Biomarkers (BMKs) have become fundamental tools in drug development, accelerating and optimizing targeted therapeutic innovation. As a bioanalytical CRO with over 15 years of experience in biomarker analysis, we’ve seen firsthand how the strategic integration of biomarkers can accelerate drug programs, help meet evolving regulatory standards, and ultimately lead to better patient outcomes.
In this first article of our Biomarker Series, we provide an overview of how biomarkers are used throughout drug development, the value they bring, and how they are measured.
The Role of Biomarkers Across the Drug Development Lifecycle
Biomarkers are not limited to a single phase of development; they are leveraged across the entire continuum:
- Discovery & Early Development: Biomarkers guide target identification and validation, helping researchers understand disease mechanisms and select promising compounds for further development.
- Preclinical Development: During nonclinical studies, biomarkers are essential for assessing pharmacodynamics (PD), and early indicators of efficacy and safety.
- Clinical Development: In clinical trials, biomarkers provide insights into how a drug behaves in the body, its efficacy, the engagement of the target, and can help identify patient subgroups most likely to benefit from the investigational new drug (IND) therapy.
Biomarkers as Powerful Tools in Drug Development and Clinical Practice
Biomarkers provide critical data that can influence strategic decisions across several steps:
- Deepen the understanding of the drug’s mode of action and the disease’s pathophysiology: This deeper insight can inform more targeted and effective therapeutic strategies.
- Enhance safety profile: By detecting early or subtle adverse effects, biomarkers can help anticipate toxicity issues and adjust dose and administration regimen.
- Demonstrate target engagement and efficacy of a drug candidate: Biomarkers confirm that a drug is interacting with its intended target and generating the expected biological response.
- Enable patient stratification and diagnostic: Identifying which patients are likely to respond to a given treatment.
- Accelerate development timelines: Integrated biomarker strategies streamline drug development, reduce the risk of failure, and can support adaptive trial designs for more flexibility.
Broad Applications Across Modalities and Disease Areas
Biomarkers are modality-agnostic and indication-flexible:
- Applicable across therapeutic modalities, including:
- Small molecules (SMs)
- Biologics
- Vaccines
- Cell & Gene Therapies (CGT)
- Replacement Therapies
- Supportive across any therapeutic area, including:
- Neurological and psychiatric disorders
- Metabolic and endocrine diseases
Depending on the modality and therapeutic area, biomarkers can be analyzed in multiple sample types, encompassing whole blood, plasma, serum, non-plasma biofluids (Cerebrospinal Fluid (CSF), Synovial Fluid (SF), Epithelial Lining Fluid (ELF), Bronchoalveolar Lavage Fluid (BALF), tear, urine), stool, and a wide variety of tissue biopsies (not limited to but including liver, kidney, brain, muscle, and nerve).
Types of Biomarkers: Qualitative, Quantitative, Soluble, and Cellular
Biomarkers can be broadly categorized based on the type of information they provide:
- Qualitative biomarkers provide presence/absence or categorical data, such as protein expression patterns or the detection of a specific pathogen or cell type.
- Quantitative biomarkers offer numerical values and allow precise tracking over time, such as cytokine concentrations, viral load, or gene expression levels.
Biomarkers can also be categorized by their biological nature:
- Soluble biomarkers are typically present in biological fluids (e.g., blood, plasma, cerebrospinal fluid) and include molecules such as proteins, peptides, metabolites, and nucleic acids. These are commonly measured using methods like LBA (Ligand binding assays), molecular biology methods (PCR), or LC-MS (Liquid chromatography coupled to mass spectrometry).
- Cellular biomarkers refer to specific cell types or intracellular markers found in tissues or cells circulating in biological fluids. They are commonly analyzed using techniques such as flow cytometry, immunohistochemistry (IHC), ELISpot, or other cell-based assays.
Biomarker Measurement Techniques
Biomarker data can be collected using a variety of traditional and emerging technologies:
Conventional Bioanalytical & Imaging Techniques include:
- LBA-based, including multiplexing, ultrasensitive, automated technologies, for quantifying proteins or antibodies
- Mass Spectrometry (LC-MS/MS) for quantification of small molecules, peptides, proteins, and metabolites
- Flow Cytometry for immune profiling, receptor occupancy assay (ROA), and cell functionality
- qPCR/dPCR for gene expression analysis
- Next-Generation Sequencing (NGS) for detailed genomic/transcriptomic insights
- PET scan and MRI imaging for functional and anatomical biomarker data
Digital Health Technologies: Expanding the Biomarker Landscape
Wearable and connected devices enable continuous, real-world monitoring of physiological parameters, providing valuable insights outside of traditional clinical settings.
Examples of digital biomarkers include heart rate irregularities detected by smartwatch sensors, fall risk prediction in Parkinson’s disease using motion sensors, or sleep duration and patterns captured through accelerometry.
FAQs: What Our Sponsors Often Ask
Over the years, our clients have raised important questions when integrating biomarkers into their development programs:
- Do you offer off-the-shelf biomarker assays?
- Are your methods validated/qualified?
- What is the difference between qualification and validation?
- Which guidelines should be followed for biomarker validation?
- How do soluble and cellular biomarkers differ, and what methods are used to analyze each?
- What factors should I consider when selecting a platform for biomarker quantification?
We will dive deeper into some of these topics in our upcoming blogs in this biomarkers series.
Final Thoughts
Biomarkers are no longer optional, they are essential tools in designing, optimizing, and accelerating modern drug development. They offer actionable insights that drive informed decision-making and increase the likelihood of clinical and commercial success.
Understanding the context of use (CoU), the biology of the biomarker, the expected levels in the target population, the pathophysiology of the disease, the anticipated impact of the drug and the final use of the biomarker- is crucial for designing the appropriate biomarker strategy is applied for accurate measurement, clinical relevance, and regulatory compliance.
With a large technological park and strong scientific expertise, we deliver personalized, regulatory-compliant biomarker strategies. We support you through every stage of the biomarker lifecycle to help drive your therapeutic program forward.
Get in touch to learn more about how we can support your biomarker strategy today.