Endocrine Abstracts

Introduction: The Global Registry for Novel Therapies in Rare Bone & Endocrine Conditions (GloBE-Reg, https://globe-reg.net/) was launched in 2022 to support studies evaluating the effectiveness and long-term safety of specific therapies. Due to gaps in knowledge about new formulations and indications, its initial focus has been on recombinant human growth hormone (rhGH),

Methods: GloBE-Reg is structured in three dataset layers: (1) internationally agreed core elements for any rare condition; (2) therapy- and diagnosis-specific selection; and (3) a minimum dataset (MDS) tailored to the specific therapy and condition. The MDS collects information on diagnosis, treatment, clinician- and patient-reported outcomes, and adverse events and is developed with input from short-life international expert working groups.

Results: Since its launch, 32 centres from 20 countries in 4 continents have enrolled 3,580 (M:F, 2,155:1,425) patients with a median age of 12.9 years (range 0.3, 67.1), of whom 3,180 (89%) are currently under 18 years of age. Among these, 1,979 (55%) were on daily rhGH, 1,572 (44%) on long-acting rhGH. In 19 cases, rhGH therapy had not been initiated while 10 cases had discontinued the treatment. Fifteen different brands of rhGH were in use across these centres for eight indications, while long-acting rhGH was prescribed for seven indications. The most common indication was short stature due to growth hormone deficiency (60%), small for gestational age (SGA) (13%), idiopathic short stature (10%), Turner syndrome (7%), Prader-Willi syndrome (PWS) (3%), and Noonan syndrome (NS) (3%). Additionally, 61 (2%) patients were receiving rhGH for conditions that were not a recognised indication. Of the 3,580 cases, 1,166 (33%) were also included in other disease registries and 1,156 (32%) of these were entered by three centres into GloBE-Reg through its bulk upload facility. Modules containing Childhood GHD, Adulthood GHD, and NS MDS are now fully operational and are currently supporting two studies led by industry and four studies led by investigators; other modules that are currently under development include PWS and SGA.

Conclusion: The GloBE-Reg project has shown that a low-cost, drug, indication and region-agnostic platform can be widely accepted and can support several long-term safety and effectiveness studies.