Flyer Check, Phase-III Portfolio

3 Introduction to the topic This check analyzes their Phase III candidates/products with regard to the existing quality of the evidence and study situation. As a result, it provides an initial assessment of the possible outcome of the early benefit assessment according to the AMNOG procedure (§ 35a SGB V) for drugs and the § 137e procedure for medical devices. As a result, an optimization propo- sal for the clinical trials up to the submission for marketing authorization (Z 1) and up to the conclusion of the benefit assessment (Z 2) and the start of reimbursement negotiations is presented. In early Phase III, there is still some time to address deficiencies and identified "shortcomings". Was the correct appropriate comparator therapy (ZVT/ZVI) selected for a future benefit as- sessment? Were endpoints relevant for patients selected in the studies or only surrogate pa- rameters? Was an adequate study design chosen for the research question? Was superiority tested? Will health services research studies be required? With the analysis of the Phase III products, one can then make an assessment of the outco- me of the benefit assessment according to § 35a SGB V for drugs or according to § 137e SGB V for medical devices. This assessment can then be used to consider a possible " step up " optimization, for example by one benefit category level. The analysis of the actual situation is then followed by a suggested maximization up to the submission of the marketing authorization (ZP 1) and up to the benefit assessment (ZP 2). Assessment of the expected additional benefit After a careful analysis of the studies performed so far and the outcomes achieved with them, a first assessment of Phase III candidates will be made. Operationalization of the Phase III Check It is necessary to specify the possible maximization. Which clinical studies can still be initia- ted, which relevant comparators can still be included, or which patient - relevant endpoints should still be considered? Is there a need for health care studies or a survey? Phase - III  Optimization of study design?  Adequately powered?  NNT and sufficient follow - up?  Choice of ZVT for phase III?  Choice of patient relevant outcomes?  Testing for superiority?  Capture of economic parameters?  Capture of relevance to care?