To Prescribe or Not to Prescribe? Consumer Access to Life-Enhancing Products.

With rapid biotechnological advances in specialty drugs and direct-to-consumer advertising, consumers are under tremendous pressure to look, perform, feel, and live better. This is often accomplished through the use of life-enhancing products, sometimes referred to as performance-enhancing products, which can be accessed only through a gatekeeper, such as a physician. Integrating consumer and medical research, this article investigates how physicians make trade-offs between objective medical and nonmedical factors to determine consumers' access to life-enhancing products by examining US pediatric endocrinologists' prescription decisions for growth hormone (GH) for healthy but short children. The results of a conjoint study indicate that consumer medical criteria have less impact on a physician's decision to prescribe GH if the consumer requests a prescription or the physician believes in the intangible product benefits, and more impact when the product is more expensive. A physician's length of experience increases the impact of consumer medical criteria and decreases the influence of a consumer's preference for a prescription on the decision to prescribe. Overall, this research shows that not all consumers have equal access to life-enhancing products; their access depends on a complex combination of medical and nonmedical factors related to the consumer, product, and the physician.

A national study of physician recommendations to initiate and discontinue growth hormone for short stature.

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.

Growth hormone and health policy.

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.

Patient, physician, and consumer drivers: referrals for short stature and access to specialty drugs.

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.

Physician decisions to discontinue long-term medications using a two-stage framework: the case of growth hormone therapy.

BACKGROUND: Physician decisions to discontinue prescription medications for chronic conditions are fundamental determinants of drug use but have been inadequately studied. The decision to stop growth hormone (GH) therapy is an important example because of high cost (approximately $26,000/y for a 48-kg child), complexity of treatment options, and expansion of patient populations. AIM: The aim of this study was to identify the factors that influence physician recommendations in the process of discontinuing therapy. DESIGN: A random sample of half of U.S. pediatric endocrinologists (n = 265) was mailed a survey that included case scenarios of GH-deficient adolescents. Decision options involved a 2-stage framework to 1) initiate change in ongoing GH therapy (by discussing discontinuing GH with the family but not yet stopping treatment), and 2) take action to discontinue ongoing GH therapy (by terminating GH or reducing the dose to adult maintenance level). MAIN OUTCOME MEASURE: Physician recommendations. RESULTS: The response rate was 83.8%. Physiological indices of growth potential (growth velocity, bone age) significantly influenced discontinuation decisions (both P < 0.001). However, family preference, child's height, and physician attitudes exerted independent effects (each P < 0.05). Treatment price had little influence. Together, these variables accounted for 60% to 70% of the variation in recommendations. Their relative influence differed by stage in the discontinuation process. CONCLUSION: The variables in our framework substantially explain discontinuation decisions. The data demonstrate the importance of both physiological and nonphysiological factors. The results suggest that physicians value even small gains as final height approaches, although an additional 20% expenditure may be needed to gain the last 1% to 3% of adult height.

Growth hormone therapy and quality of life in adults and children.

A fundamental goal of growth hormone (GH) treatment for both adults and children is improvement in quality of life (QOL). Assessments of the therapeutic role of GH depend on its effectiveness in meeting this and other goals (including improved metabolic status in adults and improved growth in children) in relation to economic parameters. However, there are difficulties in interpreting data on GH treatment and QOL. These include controversy about appropriate definitions and measures for assessing QOL, disease adaptation, comorbid conditions, and potential patient selection bias. In GH-deficient adults who have completed linear growth, there is considerable evidence that GH exerts effects on body composition, serum lipids, and bone and mineral density. Several controlled trials have also examined the effect of GH treatment on QOL in GH-deficient adults. They generally indicate improvement in QOL with GH treatment, although there are inconsistencies in the data. Caveats include differing outcome measures and instruments, instruments that are not disease specific, variation in characteristics of patient samples and treatment protocols, evidence of a placebo effect, and some inconsistency among results. Open-label trials in adults also suggest improvement in QOL with GH treatment, although interpretation is limited by potential placebo effects and patient self-selection. Studies in children have generally addressed psychological status, and relatively few specifically focus on QOL. In children with classical GH deficiency, it is intuitive that GH treatment will improve QOL, although hard data are lacking. In children with idiopathic short stature, evidence for improved QOL as a result of GH treatment is not well developed. Translating changes in QOL, together with physiological and metabolic benefits, into economic cost-benefit or cost-effectiveness analyses are needed. In doing so, it will be important to consider subgroups of patients who may derive differential benefit from GH treatment. These analyses are central to the development of a framework for research, decision making, and policy for GH treatment.

