Original Title: Arzneimittel-​​​Richtlinie/Anlage XII: Betibeglogene autotemcel (β-Thalassämie) – therapiekosten

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: Betibeglogene Autotemcel (Beti-cel) was approved for the treatment of patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. Benefit assessment is based on the pivotal HGB-207 study, the HGB-204 and HGB-205 dose-finding studies, and the LTF-303 long-term follow-up study. The pivotal study, HGB-207, is an ongoing, open-label, single-arm, multicenter, 24-month Phase III study to evaluate the efficacy and safety of Beti-cel in patients with TDT who do not have the β0/β0-genotype. All other studies were also single-arm studies. No deaths were recorded during safety follow-up in studies HGB-207, HGB-205, and HGB-204. Overall, 60-80% of study participants were found to be transfusion-independent at the time of the last study examination, which was maintained until the end of the observation period. The change in the VAS of the EQ-5D(-Y) and the quality of life questionnaires (PedsQL, SF-36, FACT-BMT) were evaluated descriptively and no assessment of clinical relevance was performed. Additionally, the duration of follow-up was short and no external control was provided. Therefore, no conclusions can be derived regarding changes in morbidity measured by EQ-5D(-Y) VAS and quality of life measured by PedsQL, SF-36, and FACT-BMT. The safety profile of Beti-cel is subject to uncertainty due to limited data on the long-term safety of Beti-cel. This is due to the small number of study participants treated with the drug and the limitations associated with the single-arm study design. The analyses were only indicative of the safety profile of Beti-cel and reflect partially the already known safety profile of the mobilization/apheresis regimen and the conditioning regimen with busulfan. Additional results for 4 participants with IVS I-110 mutation of study HGB-212 were shown in the amendment.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/506/#english

Year Published: 2020

URL for published report: https://www.g-ba.de/downloads/39-1464-4291/2020-05-14_AM-RL-XII_Betibeglogene-autotemcel_D-497_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/506/#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/