Genetic Alliance Registry Lite – Engaging citizen scientists to accelerate translational research
We strive to develop a new paradigm that accelerates translational research. Developing one drug takes almost $1B, takes about 17 years and has a 95% failure rate. The public would be appalled if they knew that the ability to be nimble and innovative in biomedical research is currently stymied by traditional structures that do not allow for the power of networks of citizen-scientists to accelerate translational research. Disease advocacy organizations (DAOs) and other citizen-scientists play important leadership and collaborative roles in research, specifically in the development and recruitment for clinical registries and biobanks.
About You
About You
First Name
Sharon
Last Name
Terry
http://twitter.com/#!/sharonfterry
Facebook Profile
About Your Organization
Organization Name
Genetic Alliance
Organization Website
Organization Phone
202.966.5557
Organization Address
4301 Connecticut Ave. NW, Suite 404
Organization Country
United States, DC
Country where this project is creating social impact
United States
Is your organization a
Non‐profit/NGO/citizen sector organization
How long has your organization been operating?
More than 5 years
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Innovation
Entry Form title
Genetic Alliance Registry Lite – Engaging citizen scientists to accelerate translational research
What change do you want to bring to the world?
We strive to develop a new paradigm that accelerates translational research. Developing one drug takes almost $1B, takes about 17 years and has a 95% failure rate. The public would be appalled if they knew that the ability to be nimble and innovative in biomedical research is currently stymied by traditional structures that do not allow for the power of networks of citizen-scientists to accelerate translational research. Disease advocacy organizations (DAOs) and other citizen-scientists play important leadership and collaborative roles in research, specifically in the development and recruitment for clinical registries and biobanks.
What are the primary activities of your project?
Genetic Alliance Registry & BioBank is a centralized clinical data registry and sample repository that enables translational research. We are planning an expansion to Registry Lite, with standardized, common data elements. This platform will include:
• a public campaign
• educational materials
• a controlled vocabulary, web-based, clinical data collection system for participant data entry
• web-based data collection and management applications
• participant recruiting tools that emphasize trust, privacy protections, data security, empowerment, and ongoing education
• a robust and dynamic process for informed decision-making, tailored to specific uses of the associated clinical information
• a robust data mining system for querying and visualizing donor statistics and cohort development
• a collaboration between citizen-scientists, advocacy organizations, academic, government, and industry partners.
Registry Lite will provide a platform for new registries, the ability to aggregate data from newly developed and existing registries, and provide support for citizen-scientists to establish a registry. We will also offer training on all aspects of the nuts and bolts of registries and biobanks, including how-to guides, manuals, boot camps and other resources. This will enhance the scientific literacy of participants, advocates, engaged consumers, and citizen-scientists.
What is innovative about your initiative? How is it a new contribution to the field?
Our project includes DAOs and citizen scientists as changemakers and drivers of translational research. These individuals have unprecedented, intrinsic knowledge of the medical condition on which they focus, as well as the community that they serve. We understand that every person is a changemaker, and as an active and engaged participant in ones own health care, one can change the paradigm. Citizens declaring ownership of their clinical data and depositing it in open access databases will create the necessary social pressures to change national public policy. Current policy has not yet embraced the participant as an active and engaged stakeholder in health and research. Open source solutions will breakdown walls and create unprecedented datasets to find treatments faster. The new paradigm of innovative, citizen-science can accelerate solutions for symptoms and diseases.
What stage is your project in?
Operating for more than 5 years
Tell us about the community that you engage? eg. economic conditions, political structures, norms and values, demographic trends, history, and experience with engagement efforts.
Genetic Alliance was founded 25 years ago as the support group for all of the disease-specific support groups who were looking for help with capacity building and ways to share their lessons learned. These organizations were also seeking ways to accelerate research efforts on their conditions of interest. Over our history, we’ve maintained strong ties DAOs and trained many on how to develop and execute research initiatives. We have also recognized that our reach is much broader than DAOs, and we have assisted individuals in healthcare, academia, industry, government and others in establishing registries and biobanks.
