Rare Disease
Expertise: Rare Disease 87 results
Rare Disease
Operationalizing Gene Therapy Trials
To achieve operational excellence in gene therapy trials, sponsors must understand – and overcome – obstacles ranging from start-up regulations and site selection to patient recruitment and retention. Gene therapy holds promise for treating a wide range of diseases, from...
Rare Disease
Adam Bloomfield, M.D.
Rare Disease
Angi Robinson
Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to...
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.
When launching your next RFP, keep this checklist on hand.
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,...