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Regulatory Pathways for Utilizing Biomarkers in Drug Development

Biomarkers can play an integral role throughout the entire drug development lifecycle, and biomarkers can improve the success rate of a drug development program, bringing new therapies to market faster. Biomarkers can be employed for a variety of purposes, including monitoring the safety of a drug or therapy, as well as helping to determine if treatments are producing desired outcomes. Biomarkers have the potential to save both cost and time during clinical trials by predicting which patients may respond better to a drug from a safety or efficacy perspective.

Figure 1. The Role of Biomarkers Throughout the Drug Development Lifecycle

Regulatory Pathways for the Integration of Biomarkers in Drug Development

FDA outlines three primary sources for biomarker evidence: scientific community consensus, drug-specific development and approval process, and the biomarker qualification program. It is important to note that while each pathway has its own strengths and limitations, some biomarkers will be a better fit for a specific pathway.

Scientific Community Consensus

When a drug developer pursues the regulatory pathway of scientific community consensus, they rely on information from published scientific literature, which provides the basis for their hypothesis regarding the use of the specific biomarker in the drug development process. Data that informs scientific community consensus is scrutinized and reviewed by multiple stakeholders and can lead to greater understanding of biologic processes or diseases. However, solely relying on scientific literature can pose challenges to drug developers looking to determine the clinical utility of a biomarker, as the studies that inform scientific literature often result from varying objectives, which can make it difficult to correlate and compare information across multiple publications.

Drug-Specific Development & Approval Process

When biomarkers are developed for a specific biologic program or investigational new drug, drug developers are encouraged to utilize the drug-specific development and approval process. The drug-specific development and approval process regulatory pathway is intended to be utilized for regulatory acceptance of biomarkers that are intended to be used for a specific candidate drug. However, it is important to note that the information provided for any specific drug program can be used by other drug developers or drug companies if the biomarker is proven to have more generalized applications.

Biomarker Qualification Program

Once a biomarker is qualified for a specific context of use through CDER’s (Center for Drug Evaluation and Research) Biomarker Qualification Program, it becomes publicly available and can be applied to any drug development program where the context of use (COU) is appropriate. In these instances, there is no need to review the information again. The Biomarker Qualification Program is intended to establish the value of a biomarker to be used in drug development for a particular COU. FDA defines context of use as a concise description of the biomarker’s specified use in drug development, containing two components: the BEST biomarker category and the biomarker’s intended use in drug development. A single COU should be identified with each biomarker qualification effort.

Figure 2. Regulatory pathways for the inclusion of biomarkers in drug development

Determining the Right Regulatory Pathway for Your Biomarker

In early development stages, it may be difficult to determine which regulatory pathway is the right fit for a specific biomarker. In these instances, FDA encourages drug developers to contemplate the reasons and circumstances for using the biomarker in drug development, what resources are available, the type of biomarker, and opportunities to collaborate with others. Selecting a regulatory pathway is always up to the discretion of the drug developer, whether the developer is a single company or a consortium because biomarker development is a voluntary activity.

Considerations for Biomarker Qualification

When biomarkers are submitted to CDER for qualification, the context of use (COU) for the biomarker must be clearly defined. For example, enrichment biomarkers enable targeted patient selection for clinical trials, identifying a patient population in which the drug candidate will likely have the greatest effect. Another example of context of use would be a surrogate endpoint biomarker, used as an endpoint in a clinical trial to determine whether the drug is effective and patients are experiencing the expected therapeutic effect.

It is important to note that the evidence required for biomarker qualification is not uniform or standard for all biomarkers that are submitted to CDER. Evidentiary criteria is dependent upon the potential impact on patients. Evidentiary criteria are different for different types of biomarkers, and when the stakes of the biomarker being correct are very high and the consequences of a wrong predication are very serious, the evidentiary criteria bar is higher. However, there are some common criteria for qualification of biomarkers regardless of context of use, including robust analytical validity.

Figure 3. Evidentiary framework for biomarker qualification

Partnering with a CRO/CDMO to Support the Use of Biomarkers in Drug Development

Choosing the right CRO/CDMO to partner with for bioanalytical support for your biomarker throughout the entire drug development lifecycle, including meeting regulatory requirements to gain approval for the use of a specific biomarker in drug development, is just as critical as selecting the right regulatory pathway. Avomeen’s Ph.D.-led team of consultative, expert scientists stand ready to support drug development from discovery through clinical trial and beyond. Our flexible teams can meet your needed turnaround time, delivering quality results on tight timelines, and our dedicated team of Project Managers facilitate consistent, open communication between clients and scientists. Are you ready to start the conversation about how Avomeen can support you throughout the drug development lifecycle? Get in touch with a scientist.

Additional Resources

Know more about the critical role biomarkers play in drug development

Explore how biomarkers are used for therapeutic drug monitoring (TDM)

Learn more about biomarkers and FDA’s BEST resource

Why do we love biomarkers so much? Check out our top 3 reasons


Our featured expert is Kevin Gorman, Ph.D., Avomeen’s Manager of Bioanalytics. Kevin’s extensive knowledge of protein biochemistry and molecular biology enables him to provide clients with expert strategic and tactical guidance to help them reach their goals. Kevin has experience in analytical method development, validation, and optimization of test methods in cGMP settings per ICH guidelines. In addition, Kevin has experience developing in-vitro diagnostics, including a serology assay for COVID-19. He is listed as a co-inventor on three patents and is highly skilled in several research techniques including LC-MS, HPLC, ddPCR, and ELISA.