Demonstration Project
Participants at the July 28–29, 2003 Design Team meeting were asked to suggest a possible
model for an NBN demonstration project. During the meeting, it became apparent that there were
many possible approaches for initiating a demonstration project. The model described below
provides a preliminary framework based on the discussion at that meeting. It is anticipated that
this framework will require further refinement.
The ultimate implementation of the National Biospecimen Network (NBN) is understood to be a
complex undertaking. It therefore will be important to evaluate the feasibility of integrating
multiple aspects of a national network by beginning with a demonstration project. The NBN
Design Team recommended establishing a 3-year demonstration project, supported by a
combination of public and private funds, with several important characteristics outlined in Table 8-1. The demonstration project should be able to perform the basic functions of the NBN,
address specific challenges discussed in this Blueprint report, and meet performance
characteristics to evaluate success. In addition, the demonstration project should be capable of
quickly expanding to operate on a national scale.
In considering the demonstration plan, the Design Team recognized the intrinsic value of
incorporating the potential of existing biospecimen resource components as a backbone for the
new system. As a starting point, the Design Team considered strengths in existing NCI resources
in tissue collection and bioinformatics, and expertise in patient privacy issues. In addition, the
expertise of private sector entities in establishing business operations and systems to collect,
process, and distribute biospecimens to the research community is also highly valued. The
Design Team considered and recommended a business model for the demonstration project that
included the use of contracting mechanisms to build on these existing resources.
One of the major challenges in managing the demonstration project will be integrating and
coordinating the diverse activities of the six proposed Business Units. Therefore, it will be
critical to identify a leader for the demonstration project to coordinate these activities. The NBN
demonstration project leader would be responsible for insuring that milestones are met
consistently, and anticipating next steps. This individual could draw upon the resources of a
biomedical incubator such as the Foundation for the NIH (FNIH) and might ultimately serve as
the Chief Executive Officer of the NBN if the demonstration project were successful.
The NBN demonstration project should begin to disseminate biospecimens and associated data to
researchers after one year of operation, and should aim to distribute 15,000–30,000 biospecimens
annually after 3 years. The Design Team estimated that a demonstration project could cost
approximately $15-20 million to develop and operate in the demonstration phase, and suggested
that the NBN identify creative partnering opportunities with the public and private sectors to
provide financial and organizational support for the initiative.
Table 8-1. Characteristics of NBN Demonstration Project
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DEMONSTRATION > > > > IMPLEMENTATION |
Module |
Basic NBN Functions |
Specific challenges |
Performance Characteristics |
Governance and Business Models |
Identify Board of Governors
Establish Operations Center, hire key executive staff
Establish oversight level QA, bioinformatics and communications functions
Coordinate establishment of all functional business units
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Develop detailed business models, examining role of reimbursements and other fees
Define milestones/performance characteristics for a formal evaluation of the NBN demonstration project by function and stage of development
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Evaluate cost recovery efforts
Evaluate contributions to publications, scientific discoveries, patents, and technology development
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Management of Ethical and Legal Issues |
Establish bioethics and legal board
Establish baseline consent procedures
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Revise/expand/optimize consent procedures as needed based on experience and any new regulations
Assess compliance with Federal, state, and local regulations
|
Assess compliance with Federal, state, and local regulations
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Biospecimen and Data Collection and Distribution |
Develop and implement Standard Operating Procedures (SOP) for collecting and annotating biospecimens
Define common data elements for biospecimen annotation
Develop criteria for distributing specimens to researchers that would eventually be adopted by the Biospecimen Utilization Committee
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Insure that SOP development is integrated with Bioinformatics and Quality Assurance efforts.
Investigate approaches (i.e. follow-up questionnaires and/or linking to tumor registries) to collect longitudinal data with biospecimens
Create incentives to encourage data submission from users
Perform genomic characterization on a subset of biospecimens and provide this information to the research community
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Assess longitudinal data collection (i.e. success rate of patient followup)
Document number of specimens collected and distributed
Evaluate the availability and utility of genomic and proteomic data
Test scalability of biospecimen collection system
|
Bioinformatics and Data Management |
Work with Biospecimen and Data Acquisition Business Unit to develop common data elements and informatics applications
Define minimal data set, associated data model, dictionary and baseline reports; deliver first functioning demonstration system
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Integrate work with Quality Assurance efforts
Integrate best functions of existing systems
Expand data model in context of advanced analysis needs
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Assess compliance with industry standards
Evaluate facilitation of all business unit operations (internal)
Assess facilitation of researcher access to clinical and specimen related info (external)
Review effectiveness of analytical tools to further the research goals of users
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Communications |
Work with Specimen and Data Acquisition Business Unit to develop informed consent documents.
Develop an NBN communications strategy that addresses marketing, public relations and outreach issues
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Sponsor focused investigations of ethical and legal issues to develop innovative approaches for obtaining consent and protecting patient privacy within the NBN
Develop incentives to increase and retain participation
Recruit more researchers, tissue donors, and (possibly) funders
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Assess patient recruitment for specimen donation
Document number of researchers applying to use specimens through the Biospecimen Utilization Committee
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In order to streamline the development and integration of disparate aspects of the NBN, the
Design Team recommended focusing the demonstration project on approximately five tumor
types. The Design Team suggested that the tumor types for the demonstration project should be
selected by weighing multiple characteristics including: incidence, mortality, tissue availability
and quantity, as well as the availability of diseased and normal (or normal adjacent) tissues. In
addition, tumors included in the demonstration project should require specimen acquisition sites
to overcome diverse technical procurement challenges, including the impact of technical
advances (such as needle biopsies and neoadjuvant therapy) upon tissue availability. After
completing the demonstration project focused on five tumor types that meet all of these criteria,
NBN specimen acquisition sites could readily expand to collect a broader range of tumors.
In addition to addressing challenges involved in biospecimen collection, the demonstration
project must develop approaches to encourage use of the resource by basic and clinical
researchers. The NBN demonstration project should initiate concerted communication efforts to
identify scientists with ongoing scientific research projects that would benefit from analyses of
biospecimens from the NBN. These researchers could provide timely feedback that could be
included as part of a formal evaluation of the demonstration project, and which would provide an
early indication of the promise for implementing the NBN on a national level.
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