Characteristics of drugs approved in Japan without conducting confirmatory clinical trials

2021 ◽  
Author(s):  
Shunsuke Izuka ◽  
Naoki Matsumaru ◽  
Katsura Tsukamoto
Keyword(s):  
Clinical Trials ◽  
Confirmatory Clinical Trials

Data-Driven Subgroup Identification in Confirmatory Clinical Trials

2021 ◽  
Author(s):  
Pierre Bunouf ◽  
Mélanie Groc ◽  
Alex Dmitrienko ◽  
Ilya Lipkovich
Keyword(s):  
Clinical Trials ◽  
Data Driven ◽  
Subgroup Identification ◽  
Confirmatory Clinical Trials

scholarly journals Recommendations on the Clinical Trial Programme for Diabetes Medicines

2021 ◽  
Vol 11 (2) ◽  
pp. 94-103
Author(s):  
I. A. Proskurina ◽  
E. A. Petraneva ◽  
D. V. Goryachev
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Treatment Effectiveness ◽  
Clinical Trial Design ◽  
Public Health Problem ◽  
The Body ◽  
Noncommunicable Diseases ◽  
The European Union ◽  
Antidiabetic Therapy ◽  
Confirmatory Clinical Trials

Diabetes is a serious public health problem and one of the major chronic noncommunicable diseases. A lengthy stepwise treatment, and the need for an individualised approach to antidiabetic therapy, pose serious challenges for medicine developers. For all new hypoglycaemic medicines, there has been a centralised authorisation procedure in the European Union (EU) since 2005, which ensures a unified approach to efficacy and safety assessment. The aim of the study was to analyse current requirements for planning clinical trials of hypoglycaemic medicines containing new active substances (except for insulin products). The recommendations for diagnosis and treatment of type 2 diabetes, prepared by the European Association for the Study of Diabetes (EASD) and the American Diabetes Association (ADA) in 2019, suggest a step-by-step approach to intensification of treatment to maintain glycaemic targets, which takes account of concomitant cardiovascular or other diseases, and clinical characteristics of patients. The analysis of EASD/ADA documents and scientific literature helped to develop recommendations on the basic principles of planning and conducting clinical trials at the final stages of hypoglycaemic medicine development. The paper describes new approaches to clinical trials, which allow for a more reliable assessment of the treatment effectiveness. The strategy for the assessment of therapeutic effect should be carefully planned, justified, and reflected in variables of interest, clinical trial design, and statistical analysis of the trial results. The main efficacy criterion in confirmatory clinical trials of hypoglycaemic medicines should be the demonstration of benefits in improving glycaemic control. The medicine’s effect on the body weight may be considered as a secondary endpoint. An essential requirement is confirmation of the medicines’ cardiovascular safety, while potential additional benefits are reduction or prevention of risks of cardiovascular disease development. The clinical trial protocol should provide definitions for intercurrent events and hypoglycaemia. A comprehensive safety study of a new hypoglycaemic medicine should involve identification of anticipated or known side effects characteristic of a particular pharmacological class. The provided recommendations may be helpful for medicine developers, and for experts who perform assessment of clinical trial programmes and regulatory submissions for hypoglycaemic medicines.           

Modified Goldilocks Design with strict type I error control in confirmatory clinical trials

2020 ◽  
Vol 30 (5) ◽  
pp. 821-833
Author(s):  
Tianyu Zhan ◽  
Hongtao Zhang ◽  
Alan Hartford ◽  
Saurabh Mukhopadhyay
Keyword(s):  
Clinical Trials ◽  
Error Control ◽  
Type I Error ◽  
Type I ◽  
Confirmatory Clinical Trials
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