Leveraging Natural History Data for Orphan Drug Development


Leveraging Natural History Data for Orphan Drug Development

Last week, I spoke at Arena’s Outsourcing in Clinical Trials event in San Francisco on the value of leveraging disease natural history data in orphan drug development. Throughout the presentation, I emphasized that a well-designed natural history study helps to establish and quantify unmet medical needs as well as increases understanding of diagnosis challenges, patient geographic distribution, clinical characteristics, genotypes, phenotypes, heterogeneity, and burden of disease.

Disease natural history data is critical to rare disease medical product development where information may be severely limited. In fact, this type of data is often required or encouraged by regulators as it can help with interpretation of both clinical trials and real-world experience post-approval.

Sponsors must be strategic in their timing of natural history data collection. The FDA advises sponsors to evaluate the depth and quality of existing natural history knowledge early in drug development, when knowledge about the disease is insufficient to guide clinical development. Such in-depth understanding of the disease helps sponsors avoid mistakes that may be costly in time and resources, and efficient study of the small number of affected patients may be better guided by greater understanding of the disease.1

There are many benefits for including disease natural history data in product development, especially for rare disease where information may be severely limited.  Has your team considered new strategies to better understand your rare disease patient populations?

UBC offers global Clinical Development & Late Stage Clinical Research services tailored to rare diseases, including clinical trial implementation and management, patient recruitment, peri and postapproval studies, registries, and value demonstration studies.

Click here to learn more about our big solutions for small patient populations, and contact us to find out how UBC can help you do more for your rare disease patients.

1. Rare Diseases: Common Issues in Drug Development Guidance for Industry. U.S. Department of Health and Human Services Food and Drug Administration. August 2015. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformat...