The FDA has released a new draft guidance document titled, “Rare Diseases: Natural History Studies for Drug Development.” The guidance document impacts pharmacy supply chain stakeholders, especially drug manufacturers and specialty interests.
What Does the Guidance Do?
According to the FDA, the Agency is publishing this draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. A natural history study collects information about the natural history of a disease in the absence of an intervention, from the disease’s onset until either its resolution or the individual’s death. Although knowledge of a disease’s natural history can benefit drug development for many disorders and conditions, natural history information is usually not available or is incomplete for most rare diseases, therefore, natural history information is particularly needed for these diseases.
Why You Should Participate:
If you are supply chain stakeholder looking to diversify your business into biological products or to serve rare disease patients, the trend is pointing towards greater utilization of specialty products. Comments are suggested to ensure that the government has your viewpoint in mind when developing further guidance and proposed rules.
There are multiple ways to respond to the “Rare Diseases: Natural History Studies for Drug Development” guidance document. Contact Frier Levitt Government Affairs to have your voice heard.