Need Help? Chat Now With a Live Representative!

QA Director Nancy Boxx Weighs In On FDA Draft Guidance On Use of Natural History Studies

QA Director Nancy Boxx Weighs In On FDA Draft Guidance On Use of Natural History Studies

QA Director Nancy Boxx Weighs In On FDA Draft Guidance On Use of Natural History Studies

April 22, 2019

In March, The FDA released a draft guidance on the use of natural history studies in rare disease clinical trials.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.

The Vital Role of Team Stability in Advancing Clinical Trials

The Vital Role of Team Stability in Advancing Clinical Trials

In the fast-paced world of clinical research, turnover rates are high and project continuity can be a challenge. However, at […]

March 29, 2024
Breaking Ground in Medicine: Our Role in the Approval of Bivigam by ADMA Biologics

Breaking Ground in Medicine: Our Role in the Approval of Bivigam by ADMA Biologics

Since 2004, ARG has been working in the immunology space, using our individualized approach to optimize the likelihood of successful […]

March 8, 2024
ARG and Humacyte Collaborate on Human Acellular Vessel (HAV) Program Advancements in Vascular Trauma

ARG and Humacyte Collaborate on Human Acellular Vessel (HAV) Program Advancements in Vascular Trauma

CHARLOTTESVILLE, VIRGINIA – ARG (Atlantic Research Group) proudly announces its ongoing collaboration with Humacyte, a leading biotechnology company, on the […]

February 23, 2024
GO TO TOP