White Paper

Registry And Natural History Studies: Vital, Contrasting Roles In Rare Disease Clinical Research

Meeting PHM Challenges

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 is essential for sponsors, patient advocates, and other stakeholders to use data on the natural history of these diseases to drive discussion and formulate drug development strategy. There are two prevalent data collection approaches:

  1. Registry studies, which may include broad collection of defined data
  2. Natural history studies, which are used for controlled, detailed collection of data that is subject to regulatory scrutiny

While registry and natural history studies both play important roles in rare disease research, it is important for sponsors to understand the differences between the two types of studies and how they can be used. In this white paper, we explore the differences between registry and natural history studies, as well as how to design and execute a natural history study to successfully support drug development.

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