Natural History or Registry Study? Conducting Rare Disease Research

Presented by

Angi Robinson and Juliet Moritz

About this talk

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 physicians who operate around the world. It’s essential that pharma companies, patient advocates, and other stakeholders use data on the natural history of these diseases to drive discussion and formulate drug development strategy. There are two prevalent data collection approaches: registry studies, which may include broad collection of defined data, and natural history studies, used for controlled, detailed collection of data that is subject to regulatory scrutiny. Key elements of this webinar will include: The importance of natural history information in rare disease research and the regulatory importance of comprehensive disease data The difference between registry and natural history studies, and how to decide which will better meet your needs How to design and conduct a registry or natural history study, and what resources are available to help you proceed

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Premier Research is a clinical research company, dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments. As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors. Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions.