Original Title: Birkenrindenextrakt (Wundbehandlung bei Epidermolysis bullosa (ab 6 Monaten))

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: Birch bark extract (Filsuvez) is approved for the treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients 6 months and older. The benefit assessment is based on the study EASE. The Study EASE is a randomized, controlled, 90-day double-blind (DB) phase 3 study with a 24-month open-label follow-up evaluating the efficacy and safety of the birch bark extract in the treatment of partial thickness wounds in subjects with inherited EB. 109 subjects were randomized to the birch bark extract gel and 114 to the control gel. The risk of bias is assessed as low at study level. The 90-day DB-controlled treatment period can be used to assess treatment effects in terms of faster wound healing. However, the duration of the study is not suitable for conclusions regarding long-term symptom improvement or and long-term side effects. For the benefit assessment, wound closure, wound infection, itching, pain and sleep disturbance as well as adverse events (AE) were considered as patient relevant endpoints. Birch bark extract was shown to be superior to the control gel in first complete closure of the target wound (low risk of bias). There were no statistically significant differences between treatment groups in wound infection, itching, pain, sleep disturbance. With the exception of wound infection, these results are associated with a high risk of bias due to the high number of missing values in the analyses. No statistically significant differences were observed in the occurrence of AE between the groups (low risk of bias). No data of health-related quality of life could be taken into account.

Authors' identified further research: epidermolysis bullosa, bitch bark, Filsuvez

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/876/#english

Year Published: 2023

URL for published report: https://www.g-ba.de/downloads/39-1464-5878/2023-02-16_AM-RL-XII_Birch-Bark-Extract_D-862_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/876/#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/