What Role do Biomarkers Play in Drug Discovery?

Robust and reliable biomarkers have become a vital tool in healthcare. They are being used to facilitate drug discovery and development, improve diagnosis, enhance techniques that monitor drug activity and therapeutic response, as well as guide the development of precision medicine techniques.

Biomarker Concept

Image Credit: ArtemisDiana/Shutterstock.com

Biomarkers are used at every stage of the drug discovery process. Here, we will review how they are used in this field and how they are paving the way to a future of precision medicine that will see breakthroughs in treating numerous diseases.

The history of biomarker use in health

Although biomarkers seem like a new and advanced concept, they have been used for centuries as indicators of various human health factors and have assisted our ancestors for generations in diagnosing disease. For example, arterial pulse was used by the ancient Chinese, Indian, Egyptian, and Greek to diagnose certain illnesses. In addition to arterial pulse, blood pressure, body temperature, and cholesterol levels have been used for many years as health indicators and are used by modern healthcare professionals globally.

Today, the term ‘biomarker’ has no official definition. However, it broadly refers to a biological parameter that can accurately and reliably be quantified or measured. The European Medicines Agency (EMA) defines a biomarker as a “biological molecule found in blood, other body fluids, or tissues that can be used to follow body processes and diseases in humans and animals”. The interpretation of the term is fairly diverse, and our currently accepted family of biomarkers includes physical parameters such as diastolic blood pressure, molecular parameters such as enzymes and blood glucose, and histologic and imaging characteristics such as angiography.

Biomarkers are not only widely used in diagnostics, but they are also becoming increasingly important to the drug discovery, development, and approval process. Biomarkers have many roles to play in the drug discovery journey, and they are yet to reach their full potential.

Biomarkers in drug discovery

Biomarkers act as clues to solving a mystery. When disease takes hold, it changes the processes in the body. As a result, various metabolites, proteins, and bodily functions can be altered. Often, studying the difference between certain biomarkers in people with a disease and those without can highlight how disease impacts the body.

We can make assumptions about the molecular pathways involved in disease depending on what we know about the certain biomarker or biomarkers that are implicated. This helps scientists to select agents that may have therapeutic value. Similarly, biomarkers are invaluable in deepening our understanding of mechanisms of action, as well as enhancing target selection and drug candidate selection.

Biomarkers can contribute to the rapid development of effective and safe therapeutics. Between 2015 and 2019, more than half of EMA and FDA drug approvals were supported by biomarker data in one or more of the drug’s development stages. In recent years, the acceptance of biomarkers has increased. For certain groups of pharmaceuticals, including immunosuppressants, immunostimulants, antithrombotic drugs, drugs used in diabetes, antineoplastic agents, and antivirals, market approval with the support of biomarkers is more common than approvals without.

One major challenge facing the biomarker field is the need for processes that can easily identify a reliable biomarker from a pool of potential biomarkers.

However, regardless of the resources and time currently required to fully develop a biomarker, drug developers are still increasingly using them due to the benefits they offer the drug discovery process, such as supporting the selection of the best and most appropriate drug candidates while significantly lowering costs and the probability that the drug may fail in clinical trials. Additionally, biomarkers facilitate regulatory and development decisions.

Biomarkers also reduce the number of patients required to demonstrate the clinical benefit of a drug in clinical trials, reducing the cost and some of the risks involved in clinical trials. In recent years, an increasing number of drugs approved by the FDA have used one or more surrogate markers (where biomarkers are used as a substitute endpoint) as primary endpoints for clinical studies. For example, two surrogate markers were used in Odefsey (treatment of HIV-1 infection) studies, which its approval was based on.

Drug Discovery

Image Credit: metamorworks/Shutterstock.com

Biomarkers and the precision medicine revolution

We cannot discuss biomarkers without discussing the emerging field of precision medicine. Naturally, biomarkers are helping to advance this field as they help scientists to understand how a particular group of patients will respond to a certain drug. For patients who have diseases that are, for some reason, resistant to traditional therapies, scientists are now beginning to develop novel drugs that are effective for these specific groups.

Currently, more than one-fifth of drugs approved by the FDA from 2014 to 2018 were categorized as personalized medicines. This percentage will likely grow over the next decade. Therefore, biomarkers will become even more important in drug discovery, as they are used to identify compounds that may be effective in precision medicine applications.


  • Davis, K.D. et al. (2020) Discovery and validation of biomarkers to aid the development of safe and effective pain therapeutics: Challenges and opportunities,” Nature Reviews Neurology, 16(7), pp. 381–400. Available at: https://doi.org/10.1038/s41582-020-0362-2.
  • Gromova, M. et al. (2020) Biomarkers: Opportunities and challenges for drug development in the current regulatory landscape,” Biomarker Insights, 15, p. 117727192097465. Available at: https://doi.org/10.1177/1177271920974652.
  • Hartl, D. et al. (2021) Translational Precision Medicine: An industry perspective,” Journal of Translational Medicine, 19(1). Available at: https://doi.org/10.1186/s12967-021-02910-6.

Further Reading

Last Updated: Jan 27, 2023

Sarah Moore

Written by

Sarah Moore

After studying Psychology and then Neuroscience, Sarah quickly found her enjoyment for researching and writing research papers; turning to a passion to connect ideas with people through writing.


Please use one of the following formats to cite this article in your essay, paper or report:

  • APA

    Moore, Sarah. (2023, January 27). What Role do Biomarkers Play in Drug Discovery?. AZoLifeSciences. Retrieved on March 02, 2024 from https://www.azolifesciences.com/article/What-Role-do-Biomarkers-Play-in-Drug-Discovery.aspx.

  • MLA

    Moore, Sarah. "What Role do Biomarkers Play in Drug Discovery?". AZoLifeSciences. 02 March 2024. <https://www.azolifesciences.com/article/What-Role-do-Biomarkers-Play-in-Drug-Discovery.aspx>.

  • Chicago

    Moore, Sarah. "What Role do Biomarkers Play in Drug Discovery?". AZoLifeSciences. https://www.azolifesciences.com/article/What-Role-do-Biomarkers-Play-in-Drug-Discovery.aspx. (accessed March 02, 2024).

  • Harvard

    Moore, Sarah. 2023. What Role do Biomarkers Play in Drug Discovery?. AZoLifeSciences, viewed 02 March 2024, https://www.azolifesciences.com/article/What-Role-do-Biomarkers-Play-in-Drug-Discovery.aspx.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of AZoLifeSciences.
Post a new comment
Azthena logo

AZoM.com powered by Azthena AI

Your AI Assistant finding answers from trusted AZoM content

Your AI Powered Scientific Assistant

Hi, I'm Azthena, you can trust me to find commercial scientific answers from AZoNetwork.com.

A few things you need to know before we start. Please read and accept to continue.

  • Use of “Azthena” is subject to the terms and conditions of use as set out by OpenAI.
  • Content provided on any AZoNetwork sites are subject to the site Terms & Conditions and Privacy Policy.
  • Large Language Models can make mistakes. Consider checking important information.

Great. Ask your question.

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.