IS/IT the prescription to enable medical group practices attain their goals.

The US spends significantly more money as a percentage of GDP on health care than any other OECD country and more importantly, this amount is anticipated to increase exponentially. In this high cost environment, two important trends have occurred: (1) the movement to managed care, and (2) large investments in Information Systems/Information Technology (IS/IT). Managed care has emerged as an attempt to provide good quality yet cost effective health care treatment. Its implications are not well discussed in the literature while, its impact on different types of medical group practices is even less well understood. The repercussions of the large investments in IS/IT on the health care sector in general and on the medical group practice in particular, although clearly of importance, are also largely ignored by the literature. This study attempts to address this significant void in the literature. By analyzing three different types of group practices; an Independent Practice Association (IPA), a Faculty Practice and a Multi Specialty Group Practice in a managed care environment during their implementation of practice management/billing systems, we are able to draw some conclusions regarding the impacts of these two central trends on health care in general as well as on the medical group practice in particular.

Patient attitudes and preferences regarding treatment: GH therapy for childhood short stature.

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.

Medical decision-making and the patient: understanding preference patterns for growth hormone therapy using conjoint analysis.

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.

Patient and hospital characteristics associated with patient assessments of hospital obstetrical care.

OBJECTIVES: The goals of this study were to examine the relationship of patient assessments of hospital care with patient and hospital characteristics. In addition, the authors sought to assess relationships between patient assessments and other patient-derived measures of care (eg, how much they were helped by the hospitalization and amount of pain experienced). METHODS: The authors surveyed 16,051 women (response rate, 58%) discharged after labor and delivery from 18 hospitals during the study period of 1992 to 1994. Patient assessments were obtained using a previously validated survey instrument, Patient Judgment of Hospital Quality, that includes eight scales assessing different aspects of the process of care (eg, physician care, discharge procedures) and other single item assessments (eg, overall quality). For this study, we utilized five of the scales (physician care, nursing care, information, discharge preparation, global assessments [willingness to brag, recommend or return to the hospital]). For analysis, items were rated on a five-point ordinal scale from poor to excellent. For scoring purposes, responses were transformed to linear ratings, ranging from 0 to 100 (eg, 0 = poor care, 100 = excellent care). RESULTS: In multivariable analyses, the authors found that patients who were older, white, not married, uninsured or had commercial insurance, and in better health status were significantly more likely to give higher assessments (P < 0.01), although very little of the variance in assessment scores was explained by these characteristics (2%-3%). In bivariate analyses, patient assessments were higher in nonteaching hospitals and those with fewer beds, fewer deliveries, lower cesarean-section (C-section) rates, fewer patients with Medicaid, and higher rates of vaginal births after C-section deliveries. When these variables were utilized as independent predictors in multivariable analyses using adjusted nested linear regression (to account for clustering of patients), few of the hospital characteristics reached a level of statistical significance. Finally, correlations between the five scales and other patient assessments of quality, such as how much they were helped by the hospitalization, were statistically significant (P < 0.01) and high in magnitude, ranging from 0.47 to 0.61. CONCLUSIONS: Although hospital scores differed according to several patient and hospital characteristics, the magnitude of the associations was relatively small. The findings suggest that, with respect to obstetric care, patient assessments may represent a robust measure that can be applied to diverse hospitals and patient casemix.

