Therapy for Childhood Short Stature Access to Emerging Treatments: Growth Hormone Insurance Coverage, Physician Recommendations, and

Beth S. Finkelstein, PhD; J. B. Silvers, PhD; Ursula Marrero, MSSA; Duncan Neuhauser, PhD; Leona Cuttler, MDContext.— There is concern in both the medical community and the generalpublicaboutmechanismsofmedicaldecisionmakingandtheinterplayofphysicianand insurer decisions in determining access to care.Objective.— To examine the medical process influencing access to growth hor-mone (GH) therapy for childhood short stature by comparing coverage policies ofUS insurers with the treatment recommendations of US physicians.Design and Participants.— Independent national representative surveys weremailed to insurers (private, Blue Cross/Blue Shield, health maintenance organiza-tions, programs for Children with Special Health Care Needs, and Medicaidprograms, n=113), primary care physicians (n=1504), and pediatric endocrinolo-gists (n=534) with response rates of 75%, 60%, and 81%, respectively. Each sur-vey included identical case scenarios. Primary care physicians were asked deci-sionsaboutreferralstopediatricendocrinologists.EndocrinologistswereaskedGHtreatment recommendations. Insurers were asked coverage decisions for GHtherapy.MainOutcomeMeasures.— InsurercoveragedecisionsforGHinspecificcasescenarios were compared with the recommendations of primary care physiciansand pediatric endocrinologists.Results.— Physician recommendations and insurance coverage decisions dif-fered strikingly. For example, while 96% of pediatric endocrinologists recom-mendedGHtherapyforchildrenwithTurnersyndrome,insurerpoliciescoveredGHtherapy for only 52% of these children. Overall, referral and treatment decisions byphysiciansresultedinrecommendationsforGHtherapyin78%ofchildrenwithGHdeficiency,Turnersyndrome,orrenalfailure;ofthoserecommendedfortreatment,28% were denied coverage by insurers. Similarly, GH therapy would be recom-mended by physicians for only 9% of children with idiopathic short stature, but in-surers would not cover GH for the vast majority of these children. Furthermore, thedata indicated considerable variation among insurers regarding coverage policiesfor GH (P,.01).Conclusions.— AccesstoGHtherapydiffersdependingonthetypeofinsurancecoverage. The deep discord between physician recommendations and insurancecoveragedecisions,exemplifiedbythesefindings,representsamajorchallengetomechanisms of health care decision making, access, and costs.