Real-World Registry Data for Long-Acting GH Analogs — SkybriGHt, SkyPASS, ANSWER, and Beyond

This episode explores how real-world registries are filling the evidence gaps left by clinical trials in GHD management, with a focus on treatment patterns, safety surveillance, and long-term outcomes for both pediatric and adult patients.

Dr. Yang introduces the SkyBright and SkyPass registries, established specifically to track real-world use of lonapegsomatropin in pediatric GHD patients. First-year data (100 patients) presented at the Pediatric Endocrine Society showed that real-world average IGF-1 was lower (+1.3 SDS) than the enliGHten trial average (+1.8 SDS), likely because clinicians in practice are more conservative with dosing than in trials. Some providers also initiated at doses below the labeled 0.24 mg/kg/week.

Dr. Garcia highlights the ANSWER registry, designed to track real-world adult GHD outcomes. He notes that cancer survivor data — a particularly vulnerable and high-need subgroup — will only emerge meaningfully from large real-world registries over time. This population’s long-term GH safety profile cannot be adequately addressed in short-duration clinical trials.

The panel draws lessons from legacy registries — KIGS and NCGS — which tracked daily GH outcomes over decades, ultimately confirming the long-term safety and efficacy of daily GH. The panel anticipates that similar maturation will occur for long-acting analogs, and that long-term registry data will be the deciding factor for clinicians who remain hesitant.

Dr. Yang notes important methodological improvements: in 2023, the FDA issued guidance to standardize registry design and data collection, improving the comparability and regulatory relevance of registry outputs. Non-pharma-sponsored registries — such as GloBE-Reg and the German INSIGHTS-GHT registry — are also capturing comparative daily vs. weekly outcomes without commercial bias.

In the next episode, “Dosing Long-Acting GH Analogs — Practical Considerations for Pediatric Patients,” the panel dives into the practical nuances of dosing all three long-acting analogs in children, including pen device limitations, rounding strategies, and IGF-1-guided titration.