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| 8066 | Strategic Plan Introduction | Overview of NCATS The National Center for Advancing Translational Sciences (NCATS), one of the 27 Institutes and Centers (ICs) at the National Institutes of Health (NIH), is transforming translational science to get more treatments to more patients more quickly. NCATS relies on the power of data, new technologies and teamwork to develop, demonstrate and disseminate innovations that reduce, remove or bypass costly and time-consuming bottlenecks in translational research. Rather than targeting a particular disease or area of fundamental science, NCATS focuses on what is common across diseases and the translational process. The Center conducts and supports research on both the scientific and operational aspects of translation to lead to more predictive and successful development of new medical interventions, such as drugs, diagnostics, and medical devices, for all human diseases. NCATS’ efforts by design complement and empower those of other NIH ICs, academic scientists, the private sector and the nonprofit community. By emphasizing collaboration, innovation, deliverables and team science, the Center serves as a catalyst to enable others in the translational research ecosystem to work more effectively. Translation and Translational Science Defining Translation and Translational Science Translation is the process of turning observations in the laboratory, clinic and community into interventions that improve the health of individuals and the public — from diagnostics and therapeutics to medical procedures and behavioral changes. Translational science is the field of investigation focused on understanding the scientific and operational principles underlying each step of the translational process. NCATS studies translation on a system-wide level as a scientific and operational problem. Translational Science Spectrum The translational science spectrum, as illustrated in the graphic above, represents each stage of research along the path from the biological and chemical basis of health and disease to interventions that improve the health of individuals and the public. The spectrum is not linear or unidirectional; each phase builds upon and informs the others. At all stages of the spectrum, NCATS develops new approaches, demonstrates their usefulness, and disseminates the findings. Patient involvement is a critical feature of all phases in translation. Basic Research — Basic research involves scientific exploration that can reveal fundamental mechanisms of biology, disease or behavior. Every stage of the translational research spectrum builds upon and informs basic research. NCATS scientists typically do not conduct basic research; however, insights gained from the Center’s studies along the translational spectrum can inform basic research. Preclinical Research — Preclinical research connects the basic science of disease with human medicine. During this stage, scientists develop model interventions to further understand the basis of a disease or disorder and find ways to treat it. Testing of medical interventions is carried out using cell or animal models of disease; samples of human or animal tissues; or computer-assisted simulations of drug, device or diagnostic interactions within living systems. Clinical Research — Clinical research includes studies to better understand a disease in humans and relate this knowledge to findings in cell or animal models; testing and refinement of new technologies in people; testing of interventions for safety and effectiveness in those with or without disease; behavioral and observational studies; and outcomes and health services research. The goal of many clinical trials is to obtain data to support regulatory approval for an intervention. Clinical Implementation — The clinical implementation stage of translation involves the adoption of interventions that have been demonstrated to be useful in a research environment into routine clinical care for the general population. This stage also includes implementation research to evaluate the results of clinical trials and to identify new clinical questions and gaps in care. Public Health — In this stage of translation, researchers study health outcomes at the population level to determine the effects of diseases and efforts to prevent, diagnose and treat them. Findings help guide scientists working to assess the effects of current interventions and to develop new ones. Center Organization NCATS’ divisions and offices span the entire spectrum of translational science. Through programs in its Division of Preclinical Innovation, the Center drives advances in early stages of the translational process, from target validation to first-in-human studies. Through its Division of Clinical Innovation, NCATS supports clinical and translational research, creating and sharing the expertise, tools and training needed to develop and deploy effective treatments in people. Cross-cutting programs in rare diseases, translational technologies, strategic alliances and other emerging areas address scientific and operational barriers to enable faster and more effective interventions that tangibly improve human health. Bridging the Gap: Scientific and Operational Challenges and Opportunities in Translational Science Several thousand genetic diseases affect humans, of which only about 500 have any treatment. A novel drug, device or other intervention can take about 14 years and cost $1 billion or more to develop, and about 95 percent never make it past clinical trials. Even when a new drug or other intervention is developed and shown to be effective in clinical trials, many years may pass before all patients who could benefit from it are identified and treated. Numerous scientific and organizational roadblocks can limit the speed of progress. Obstacles along