Endocrine Abstracts

JOINT2496

Introduction: The Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions (GloBE-Reg, https://globe-reg. net/) project was launched in 2022 with the aim of supporting studies that focus on effectiveness and long-term safety of specific therapies. The project’s initial focus has been on recombinant human growth hormone therapy (rhGH), given the existing knowledge gaps associated with the introduction of new indications and novel forms of rhGH.

Methods: The GloBE-Reg registry consists of three layers of datasets: the first includes internationally agreed-upon core data elements applicable to any rare condition; the second allows for the selection of a specific therapy and diagnosis; and the third comprises a therapy- and diagnosis-specific minimum dataset (MDS), which collects information on diagnosis, therapy, clinician-reported outcomes, patient-reported outcomes, and adverse events. The fields within the MDS are developed following guidance from short-life expert working groups.

Results: Since its launch, 29 centres from 17 countries in 4 continents have enrolled 2, 798 (M:F, 1, 679:1, 119) patients with a median age of 12. 8 years (range 0. 2, 64. 6), of whom 2, 481 (89%) are currently under 18 years of age. Among these patients, 1, 777 (64%) were on daily rhGH, 1, 011 (36%) on long-acting rhGH, and in 10 cases, rhGH therapy had been discussed but not initiated. Twelve different brands of rhGH were in use across these centres for eight indications, while long-acting rhGH was prescribed for six indications. Additionally, 41 (1%) patients were receiving rhGH for other conditions associated with short stature or growth retardation. The most common indication was growth hormone deficiency (59%), followed by small for gestational age (15%), idiopathic short stature (9%), Turner syndrome (8%), Prader-Willi syndrome (4%), and Noonan syndrome (2%). Of the 2, 798 cases, 1, 064 (38%) were also included in other disease registries and 923 (33%) of these were entered by three centres into GloBE-Reg through its bulk upload facility. Modules containing Childhood GHD, Adulthood GHD, and Noonan syndrome MDS are now fully operational and supporting studies in these fields; other modules are under development.

Conclusion: GloBE-Reg has demonstrated that a relatively low-cost, brand-agnostic platform can achieve sufficient stakeholder acceptability and versatility for collecting data to support long-term safety and effectiveness studies conducted by investigators from both academia and industry. The preliminary data collected on rhGH underscore the platform’s long-term utility in evaluating the safety and effectiveness of a wide range of drugs.