Rare Disease Patient Registries Beneficial for Research, Drug Development

A new paper written by member of the National Organization for Rare Disorders (NORD) and published in the journal Pharmaceutical Medicine, argues that establishing rare disease patient registries is beneficial for rare disease research and drug development.

The researchers argue that patient registries can be an effective tool in overcoming many of the obstacles that rare disease-specific patient populations face, including limited funding, dispersed and small populations, and diagnostic delays that are often the result of complex and evolving knowledge.

In addition to documenting the natural history of a disease and keeping clinicians informed on clinical trial progress, the researchers believe that rare disease patient registries act to unite the rare disease community.

“Incorporating collaborative research processes that include a variety of stakeholders such as patient organizations, researchers, clinicians, industry, and government agencies in the planning and design of patient registries is essential for rare disease research,” wrote the researchers.

Reference

Boulanger V, Schlemmer M, Rossov S, et al. Establishing patient registries for rare diseases: rationale and challenges. Pharm Med (2020). https://doi.org/10.1007/s40290-020-00332-1.