FDA Publishes Guidance on the Rare Disease Treatments

Published on: 

The new guidance, Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings Guidance for Industry, discusses the planning of pre-IND meetings with FDA and sponsors.

FDA published draft guidance on October 15, 2018 to assist sponsors developing treatments for rare diseases plan for pre-investigational new drug application (pre-IND) meetings with FDA. In the guidance, the agency describes topics that should be considered in early drug development and pre-IND meetings.

The agency states that while issues discussed at pre-IND meetings may vary depending on the drug, development stage, and targeted disease, sponsors should consider quality, nonclinical evaluation, clinical pharmacology, early phase study designs, and statistical analysis plans when preparing a pre-IND meeting package. In addition to these topics, the guidance also addresses expedited programs for serious conditions, companion diagnostics, orphan drug product incentives, pediatric studies, and data standards and electronic submissions.


Source: FDA