This March, CDER, CBER, and the Office of Orphan Products (OOP) issued a draft guidance describing their current thinking on the performance of natural history studies. This type of study is used to collect information on the course of a particular disease and to identify demographic, genetic, environmental, and treatment variables

that correlate with the disease’s development and outcomes. The focus of the guidance is on preplanned natural history studies of rare diseases, but the principles may still apply to nonrare diseases. The guidance focuses on the use of data from preplanned natural history studies to support drug development for rare diseases. Such studies can help identify and stratify the patient population, develop reliable clinical outcome assessments, identify biomarkers for the disease and potentially support the design of a clinical study using an external control group. The guidance points to additional benefits for rare disease patients such as establishing communication pathways, establishing disease centers of excellence, evaluating the current standard of care practices and finding ways to improve patient care. The document describes types of natural history studies and provides recommendations on study protocols, data elements, research plans, data collection/storage/dissemination, and human subject protection. I thought the points on planning for future use of data and samples in the Informed Consent section particularly foresighted. The draft guidance is open for comment until 24MAY2019.

Text Copyright © 2019 Katrina Rogers