There are more than 7,000 genetic diseases touching everyone in the world, from heart disease, cancer, and diabetes to the 6,000 or so rarer diseases. These diseases do not discriminate; all socioeconomic groups, demographics, races and ethnic groups are affected equally. Registry Lite has the ability to inform and accelerate research in many communities, including those who are underserved.
Share the story of the founder and what inspired the founder to start this project
Genetic Alliance was founded by a social worker, Joan Weiss, when her son was diagnosed with a heart condition as a baby. She felt no parent should feel as alone as she did then. I learned of Genetic Alliance when my two children were diagnosed with pseudoxanthoma elasticum (PXE), and I founded PXE International, a disease group to accelerate research on PXE. I was aided by the guidance Genetic Alliance offered. At that time it was limited to helping individuals start support groups.
As I worked to create a research consortium for PXE, I met huge obstacles created by a lack of collaboration on the part of academic and industry researchers. When I learned that on average drug development costs almost a billion dollars per project, takes 17 years, and has a 95% failure rate, I was inspired to create a new paradigm. I realized that it would take a team of teams to move research along for any disease – and that the current siloed models, rife with competition would continue to fail. Genetic Alliance Registry & BioBank was born of this passion – my premise was: if disease advocacy organizations, using a long tail aggregation approach, would collaborate in a meaningful way, then we could accelerate this timeline and build in cost efficiencies. I have evidence that this first phase is successful and believe the next one will really accelerate the pace of research. It is time for the efficiencies and technologies that we have seen transform other industries (music, publishing, computers) coupled with consumer engagement transform the biomedical research landscape.
Social Impact
This Entry is about (Issues)
Please describe how your project has been successful and how that success is measured
Currently 6 DAOs are utilizing the current Genetic Alliance Registry & BioBank infrastructure to actively engage in translational research. This has led to the participation of more than 5,000 citizen scientists, and numerous research collaborations. Some of the success metrics include two member organizations discovering the gene associated with their conditions, and another has developed and licensed a diagnostic test, conducted an epidemiological study, a genotype-phenotype association study, and begun enrolling for a clinical trial to test a potential treatment. One member has just made the largest single collection of psoriasis and psoriatic arthritis samples available to researchers to further elucidate the genetics of the complex disease. Another member is utilizing the infrastructure for adverse event reporting to the FDA. Our members are collecting robust clinical data that is not currently being collected by other studies and are using the infrastructure for functional studies and biomarker identification. Genetic Alliance Registry & BioBank is part of a paradigm shift, where DAO’s are driving participatory research, engaging participants within their community, and convening researchers to accelerate translation. Registry Lite has the ability to engage additional groups and community members, and accelerate research for numerous conditions.
How many people have been impacted by your project?
1,001- 10,000
How many people could be impacted by your project in the next three years?
More than 10,000
Winning entries present a strong plan for how they will achieve growth. Identify your six-month milestone for growing your impact
Registry Lite solution will be scoped, developed and operational within the first six months.
Task 1
Complete requirements analysis for Registry Lite.
Task 2
Build Registry Lite to required specifications identified in Task 1.
Task 3
Continue to develop tools and training materials for DAOs and others to drive and accelerate research.
Identify your 12-month impact milestone
Twelve DAOs will be participating in Registry Lite, with the addition of approximately two new groups per month.
Task 1
Conduct outreach campaign to DAOs in Genetic Alliance’s network about Registry Lite.
Task 2
Educate and train a minimum of twelve DAOs for participation in Registry Lite.
Task 3
Launch a public campaign of importance of DAO and citizen scientist participation in research.
How will your project evolve over the next three years?
The next three years will allow for the participation of additional DAOs and citizen scientists in Registry Lite. This will enable more conditions to be represented and more individuals to accelerate translational research. Within three years, we will reach a critical mass that enables researchers to analyze the data within Registry Lite as well as to begin to build cohorts for clinical trials. We will also have the opportunity to educate communities about the importance of public involvement in translational research, as well as to help increase science literacy in the communities we serve.
Sustainability
What barriers might hinder the success of your project and how do you plan to overcome them?