Insurance coverage, physician recommendations, and access to emerging treatments: growth hormone therapy for childhood short stature.

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.

Short stature and growth hormone therapy. A national study of physician recommendation patterns.

OBJECTIVE: To determine current expert opinion and recommendations regarding the controversial issue of the use of growth hormone (GH) to treat short children who do not have classical GH deficiency (non-GHD children). STUDY DESIGN: Analysis of a national survey mailed to 534 US physician experts on the management of short stature (pediatric endocrinologists) with a response rate of 81.3%. MAIN OUTCOME MEASURE: The experts' GH treatment recommendations. RESULTS: The physicians reported that approximately 58% of their current patients undergoing GH therapy have classical GH deficiency, while 42% have other conditions. The proportion of physicians who recommended GH treatment of short non-GHD children ranged from 1% to 74% over all case scenarios presented. The likelihood of GH being recommended depended on the physiological growth characteristics of the child (ie, the child's height, growth rate, and predicted adult height), contingency factors (ie, strong family wishes or a reduction in GH cost), and physician beliefs (ie, the impact of short stature on well-being, the effectiveness of GH therapy). Each of these factors exerted highly significant, independent, and additive effects on decisions to recommend GH. CONCLUSION: Our results indicate that many pediatric endocrinologists consider GH treatment appropriate for selected short non-GHD children, going beyond current Food and Drug Administration-approved indications for GH. Decisions to recommend GH for a non-GHD child rest on a combination of medical, social, and perceptual factors; variations in treatment patterns stem from variations in these influences. Future GH use will likely be determined not only by the results of controlled trials, but also by family preferences, producer pricing, and physician perceptions of the value of height and GH therapy.

Cost-effectiveness of emergency intraarterial intracerebral thrombolysis: a pilot study.

PURPOSE: To assess the clinical efficacy and cost-effectiveness of emergency thrombolysis as a treatment strategy for thromboembolic intracerebral events. METHODS: Thirty-four patients with symptoms suggestive of middle cerebral artery occlusion were included. Eight of these patients were treated with intraarterial urokinase. Effectiveness was determined by comparing the admission National Institutes of Health stroke score to the 24-hour National Institutes of Health stroke score. The cost and length of stay of both populations were derived and used as measures of direct cost. The likelihood of admission to extended care facilities and estimated length cost of admission was used as a measure of indirect cost. RESULTS: The control population became slightly worse, with a change in National Institutes of Health score of -0.5, whereas the treated population improved slightly, with a change in National Institutes of Health score of +5.12. Analysis of the direct costs data between the two populations revealed a slight increased mean for the treated population ($15,202) as compared with the control population ($13,478). The unpaired t test, however, revealed no significant cost difference between the two groups. By reducing the number of completed strokes by one third or by decreasing the severity by the same factor (as shown in our study), the likelihood of admission to an extended nursing facility also is decreased. The cost saving per patient from extended care facilities is approximately $3435. CONCLUSION: The emergency application of intraarterial thrombolysis with urokinase results in a statistically significant positive change in National Institutes of Health score by at least five points. A statistically significant benefit is realized through the use of intraarterial urokinase. A statistically insignificant additional cost is shown by this study. This insignificant cost is more than offset by the saved nursing home costs.

Cost justification of a community health information network: the ComputerLink for AD caregivers.

Recent studies examined the perceived benefits of the ComputerLink, a specialized computer network for caregivers of persons with Alzheimer's Disease. The ComputerLink existed as a special service within a free, public access community health information network. The issue of "free", however, is debatable. Though usage costs were not incurred by the AD caregivers who used ComputerLink, there are direct and indirect costs associated with these and other community networks. This study will focus on costs incurred at the individual level, recognizing that organizations and society also bear community network costs. At this level, the primary cost savings occur with the caregiver. The biggest factor determining net economic benefits is the extent to which this technology intervention delays or prevents institutionalization. Results of this analysis suggest that community health networks do return economic and social benefits to their users.