Tag: natural history studies

Medical and Regulatory Affairs

The FDA’s New Guidance on Natural History Studies in Rare Diseases: What You Need to Know

In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack...

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, Vice...

Clinical Research: Phase 1 - Phase 4

Choices, Choices: Which Type of Natural History Study Meets Your Needs?

When you’re looking into conducting a natural history study, you’ve got two things to consider up front: the premise that underlies all of these studies, and which of several types suits your purpose. We tackled these and other topics in our last rare disease webinar. Natural History vs. Registry Studies in Rare Disease considers a...

Clinical Research: Phase 1 - Phase 4

Registry and Natural History Studies: Vital, Contrasting Roles in Clinical Research

Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success. Sound drug development requires a comprehensive understanding of the disease being treated. To design reliable clinical...

Clinical Research: Phase 1 - Phase 4

Natural History Studies: A Growing Force in Treating Rare Diseases

Knowledge of a disease’s natural history is essential to building the scientific foundation for an effective clinical development program. That’s especially the case when developing drugs to treat rare diseases, which by their very nature are poorly understood. Natural history studies don’t just track the course of diseases over time, but identify demographic, genetic, environmental,...

Clinical Research: Phase 1 - Phase 4

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...

Clinical Research: Phase 1 - Phase 4

Natural History or Registry Study? Conducting Rare Disease Research

Sound drug development requires a comprehensive understanding of the disease being treated. To design reliable clinical studies and achieve meaningful outcome measures, researchers must apply known etiology and thorough knowledge of the disease’s progression. For rare diseases, this information is often minimal at best: patient numbers are small and historical data is spread across treating...