Natural History Studies In Gene Therapy Trials: Benefits, Timing, And Execution

By Raul P. Lima, Executive Vice President, Clinical Operations, inSeption Group

A traditional regulatory pathway requires being able to accurately describe the normal clinical course of the disease. This can be difficult with rare diseases because existing academic data is incomplete and/or poorly controlled. Moreover, data rarely exists that is relevant to specific, high-sensitivity endpoints needed for a gene therapy study.

Clinical trial researchers not only need to understand the course of the disease broadly, they need to understand how measurements should be interpreted throughout the course of that disease. But, while clinical trial researchers have access to a wealth of tests and equipment, practicing physicians generally do not hook up patients to finely tuned instrumentation or require patients to fill out numerous patient-reported outcome (PRO) forms.

It is critical to consider the benefits and impacts of an NHS as early as possible. Considering it early allows it to be in the funding plan. This has many benefits to the program as whole, including collecting important contextual data that may not exist, taking a dry-run of the expected clinical study visits, testing deployment of sensitive equipment, and testing endpoint sensitivity.