Original Title: Arzneimittel-Richtlinie/Anlage XII: Selumetinib (Neurofibromatose (≥ 3 bis < 18 Jahre, Typ 1))

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: Selumetinib is approved for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above. Benefit assessment is based on the study SPRINT, an open-label, single-arm, multicentre trial on the safety and efficacy of Selumetinib in children and adolescents with symptomatic, unresectable PN due to NF1. No deaths were recorded in either phase I or phase II. Within phase II of the SPRINT trial, improvements over time were seen for worst pain and GIC-reported global assessment of clinical change for participants aged 8 to 18 years. No (separate) evaluations are available for younger study participants. Furthermore, an improvement in general health-related quality of life measured by PedsQL total score was observed over the course of the study. However, bias in favour of Selumetinib is possible due to nonresponse of the school function subscale. Severe AEs occurred in about two-thirds of participants (62%), SAEs in about one-quarter (24%). A small proportion of patients (approx. 12%) discontinued therapy due to AEs. An interpretation and assessment of the changes that occurred in the study is not possible due to the lack of a control group. The indirect comparison based on external control studies was considered inappropriate for benefit assessment and was not considered for benefit assessment due to lack of comparability of the patient populations. The effect of Selumetinib on mortality, morbidity, quality of life and safety cannot be conclusively evaluated on the basis of the single-arm trial submitted for assessment.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/726/#english

Year Published: 2022

URL for published report: https://www.g-ba.de/downloads/39-1464-5266/2022-02-03_AM-RL-XII_Selumetinib_D-714_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/726/#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/