Sustainable finance could hinder the success of the project. In the current Genetic Alliance Registry & BioBank, DAOs that bank clinical information and samples provide the majority (70%) of funding, and Genetic Alliance provides the remainder (30%). For Registry Lite we will obtain funding from additional sources. At this time we are pitching the concept to pharmaceutical and biotech companies that wish to use the resource. We also expect Registry Lite to attract smaller organizations that may not have the capacity to sustain a registry, and they will make a small contribution.
We have mentored dozens of organizations in building registries and biobanks, and our most important learnings have been the realization that not all organizations can develop and sustain research initiatives. In our current strategy, we are educating the greater community, as “rising tides lift all boats”, and helping individuals ask the questions needed to establish and maintain a successful research program. Registry Lite will provide an entry point for citizen-scientists to enter the research space that does not currently exist. This will bridge this time when activated citizens in a team of teams approach, and much less a top down structure, are in need of support and resources.
Tell us about your partnerships
As an organization, Genetic Alliance has a network that includes more than 10,000 organizations, ranging from advocacy organizations to academia to industry. Genetic Alliance Registry & BioBank has formal partnerships with 6 member organizations and relationships with numerous vendors to provide services, including Ocimum Biosolutions, ExamOne, LabCorps, Private Access, and others. In other areas of our work, Genetic Alliance has had as many as 500 formally associated partners in the past 5 years alone.
Current annual budget of project, in US dollars
$250,001‐500,000
Explain your selections
Genetic Alliance Registry & BioBank is currently funded by our 6 member organizations and Genetic Alliance. As described above, we are currently seeking funding from a variety of diverse sources to build and maintain Registry Lite.
How do you plan to strengthen your project in the next three years?
This project will be strengthened by integrating Registry Lite into Genetic alliance’s suite of translational research initiatives. It is a vital component of Gateway to Rare and Neglected Disease Research (GRANDRx), a project that forms the overarching work of accelerating research. It will also support MatchRx, the ‘dating game’ for researchers and diseases and GaugeRx, a tool to help researchers and disease organizations assess the best way forward, since, in general there are no sign posts to help determine where to put resources in the quest for a diagnostic or a treatment for a disease. It is a partner and building block of Disease Infosearch (DIS), a system that connects individuals with hard-to-find information and other people experiencing the same difficulties, ameliorating some of the anguish of a serious diagnosis in a single click.
Challenges
Which barriers to health and well-being does your innovation address?
Please select up to three in order of relevancy to your project.
PRIMARY
Other (Specify Below)
SECONDARY
Lack of access to targeted health information and education
TERTIARY
Limited diagnosis/detection of diseases
Please describe how your innovation specifically tackles the barriers listed above.
Registry Lite has the ability to accelerate translational research, potentially providing access to new treatments for a variety of conditions. Registry Lite is a vehicle for DAOs to help educate the communities they serve on health and the research enterprise. Registry Lite may also help facilitate the development of new diagnostics, leading to increased diagnosis and detection of disease.
How are you growing the impact of your organization or initiative?
Please select up to three potential pathways in order of relevancy to you.
PRIMARY
Grown geographic reach: Global
SECONDARY
Leveraged technology
TERTIARY
Influenced other organizations and institutions through the spread of best practices
Please describe which of your growth activities are current or planned for the immediate future.
Registry Lite has the ability for global reach, as DAOs reach individuals in many geographic areas. Registry Lite leverages technology in a long-tailed approach to serve multiple conditions and disease commuities worldwide. We are also able to share our knowledge and best practices for accelerating translational research that will benefit those in other organizations and institutions.
Do you collaborate with any of the following: (Check all that apply)
Government, Technology providers, NGOs/Nonprofits, For profit companies, Academia/universities.
If yes, how have these collaborations helped your innovation to succeed?
In Genetic Alliance Registry & BioBank, we currently collaborate with other nonprofit organizations, technology providers, and for profit companies. Our members collaborate with other nonprofits as well and individuals in Academia and Industry. We also work closely with the Office of Rare Diseases and share best practices for registry management.
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