Biomarkers – Why are biomarkers so important for drug development?

Zubair Israr - 7 September 2021

Biomarkers are used at every stage of drug discovery and development, but why are they so important?

Biomarkers are used at every stage of drug discovery and development.

Biomarkers are biological characteristics that are measured and evaluated as indicators of normal biological processes, pathological or disease processes, or in response to a therapeutic intervention. A good biomarker must therefore fulfil three key criteria; the biomarker must be present in the body; the biomarker must be easily detectable using assays that are both affordable and robust; and the biomarker must reflect a change in stimuli upon the body (i.e., disease progression, drug response etc.).

Drug development programme

Any drug candidate must pass through several development processes for regulatory requirements to ensure the drug is safe, efficacious and beneficial. According to the U.S. Food & Drug Administration (FDA), there are 5 stages of the development process:

Stage 1. Discovery and development – laboratory-based research on the new drug

Stage 2. Preclinical research – laboratory and animal testing on the drug to address basic questions such as safety

Stage 3. Clinical Research – Drugs are tested in patient populations to test safety and efficacy

Stage 4. Review – review and assessment by a regulatory authority

Stage 5. Post-market safety monitoring – drug monitoring once the drug is available to the public

The conventional programme of drug development is problematic. The conventional approach measures drug response using clinical outcomes (e.g., mortality) and therefore obtaining such clinical endpoints may take many years. Drugs that have gone through the pre-clinical and animal testing processes still face 90% failure rate once they are evaluated in people because people don’t respond to drugs in a uniform way.  This means that less than 1 in 10 drugs actually have a chance of getting on the market. This reduces treatment availability, is very costly and time-consuming.

Why are biomarkers so important?

Biomarkers are crucial in preventing the high attrition rates that occur with conventional drug development programmes. Biomarkers are used at every stage of drug discovery and development. During preclinical studies, biomarkers are used to understand molecular pathways of disease, mechanisms of action, selecting drug candidates, dose selection and nonclinical safety. The effect of biomarkers is measured in patients during clinical trials to assess for drug efficacy, safety and toxicity, allowing for earlier and more successful judgement on whether a drug will be beneficial for a particular patient in a particular clinical application. Even after the new drug is approved, biomarkers are used for monitoring therapeutic response and clinical benefit.

According to the database, more than 33,000 clinical trials were involving biomarkers, including around 4000 in Phase 3 and Phase 4. Furthermore, between 2015 and 2019, more than half the drug trials approved by the FDA and EMA were involving biomarkers.

Despite biomarker development, validation and qualification being time- and resource-consuming, it is evident that many drugs are approved with the support of biomarkers.  There are several reasons for this.   The benefits of using biomarkers for drug development and launch are for the patients and drug companies and healthcare payors alike:

  • Biomarkers are able to select the best drug candidates, therefore reducing time and cost at later stages
    Biomarkers allow the use of surrogate endpoints for clinical trials, allowing for clinical prediction over time
    Biomarkers increase the success rate of the drug development timeline and therefore accelerate the availability of new drugs
    Biomarkers can be used to predict or select patients who respond better to drugs using precision medicine approaches


The U.S Food & Drug Administration (FDA), 2018, The Drug Development Process,

Further Reading

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