the path to translation include: Lack of understanding about the science of translation and insufficient rigor in conducting translational research, leading to unpredictability and frequent failure of possible interventions A shortage of qualified translational investigators Organizational structures and incentives that do not encourage the teamwork essential to translational science Inflexible and inefficient clinical trial implementation and low participation in studies A lack of data interoperability Insufficient tools and technologies to predict toxicology and efficacy in safety assessment A shortage of qualified biomarkers for application in disease diagnosis and measurement of treatment response Inadequate development and measurement of appropriate clinical outcome measures or endpoints, including patient reported outcomes | /sites/default/files/Spectrum_Graphic_900px.jpg | Strategic Plan Introduction | By emphasizing collaboration, innovation, deliverables and team science, NCATS serves as a catalyst to enable others in the translational research ecosystem to work more effectively. | /sites/default/files/Spectrum_Graphic_900px.jpg | Strategic Plan Introduction | |||
| 8065 | NCATS Strategic Principles | NCATS’ strategic principles represent its philosophy. To accomplish the overarching goal of bringing more treatments to more patients more quickly, NCATS programs are guided by the following principles: Catalytic: NCATS is a catalyst that enables others to perform more efficient and effective translation. Generalizable Principles: NCATS uncovers fundamental principles shared among diseases and translational processes; widespread implementation of such generalizable principles will accelerate translation. Innovative: NCATS programs lead to profound improvements in translational understanding and effectiveness, producing innovation that establishes fundamentally new ways of doing translation that are multiplicative in their effects. Collaborative: Translational research endeavors require the expertise of multiple people and groups, particularly as the research is carried across through different phases of the translational science spectrum. NCATS approaches translation as a “team sport.” Patient-focused: At all phases of translational science, NCATS is committed to patients and their communities and looks for opportunities to include the patient perspective. The ultimate goal of translation is tangible improvement in health, so the perspectives of and partnerships with patients are crucial. Measurable: NCATS continuously improves translational effectiveness, so programs must be designed and implemented with explicit indicators of success for translational progress. | To accomplish the overarching goal of bringing more treatments to more patients more quickly, NCATS programs are guided by these principles. | /sites/default/files/strategic-plan_900px.jpg | NCATS Strategic Principles | To accomplish the overarching goal of bringing more treatments to more patients more quickly, NCATS programs are guided by these principles. | /sites/default/files/strategic-plan_900px.jpg | NCATS Strategic Principles | ||
| 8064 | Strategic Plan Development | Internal Process: Strategic Principles NCATS employees were engaged in the early stages of the Center’s strategic planning process to provide their perspectives on the fundamental characteristics that guide activities at NCATS, both current and aspirational. Through a series of small group discussion sessions, the opinions and insights of nearly all NCATS employees were gathered. These diverse perspectives contributed to the development of the request for external input as well as the final strategic principles that are presented in the NCATS Strategic Principles section. Gathering External Input: Focus Groups To identify the key strategic planning topics on which NCATS would seek public input, NCATS created a series of focus groups composed of NCATS researchers, program leaders, other staff, and members of NCATS’ two advisory committees: the Advisory Council and the Cures Acceleration Network (CAN) Review Board. Each focus group was asked to identify priorities and challenges in each of six pre-defined overarching areas of NCATS’ research and operational activities. These focus areas were: Preclinical Innovation — Improving the Drug Development Process; Preclinical Innovation — Testing and Predictive Models; Repurposing Drugs; Re-Engineering the Clinical and Translational Process; Accelerating and Supporting Rare Diseases Research; and Building Partnerships with Stakeholder Groups. All focus groups also were asked to consider two cross-cutting questions: (1) how NCATS could more effectively engage with patients and community members in each phase of translation, and (2) how the strategic plan should address the role of “big data” and informatics in translational science. Stakeholder Engagement: RFI and Webinars After receiving additional input from NCATS Advisory Council and CAN Review Board members at a public meeting in early September 2015, NCATS launched the stakeholder engagement phase of the strategic planning process in October 2015 to solicit feedback from the broader public. The goal of this phase was to identify areas of opportunity, challenges and research needs in translational science to help set the Center’s strategic priorities. NCATS’ stakeholders include, but are not limited to, patients and members of the health advocacy community; basic, translational and clinical scientists at universities and research institutions; health care providers; biotechnology, venture capital and pharmaceutical industry members; colleagues at other NIH Institutes, Centers and Offices; partners at other government agencies (e.g., the Food and Drug Administration and other Department of Health and Human Services agencies, the Environmental Protection Agency, and