FEATURED ARTICLES

• In Silico Modeling Unveils A New Era In Rare Disease Drug Development

  Gain insights into how innovative silico modeling is revolutionizing rare disease drug development and opening new possibilities for accelerated research.

• Evaluating A CRO For Rare Disease Studies: A Checklist For Success

  When launching an RFP, refer to this checklist to ensure comprehensive coverage of all aspects when evaluating potential research partners for the rare disease study.

• Revolutionary CAR-T Therapies Pave Way To Conquer Solid Tumors

  Get a comprehensive and insightful overview of the latest advancements in CAR-T cell therapy and their potential to transform cancer treatment.

• Rescuing A Phase 1 Oncology Trial: Long-Standing Experience Saves Study Momentum

  Study transitions are inherently challenging. Here’s what teams should know to stay informed, keep processes aligned, and successfully transfer data.

• To Placebo Or Not To Placebo? The Great Debate In Rare Disease Trials

  Consider the severity of the disease, the availability of existing therapies, and more when deciding whether or not to use placebo control in rare disease trials

• 5 Things To Know About Bayesian Response-Adaptive Design

  For sponsors considering a Bayesian response-adaptive style design, review this guide for tips to consider prior to developing a clinical study protocol.

• Patient Centricity In Rare Disease: 5 Ways To Maximize Engagement

  Sponsors looking to differentiate their trials and inspire patient interest should consider these five tips to proactively demonstrate their commitment to addressing unmet therapeutic needs.

• Executing A Dual-Submission Study

  Dual submissions can pose a range of difficulties for clinical trials. Gain insight into how multiple requirements can be addressed successfully with the right strategy.

• Establishing Clinical Utility Of Molecular Diagnostics For Precision Medicine: 5 Tips On RCT Conduct

  Explore five ways to approach designing and conducting a randomized clinical trial to demonstrate clinical utility.

• IVDR Survival Guide Part 1: Classification, Clinical Evidence, & Performance Requirements

  Have common questions concerning the IVDR product classification system addressed and learn how to comply with new clinical evidence and performance expectations.

• Rare Disease And Precision Medicine: A Road To Growth

  As precision medicine advances, discover how the timing of rare disorder diagnosis could be improved so that treatment can begin at earlier stages of the illness.

• Rare Disease Patients As Partners: Value Beyond Enrollment

  Learn why teams are putting an increased emphasis on creating processes and frameworks to encourage sustainable patient partnerships and establishing a paradigm of patient-centered research.

• How Patient & Caregiver Journeys Guide Rare Disease Study Designs

  Discover how designing trials that account for both patient and caregiver needs can improve the overall experience of study participation and may contribute to improved patient retention and higher-quality data.

• Can Your Protocol Flex? The Importance Of Adaptive Trial Designs In Precision Oncology Studies

  Discover how adaptive trial designs work to limit the number of patients exposed to ineffective doses or treatments and accelerate detection of efficacy signals.

• 9 Design & Operationalization Tips For Early-Stage Precision Oncology Studies

  Early-stage clinical trials tend to focus on toxicity assessment and dose selection, which requires careful planning in study design and execution. Explore nine tips to improve precision oncology study processes.