SEP 30, 2015 1:30 PM PDT

Biomarkers: An Auspicious Beginning and a Portentous Future?

Speaker
  • Anna Barker, PhD

    Co-Director, Complex Adaptive Systems Initiative, Director, Transformative Healthcare Networks, Professor, School of Life Sciences, Arizona State University (ASU)
    BIOGRAPHY

Abstract

Biomarkers, with their associated “tsunami” of publications, represent what is arguably one of the largest areas of scientific inquiry in the history of biomedicine. Touted as the foundation for “precision medicine”, biomarkers do represent an unparalleled “value proposition”, as they are central to transitioning from a 20th century “one size fits all” approach to healthcare to a 21st century molecularly based model. Based on individual patient molecular (and other) biomarkers, this model would connect research to patient care in a seamless process. Early in the history of biomarkers, in areas such as cardiovascular disease and cancer, it appeared that the discovery and development of successful disease biomarkers might indeed be a straightforward process. However, that vision has proven elusive. Although to date, literally thousands of biomarkers exist in the literature as “discovered”, in the specific case of protein biomarkers, the FDA has approved less than one per year since 1993. This lack of validated biomarkers also contributes to the dismal success rate of late stage clinical trials in oncology and other complex diseases. Large-scale projects in whole genome sequencing (such as The Cancer Genome Atlas and other international efforts) are producing unprecedented – and often unmanageable – multi-dimensional data sets in cancer and other diseases. These efforts promise to inform and enable biomarker discovery by identifying the molecular changes associated with complex diseases. Questions as to whether these molecular changes are causative or merely associated with a given disease generally remain unanswered; and it undoubtedly take time to understand the intricacies of genomic and other molecular changes that characterize complex diseases. In parallel to these profound scientific questions that underlie biomarker science, there are real problems associated with the discovery and development of biomarkers that require a unified effort by the affected communities to achieve real solutions. For example, the lack of standards for biomarker discovery and development in areas ranging from the relevance of the original clinical question, to the quality of biospecimens, to the provenance of data and appropriate analytics all contribute to biomarker failure. Furthermore, end-to-end regulatory pathways for biomarker qualification and validation have lacked specificity in terms of required evidentiary standards. The FDA is currently addressing many of these issues through its increased emphasis on community engagement in biomarker qualification. Assuming that knowing the molecular underpinnings of complex diseases will enable success in the discovery of clinically relevant biomarkers, most, if not all, of the other problems are solvable. The National Biomarker Development Alliance (NBDA) is a non-profit, multidisciplinary and trans-sector network organization that is dedicated to achieving solutions to many of the systemic problems that plague the field. The presentation will focus on the state of the biomarker field and progress by the the NBDA and other efforts to create end-to-end biomarker discovery and development approaches dedicated to patient benefit.


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SEP 30, 2015 1:30 PM PDT

Biomarkers: An Auspicious Beginning and a Portentous Future?



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