Reviewing FDA Draft Guidance On Biomarker Qualification And Validatio

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biomarker assays

Teaser: A holistic post on the critical aspects of the latest FDA guidance on biomarker qualification. With a special focus on the Cure Act and Precision Medicine Initiative.

The U.S. Food and Drug Administration issued a new guidance document in 2018 on biomarker qualification. The new guidelines include recommendations that match the requirements of the 21st Century Cures Act, 2016.

This document guides the industry on biomarker assays is necessary for the Precision Medicine Initiative. This is a national-level effort that preceded the Cures Act by a year.

The Cures Act is really a formalization of FDA recommendations. For qualifying molecular drug development tools and clinical outcome assessments.

In its latest guidance document, FDA presents the evidentiary framework within which to conduct biomarker testing assays. For compliance with the Cures Act.

Before we discuss the evidentiary framework, let us revisit how biomarker testing relates to PMI.


PMI And Biomarkers: The Connection


The Precision Medicine Initiative (PMI) derives from the patient-centered care model. The aim of PMI is to deliver “the right treatment to the right patient at the right time.”


Healthcare professionals can now figure out the exact molecular aberrations of every individual patient. Technological advances have made that possible. That enables targeted treatment, in turn.


This is where biomarker testing has a critical role to play. Biomarker tests are necessary for molecularly targeted therapies.
PMI has the potential to radicalize treatments for improved health outcomes. Its adoption, however, has been slow, owing to a variety of barriers.


Oncology has been the most proactive in the adoption of PMI.


The Linkage With Drug Development


The precision medicine model also offers an immense opportunity for the pharmaceutical industry. However, a mindset shift towards drugs that target smaller subgroups of patients needs to happen.


Cancer therapeutics has been the most adoptive. But barriers remain in other areas of drug development. Financial resources pose a problem. So do the risks involved in finding and testing biomarkers. Big data handling is a challenge, as well.


The industry needs to understand the FDA’s latest guidance on biomarker testing and biomarker validation against this backdrop.

Key Points From The FDA’s Evidentiary Framework


The Cures Act promotes an era of therapies based on the molecular comprehension of a disease. The evidentiary framework recommends processes to develop molecular therapeutics to assist such treatment procedures.


Different components determine the kind and level of evidence necessary for qualifying a biomarker.


Needs Assessment
The evidentiary framework should explain why it is necessary to develop a particular drug.


The Context of Use (COU)
It is necessary to specify the category of the biomarker. Also its proposed use in the development of the new therapeutic.

Potential Benefits
How qualifying this biomarker will add value to the drug development process.

Potential Risks
What are the probable risks of the biomarker does not suit its intended use?


Evidence Necessary for Biomarker Qualification
Evidence should include, but is not limited to:
● The biological rationale for choosing the biomarker
● Data that supports the correlation between the proposed biomarker and the intended clinical outcome
● The analytical performance during the biomarker validation process

The biomarker validation process needs to assess the performance features of the biomarker assays used. It is important to demonstrate that the biomarker assays used are suitable for the COU.

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