Newsletter for Statistical Programmers and Biostatisticians #48

Winners from PHUSE 2022 in Belfast

Application Development (AD) – Analysis and Reporting Table Translation (ARTT) – The Art of Automated Translation: Purusottam Maharana & Qian Wang , MSD Presentation Video

Analytical Risk-based Monitoring (AR)?– Quality Tolerance Limits: Statistical and Programming Consideration: Nathalie van Borrendam , Janssen Research & Development, Annett Keller , Boehringer Ingelheim & Patrice Benner-Bator , MSD Video Presentation Paper

Analytics & Statistics (AS)?– ?Non-inferiority Trial – Growing Significance - Anubrata Kundu , Novartis Presentation

Coding Tips & Tricks (CT)?– CALL EXECUTE Will FIGURE It Out: Data-driven Figure Programming - Mitchikou Tseng, OCS Life Sciences Presentation Paper

Data Handling & Data Engineering (DH)?– DataSet-JSON – Smaller than XML, But Just as Powerful - Fabian Noelle , GSK Presentation

Data Standards & Governance (DS)?– Cross-functional Standardisation of Controlled Terminologies Through Knowledge Graphs - Daniela Bergann & Ena Kolundzic , Bayer Video Presentation

Data Visualisation (DV)?– Pixel Plots: Identify Missing Data, See High-level Patterns and Break Axis Constraints - Paul Grimsey, Roche Presentation Paper

Leadership & Innovation (LI)?– “Co-opetition”: The New Leadership Buzzword for Driving Innovation! - Bhavesh S Patkar , Syneos Health Presentation Paper

Machine Learning & Connected Health (ML)?– How Will Knowledge Graphs Improve Clinical Reporting Workflows? - Alexey Kuznetsov & Jorine Putter , GSK Video Presentation

Professional Development (PD)?– Evolving into Data Science. What Has Changed? Sarah Kirk , Roche Video Presentation

Personnel Management (PM) – Culture Club: Our Experience of Creating an Environment where Individuals Feel Psychologically Safe, Fulfilled, Valued, Valuable and Impactful - Ivan Robinson & Huw Mason , Roche Video Presentation

Poster Presentations (PP)?– What Do You Aspire to Be? A Cloud-based Clinical Analytics Platform Transforming the Clinical Data Flow - Florence Ratcliffe , Instem Presentation

Real-world Evidence (RE)?– Using Individual-level Real-world Data to Inform Disease Area Data Models Employed for Clinical Trial Simulation and Design - Sheila McCartan, PhD , Exploristics Video Presentation

Submissions & Agencies (SA)?– Going through an AdCom with the FDA: The Ultimate Programming Experience - Frederikke H?yer , Ferring Pharmaceuticals Presentation Paper

Software Demonstrations (SD)?– ‘Verify Outputs’ Product Demonstration: Validation of Statistical Outputs Using Automation - Noa Sharon & Roi Kain , Beaconcure Presentation

Standards Implementation (SI)?– Who is Subject X?: Vicky Poulsen & Henning Pontoppidan F?h , Novo Nordisk Video Presentation

Scripts & Macros (SM)?– DOT Language for Consort Flow Diagrams - Nicolas Guerro , Idorsia Pharmaceuticals Presentation Paper

Trends & Technology (TT)?– Building a Trial Pipeline with R Markdown and RStudio Connect - Ari Siggaard Knoph , Novo Nordisk Video Presentation

More for statistical programmers:

How to convert a character value to numeric in SAS. One of the first skills that new SAS programmers learn is how to read and convert raw character data into meaningful numeric data that can be used for calculations and statistics - Chris Hemedinger - Link

Jagadish K. has developed an R Shiny application to view the components of the R package. ?The main important components of the R package are datasets and functions. Through this app, we can view the datasets and functions. It will list all the datasets and functions with in the package. Also, we can select and view individual dataset within the package. Check this link

Hengwei Liu has created a short video on how to customize the jar file for shinyproxy. Check Video

Ben Straub has shared a post on a specialization from Coursera and DeepLearning.AI. Fantastic overview of deploying ML models into production and all its challenges and possibilities. Link

Eduard Parsadanyan has shared a post on How to import CSV into MySQL: four techniques. This step-by-step tutorial focuses on four different import methods:?with the help of a Graphical User Interface like MySQL Workbench, the SQL queries and no-code platforms.?It will benefit both beginners and more experienced professionals. Link

