Original Title: Arzneimittel-​​​Richtlinie/Anlage XII: Caplacizumab (neues Anwendungsgebiet: erworbene thrombotische-thrombozytopenische Purpura, 12 bis < 18 Jahre)

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: Caplacizumab is approved for adults and adolescents aged 12 years and older with a body weight of at least 40 kg who have an episode of acquired thrombotic thrombocytopenic purpura (aTTP). The patients covered by the benefit assessment are adolescent patients aged 12 years and older with a body weight of at least 40 kg who are addressed by an extension of the previous marketing authorisation regarding adults. Caplacizumab is used in combination with plasmapheresis and immunosuppression. Among other data, the pharmaceutical company submitted the HERCULES study (ALX0681-C301) and ALX0681-MS-013 for an evidence transfer from adults to adolescents. In a previous benefit assessment, a non-quantifiable additional benefit was determined for caplacizumab in adult patients with aTTP based on results from the HERCULES trial. ALX0681-MS-013 is a PK/PD modelling or simulation study. The HERCULES study, which only included adults, and the PK/PD modelling and simulation studies ALX0681-MS-013 are not accepted for an evidence transfer within the scope of the benefit assessment because there are no clinical studies in which the population covered by the extension of the marketing authorisation (adolescents with aTTP) was investigated. Therefore, the evidence transfer is not regarded as valid for a benefit assessment, in particular, because no or no meaningful data is available to conclude on the comparability of study-specific characteristics as well as efficacy and safety of caplacizumab between adults and adolescents (12 years and older and weighing 40 kg or more).

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/567/#english

Year Published: 2019

URL for published report: https://www.g-ba.de/downloads/39-1464-4653/2021-01-07_AM-RL-XII_Capalcizumab_D-566_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/567/#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/