04.14.23 -- Progression For Patients Needing Precision Medicine

Competition in clinical trials has only gotten worse, as industry interest in rare diseases grows. Sponsors looking to differentiate their trials and inspire patient interest should consider these five tips to demonstrate their commitment to addressing unmet therapeutic needs.

Regulatory agencies typically require that a sponsor conduct a randomized controlled trial, with the control group being either a placebo, an active control, or a control arm using the standard of care. Discover how in silico trials meet ethical codes and can be used to evaluate drug candidates.

Rare diseases have a low prevalence per person, yet these unusual conditions tend to influence society on a much larger scale. As precision medicine advances, understand how the timing of rare disorder diagnosis could be improved so that treatment can begin at earlier stages of the illness.

To develop a successful gene therapy, sponsors must conduct research studies that balance the need for safety oversight and robust clinical evidence with the challenges of finding the right patients. In this article, uncover several key considerations for designing gene therapy trials.

During Phase 2 studies, it can be a challenging and lengthy process to assess dose-effect signals when multiple doses and a placebo are involved. For sponsors considering a Bayesian response-adaptive style design, review this guide for tips to consider prior to developing a clinical study protocol.