Thought Leadership

Digital Measures: A Paradigm Shift Advancing Drug Development

February 8, 2024
Digital Measures: A Paradigm Shift Advancing Drug Development

Digital measures, the cornerstone of digital biomarkers, hold immense promise for revolutionizing drug development and advancing our understanding of treatment efficacy. Yet despite their transformative potential, their full utilization remains hindered by standardization hurdles, regulatory ambiguity, and limited awareness among patients and healthcare providers. Biofourmis and HealthXL recently set out to analyze the state of digital measures in drug development. We surveyed experts in pharma and digital health to uncover the current benefits and challenges associated with integrating digital biomarkers into drug development, and outline strategic approaches for their widespread adoption.

Get the full report: Unlocking the Value of Digital Measures in Drug Development

Defining Digital Biomarkers: Unveiling a World of Objective Insights

Before delving into their benefits and challenges, it is important to align on what digital biomarkers are. The research found that differing terms are often used interchangeably, and definitions vary between stakeholders. But we also found some consensus - 81% of those surveyed said that they themselves, or their organization, have adopted the FDA’s definition of a digital biomarker: “a characteristic or set of characteristics, collected from digital health technologies, that is measured as an indicator of normal biological processes, pathogenic processes, or responses to an exposure or intervention, including therapeutic interventions.”

When exploring the ways to create added value from the data collected via digital health technologies, it is digital biomarkers that we feel could offer the greatest potential. They bring invaluable insights into both normal and pathologic processes, fostering a more holistic understanding of disease progression and treatment responses.

Harnessing the Power of Digital Biomarkers in Drug Development

The integration of digital biomarkers into drug development is a natural fit. They serve as sensitive gauges of physiological changes, enabling a clearer assessment of treatment efficacy. This enhanced insight is an opportunity to augment drug development in a way that could bring more individualized treatments to patients in need.

Moreover, digital biomarkers can make use of passive and continuous data collection, capturing real-world health insights outside of traditional clinical visits. This comprehensive data can deliver a nuanced understanding of a patient’s condition, enabling researchers to tailor treatment strategies accordingly.

By employing digital biomarkers as primary endpoints in clinical trials, pharmaceutical companies can enhance their asset development, obtaining early indications of efficacy which may mitigate the risk of lengthy and costly trials. Additionally, the FDA has recently laid out their framework for the use of digital health technologies in drug and biological development, which will ultimately include publishing guidance on how prescription drug use-related software such as digital biomarkers could be used to substantiate label claims.

Navigating the Challenges: Addressing Standardization, Regulatory Hurdles, and Awareness Gaps

Despite their compelling benefits, several challenges impede the widespread adoption of digital biomarkers in drug development. A primary obstacle is the lack of standardization, with evolving definitions and measurement standards hindering comparisons across studies. Global regulatory uncertainty further complicates the landscape, with the FDA and EMA issuing guidelines but lacking unified evidentiary standards for approval. The absence of clear regulatory pathways creates uncertainty for pharmaceutical companies, deterring them from investing in the development of digital biomarkers.

Another significant challenge is the limited awareness among patients and healthcare providers regarding the potential benefits of digital biomarkers. Educating these key stakeholders about the utility of digital biomarkers and providing evidence of their tangible benefits, such as improved treatment outcomes and more efficient drug delivery, will be key in gaining buy-in from these critical stakeholders.

Overcoming these challenges demands concerted efforts to educate patients and healthcare providers about the immense potential of digital biomarkers. Collaboration, standardization, and addressing technical and cost barriers are crucial steps in driving acceptance.

Spearheading Adoption: A Collaborative Call to Action

Pharmaceutical companies play a pivotal role in surmounting these challenges and driving the adoption of digital biomarkers. Collaboration and standardization are essential. By working through industry consortiums such as DiMe and sharing validation data, stakeholders can create a common framework for developing fit-for-purpose digital clinical measures.

These open-source collaboration and pre-competitive initiatives are vital for advancing the field. Instead of proprietary metrics, companies should focus on developing rigorously validated measures that resonate with end-users. Prioritizing meaningful measures of patient health to guide algorithm development is also crucial.

Unveiling Opportunities in Key Therapeutic Areas

Digital biomarkers hold great potential to transform an array of therapeutic areas. While respiratory and neuromuscular conditions have witnessed significant research milestones, oncology and cardiovascular diseases look to be poised to experience the greatest uptake in the next 3-5 years. These areas present unique opportunities for developing and validating digital biomarkers to enhance disease monitoring and treatment outcomes.

One headlining use case is the work that Biofourmis and Vanderbilt have undertaken using DHTs and remote monitoring to measure the safety and efficacy of cancer immunotherapy treatment delivered in a home-like outpatient setting. Key to developing this new standard of care is creation and validation of an algorithm to assess individual risk of developing Cytokine Release Syndrome (CRS) and allow for early detection of onset.

CRS has emerged as a notable concern in cancer immunotherapy, particularly with chimeric antigen receptor T-cell (CAR-T) and immune checkpoint inhibitors. CRS is a systemic inflammatory response resulting from the activation and release of cytokines, triggered by the immune system's robust reaction to cancer cells.Its prevalence has increased as these innovative cell therapies gain traction for their remarkable efficacy against various malignancies.

Early detection of CRS is paramount, as its symptoms can range from mild flu-like discomfort to severe, life-threatening complications. Timely identification utilizing continuous remote patient monitoring and purpose-built algorithms could allow for prompt intervention and management strategies, reducing the risk of CRS-related complications and enhancing the safety and accessibility of cancer immunotherapy.

The Path Towards a New Era of Medicine

Digital biomarkers are poised to revolutionize mainstream drug development and amplify our understanding of treatment efficacy. Overcoming challenges such as standardization, regulatory uncertainty, and limited awareness requires collaborative efforts, patient-centric approaches, and investment in algorithm development. By leveraging the benefits and addressing challenges, we can usher in a new era of medicine, characterized by personalized, effective, and data-driven treatments.

Biofourmis’ was founded on the idea of using data to outpace disease. Through collaboration with our academic and industry partners, we are driving innovation forward that promises to bring new methods of measuring treatment efficacy, mitigating safety risk, and expanding the availability of life changing therapies by reducing the burden on patients receiving care.

Download the full report “Unlocking the Value of Digital Measures in Drug Development” from HealthXL, and visit us a to connect with our experts and learn how we can help advance your drug development.