Small patient populations. Limited data on disease. Patient enrollment and retention challenges. Narrow site pools. These are some of the most common hurdles sponsors face in rare disease drug development. The right CRO partner can help turn these challenges into...
Expertise: Rare Disease 89 results
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...
As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders,...
MORRISVILLE, N.C., MILAN, ITALY, TRIESTE, ITALY, March 13, 2023 — Premier Research, whose mission is to help the most innovative biotech and device companies take their best ideas from concept to commercialization, and InSilicoTrials, a leader in the use of artificial...
Individually, rare diseases by definition have a low prevalence, but collectively, the societal burden and impact of these uncommon conditions is significant. The majority of rare diseases are genetic in origin, and advances in genomic sequencing tools and technologies have...
In 2020, the European Joint Programme for Rare Diseases developed a guide on patient partnerships in rare disease research projects. This guide still rings true today, and we summarize in this blog the key points for sponsors of rare disease...
It comes as no surprise that putting patients first is imperative for achieving success in rare disease development programs. However, to what extent can sponsors integrate patient input into their study design while still securing the high-quality data they need? ...
Leveraging Rare Disease Insights Powered by the CENTOGENE Biodatabank and Centralized Multiomic Laboratories in Clinical Trials MORRISVILLE, N.C., and CAMBRIDGE, Mass., ROSTOCK, Germany, and BERLIN, January 9, 2023 — Premier Research, whose mission is to help the most innovative biotech...
Increasing emphasis on rare diseases has led to a greater need to accurately define the profile, characteristics, and outcomes of patients living with those conditions. The development of safe and effective treatments of rare diseases requires a deep understanding of...
Natural history studies play a crucial role in rare disease drug development, providing insights that advance discovery and shape clinical trial design. The benefits of these studies also extend to understanding patient journeys, clarifying the standard of care, identifying disease-specific...