policies covered GH therapy for only 52% of these children. Overall, referral and treatment decisions by physicians resulted in recommendations for GH therapy in 78% of children with GH deficiency, Turner syndrome, or renal failure; of those recommended for treatment, 28% were denied coverage by insurers. Similarly, GH therapy would be recommended by physicians for only 9% of children with idiopathic short stature, but insurers would not cover GH for the vast majority of these children. Furthermore, the data indicated considerable variation among insurers regarding coverage policies for GH (P<.01)., Conclusions: Access to GH therapy differs depending on the type of insurance coverage. The deep discord between physician recommendations and insurance coverage decisions, exemplified by these findings, represents a major challenge to mechanisms of health care decision making, access, and costs.