Today, the FDA released a blog post about a new tool for drug developers. The FDA writes, “With the vast majority of rare diseases still without FDA-approved treatments, we have recently released a new resource for drug developers — draft guidance document — designed to help them navigate the difficult and unique challenges of developing and bringing to market new FDA-approved drugs to treat rare diseases.”
The FDA intends for its draft guidance to help drug developers create more accurate and timely drug development programs by encouraging
- a focus on understanding a disease’s “natural history,”
- creation of study designs with clinically meaningful endpoints,
- development of evidence needed to establish safety and effectiveness,
- and the establishment of drug manufacturing specifications to ensure quality.
To read the draft guidance, visit: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf.