Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of…
Abie Ekangaki
Small patient populations. Geographic dispersion. Competing studies. Sound familiar? If you’re a sponsor of a rare disease study, you know all too well the challenges that come with recruiting patients. These challenges lead to immense pressure to design and implement studies that limit the burden of participation, while keeping patients engaged and generating high-quality data…
Oncology & Hematology
9 Tips for Designing and Operationalizing Early-Stage Precision Oncology Studies
Traditionally, early-stage clinical trials focus on toxicity assessment and dose selection. Today, a growing number of Phase 1/2a trials are designed to draw inference about preliminary response rates due, in part, to the use of biomarkers and adaptive design approaches that enhance the early detection of efficacy signals. These approaches may add to study complexity…
Cancer is one of the most scientifically complex and dynamic diseases. Even with significant advances in our understanding of the genetic and molecular mechanisms that lead to cancer, only 10 percent of approved oncology drugs demonstrate an overall survival benefit.1 In recent years, there has been a shift in how early-phase oncology studies are conducted…
Traditionally, Phase 1 trials commonly utilize 3+3 designs to determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose (RP2D). Studies have shown, however, that two out of three trials employing a 3+3 design failed in identifying the MTD, and better approaches are needed.1 During Premier Research’s recent webinar Alternative Designs to the…
Oncology & Hematology
Considerations for Applying Adaptive Design Approaches to Early Oncology Studies
By utilizing accumulating data to modify the operating characteristics of an active trial in accordance with pre-specified rules, adaptive designs can make clinical trials more flexible, efficient, and informative than fixed-sample designs.1 Adaptive design approaches can be applied across all phases of clinical development, including early oncology studies. These designs introduce real-time flexibility while a…
Oncology & Hematology
Minimizing Early-Stage Oncology Failures with Adaptive Design Approaches
Despite advances in our understanding of the complex genetic, molecular, and immunological factors which lead to cancer, the success and likelihood of approval rates for oncology remain low. According to a recent study performed by the Biotechnology Innovation Organization (BIO), nearly one-third of drugs entering phase 2 studies between 2006 and 2015 failed to progress….
In response to the newly released U.S. Food and Drug Administration (FDA) guidance on COVID-19, the Statistics Department at Premier Research has committed to highlighting specific and necessary actionable considerations that directly address key FDA recommendations in the guidance. These considerations call attention to some general actions required to ensure the operational integrity and scientific…
They say that great leaders aren’t born, but grow into their positions through sweat and tears. No matter what stage in life, industry, or phase of your career you’re at, it is important to continuously strengthen your skills, and this ongoing development is one of the key values we look to foster in our team…
Oncology & Hematology
6 Early Phase Dose-Finding Trial Designs for Oncology Therapeutics
Phase I and II trials may have different overall goals (i.e., demonstrating safety vs. efficacy), but the two both struggle with a major challenge in oncology study design: finding the right dose. Luckily, decades of data and innovations have given researchers the tools necessary to plan a successful dose-finding trial. Read on for a look…