It is an unfortunate reality that many diseases and conditions affect such small numbers of patients that, when a sponsor develops a drug or biological product to treat them, relatively little return on investment is generated to offset the high development costs....
Expertise: Rare Disease 89 results
Rare Disease
Daniel Zamfir, MBA
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...
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.