Randomized Clinical Trial Design for Assessing Noninferiority When Superiority Is Expected

2007 ◽  
Vol 25 (31) ◽  
pp. 5019-5023 ◽  
Author(s):  
Boris Freidlin ◽  
Edward L. Korn ◽  
Stephen L. George ◽  
Robert Gray
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Tolerability Profile ◽  
Modest Improvement ◽  
Noninferiority Trials ◽  
New Treatment ◽  
Formal Test ◽  
Hybrid Trial

The randomized clinical trial (RCT) is the gold standard for definitive evaluation of new therapies. RCTs designed to show that the therapeutic efficacy of a new therapy is not unacceptably inferior to that of standard therapy are called noninferiority trials. Traditionally, noninferiority trials have required very large sample sizes. Sometimes, a new treatment regimen with a favorable toxicity and/or tolerability profile is also expected to have some modest improvement in efficacy. In such specialized settings we describe a hybrid trial-design approach that requires a dramatically smaller sample size than that of a standard noninferiority design. This hybrid design can naturally incorporate a formal test of superiority as well as noninferiority.

Value of Biomarker Expression for Randomized Clinical Trial Design: One (More) Missed Opportunity

2020 ◽  
Vol 38 (6) ◽  
pp. 649-651 ◽  
Author(s):  
Francisco E. Vera-Badillo ◽  
Andrew J. Robinson ◽  
David M. Berman ◽  
Christopher M. Booth
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Missed Opportunity

scholarly journals General concepts in biostatistics and clinical epidemiology: Experimental studies with randomized clinical trial design

Medwave ◽  
2020 ◽  
Vol 20 (03) ◽  
pp. e7869-e7869
Author(s):  
Sebastián Estrada ◽  
Marcelo Arancibia ◽  
Jana Stojanova ◽  
Cristian Papuzinski
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Trial Design ◽  
Clinical Epidemiology ◽  
Experimental Studies ◽  
Clinical Trial Design

scholarly journals Use of a randomized clinical trial design to study cognitive rehabilitation approaches to enhance warfighter performance

2020 ◽  
Vol 20 ◽  
pp. 100660
Author(s):  
Ida Babakhanyan ◽  
Melissa Jensen ◽  
Rosemay A. Remigio-Baker ◽  
Paul Sargent ◽  
Jason M. Bailie
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Cognitive Rehabilitation ◽  
Trial Design ◽  
Clinical Trial Design

scholarly journals The Glaucoma Intensive Treatment Study (GITS), a randomized clinical trial: design, methodology and baseline data

2018 ◽  
Vol 96 (6) ◽  
pp. 557-566 ◽  
Author(s):  
Boel Bengtsson ◽  
Anders Heijl ◽  
Gauti Johannesson ◽  
Sabina Andersson-Geimer ◽  
Johan Aspberg ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Trial Design ◽  
Design Methodology ◽  
Clinical Trial Design ◽  
Baseline Data ◽  
Intensive Treatment

Clinical Trials in Childhood Cancer

2020 ◽  
pp. 38-45
Author(s):  
Maria Grazia Valsecchi ◽  
Stefania Galimberti ◽  
Pamela Kearns
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Rare Diseases ◽  
Treatment Strategy ◽  
Trial Design ◽  
Methodological Approach ◽  
Clinical Trial Design ◽  
New Treatment ◽  
Diagnosis Therapy ◽  
Collaborative Efforts

Many important improvements have been achieved in the last decades in the diagnosis, therapy, and prognosis of paediatric cancers. These were possible through clinical trials that were mainly the result of collaborative efforts of national and international groups. For a clinical trial to have the best chance of success, it must ask a clear question that is seen as important both to the clinicians who will implement the trial and to the patients who will participate. Moreover, the design of such trials needs a sound methodological approach in order to provide reliable evidence. This chapter has the following aims: (a) to review the most common conventional approaches that allow to reliably define the benefit of a new drug or a new treatment strategy, and (b) to provide information on novel strategies for clinical trial design that can be useful in the context of rare diseases such as paediatric cancers.

scholarly journals Development of New Antimicrobials for Urogenital Gonorrhea Therapy: Clinical Trial Design Considerations

2019 ◽  
Vol 70 (7) ◽  
pp. 1495-1500 ◽  
Author(s):  
Edward W Hook ◽  
Lori Newman ◽  
George Drusano ◽  
Scott Evans ◽  
H Hunter Handsfield ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Antimicrobial Resistance ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Evaluation Process ◽  
Antibiotic Resistant ◽  
Noninferiority Trials ◽  
New Antibiotics ◽  
Public Health Challenge ◽  
New Treatments

Abstract Gonorrhea remains a major public health challenge, and current recommendations for gonorrhea treatment are threatened by evolving antimicrobial resistance and a diminished pipeline for new antibiotics. Evaluations of potential new treatments for gonorrhea currently make limited use of new understanding of the pharmacokinetic and pharmacodynamic contributors to effective therapy, the prevention of antimicrobial resistance, and newer designs for clinical trials. They are hampered by the requirement to utilize combination ceftriaxone/azithromycin therapy as the comparator regimen in noninferiority trials designed to seek an indication for gonorrhea therapy. Evolving gonococcal epidemiology and clinical trial design constraints hinder the enrollment of those populations at the greatest risk for gonorrhea (adolescents, women, and persons infected with antibiotic-resistant Neisseria gonorrhoeae). This article summarizes a recent meeting on the evaluation process for antimicrobials for urogenital gonorrhea treatment and encourages the consideration of new designs for the evaluation of gonorrhea therapy.

scholarly journals Randomized Clinical Trial Design to Assess Abatacept in Resistant Nephrotic Syndrome

2018 ◽  
Vol 3 (1) ◽  
pp. 115-121 ◽  
Author(s):  
Howard Trachtman ◽  
Debbie S. Gipson ◽  
Michael Somers ◽  
Cathie Spino ◽  
Sharon Adler ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Nephrotic Syndrome ◽  
Randomized Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design
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