Can Drug Development be Accelerated? FDA Wants Help

Regulatory NewsRegulatory News | 12 February 2015 |  By 

The US Food and Drug Administration (FDA) is soliciting input on how it might accelerate the development of certain drug products through the use of new and emerging biomarkers.


In FDA's telling, a qualified biomarker—"an objective characteristic that is measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or pharmacologic responses to treatment"—can help drug development in several ways.

For example, a biomarker might allow a company to more easily identify which patients would—and which would not—benefit from an experimental treatment. A biomarker might also allow a company to identify which patients would be harmed by a drug, or which patients need a different dose of a drug in order to benefit.

At FDA, the effort to identify appropriate biomarkers has traditionally focused on two areas: the identification of the biomarker as being useful, and the development of tools to capitalize on the identification of the biomarker.

FDA's DDT Qualification Program

FDA's argument has essentially been that while the identification of a useful biomarker is important, even more important is the application of that information to the development of new products.

To address this, in 2012 FDA created a new program known as the Drug Development Tools Qualification Program to qualify—i.e. validate as being useful and appropriate—new biomarkers, clinical outcomes, assessments and animal models.

The hope of the program is that the pharmaceutical industry will be able to accelerate the development of new therapies. For example, a company might use a biomarker to replace a drug's clinical efficacy endpoint or accelerate a testing phase.

"The path to development of promising therapeutics can be enabled by the availability of biomarkers that are analytically validated and clinically qualified for a specific context of use (i.e., a comprehensive, clear, and precise statement that describes the manner of use, interpretation, and purpose of use of a biomarker in drug development)."

FDA has said it also hopes the program will "foster" the development of new biomarkers and better integrate the use of qualified biomarkers into the regulatory review process.

Biomarker Tools

To date, FDA has qualified four drug development tools based on biomarkers:

Biomarker(s) Referenced in GuidanceIssuance Date with Link to Specific GuidanceSupporting Information
Urinary biomarkers: Albumin, β2- Microglobulin, Clusterin, Cystatin C, KIM-1, Total Protein, and Trefoil factor-34/14/2008 Drug-induced Nephrotoxicity Biomarkers Reviews
Urinary biomarkers: Alpha-glutathione S-transferase (αGST), Clusterin, Renal Papillary Antigen (RPA-1)9/22/2010 Drug-induced Nephrotoxicity Biomarkers Reviews
Serum/plasma biomarkers: Cardiac troponins T (cTnT) and I (cTnI)2/23/2012 Drug-induced CardiotoxicityBiomarkers Reviews
Serum/bronchoalveolar lavage fluid biomarker: Galactomannan10/24/2014Invasive Aspergillosis Biomarker Reviews

The biggest problem, FDA explains in a new Federal Register notice, is that it just doesn't have the resources to validate biomarkers on its own. To overcome this, the agency says it wants to work with private entities to help catalyze progress in the area.  "Because of limitations in resources, such efforts must be focused on the opportunities that offer the greatest potential for impact."

"FDA intends to facilitate identification of the most promising biomarkers and the areas important to drug development and to promote efforts that will aid in the qualification and regulatory adoption of the drug development framework," it added.

Identifying Promising Areas

The agency also wants the public's help to "identify promising biomarker candidates in areas important to drug development and to identify considerations for evidence needed to qualify various types of biomarkers for specific contexts of use."

Responses should include information about the biomarker, as well as a discussion about the specific aspects of drug development the biomarker could "enhance" and why the biomarker represents the "greatest near-term opportunity to establish utility in drug development."

Comments are due in 60 days.


FDA Request Notice


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