the Department of Defense); policymakers; and the general public. A public request for information (RFI) and a series of four “town hall” webinars were the principal vehicles used to solicit feedback from these individuals and groups. The RFI was publicly disseminated in October 2015 and was open for comment until February 2016, and the public informational webinars took place in October and November 2015. Through the RFI, NCATS encouraged stakeholders to comment on any issues of interest that apply across the translational science spectrum, including: Breaking down professional, cultural and scientific silos across the translational science spectrum Focusing on inter-operability of data systems (such as integrating patient data and electronic health records into preclinical research) Expanding research efforts at NCATS into new therapeutic modalities Focusing on patient-driven research and patient/community engagement Forming innovative partnerships with a wide variety of stakeholders Identifying skillsets and competencies needed for training the next generation of translational scientists Using modern communication and dissemination tools to expand awareness of translational science to a wide variety of stakeholders NCATS received 54 responses to the RFI from individuals, organizations and institutions representing academia, government, industry, patient advocates, and health care providers. A total of 119 unique participants from around the country and across multiple sectors signed into the webinars to learn more about the NCATS strategic planning process and how best to contribute comments about the Center’s strategic priorities via the RFI. The analysis of RFI responses involved carefully reviewing each response, along with any attached supplementary materials, and identifying specific recommendations and actionable items provided. The responses addressed a broad array of priority issues across both the scientific and operational domains of translation, and directly informed the development of the objectives and example approaches within each strategic goal. | NCATS developed its Strategic Plan by creating strategic principles and engaging focus groups and stakeholders. | /sites/default/files/strategic-plan_900px.jpg | Strategic Plan Development | NCATS developed its Strategic Plan by creating strategic principles and engaging focus groups and stakeholders. | /sites/default/files/strategic-plan_900px.jpg | Strategic Plan Development | ||
| 8063 | Structure of the Strategic Plan | The Strategic Plan is organized into four overarching themes of translational science, collaboration and partnerships, education and training, and stewardship. Each theme is captured within a strategic goal, and collectively, they provide an overview of what NCATS plans to accomplish to achieve its mission. The objectives listed under each Strategic Goal offer greater context to the high-level themes and will be used to guide NCATS in developing, evaluating and refining its research and operational agenda over time. Example approaches are potential ways for NCATS to achieve these objectives. In brief, the strategic goals reflect NCATS’ overarching mission, the objectives provide context for each goal, and the example approaches offer more specific tactics for undertaking the objectives. | The Strategic Plan is organized into four overarching themes of translational science, collaboration and partnerships, education and training, and stewardship. | /sites/default/files/strategic-plan_900px.jpg | Structure of the Strategic Plan | The Strategic Plan is organized into four overarching themes of translational science, collaboration and partnerships, education and training, and stewardship. | /sites/default/files/strategic-plan_900px.jpg | Structure of the Strategic Plan | ||
| 8062 | Strategic Plan Acknowledgments | NCATS appreciates the extensive input from its staff, the NCATS Advisory Council, the Cures Acceleration Network Review Board, and members of the translational science community in the development of this strategic plan. In particular, NCATS would like to thank the members of the Strategic Plan Working Group for leading the development of this plan: Penny Burgoon, Christine Cutillo, C. Taylor Gilliland, Cindy McConnell and Dorit Zuk. | NCATS appreciates the extensive input from many members of the community in the development of this Strategic Plan. | /sites/default/files/strategic-plan_900px.jpg | Strategic Plan Acknowledgments | NCATS appreciates the extensive input from many members of the community in the development of this Strategic Plan. | /sites/default/files/strategic-plan_900px.jpg | Strategic Plan Acknowledgments | ||
| 8061 | Megan O'Boyle (2019) | Ms. O’Boyle is the Principal Investigator for the PCORI funded Phelan-McDermid Syndrome (PMS) Data Network (PMS_DN, PCORnet) and the Phelan-McDermid Syndrome International Registry (PMSIR). She is also a parent of a child with Phelan-McDermid Syndrome. This diagnosis includes autism, intellectual disabilities, epilepsy, ADHD, and other medical conditions. The web-based PMSIR was launched in May 2011, and currently has more than 70% of the PMS Foundation families enrolled. Ms. O’Boyle also directed a biosample collection for PMS families that is now part of the NIMH Stem Cell Center at the Rutgers University Cell and DNA Repository. She is passionate about the value of the patient’s voice in research, drug development, clinical trial design, development of related legislation, and quality of life decisions. She advocates for data sharing, developing international patient registries, improving patient engagement, sharing resources and streamlining IRB practices and policies. | Ms. O’Boyle is the Principal Investigator for the PCORI funded Phelan-McDermid Syndrome (PMS) Data Network (PMS_DN, PCORnet) and the Phelan-McDermid Syndrome International Registry (PMSIR). | Megan O’Boyle | Ms. O’Boyle is the Principal Investigator for the PCORI funded Phelan-McDermid Syndrome (PMS) Data Network (PMS_DN, PCORnet) and the Phelan-McDermid Syndrome International Registry (PMSIR). | Megan O’Boyle | ||||
