Original Title: Arzneimittel-​​​Richtlinie/Anlage XII: Burosumab (neubewertung nach fristablauf: hypophosphatämie)

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: CRYSVITA is indicated for the treatment of X-linked hypophosphataemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons. The benefit assessment is based on the pivotal study UX023-CL301, a multicentre, randomized controlled, open-label phase III study evaluating the efficacy and safety of CRYSVITA compared to oral intake of phosphate and active vitamin D in children with XLH aged 1 to ≤ 12 years. The study population included 61 patients, 29 in the intervention arm and 32 in the control arm, who were randomised based on the three stratification factors rickets severity, age and region. The study population was characterised by a nearly balanced proportion of girls and boys. The study was divided into a screening, treatment and extension period and included data up to week 64. Maximum study duration was 140 weeks. Primary endpoint was a comparison of changes in RGI-C total score from baseline to week 40 between intervention and control arm. Deaths were documented during safety recording. No person died during the course of the study. There were statistically significant advantages for CRYSVITA over the control group in the change in z-score of body height/recumbent length and in the absolute walking distance of the 6MWT. No differences were reported between study arms in the percentage of expected walking distance, pain interference, physical function and fatigue by PROMIS, and pain intensity by FPS-R. Due to limitations in the psychometric properties, results on quality of life could not be taken into account. More adverse events occurred in the intervention arm compared to the control arm for the same observation periods, yet the trustworthiness of these results was substantially affected by several factors. A conclusive assessment of the safety profile was not possible. No data was available for the population of 13- to 17-year-old XLH-patients.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/497/#english

Year Published: 2020

URL for published report: https://www.g-ba.de/downloads/39-1464-4239/2020-04-02_AM-RL-XII_Burosumab_D-492_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/497/#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/