Biostatistics

Accurate Collection of Reasons for Treatment Discontinuation to Better Define Estimands in Clinical Trials has been published online in Therapeutic Innovation & Regulatory Science. This research provides insight and directions on how to optimize the CRF for recording treatment discontinuation. From Yongming Qu , Link

A flexible analytical framework for reference-based imputation, delta adjustment and tipping-point stress-testing. This paper addresses the challenge of implementing the treatment policy strategy when subjects are not followed up after treatment discontinuation. This problem can be addressed using reference-based imputation, delta adjustment, and tipping-point analysis. From Alberto Garcia Hernandez, Teresa Pérez, María del Carmen Pardo,?Dimitris Rizopoulos. Link

Bayesian Strategies in Rare Diseases. Bayesian strategies for planning and analyzing clinical trials have become a viable choice, especially in rare diseases where drug development faces many challenges and stakeholders are interested in innovations that may help overcome them. Authors ?introduce concepts of borrowing, extrapolation, adaptation, and modeling and illustrate them with examples that have been discussed or developed with regulatory bodies to show how Bayesian strategies can be applied to drug development in rare diseases. Ursula Garczarek,?Natalia Mühlemann,?Frank Richard, MD, MBA,?Pranav Yajnik,?Estelle Russek-Cohen. Link

Yannis Jemiai , Chief Scientific Officer at Cytel shared his insights on flexible, adaptive, and novel designs,?simulation-guided design, quantitative bias analysis, estimands, dose-finding in?oncology?clinicaltrials, and more. Link Also from Yannis, a paper Advancing Clinical Trials with Digital Tools: Simulation-Guided Design. Link

Zhiheng Xu , the president of FDA Statistical Association has shared his insights on - New Hope for Alzheimer's Disease Patients. - ?mRNA Cancer Vaccine. - Decreasing Number of Cancer Deaths. -? CAR-T therapy patent fight. -?Over-the-counter (OTC) Hearing Aids. -? Growth of Digital Health. Check his post under this link.

Prateek Shukla has published his paper at Indian Society of Hematology and Blood Transfusion. A Phase 2 Randomized Controlled Trial of Single-Agent Hydroxyurea Versus Thalidomide Among Adult Transfusion Dependent β Thalassemia Patients. Link

Bayesian design of multi-regional clinical trials with time-to-event endpoints. Sponsors often rely on multi-regional clinical trials (MRCTs) to introduce new treatments more rapidly into the global market. Many commonly used statistical methods do not account for regional differences, and small regional sample sizes frequently result in lower estimation quality of region-specific treatment effects. The International Council for Harmonization E17 guidelines suggest consideration of methods that allow for information borrowing across regions to improve estimation. In response to these guidelines, authors develop a novel methodology to estimate global and region-specific treatment effects from MRCTs with time-to-event endpoints using Bayesian model averaging (BMA).? Nathan Bean,?Joseph Ibrahim,?Matthew Psioda. Link

Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies. Traditional vaccine efficacy trials usually use fixed designs with fairly large sample sizes. Recruiting a large number of subjects requires longer time and higher costs. Furthermore, vaccine developers are more than ever facing the need to accelerate vaccine development to fulfill the public's medical needs. A possible approach to accelerate development is to use the method of dynamic borrowing of historical controls in clinical trials. In this paper, we evaluate the feasibility and the performance of this approach in vaccine development by retrospectively analyzing two real vaccine studies. Andrea Callegaro,?Naveen Karkada,?Emmanuel Aris,?Toufik Zahaf. Link

Events and webinars

The 5th?Conference of the?Central European Network?will take place from. 3-7 September, 2023, at the?Biozentrum?of the?University?of?Basel, Switzerland. Established in 2008, the Central European Network’s main purpose is the scientific exchange of information and the strengthening of biometry in Central Europe and around the Alps.?The initial members of the Network include the Austrian Swiss Region, The German Region and the Polish Region.??Joint conferences are held in turn in each region every 3 or 4 years.?Link

The draft agenda for the 2023?PHUSE?US Connect is up writes Michael Rimler . 14 streams plus workshops, panels, and posters.?And on top of that you will find exciting social events:

>> Sunday - Speed Networking >> Monday -?#PHUSE?Connect Dinner >> Tuesday - Poker Night, Top Golf, Whiskey Social

See Michel's post here: Link