| 8060 | Daniel Hartman (2019) | Daniel L. Hartman, M.D., joined the Bill & Melinda Gates Foundation in 2012 as the director of Integrated Development and leads a team that provides technical expertise in drug and diagnostic development. Under his leadership, Integrated Development works closely with the Foundation’s strategy teams to manage product pipelines through clinical trials and to provide help with manufacturing, compliance and regulatory strategy/intelligence. Hartman joined the Foundation after four years as president and chief executive officer of Great Lakes Development, Inc., a consulting company that provides strategic and operational support for early drug development projects. Previously, Hartman served as senior vice president of product development at deCODE genetics, executive director of Pfizer Global Research and Development and vice president of Global Clinical Development at Esperion Therapeutics. He also has held senior clinical research positions at Eli Lilly & Company and is a member of the Scientific Medical Advisory Board at Hopen Life Science Ventures. Hartman received his bachelor’s degree from Calvin College and his medical degree from Wayne State University. He trained in internal medicine and completed a fellowship in pulmonary medicine at Indiana University, where he was chief medical resident. | Dr. Dan Hartman joined the Bill and Melinda Gates Foundation in 2012 as the Director of Integrated Development and leads a team that provides technical expertise in drug and diagnostic development. | Daniel Hartman | Dr. Dan Hartman joined the Bill and Melinda Gates Foundation in 2012 as the Director of Integrated Development and leads a team that provides technical expertise in drug and diagnostic development. | Daniel Hartman | ||||
| 8059 | Alan Palkowitz (2019) | Alan Palkowitz, Ph.D., is currently senior research professor of medicine in the Department of Clinical Pharmacology at the Indiana University (IU) School of Medicine. He is a key contributor to multiple translational programs focusing on Alzheimer’s disease and pediatric cancers as part of the IU Precision Health Initiative. Palkowitz most recently was the vice president of discovery chemistry research and technologies at Eli Lilly and Company, where he worked for 28 years. In his role as vice president, Palkowitz was responsible for the global small-molecule strategy and delivery of clinical candidates in all areas of disease focus, including cancer, diabetes, immunology, pain and neurodegenerative disorders. Palkowitz oversaw a large, multisite global research enterprise that included medicinal chemistry, computational and biophysical/structural sciences, molecular and cellular pharmacology, analytical technologies, synthetic technologies, and automation sciences. Palkowitz championed the creation of multiple molecular discovery strategies to diversify approaches to drug discovery and successfully expand therapeutic innovation. In addition, he designed and implemented unique business models aimed at expanding global access to diverse talent and capabilities in partnership with academic centers, small biotech companies and government researchers. As a member of the Lilly Research Laboratories leadership team, Palkowitz participated in setting strategic direction for the company, along with technical governance of the discovery and early clinical development pipeline. He currently serves on several advisory groups, including the NCATS Advisory Council and the National Academy of Sciences Board on Chemical Sciences and Technologies. | Dr. Palkowitz is the Vice President of Discovery Chemistry Research and Technologies at Eli Lilly and Company. | Alan Palkowitz | Dr. Palkowitz is the Vice President of Discovery Chemistry Research and Technologies at Eli Lilly and Company. | Alan Palkowitz | ||||
| 8058 | NCATS-Supported Researchers Recruit Citizen Scientists to Help Mine Biomedical Literature | Biomedical scientists are publishing new discoveries at a rapid rate. Currently, PubMed, the primary database for biomedical literature housed by NIH’s National Center for Biotechnology Information, contains more than 26 million articles and is expanding by more than 1 million articles per year — that’s two articles per minute. The research boom is great news for biomedical science, but it is challenging for humans to keep up with such massive amounts of information. Enter a team of bioinformatics scientists from the Scripps Translational Science Institute (STSI) at The Scripps Research Institute (TSRI) in La Jolla, California, an NCATS Clinical and Translational Science Awards (CTSA) Program hub. Led by Andrew Su, Ph.D., associate professor in the Department of Molecular and Experimental Medicine at TSRI and director of computational biology at STSI, the group invented a web-based technology platform to arrange biomedical literature into a format that is easier for computers to organize and analyze. Because of the size of such a task, the platform, called Mark2Cure, is designed to employ crowdsourcing. Crowdsourcing is the practice of recruiting large numbers of people to help solve a complex problem—in this case, sorting and organizing thousands of biomedical papers. Su and his bioinformatics colleagues Max Nanis, Ginger Tsueng, Ph.D., and Benjamin Good, Ph.D., hope that Mark2Cure can make the biomedical literature more manageable and useful by enabling scientists to rediscover buried knowledge that can spur new research hypotheses. “The Mark2Cure platform exemplifies the CTSA Program’s mission to develop innovative solutions that will improve the efficiency, quality and impact