There may be as many as 7,000 rare diseases. The European Union defines a disease as rare when it affects less than one in 2,000 people. According to the Orphan Drug Act, a rare disease affects less than 200,000 people in the United States. Support from FDA, EMA and other regulatory agencies, combined with technological and scientific advances, have made it more feasible for pharmaceutical companies to give dedicated attention to orphan drug development. In doing so, they're developing medications for rare diseases that often have no cure and no available treatment. To ensure novel therapies advance from the lab to regulatory approval, a blockbuster-drug approach won't work. Managing rare disease studies to completion requires innovative thinking in nearly every phase of program development. Those willing to become trailblazers have the highest odds of success.
Biorasi’s Oncology Center of Excellence remains at the forefront of this exciting field. Our team of clinicians, program managers and other experts stay up to date on the latest developments, from Tumor Infiltrating Lymphocyte (TIL) therapy to gene therapy advances. This experience, combined with Biorasi’s worldwide site network and data-driven approach, allows us to guide oncology programs to success—on time and on budget.
It's rare for any large project that involves hundreds of individuals and multiple locations to proceed without some sort of hiccup. Budgets get stretched, schedules run [...]
Movement disorders such as Parkinson’s disease, ataxias, tremor, dystonia, tics, Tourette’s, and dozens of rare diseases, affect nearly 40 million Americans. They're usually chronic, often progressive, [...]
Have you ever put off talking to your significant other about expensive-but-necessary home repairs, for fear they'll hit the (leaking) roof? Most of us have, and [...]
Each year, doctors diagnose about 1,000 Americans with cystic fibrosis, a rare hereditary disease that affects about 30,000 people in the United States. Thanks to advances [...]