Original Title: Arzneimittel-Richtlinie/Anlage XII: Maribavir (Cytomegalievirus-Infektion (therapierefraktär))

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: Maribavir is approved for the treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant or solid organ transplant. The benefit assessment of Maribavir is based on the pivotal study SHP620-303, a multicenter, open-label, randomized controlled phase III study to determine the safety and efficacy of Maribavir compared to an investigator assigned anti-CMV treatment (ganciclovir, valganciclovir, cidofovir or foscarnet) in patients with refractory CMV-infection (with or without resistance) after haematopoietic stem cell transplant or solid organ transplant. In terms of all-cause mortality, there is no statistically significant difference between maribavir and investigator assigned anti-CMV treatment. For the morbidity endpoint of confirmed CMV viremia clearance at week 8 and maintenance of CMV viremia clearance from week 8 until the end of the study at week 20, there is a statistically significant difference in favour of maribavir compared to investigator assigned anti-CMV treatment (RRweek 8=2.37; 95% CI:1.69; 3.34; RRweek 20=2.10; 95% CI: 1.14; 3.89). However, the proportion of responders decreases over the course of the study and the potential for bias for the endpoint is considered high due to missing values and intercurrent events. New-onset tissue-invasive disease and graft outcomes are presented descriptively and have a high potential for bias. The results of the EQ-5D-VAS and the SF-36 are not presented in the benefit assessment because the response rates were too low and showed large differences between the study arms. A conclusive assessment of safety is not possible due to the lack of blinding and inadequate analyses. The potential for bias for adverse events is classified as high.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/906/#english

Year Published: 2023

URL for published report: https://www.g-ba.de/downloads/39-1464-6032/2023-06-01_AM-RL-XII_Maribavir_D-898_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/906/#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/