In the complex and uncharted territory of rare disease research, particularly cell and gene studies, Premier offers significant experience with natural history studies, precision medicine, and real-world data planning, trial design, and regulatory strategy to guide rare disease product development.
Expertise: Rare Disease 72 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 studies that limit the burden of participation, while keeping patients engaged and generating high-quality data…
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, sponsors need to be proactive in demonstrating their commitment to addressing unmet therapeutic needs. Beyond…
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 intelligence (AI) and computational modeling and simulation (CM&S) to accelerate development of new therapies and…
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 driven an increase in the identification of rare disorders. Currently, one out of every 10…
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 studies to consider when engaging patients and their representatives in study planning. If you’re developing…
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? Although a bit of a balancing act, it is possible to design a study that…
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 and medtech companies take their best ideas from concept to commercialization, and Centogene N.V. [Nasdaq:…
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 the disease of interest, including its frequency, clinical features, risk factors, outcomes, burden, and evolution….