The CDER/CBER divisions of the FDA released a draft guidance in February 2019 describing various approaches to addressing challenges common in rare disease drug development. This latest guidance focuses on issues faced by sponsors in clinical trials; issues in early drug development/pre-IND are discussed in a draft guidance issued last year. The latest guidance discusses the role of natural history studies in clinical trial design and conduct, what is appropriate for biomarkers, in-trial measurements, and efficacy endpoints, how to use nonclinical studies to support the review process, and considerations for pharmaceutical quality issues. The role of patients and caregivers in the trial process is encouraged, as are the use of breakthrough designations and consideration for pediatric populations. The draft guidance docket is open for comments until 02APR2019.
Text Copyright © 2019 Katrina Rogers