TY - JOUR

T1 - Early benefit assessment (EBA) in Germany

T2 - analysing decisions 18 months after introducing the new AMNOG legislation

AU - Ruof, Jörg

AU - Schwartz, Friedrich Wilhelm

AU - Schulenburg, J. Matthias

AU - Dintsios, Charalabos Markos

N1 - Funding Information: Medical writing services were provided by nspm ltd, Meggen, Switzerland, with financial support from Roche Pharma AG.

PY - 2014/7

Y1 - 2014/7

N2 - Objectives: Since the introduction of the German health care reform in January 2011, an early benefit assessment (EBA) is required for all new medicines. Pharmaceutical manufacturers have to submit a benefit dossier for evaluation by the Institute for Quality and Efficiency in Health Care (IQWiG). A final decision is made by the Federal Joint Committee (G-BA). The aim of this investigation was to analyse the outcomes 18 months after introduction of the new legislation and to identify critical areas requiring further discussion and development. Methods: All EBAs commenced prior to June 2012 were included. The G-BA website was used to obtain manufacturers' benefit dossiers, IQWiG assessments, and G-BA decisions. Four areas of interest were analysed: levels of additional benefit, appropriate comparative therapy (ACT), patient-relevant endpoints, and adverse events. Results: Twenty-seven EBAs were analysed. IQWiG stated a benefit in 50 % of EBAs, whereas G-BA stated a benefit in 63 %, but only in 50 % of identified subgroups and 40 % of patients involved. In 12 EBAs, the ACT suggested by G-BA differed from the comparator used in phase III trials. The G-BA reported no benefits on health-related quality of life. Discrepancies arose in morbidity outcomes such as 'progression-free survival' and 'sustained virological response'. Categorisation and balancing of adverse events was conducted within various assessments. Conclusions: Considerable variance was observed in the levels of additional benefit reported by pharmaceutical manufacturers, IQWiG and G-BA. The areas of disagreement included ACT selection, definition of subgroups and patient-relevant endpoints, and classification and balancing of adverse events.

AB - Objectives: Since the introduction of the German health care reform in January 2011, an early benefit assessment (EBA) is required for all new medicines. Pharmaceutical manufacturers have to submit a benefit dossier for evaluation by the Institute for Quality and Efficiency in Health Care (IQWiG). A final decision is made by the Federal Joint Committee (G-BA). The aim of this investigation was to analyse the outcomes 18 months after introduction of the new legislation and to identify critical areas requiring further discussion and development. Methods: All EBAs commenced prior to June 2012 were included. The G-BA website was used to obtain manufacturers' benefit dossiers, IQWiG assessments, and G-BA decisions. Four areas of interest were analysed: levels of additional benefit, appropriate comparative therapy (ACT), patient-relevant endpoints, and adverse events. Results: Twenty-seven EBAs were analysed. IQWiG stated a benefit in 50 % of EBAs, whereas G-BA stated a benefit in 63 %, but only in 50 % of identified subgroups and 40 % of patients involved. In 12 EBAs, the ACT suggested by G-BA differed from the comparator used in phase III trials. The G-BA reported no benefits on health-related quality of life. Discrepancies arose in morbidity outcomes such as 'progression-free survival' and 'sustained virological response'. Categorisation and balancing of adverse events was conducted within various assessments. Conclusions: Considerable variance was observed in the levels of additional benefit reported by pharmaceutical manufacturers, IQWiG and G-BA. The areas of disagreement included ACT selection, definition of subgroups and patient-relevant endpoints, and classification and balancing of adverse events.

KW - (Early) benefit assessment

KW - AMNOG

KW - Appropriate comparative therapy

KW - Health care reform

KW - Market access

UR - http://www.scopus.com/inward/record.url?scp=84905568232&partnerID=8YFLogxK

U2 - 10.1007/s10198-013-0495-y

DO - 10.1007/s10198-013-0495-y

M3 - Article

C2 - 23771769

AN - SCOPUS:84905568232

VL - 15

SP - 577

EP - 589

JO - European Journal of Health Economics

JF - European Journal of Health Economics

SN - 1618-7598

IS - 6

ER -