Regulatory Focus™ > News Articles > First-Ever Drug Development Tool Tentatively Clears FDA's Qualification Process

First-Ever Drug Development Tool Tentatively Clears FDA's Qualification Process

Posted 13 January 2014 | By Alexander Gaffney, RAC

Just days after releasing a guidance document extolling the virtues of its drug development tool (DDT) qualification process, the US Food and Drug Administration (FDA) has tentatively released the text of its first qualified clinical outcome assessment (COA) tool, one intended to expedite the development of products to treat chronic bronchitis.

Background

The DDT program, as explained in a final guidance released on 6 January 2013, is intended to help guide the development of a product for a population in which the product can create the most benefit.

For example, a drug intended to treat a rare genetic condition would benefit from a test that would validate the presence of that genetic marker in a patient-a so-called biomarker. But within industry, there is presently a problem of scale. Many drug makers will create and validate DDTs for their specific products, but not more broadly, slowing the development of other therapies that might stand to benefit from the same tool in the future.

FDA's DDT Qualification Program aims to change that paradigm by reviewing tools-biomarkers, COAs and pathways under the Animal Rule-and qualifying their use for any entity that wishes to utilize them.

First Tool Clears Process

Now, the first of those COA qualifications has been released by FDA as a draft attachment to its Guidance on Qualification Process for Drug Development Tools.

The attachment, called Qualification of Exacerbations of Chronic Pulmonary Disease Tool for Measurement of Symptoms of Acute Bacterial Exacerbation of Chronic Bronchitis in Patients With Chronic Obstructive Pulmonary Disease, is a COA tool known as EXACT (Exacerbations of Chronic Pulmonary Disease Tool).

The tool was notably the first to enter into the DDT qualification process, and was developed under the EXACT-PRO (for patient-reported outcome) initiative to help assess COPD patients.

FDA explains in the guidance that its regulators have determined that the tool is "qualified as a measure of symptoms of acute bacterial exacerbation of chronic bronchitis in patients with chronic obstructive pulmonary disease (ABECB-COPD)."

However, its use is not unlimited. Rather, there are specific contexts of use (COU) in which the use of the tool is appropriate. For example, it is qualified for use in Phase 2 studies as either a primary or secondary endpoint, and is intended to support labeling claims related to symptom changes in a patient.

Other caveats: The tool is intended for superiority trials enrolled with COPD patients with acute bacterial exacerbations of chronic bronchitis. The tool has not yet been validated for use over the course of an exacerbation, FDA wrote.

To be fair, there are other DDTs that have been qualified, such as several biomarkers, but the EXACT appears to be the first COA yet approved by FDA-a major step not just for the DDT program, but for anyone wishing to develop new COPD therapies as well.

The guidance remains in draft form, and FDA will be accepting comments on it until 10 April 2014.


Qualification of Exacerbations of Chronic Pulmonary Disease Tool for Measurement of Symptoms of Acute Bacterial Exacerbation of Chronic Bronchitis in Patients With Chronic Obstructive Pulmonary Disease

Federal Register


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