7194 The Use Of GloBE-Reg As A Global Platform For Performing Safety & Efficacy Studies Of rhGH Therapy - First Year Results

Abstract Disclosure: M. Alimussina: None. J. Bryce: None. M. Chen: None. S. Koley: None. A. Al-Agha: None. N. Amin: None. N. Atapattu: None. J. Chen: None. Y. Deyanova: None. A. Fu: None. C. Gong: None. V. Iotova: None. D. Janus: None. A. Jorge: Consulting Fee; Self; Novo Nordisk. Grant Recipient; Self; BioMarin. R. Markosyan: None. S. Seneviratne: None. M.G. Shaikh: None. S. Shenoy: None. S. Shepherd: None. L. Tack: None. M. Wasniewska: None. S. Chen: Research Investigator; Self; Pfizer, Inc. S.F. Ahmed: Grant Recipient; Self; GenSci, Novo Nordisk, Pfizer, Inc.. Speaker; Self; Ipsen. 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). Although safety information on rhGH therapy has now been collected for several years, there are still existing gaps in our knowledge, especially with the introduction of new indications and novel forms of rhGH. Methods: The GloBE-Reg registry has three layers of datasets, with the first dataset consisting of internationally agreed core data elements that can apply to any rare condition, another set of data elements that allows the selection of a specific therapy and diagnosis, a third set of additional data elements that is made up of the therapy and diagnosis-specific minimum dataset (MDS) and an option to have a more extended dataset. The minimum dataset collects information on the diagnosis, therapy, clinician reported outcomes, patient reported outcomes and adverse events. The platform and the registry received ethics/IRB approval in the UK and is now extending internationally. The fields within the MDS are developed following guidance from short-life expert working groups (EWG). Results: Since its launch in November 2022, 51 centres from 29 countries have registered their interest to use the platform and of these, 14 centres from 11 countries in 3 continents have already included 444 (M:F, 282:162) patients with a median age of 11 yr (range <1, 29). Of these, 317 (71%) were on daily rhGH, 126 (28%) were on long-acting rhGH, one was on daily rhGH as an adult and in one, rhGH therapy had been discussed but not started. Nine different brands of rhGH were being used across these centres for 9 groups of indications, whereas long-acting rhGH was being used for only 4 indications. In addition, there were 11 (3%) cases receiving rhGH for other conditions associated with short stature or growth retardation. The largest indication group was GHD (65%) followed by SGA (10%), TS (7%), PWS (7%), ISS (5%) and others. Of the 444 cases, 67 (15%) had also been entered in other disease registries and 18 (27%) of these 67 had been entered by one centre into the GloBE-Reg through its bulk upload facility. A Childhood GHD MDS is now fully operational and MDS for other conditions are under development. Conclusion: Since its launch, GloBE-Reg has shown that a relatively low-cost platform operated through an academic institution can have sufficient stakeholder acceptability and versatility for collecting information that can support the development of long-term safety and effectiveness studies. The preliminary data that have been collected on rhGH show the long-term utility of the platform for collecting global brand and diagnosis-specific data. Presentation: 6/2/2024