of the process for turning observations in the laboratory, clinic and community into interventions that improve the health of individuals and the public,” said NCATS Director Christopher P. Austin, M.D. Citizen Scientists as Research Partners Citizen scientists Judy and A.J. Eckhart share their motivations for contributing to the project. Citizen scientists contribute much more than data, offering valuable insight, suggestions and feedback on how to improve a project. The Eckharts and many other participants provided feedback on how to improve Mark2Cure’s tutorials and interface. (Su Lab, The Scripps Research Institute) Humans are better than computers at certain tasks, such as scanning text and recognizing keywords and the relationships between them. “Computers have trouble interpreting the free text in a scientific article very well. We call this an information extraction problem,” Su explained. “On the other hand, humans have a well-developed sense of how to parse language, understand grammar and infer meaning, even when the language is technical and jargon-filled.” With this knowledge, the team set out to build a platform by which volunteers from the general public, whom they call “citizen scientists,” help solve the information extraction problem. After logging onto the web-based platform and undergoing a brief training exercise to become comfortable with scientific language and concept identification, the citizen scientists complete a two-step process. First they identify relevant concepts, such as genes, proteins, drugs or diseases, in the text, and then they define the relationships between concepts. For example, a relationship may be expressed by stating that a particular drug treats a certain disease; for example, insulin (drug) treats diabetes (disease). “If we carried out those two steps for every article published, we would have a powerful knowledge base that computers could mine very effectively,” Su said. In an initial experiment, Su and his team tested the effectiveness of the Mark2Cure approach by comparing the citizen scientists’ efforts with those of experts performing the same task. The Scripps researchers found that, in the aggregate, the citizen scientists performed the task of highlighting disease mentions within biomedical text with very high accuracy, comparable to that of the experts. The researchers also found, through survey responses, that the citizen scientists had high levels of desire and motivation to volunteer for Mark2Cure; most respondents cited advancing science or learning as their motivation for participating. The Scripps scientists are publishing these findings in Citizen Science: Theory and Practice. Using Mark2Cure to Study a Rare Disease A knowledge network constructed from concepts identified by citizen scientists within months after Mark2Cure’s launch. Blue text indicates disease-related terms, green text indicates gene-related terms, and pink text indicates treatment-related terms. Many highly motivated citizen scientists were concerned about the quality of their work and were reassured to see that high-quality annotations dominated knowledge networks generated from their work. (Su Lab, The Scripps Research Institute) Su and his group now have turned to testing Mark2Cure in the context of an actual disease. To start, they are focusing on N-glycanase 1 (NGLY1) deficiency, an extremely rare inherited disorder that affects multiple organs, causing developmental delays, movement problems and seizures, among other symptoms. According to Su, rare diseases are an ideal starting point, because the literature base is relatively small and members of patient groups are often well-informed and highly motivated to help with research. Indeed, one of the ways NCATS seeks to improve the process of developing interventions is by engaging patient communities and advocacy groups and partnering with them to carry out research. Already, the citizen scientists have identified a potential treatment for NGLY1 deficiency that is not typically associated with the condition: adrenocorticotropic hormone. The hormone improved some of the symptoms in one patient, and while it ultimately was not a viable treatment option, the discovery points to the potential of the Mark2Cure approach. Beyond NGLY1 Deficiency Mark2Cure is designed to help explore virtually any disease, rare or common, and the Scripps team plans to expand beyond NGLY1 deficiency in future projects. It is open-source, meaning that the software and data are available for anyone to access, but the researchers caution that the technology remains experimental. Still, the hope is that Mark2Cure will enable researchers to make unexpected connections between various diseases, their underlying mechanisms and potential treatments. “By looking for commonalities across diseases, scientists have the potential to accelerate the development and demonstration of treatments for multiple diseases at once,” Austin said. “Mark2Cure’s use of crowdsourcing, engaging citizens and the rare diseases community, and identifying unexpected biological connections are all shared aspects of NCATS’ approach to speed translation to get more treatments to more patients more quickly.” The work was supported in equal parts by the Scripps CTSA and NIH’s Big Data to Knowledge program. Posted December 2016 | A team of bioinformatics scientists invented a web-based technology platform to arrange biomedical literature into a format that is easier for computers to organize and analyze. | Researchers Recruit Citizen Scientists to Mine Biomedical Literature | A team of bioinformatics scientists invented a web-based technology platform to arrange biomedical literature into a format that is easier for computers to organize and analyze. | Researchers Recruit Citizen Scientists to Mine Biomedical Literature |
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