Original Title: Arzneimittel-Richtlinie/Anlage XII: Somatrogon (Wachstumsstörung durch Wachstumshormonmangel, ≥ 3 bis < 18 Jahre)

Authors' objectives: The Federal Joint Committee [Gemeinsamer Bundesausschuss (G-BA)] has had the legal task of carrying out an (additional) benefit assessment for all newly approved drugs with new active ingredients immediately after market entry (§ 35a SGB V). The result of this assessment is the basis for deciding how much the statutory health insurance pays for a new drug with a new active ingredient. The G-BA was commissioned to carry out the benefit assessment through the Pharmaceuticals Market Reorganisation Act [Gesetz zur Neuordnung des Arzneimittelmarktes (AMNOG)]. In the context of the early benefit assessment of medicinal products containing new active substances, the following rules apply to orphan drugs: According to the legal requirements (§ 35a SGB V), the additional medical benefit of these drugs is already considered to be proven by the approval. The G-BA determines the extent of the additional benefit for orphan drugs that do not exceed a turnover of 50 million Euros in the last twelve calendar months, on the basis of the approval and the studies justifying the approval.

Authors' results and conclusions: Somatrogon (Ngenla®) is indicated for the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone (GH). The benefit assessment is based on the open-label, randomised, controlled phase III CP-4-006 study with data cut-off in month 12. In the study, patients were randomised in a 1:1 ratio to the intervention arm (weekly dose of somatrogon; N = 109) or the comparator arm (daily dose of genotropin; N = 115). The study enrolled paediatric patients aged 3 to < 11 years (for girls) and < 12 years (for boys). The bone age of the study participants is not older than the chronological age and should be < 10 years for girls and < 11 years for boys. The enrolled patients had to have either an isolated growth hormone deficiency (GHD) or a GHD as part of a multiple pituitary hormone deficiency. Confirmation of the diagnosis of GHD was made by two different GH stimulation tests at a maximum GH concentration of ≤ 10 ng/ml measured by a local or central laboratory. The primary endpoint was the annualized growth velocity in cm/year after 12 months of treatment, which was only presented additionally in the benefit assessment due to lack of patient relevance. There were no deaths in the study. Regarding morbidity, the endpoint of body height (z score), showed no statistically significant difference between the treatment arms. Quality of life was assessed by the Quality of Life in Short Stature Youth Questionnaire (QoLISSY). No statistically significant difference was found between treatment arms for the QoLISSY. For the endpoint category side effects, no statistically significant difference was observed between the treatment arms for serious and severe adverse events (AE). In the study, one subject in the somatrogon arm discontinued treatment due to AE. However, this was not statistically significant different between treatment arms. In total, the potential of bias is assessed as high due to the open-label study design. Uncertainties remain due to the cut-off value used in the CP-4-006 study to diagnose GHD.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/815/#english

Year Published: 2022

URL for published report: https://www.g-ba.de/downloads/39-1464-5619/2022-09-15_AM-RL-XII_Somatrogon_D-800_EN.pdf

Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/815/#nutzenbewertung

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Germany

Organisation Name: The Federal Joint Committee

Contact Address: Gutenbergstr. 13, 10587 Berlin, Germany

Contact Name: Fachberatung Medizin [Department of Medical Consultancy]

Contact Email: Fachberatung-Medizin@g-ba.de

Copyright: https://www.g-ba.de/sys/impressum/