Tag: patient registry

Clinical Research: Phase 1 - Phase 4

Premier Voices #2: Registry Studies & Natural Histories in Rare Disease Trials w/ Angi Robinson & Juliet Moritz

Patient registries and natural history studies are vital tools for orphan drug researchers, who often struggle with limited understanding of diseases and sparse data due to small patient numbers. In the second installment of the Premier Voices podcast with Angi Robinson, Vice President, Clinical Development Services, Rare Disease & Pediatrics, and Juliet Moritz, we...

Natural History vs. Registry Studies in Rare Disease

Patient registry and natural history studies are often thought of as interchangeable, but there are major differences in these approaches to clinical drug development. Understanding the role of each is important, as well as understanding the distinctions on how these study designs are used in pursuing treatments for rare diseases. Patient Registries A patient registry...