NBDA Launch 2014

A New National Alliance to Transform Biomarker Discovery and Development

National Press Club

January 23, 2013

Summary of Panelists’ Remarks

January 13, 2013, National Press Club (Washington, DC) – Nearly two years in development, today the National Biomarkers Development Alliance (NBDA) was launched to address what is perhaps the major barrier to achieving precision/personalized medicine: the discovery and development of successful, clinically relevant biomarkers. The mission of the NBDA is to create standards-based, end-to-end systems approaches to biomarker research and development (R&D). Bringing together key stakeholders from academia, pharmaceutical and diagnostics companies, payers, and patient and advocacy groups under the umbrella of an independent, non-profit organization, the NBDA intends to change the current dismal success rate of biomarker discovery, development, and validation.

Introduction:  In her opening remarks, Dr. Anna Barker, Director and Co-Founder of the NBDA (Co-Director of Complex Adaptive Systems and Professor, ASU, Former Deputy Director, National Cancer Institute [NCI]) stated, “Creating the standards and systems for successful biomarker development is complex but achievable through a new generation of networks of stakeholders that integrate knowledge to solve critical problems of this scale.” She added, “The NBDA was developed not just to relegate the flawed and fragmented approaches to biomarker development processes to the junk pile of history but also to serve as a working example of what purposeful convergence of scientific knowledge and multi-sector collaboration can accomplish.”

The NBDA is comprised of a "coalition of the willing" who understand the fact that the status quo is not working for both scientific and cultural reasons. “We’re declaring a war on failure,” said Dr. Barker. “This is a war we can win and we know how to win it. It will take some new science, but for the most part winning will take bringing together the parties with the will and the resources to create standards-based approaches for biomarker R&D.”

The Problem:  We are living in a remarkable time, one that stands on the precipice of great advances in medicine thanks to genomics and proteomics and the other tools of modern biomedical science, but also a time when the costs of the current health care system is unsustainable. Healthcare costs are projected to increase to $4.4 trillion dollars, or 19.8 percent of the U.S. gross domestic product, by 2020. The much-heralded concept of molecularly (personalized or precision) based medicine is a potentially transformative approach that promises to reduce costs; however, the lack of successful biomarkers—measurable signals of normal or disease-related processes or responses to therapy—currently represents the major barrier to realizing this vision. 

The human genome has been sequenced and a number of pathways identified as implicated in disease, so why is the successful development of clinically useful biomarkers still such a dismal story? “The problem stems from the irreproducibility of much of biomarker science, one might even say it's sloppy science,” said Dr. George Poste, interim Chief Science Officer of the NBDA, Co-Director of Complex Adaptive Systems and Regents Professor at ASU, and Former President of Research & Development for SmithKline Beecham (now GlaxoSmithKline). “It’s about a culture that worries more about publication than replication, that neglects the use of standards and sound statistics, and uses biological specimens that are not fit for the purpose of conducting high quality discovery research.”  

According to the literature, there is no shortage of biomarkers by Dr. Poste’s accounting:  researchers had published over 150,000 papers by 2012 reporting the discovery of new biomarkers. However, the U.S. Food and Drug Administration (FDA) has approved less than one proteomic biomarker per year since the early 1990s, and in the case of cancer, fewer than one hundred are routinely used in the clinic. This is a major trans-sector problem that can and must be fixed!

The Solution:  The NBDA was founded to bring standards and systems thinking to create successful end-to-end biomarker development approaches. “Experience in science and engineering demonstrate repeatedly that standards can enable radical advances,” said Dr. Carolyn Compton, the NBDA's Chief Medical Officer, Professor at ASU, and former President and CEO of the Critical Path Institute. “Standards are well-accepted in the drug development world and are at the heart of the regulatory approval process. However, there is a complete absence of a system to enact and monitor standards in the biomarker arena.”

Dr. Steven Averbuch, Vice President for Translational Clinical Development and Pharmacodiagnostics at Bristol Myers Squibb, reiterated the session's theme: “The biopharmaceutical industry is clearly invested in precision medicine, but we need to apply the same principles of evidence-based standards to biomarker development that we apply to drug development. Applying the standards that the NBDA is developing will allow the promise of biomarkers to become reality.”

 Dr. Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research, said that every day she hears comments from patients such as “why is it taking so long to develop new therapies? Why can’t you tell me if my medicine is working? “And my answer is always the same: we don’t have the tools yet. Biomarkers are the key tools that we need.”

 Dr. Laura Esserman, Director of the Carol Franc Buck Breast Cancer Center and Professor of Surgery and Radiology at the University of California, San Francisco, stated that biomarkers are a critical component of the game-changing I-SPY clinical trials that aim to dramatically shorten the time and expense for approval of drugs and biomarkers. “The goal is to develop a biomarker for use with every drug that gets approved,” she explained.

Clifton Leaf, Assistant Managing Editor of Fortune Magazine, a cancer survivor, and author of The Truth in Small Doses: Why We’re Losing the War on Cancer – and How to Win It, said that the country has wasted billions of dollars in research funds because of the shoddy state of biomarker science and its lack of adherence to standards. “If you have a bad biomarker it’s often more costly than if you have no marker at all,” he noted. The solution, he said, lies with both standards and critical mass. “Without standards, we wouldn’t have the technologies that we marvel at today, and without a critical mass of people buying into the critical importance of standards, we’ll never gain the momentum we need to solve this problem.” He added that in his opinion, "The NBDA offers a unique opportunity to address both of these issues by developing evidence-based standards and creating the necessary critical mass of people, companies, and institutions that value the use of these standards."

Audience Discussion:  During the question and answer period that followed the prepared statements, representatives from several patient advocacy groups voiced their support for the NBDA’s efforts. Among them, Dr. Margaret Foti, Chief Executive Officer of the American Association for Cancer Research (AACR), said, “AACR will absolutely be an enthusiastic supporter of the NBDA.” Audience comments also included suggestions to get journal editors involved as enforcers of standards and to create training programs to educate the research community about the critical importance of using standards in their work, suggestions that Dr. Barker said the NBDA is already at work to implement.

 

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