Varying results of early benefit assessment of newly approved pharmaceutical drugs in Germany from 2011 to 2017: A study based on federal joint committee data

2019 ◽  
Vol 12 (1) ◽  
pp. 9-15 ◽  
Author(s):  
Frank Peinemann ◽  
Alexander Labeit
Keyword(s):  
Benefit Assessment ◽  
Pharmaceutical Drugs ◽  
Federal Joint Committee ◽  
Early Benefit Assessment ◽  
Early Benefit

INDUSTRY'S EXPERIENCES WITH THE SCIENTIFIC ADVICE OFFERED BY THE FEDERAL JOINT COMMITTEE WITHIN THE EARLY BENEFIT ASSESSMENT OF PHARMACEUTICALS IN GERMANY

2018 ◽  
Vol 34 (2) ◽  
pp. 196-204 ◽  
Author(s):  
Charalabos-Markos Dintsios ◽  
Sara Schlenkrich
Keyword(s):  
Small Sample Size ◽  
Small Sample ◽  
Benefit Assessment ◽  
Positive Trend ◽  
Scientific Advice ◽  
Federal Joint Committee ◽  
Active Involvement ◽  
Early Benefit Assessment ◽  
Early Benefit

Objectives: Optional scientific advice (SA) for the early benefit assessment of pharmaceuticals is offered by the German decision maker, the Federal Joint Committee (FJC). The aim of this study was to elicit manufacturers’ experiences with the SA procedures offered by the FJC to date.Methods: A preliminary survey on a small sample size was conducted. Subsequently, a questionnaire comprising eight items, which was developed on the basis of that survey, was used. Data were analyzed using qualitative and quantitative approaches.Results: The elicitation, including a sample of 25 percent of the completed advice, highlighted the following, regarding the process as well as to the content shortcomings of the SA procedures from an industrial perspective: inconsistencies, FJC's lack of expertise in conducting clinical trials, partially incomplete answers. and a low willingness of the FJC to engage in dialogue with industry were criticized. On the other hand, the majority of respondents expressed a positive attitude concerning unambiguousness, completeness, traceability, discussion atmosphere, and the protocol of the advice. Early SA, before pivotal trials start, showed a significantly higher completeness compared with late SA with respect to endpoints and study duration. Within 4 years the quality of FJC's propositions on some topics improved significantly.Conclusions: Only a few statistically significant differences were detectable between early versus late SA. A positive trend in industry's perception of the SA can be observed over time. A more active involvement of additional stakeholders and the incorporation of procedural elements from other healthcare systems could improve the quality of the SA offered by the FJC.

scholarly journals PCN243 Price Changes after Indication Extension in the German EARLY Benefit Assessment

2020 ◽  
Vol 23 ◽  
pp. S466
Author(s):  
M. Freiberg ◽  
M. Karimi ◽  
D. Böckmann ◽  
P. Wagner
Keyword(s):  
Benefit Assessment ◽  
Price Changes ◽  
Early Benefit Assessment ◽  
Early Benefit

scholarly journals Impact of the Early Benefit Assessment on A Pharmaceutical's Real Consumption in Germany

2014 ◽  
Vol 17 (7) ◽  
pp. A416
Author(s):  
W. Kotowa ◽  
B. Häussler ◽  
S. Reindl ◽  
A. Höer
Keyword(s):  
Benefit Assessment ◽  
Early Benefit Assessment ◽  
Early Benefit

OP135 Confirmatory Versus Explorative Endpoints In Drug Approval Versus Health Technology Assessment

2017 ◽  
Vol 33 (S1) ◽  
pp. 63-64
Author(s):  
Ines Niehaus ◽  
Charalabos-Markos Dintsios
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Drug Approval ◽  
Health Technology ◽  
Benefit Assessment ◽  
Benefit Ratio ◽  
Early Benefit Assessment ◽  
The Impact ◽  
Early Benefit

INTRODUCTION:The early benefit assessment of drugs in Germany and their preceded market authorization pursue different objectives, resulting in divergent decision-making strategies. This is reflected inter alia by the diverse inclusion of confirmatory endpoints within the assessments of oncological drugs. The pharmaceutical manufacturers are facing the challenge of meeting the requirements for both evaluation processes by the available evidence and avoiding hereby negative early benefit assessments. This is mainly due to the concept of mutually relevant clinical trials.METHODS:Identification and gathering of the endpoints is based on a specifically developed guide. The extracted data from the documents of completed assessments up to July 2015 are used to estimate both separately and together the impact of explorative in relation to confirmatory endpoints on the drug approval and early benefit assessment, by contrasting the European Medicines Agency's risk-benefit-ratio and the benefit-harm-balancing of the national Health Technology Assessment (HTA) jurisdiction.RESULTS:Twenty-one of fourty-one studies’ oncological assessments could be included in the endpoint analysis. From a procedural point of view both the drug approval and the early benefit assessment seem to be not confirmatory since they include explorative endpoints as well. Yet, drug approval is in terms of quality of endpoints more confirmatory than early benefit assessment since it contains a higher proportion of primary endpoints. The latter implies only in 67 percent of the assessments a primary endpoint to be relevant for the benefit-harm-balancing. Moreover, explorative mortality endpoints reached the highest agreement and explorative endpoints capturing health-related quality of life no agreement, referring to the mutual relevance of endpoints for the risk-benefit-ratio and the benefit-harm-balancing.CONCLUSIONS:The missing information transparency of the assessment reports compared to the information offered within the early benefit assessment makes an assignment of endpoints with respect to the mutually relevant clinical trial sometimes troublesome. To warrant, in the long run, a broader confirmatory basis for decisions in health care supported by HTA, a closer inter-institutional cooperation of approval authorities and German HTA jurisdictions seems favorable.

Frühe Nutzenbewertung neu zugelassener Arzneimittel – Methodische Anforderungen an die Daten zur Lebensqualität

2017 ◽  
Vol 22 (03) ◽  
pp. 125-125
Author(s):  
Helena Thiem
Keyword(s):  
Quality Of Life ◽  
Qualitative Study ◽  
New Drugs ◽  
Benefit Assessment ◽  
Health Econ ◽  
Life Data ◽  
Early Benefit Assessment ◽  
Frühe Nutzenbewertung ◽  
Early Benefit

Blome C et al. Four years of early benefit assessment of new drugs in Germany: a qualitative study on methodological requirements for quality of life data. Eur J Health Econ 2017; 18: 181–193 Der Gemeinsame Bundesausschuss (G-BA) führt für alle neu zugelassenen Arzneimittel eine frühe (Zusatz-)Nutzenbewertung durch. In die Entscheidung mit einbezogen wird die Auswirkung auf unterschiedliche Endpunkte, u. a. auch auf die Lebensqualität (QoL). Die Autoren haben für die zwischen den Jahren 2011–2014 veröffentlichten Nutzenbewertungen analysiert, inwieweit die Daten zur Lebensqualität bei der abschließenden Bewertung zum Tragen kommen.

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