In March, The FDA released a draft guidance on the use of natural history studies in rare disease clinical trials. Nancy Boxx, ARG’s Director of Quality Assurance, has weighed in on the guidance document with a few brief comments.

What is the overall purpose of the document?

The purpose of the guidance is to assist in trial design and support in all phases of drug development for rare diseases.

Specifically, what is delineated?

It outlines the advantages and disadvantage of various types of natural history study designs, for example, although longitudinal studies may provide the most information on disease onset and progression, phenotypes and sub-groups and prognostic factors they are more lengthy and resource intensive than cross-sectional trials.

What is the message to rare disease patients and their access to future opportunities?

The guidance also describes how patients with rare diseases can be encouraged to be participants in these trials and ultimately involved in the drug development process.

Please see Nancy’s previous on FDA guidances on oncology trials here.