Original Title: Arzneimittel-Richtlinie/Anlage XII: Imlifidase (Desensibilisierung bei Nierentransplantation)

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: Imlifidase is indicated for desensitization treatment of highly sensitized adult kidney transplant patients with positive crossmatch against an available deceased donor. The use of imlifidase should be reserved for patients unlikely to be transplanted under the available kidney allocation system including prioritization program for highly sensitized patients. The benefit assessment was based on the pivotal studies 13-HMedIdeS-03 (S03), 14-HMedIdeS-04 (S04) and 15-HMedldeS-06 (S06), the follow-up study 17-HMedldeS-14 (S14) as well as 13-HMedldeS-13 (S13). Study S03 (N=5), S04 (N=17), S06 (N=19) were open label non-randomized phase II (S04 phase I/II) studies with a study duration of 180 days. A retrospective study (S13) was conducted to collect additional baseline data on donors and recipients and on specific endpoints (e.g., renal function) for study S03. Study S14 is an ongoing prospective, non-randomized, long-term observational study of participants who received imlifidase prior to transplantation with a planned duration period of 5 ½ years. Interim-results were presented for data cut point September 30th, 2019. No deaths occurred during the observation period of 180 days. However, 2 subjects died 10.5 and 12 months after completion of the studies S-04 and S-06. In study S14, none of the subjects actively enrolled died until the data cut point. By month 6, one and two graft losses were observed in study S04 and S06, respectively and three graft losses occurred between the end of study S04 and inclusion into study S14. The proportion of subjects (n=1-2) with an estimated glomerular filtration rate

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/661/#english

Year Published: 2021

URL for published report: https://www.g-ba.de/downloads/39-1464-5002/2021-09-02_AM-RL-XII_Imlifidase_D-647_BAnz.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/661/#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/