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,...
Expertise: Rare Disease 77 results
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...
A key challenge of developing therapies for rare diseases is the availability of relevant data. With small, often geographically-dispersed patient populations, natural history information is typically sparse or even unknown. Without this data, it may be difficult to identify biomarkers...
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...
Rare Disease
The Expanding Role of RWE in Rare Studies
Real-world evidence is increasingly important in drug development as the medical and pharma communities recognize the data’s potential for predicting the benefits of treatment interventions.
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...
Successful drug development requires a deep understanding of the disease of interest—its etiology, epidemiology, presentation, manifestations, and progression. In rare diseases, however, much of this information may be unknown. Patient populations are small and historical data are collected inconsistently and...