PCN172 - HR-QOL Data in the Amnog Early Benefit Assessment: Industry and IQWIG/GBA Perspective

Abstract

To explore how often and which types of quality of life (QOL) data are included in the dossiers submitted for the AMNOG early benefit assessment and analyze how a drug’s effect in QOL is interpreted by the industry and the evaluating committee. The published value dossiers up to June 15, 2013 and the corresponding benefit assessments were reviewed. Types of QOL data and the statements concerning QOL in the value dossier and the benefit assessments were collected and summarized. A total of 46 cases were analyzed. In 6 cases no dossier was submitted. Of the rest, 13 dossiers (32.5%) did not include any QOL data, 13 (32.5%) included disease-specific QOL data only, 5 (12.5%) included generic QOL data only, and 9 (22.5%) both types. The QOL instruments used were considered as valid by the assessors. However, outcomes such as pain severity, fatigue, work productivity, and health care resource use were not regarded as QOL measures. Of 27 dossiers that have included QOL data companies have claimed an added benefit in QOL, either for the whole population or a specific subgroup, in 15 (56%) cases. However, the assessors recognized the added benefit claim in 2 cases only. In an additional 2 cases no clear scoring was provided for the QOL outcome and in the remaining 11 cases no additional benefit was acknowledged. The main reasons were missing statistical significance of results or the submitted QOL data were regarded as not usable. In a majority of value dossiers companies included QOL data and also most often claimed an added benefit in QOL. However, the assessors have accepted this added value in exceptional cases only. In order to get an added value recognized showing both statistical and clinical significance of results is required.