Newsletter for Statistical Programmers and Biostatisticians #47

??????????????????????????????????????????????????

Statistical Programming

Presentation from Sunil Gupta fromPhUSE SDE. Sunil distributed a CDISC 360 SDTM Design and Automation flyer which focuses on leveraging metadata. You can contact him for a copy of this general SDTM process flowchart. Link

An Integrated Summary of Safety is required by the FDA for any New Drug Application or similar market approval request. An Integrated Summary of Effectiveness may also be required.?Angelo Tinazzi?discusses these summaries and ways to approach your?data integration strategy with examples from his recent?PHUSE?EU presentation. Link

Jagadish K. developed a Tendril application using the Tendril?package. The tendril plot algorithm is inspired by a striking piece of work by Stefaner et al. The tendril plot is a visualization that summarizes the overall AE profile in a study, clearly shows the AEs of major importance, and, in addition, illustrates the time course of reported AEs. Please, check this very interesting piece of work with this link.

6 hour long SAS Macro Video tutorial from Kiran Venna , in this tutorial he covers following topics. 1. Macro Introduction. 2. Macro variables and %let statements. 3. Macro and Macro parameters. 4. Conditional and iterative macro statement. 5. Creating macro variables with Proc SQL and three ways to use them. 6. CALL SYMPUT/CALL SYMPUTX and comparison with PROC SQL into 7a. Macro Quoting functions 7b.Evaluation, %sysfunc and character based Macro functions. 8. Macro Libraries. 9. Macro facility interaction with Call execute. 10. Scope of Macro Variables. Link

R package for clinical trial simulation. Alex Dmitrienko has shared a new module to his company's open-source software tool for clinical trial simulation (MedianaDesigner package). The module supports a new class of trial designs, namely, cluster-randomized designs. Designs of this kind are commonly used in global health studies, e.g., in community intervention studies, and this module streamlines the process of designing cluster-randomized designs. The latest version of the MedianaDesigner package is available on the CRAN site: Link

If you are curious about best in stream PHUSE 2022 winners, please check PHUSE website for details. Most of the winners are on LinkedIn and might be willing to share their work! Link

????????????????????????????????????????????????????

Biostatistics

On December 2, 2022, FDA published the draft guidance for industry entitled Statistical Approaches to Establishing Bioequivalence. Link

The use of external controls: To what extent can it currently be recommended? This paper provides an overview on types of external control, their applications and the different sources of bias their use may incur, and discuss potential mitigation steps. It will also give recommendations on how the use of external controls can be justified. From Hans Ulrich, Burger,?Christoph Gerlinger,?Chris Harbron,?Armin Koch,?Martin Posch,?Justine Rochon,?Anja Schiel. Link

Comparing algorithms for characterizing treatment effect heterogeneity in randomized trials. In this work, authors review some existing simulation work and perform a simulation study to evaluate recent methods for identifying and estimating the heterogeneous treatments effects using various metrics and scenarios relevant for drug development.?From Marc Vandemeulebroecke and Sun Hui (Sophie) . Link

Design considerations for two-stage enrichment clinical trials. . In this work, authors review some existing simulation work and perform a simulation study to evaluate recent methods for identifying and estimating the heterogeneous treatments effects using various metrics and scenarios relevant for drug development. The focus is not only on a comparison of the methods in general, but on how well these methods perform in simulation scenarios that reflect real clinical trials. Rosamarie Frieri, William F. Rosenberger,?Nancy Flournoy,?Zhantao Lin. Link

A modification of location commensurate power prior in clinical trials. Our simulation results show that in the presence of large conflicts (a conflict is defined as an average of historical data minus current control data), all Bayesian approaches do not perform well especially in the presence of negative large conflicts, all Bayesian approaches have much lower power compared to frequentist. To address this issue, we propose a modification of location commensurate power prior (mLCPP) that leverages frequentist, traditional Bayesian and LCPP. The proposed approach results in better performance across all conflict profiles. From Hong Tran

????????????????????????????????????????????????????

Events & Webinars

Paolo Eusebi and Dr. Alexander Schacht have created a new podcast The Effective Data Scientist! After having published over 250 episodes of The Effective Statistician podcast, they want to broaden the audience and get many learnings from one podcast over to the other. Check their new Linkedin website: Link

Cytel's webinars. Design considerations for early phase clinical trials of immuno-oncology drug. With Mike Fossler and James Matcham ,?from the Cytel's Strategic Consulting team. 5th January at 8:00am ET. Sign up: Link.?

The Duke-Industry Statistics Symposium (DISS)will take place March 29-31, 2023?with the theme?Empower Clinical Development by Harnessing Data from Diverse Sources.?Registration Link

Recording of the?IDDI - Biostatistics & eClinical Experts?webinar: Why do so many phase III trials fail?, by Marc Buyse and @Everardo Saad (6 December 2022). Learn on statistical explanations for a large proportion of Phase III clinical trials failures, and how to address both the overestimation of treatment effects, and the misinterpretation of P-values via Bayesian methods. Link

Agenda, presentation and recording of Joint EFSPI & BBS virtual event that took place on the 8th and 15th December 2022. Addressing intercurrent events: Treatment policy and hypothetical strategies. How to implement a treatment policy strategy if data after intercurrent events (ICEs) are frequently missing? When is a hypothetical strategy warranted and acceptable to regulators? Is it mandatory to remove data after the ICE for implementing a hypothetical strategy? How to estimate treatment effects for estimands with multiple intercurrent events and mixed estimand strategies? Link