What We Do
Reaping the benefits envisioned for molecularly-based precision (used synonymously with personalized) medicine on a broad scale will depend on converting scientific knowledge of molecular alterations in disease into biomarkers that can inform the development of new diagnostics, preventives, and therapeutics. Biomarkers represent specific indicators (markers) of normal or disease-related processes or measures of pharmacologic response to therapy.
The discovery of biomarkers has become a major focus for researchers in both the academic and private sectors. Through 2011 over 150,000 publications claimed the discovery of thousands of biomarkers (Poste G. Bring on the Biomarkers. Nature 469, 2011, 156-157). Unfortunately, in the face of this virtual “tsunami” of potential disease biomarkers, their successful advancement to the clinic has proven difficult at every stage. For example, although protein biomarkers offer the greatest promise to drive personalized medicine, FDA approval of new protein biomarkers has been essentially flat for the past 15 plus years, averaging ~1 new protein biomarker per year since 1994. (Polanski and Anderson, Biomarker Insights 2006: 2:1– 48). The impact of this critical barrier is further reflected in the cost of developing a new drug which can be well over a billion dollars and take up to 15 years. A 10% improvement in predicting failure would save $100 million/drug—a need that high quality biomarkers could address.
Even though in an area such as technology standards a great deal of work has been published. There are currently no widely accepted and/or adopted best practices, guidelines, standards, etc., for the development of biomarkers. Consequently there is no real evidence-based, predictable end-to-end processes that address these major barriers. Lack of any real effort to unite the stakeholders to solve these problems has led to highly publicized examples of publication of data that cannot be reproduced, which is undoubtedly tied to the failure of a number of biomarker driven clinical trials. The need for such a trans-sector network of affected organizations is obvious—and the timing for its implementation is now.
The National Biomarker Development Alliance (NBDA) is a big idea with audacious goals, but one that is necessary (and possible) if we are to meet these challenges and deliver on the promise of precision medicine. The NBDA is bringing together the major stakeholders in biomarker development (disease agnostic) to identify the specific barriers—and possible solutions—at each stage of biomarker development. this NBDA "due diligence: will as a basis for the creation of evidence-based, end-to-end predictable biomarker development processes.
In brief, the NBDA team, through a unique management construct, will create knowledge networks that are committed to assembling and creating NBDA Standards*, the guidelines, best practices, and processes necessary to effectively advance biomarkers from discovery; through clinical trials; and successful regulatory review and approval. The NBDA will be agnostic as to disease and technologies: all diseases where biomarkers could make a real difference in diagnosis, treatment, prevention, and patient management will receive careful consideration by the NBDA. Achieving these goals will involve a number of strategies, new trans-sector networks, workflows, and broad community engagement. Some examples of the types of activities the NBDA will undertake are as follows:
- Conduct needed forums, workshops, and think tanks to analyze the end-to-end biomarker development process.
- Identify the major “modules” and critical interfaces for biomarker development.
- Convene consensus groups, as needed, to agree on the NBDA Standards*/guidelines and/or best practices are needed to address each barrier identified in the due diligence process.
- Undertake the necessary integration of existing material, outcomes, new research, and/or demonstration projects needed to address each barrier.
- Create education approaches with NBDA partners to broadly inform affected communities about the value proposition of successful biomarkers to advancing precision medicine.
- Engage with partners from all sectors to identify classes of biomarkers (or individual biomarkers) that require a clear end-to-end evidence base for their rational development.
- Insofar as possible make findings, recommendations, workflows, processes, and data generally available.
- Create community resources that are based on clear standards (including a potential NBDA biobank and biomarker database).