Barriers to growth hormone access in pediatric patients at an academic medical center

Evaluating barriers that may delay or limit human growth hormone (hGH) access is important to ensure timely and equitable treatment. We examined time to hGH access and whether time to approval was associated with patient factors and impacts on patient growth.

This was a single-center, retrospective review of the electronic medical records of pediatric patients starting hGH treatment for an indication approved by the FDA at the Vanderbilt Pediatric Endocrinology Clinic between January 1, 2018, and December 31, 2020. A Cox proportional hazards model was used to assess factors associated with time to access. Linear regression analysis was used to assess factors impacting patients' change in height z score from baseline to 1 year after the decision to treat (DTT).

The inclusion criteria were met by 374 patients. Patients had a median age of 11 years (interquartile range [IQR], 8-13 years), 66% were male, 80% identified as White, and the median pretreatment height z score was -2.5 (IQR, -3 to -2). The median time to access was 3 days (IQR, 1-6 days) with a prior authorization, 34 days (IQR, 22-68 days) with an appeal, 84 days (IQR, 59-122 days) with a patient assistance program, and 94 days (IQR, 46-155 days) with cash pay. Diagnosis (P = 0.002), additional testing (P < 0.0001), and method of final access (P < 0.001) were significantly associated with the time to access. Height z score at DTT (P < 0.001), diagnosis (P = 0.021), and race (P = 0.045) were significantly associated with the change in height at 1 year after DTT.

Diagnosis, insurance, additional testing, and method of medication approval were associated with time to access. Time to hGH initiation did not impact patient growth 1 year after DTT.