Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0268596 (EMA)
 2,520 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

No doubt, the public should be involved in healthcare decision making, especially when decision makers from politics and self-government agencies are faced with the difficult task of setting priorities. There is a general consensus on the need for a stronger patient centeredness, even in HTA processes, and internationally different ways of public participation are discussed and tested in decision making processes. This paper describes how the public can be involved in different decision situations, and it shows how preference measurement methods are currently being used in an international context to support decision making. It distinguishes between different levels of decision making on health technologies: approval, assessment, pricing, and finally utilization. The range of participation efforts extends from qualitative surveys of patients' needs (Citizen Councils of NICE in the UK) to science-based documentation of quantitative patient preferences, such as in the current pilot projects of the FDA in the US and the EMA at the European level. Possible approaches for the elicitation and documentation of preference structures and trade-offs in relation to alternate health technologies are decision aids, such as multi-criteria decision analysis (MCDA), that provide the necessary information for weighting and prioritizing decision criteria.
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PMID:[Involving patients, the insured and the general public in healthcare decision making]. 2687 34

The early benefit assessment of pharmaceuticals in Germany and their preceding market authorization pursue different objectives. This is reflected by the inclusion of varying confirmatory endpoints within the evaluation of oncology drugs in early benefit assessment versus market authorization, with both relying on the same evidence. Data from assessments up to July 2015 are used to estimate the impact of explorative in comparison to confirmatory endpoints on market authorization and early benefit assessment by contrasting the benefit-risk ratio of EMA and the benefit-harm balance of the HTA jurisdiction. Agreement between market authorization and early benefit assessment is examined by Cohen's kappa (k). 21 of 41 assessments were considered in the analysis. Market authorization is more confirmatory than early benefit assessment because it includes a higher proportion of primary endpoints. The latter implies a primary endpoint to be relevant for the benefit-harm balance in only 67% of cases (0.078). Explorative mortality endpoints reached the highest agreement regarding the mutual consideration for the risk-benefit ratio and the benefit-harm balance (0.000). For explorative morbidity endpoints (-0.600), quality of life (-0.600) and side effects (-0.949) no agreement is ascertainable. To warrant a broader confirmatory basis for decisions supported by HTA, closer inter-institutional cooperation of approval authorities and HTA jurisdictions by means of reliable joint advice for manufacturers regarding endpoint definition would be favorable.
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PMID:Confirmatory versus explorative endpoint analysis: Decision-making on the basis of evidence available from market authorization and early benefit assessment for oncology drugs. 2960 27