artbin: Extended sample size for randomized trials with binary outcomes.

We describe the command artbin, which offers various new facilities for the calculation of sample size for binary outcome variables that are not otherwise available in Stata. While artbin has been available since 2004, it has not been previously described in the . artbin has been recently updated to include new options for different statistical tests, methods and study designs, improved syntax, and better handling of noninferiority trials.

Real-World Evidence Utilization in Clinical Development Reflected by US Product Labeling: Statistical Review.

The US Food and Drug Administration (FDA) has shown scientific discretion in interpreting the substantial evidence requirement for the approval of new drugs with its considerations on the use of single controlled or uncontrolled trials (Federal Food, Drug, and Cosmetic Act § 505(d), 21 USC 355(d), 1962). With the passage of the 21st Centuries Cures Act (21st Century Cures-patients. House, Energy and Commerce Committee, Washington, DC, 2019 available at: https://energycommerce.

Development and validation of the Detail and Flexibility Questionnaire (DFlex) in eating disorders.

Whilst neuropsychological testing provides the most accurate profile of cognitive functioning, the time consuming nature of individual assessment deems it impossible for many research and clinical settings. This paper presents the development and validation of the Detail and Flexibility Questionnaire (DFlex), a 24-item self-report scale measuring two aspects of neurocognitive functioning; cognitive rigidity (difficulty with set-shifting/flexibility) and attention to detail (weak coherence). Exploratory factor analysis extracted two subscales, further confirmed and refined by item response analysis.

Designs for clinical trials with time-to-event outcomes based on stopping guidelines for lack of benefit.

Background: The pace of novel medical treatments and approaches to therapy has accelerated in recent years. Unfortunately, many potential therapeutic advances do not fulfil their promise when subjected to randomized controlled trials. It is therefore highly desirable to speed up the process of evaluating new treatment options, particularly in phase II and phase III trials.

Speeding up the evaluation of new agents in cancer.

Despite both the increase in basic biologic knowledge and the fact that many new agents have reached various stages of development during the last 10 years, the number of new treatments that have been approved for patients has not increased as expected. We propose the multi-arm, multi-stage trial design as a way to evaluate treatments faster and more efficiently than current standard trial designs. By using intermediate outcomes and testing a number of new agents (and combinations) simultaneously, the new design requires fewer patients.