Patient, Physician, and Consumer Drivers

Abstract

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.

Short Stature in Childhood — Challenges and Choices

“…Management decisions often evolve from primary care physicians’ threshold for specialist consultation to rule out pathologic causes of shortness, pediatric endocrinologists’ perspective about use of growth-promoting medications, insurance, 13–16 and parents concerned that their child is “noticeably shorter than the other kids” or “teased because of his/her size.” Their potentially valid concern - “my child is short and needs help to be taller”- ranges in meaning from “will s/he be disabled by short stature as an adult?” to “will s/he be disadvantaged in social and career success?” 17 to “would s/he feel better if s/he were taller?” 18 …”

Warning about Warnings: Weighing Risk and Benefit When Information Is in a State of Flux

“…In the case of decisions regarding rhGH, there is evidence that factors such as parental concerns and physician attitudes [13,14] heavily influence clinical management. In the case of children with ISS, pediatricians are far more likely to make referrals to endocrinologists for possible use of rhGH when families express concern.…”

“…Beliefs about the quality-of-life benefits of rhGH treatment continue to influence conversations between providers, young patients, and their parents. Attitudes toward short stature held by the general public and physicians are informed by negative stereotypes [9,10,11,12,13,14], despite the absence of evidence for problems in psychosocial adaptation among youths with idiopathic short stature [15,16]. The known social consequences of short stature (e.g.…”

“…In the case of children with ISS, pediatricians are far more likely to make referrals to endocrinologists for possible use of rhGH when families express concern. They are also more likely to refer if they hold beliefs that short stature negatively affects a child’s emotional well-being [13]. …”

Short Stature: Psychosocial Interventions

“…Since the clinical diagnosis and endocrine management of SS have expanded dramatically since the advent of biosynthetic GH, it is possible that clinicians’ personal beliefs regarding the psychosocial liabilities associated with SS figure prominently in treatment decisions [4,5]. These beliefs have been shaped by older clinical research focusing on psychological adaptation among highly selected samples of patients (e.g., panhypopituitarism, achondroplasia, Turner syndrome) for whom SS was one feature of an otherwise complex medical condition (for a review, see Meyer-Bahlburg [6]).…”