Rare Disease
Expertise: Rare Disease 69 results
Rare Disease
Operationalizing Gene Therapy Trials
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 evaluate large complex databases and to integrate information in useful ways. One exciting application of…
There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease.
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.
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…