Jörg Ruof1, Dietrich Knoerzer2, Anja-Alexandra Dünne2, Charalabos-Markos Dintsios3, Thomas Staab2, Friedrich Wilhelm Schwartz4. 1. Roche Pharma AG, Grenzach-Wyhlen, Germany; Medical School of Hanover, Hanover, Germany. Electronic address: joerg.ruof@roche.com. 2. Roche Pharma AG, Grenzach-Wyhlen, Germany. 3. German Association of Research-based Pharmaceutical Companies (vfa), Berlin, Germany; Department of Public Health, Heinrich Heine University, Düsseldorf, Germany. 4. Medical School of Hanover, Hanover, Germany.
Abstract
BACKGROUND AND AIMS: In Germany, a mandatory early benefit assessment (EBA) by the Federal Joint Committee (G-BA) is required for reimbursement of new marketing-authorised medicines. Additional benefit is based on patient-relevant endpoints in mortality, morbidity and health-related quality of life (HRQoL). We aimed to compare endpoints and related benefit categories used in marketing authorisation to those considered by G-BA in the field of oncology. METHODS: We evaluated EBAs in oncology commencing prior to 31 December 2013. Endpoints for the appropriate medicines, derived from European Medicines Agency's (EMA) Summary of Product Characteristics (SPC), manufacturers' value dossiers and G-BA decisions, were grouped into the three benefit categories. RESULTS: Of 23 oncology medicines evaluated, primary clinical trial endpoints were included in only 12 G-BA value decisions. Mortality endpoints were generally accepted by EMA and G-BA. However, G-BA excluded 80% of (co-)primary morbidity endpoints. Only 5 SPCs reported HRQoL instruments. G-BA accepted applied instruments in 15 medicines, but the manufacturers' analyses only in 5 medicines, of which 2 indicated an additional benefit. CONCLUSIONS: Mortality endpoints are accepted by EMA and G-BA. EMA accepted well established and clinically relevant morbidity endpoints (e.g. progression-free survival and response rate), which were mostly excluded by G-BA from their value decisions. The applicability of methods used for benefit assessments to HRQoL differs from the mortality and morbidity categories, and requires further clarification.
BACKGROUND AND AIMS: In Germany, a mandatory early benefit assessment (EBA) by the Federal Joint Committee (G-BA) is required for reimbursement of new marketing-authorised medicines. Additional benefit is based on patient-relevant endpoints in mortality, morbidity and health-related quality of life (HRQoL). We aimed to compare endpoints and related benefit categories used in marketing authorisation to those considered by G-BA in the field of oncology. METHODS: We evaluated EBAs in oncology commencing prior to 31 December 2013. Endpoints for the appropriate medicines, derived from European Medicines Agency's (EMA) Summary of Product Characteristics (SPC), manufacturers' value dossiers and G-BA decisions, were grouped into the three benefit categories. RESULTS: Of 23 oncology medicines evaluated, primary clinical trial endpoints were included in only 12 G-BA value decisions. Mortality endpoints were generally accepted by EMA and G-BA. However, G-BA excluded 80% of (co-)primary morbidity endpoints. Only 5 SPCs reported HRQoL instruments. G-BA accepted applied instruments in 15 medicines, but the manufacturers' analyses only in 5 medicines, of which 2 indicated an additional benefit. CONCLUSIONS: Mortality endpoints are accepted by EMA and G-BA. EMA accepted well established and clinically relevant morbidity endpoints (e.g. progression-free survival and response rate), which were mostly excluded by G-BA from their value decisions. The applicability of methods used for benefit assessments to HRQoL differs from the mortality and morbidity categories, and requires further clarification.
Authors: Oriana Ciani; Bogdan Grigore; Hedwig Blommestein; Saskia de Groot; Meilin Möllenkamp; Stefan Rabbe; Rita Daubner-Bendes; Rod S Taylor Journal: Med Decis Making Date: 2021-03-10 Impact factor: 2.583
Authors: Rolf A Stahel; Denis Lacombe; Fatima Cardoso; Paolo G Casali; Anastassia Negrouk; Richard Marais; Anita Hiltbrunner; Malvika Vyas Journal: ESMO Open Date: 2020-03