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7 Opportunities and Options for Enhancing Autoimmune Disease Research at the National Institutes of Health
Pages 411-442

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From page 411...
... population, with consequences that compromise quality of life for those afflicted. Many autoimmune diseases affect women predominantly, and the incidence and prevalence of these diseases appears to be rising in certain groups, such as children and adolescents in the case of two of the most common autoimmune diseases, namely inflammatory bowel disease and type 1 diabetes.
From page 412...
... For this area of inquiry to advance, a multidisciplinary approach will be needed that draws upon the expertise of researchers from across the NIH. Chapter 5 describes the NIH research grant process and coordination of autoimmune disease research.
From page 413...
... Committee members reviewed IC strategic plans, websites, and other information to obtain insights into the autoimmune research priorities of the ICs reviewed and to identify which autoimmune diseases might be relevant to their missions. The committee notes that the lack of a central repository for autoimmune disease research at the NIH hampers the ability of individuals in and outside of NIH, and in and outside of the research enterprise, to readily access this information and consequently limits transparency and communication with the public and other stakeholders.
From page 414...
... A crosscutting strategic plan to guide current research activities with details on specific goals, objectives, timelines and milestones would have provided an important reference point to guide the assessment of the current portfolio. Developed 20 years ago, the last autoimmune disease research plan lacked such details.
From page 415...
... Autoimmune diseases are complex and share commonalities that would benefit from a coordinated, multidisciplinary research approach to better understand basic mechanisms, etiology, risk factors, and to support the development of interventions to mitigate the impact of autoimmune diseases and associated co-morbidities and complications across the lifespan. Current NIH autoimmune disease research efforts are guided by a dated research plan and efforts to coordinate autoimmune disease research are minimal.
From page 416...
... However the committee believes that any centralized entity, to be effective, will require new resources; and that a broader and better research enterprise on autoimmune disease has a greater potential to bring relief to patients and their families. The committee considered five options for enhancing autoimmune disease research and resulting outcomes: increased funding, two options for coordinating research, establishing a national strategic plan, establishing a new institute, and creating a special office for autoimmune disease research.
From page 417...
... Such an office would be positioned to respond to the committee's concerns by: • Facilitating cross-NIH multidisciplinary collaboration and stimu lating innovation around autoimmune disease research; • Engaging in priority setting, strategic planning, and implemen tation; • Budgeting for and allocating available research funds in align ment with the strategic plan; • Managing and evaluating research efforts; • Communicating with key stakeholders; and • Providing visible leadership on autoimmune disease research. Option 1: Increased Funding for Autoimmune Disease Research Given the flat line of research spending levels over the past 12 years, the committee believes that increased funding will be an indispensable component of any effort to enhance autoimmune disease research opportunities, and thus it lists that option first.
From page 418...
... Cons: Potential • Without major new funding, new investments in one area may be Concerns and offset by cuts in another. Disadvantages • There may be opposition to earmarking funding for autoimmune disease research.
From page 419...
... Autoimmune Disease Coordinating Committee • Would raise the visibility of autoimmune diseases to the cabinet level. • Engages and potentially leverages other HHS agencies, including their expertise, resources, and reach.
From page 420...
... • Elevates visibility, provides sustained leadership, and establishes a clear focal point for autoimmune disease research within NIH and externally through extramural, intramural, training, communica tion and outreach activities. • De facto coordinating body that is crosscutting rather than autoimPros: Potential mune disease specific.
From page 421...
... Increased funding directed to certain focused autoimmune disease research activities and initiatives is another alternative. Several focused activities, such as the Autoimmunity Centers of Excellence (ACEs)
From page 422...
... Given these considerations, the committee notes that funding by itself is necessary but not sufficient for enhancing the research enterprise for autoimmune diseases; it does not address issues of coordination, prioritization, or workforce development. Option 2a: Coordination by Revitalizing ADCC The ADCC, NIH's current coordinating mechanism for research on autoimmune diseases, was most active between 1998 and 2005, when it produced reports summarizing the state of autoimmune disease research and funding (2000)
From page 423...
... SAMHSA could be informed and benefit from NIH research on co-occurring mental health issues related to autoimmune diseases and could provide information on potential areas needing NIH research. An added benefit of elevating this issue to the cabinet level is that it would PREPUBLICATION COPY -- Uncorrected Proofs
From page 424...
... While the committee believes NIH would benefit from engaging more with sister agencies, it may be that there are less formalized and more issue-specific ways of doing this than the creation of a new HHS body. Option 3: Develop a Congressionally Mandated National Autoimmune Diseases/Autoimmunity Research Plan The committee considered and believes that there may be significant value in a congressionally mandated National Autoimmune Diseases/ Autoimmunity Research Plan that could be sited in any one of a number of organizational entities.
From page 425...
... In some cases, research is in its infancy, treatments are limited, and there are no cures. Most importantly, no overall architecture for Alzheimer's disease research existed before developing the National Plan, and one does not exist for autoimmune disease research.
From page 426...
... NIADAR would establish a clear focal point for autoimmune disease research for individuals within NIH and for external constituencies. The Institute would function as a clear and empowered convener, coordinator, and accountable focal point for autoimmune disease and autoimmunity research.
From page 427...
... The committee found that the 1999 bill addressed several concerns that the committee had raised in its deliberations including the need for a central entity to: promote coordination and cooperation among ICs and other entities, develop an agenda for conducting and supporting autoimmune disease research, promote the appropriate allocation of NIH for autoimmune disease research, report on research activities and identify future research opportunities, and to provide leadership on autoimmune disease to HHS, the NIH Director and other relevant agencies. As governmental units at NIH and elsewhere persuasively argue to the Congress, the impact of a research organization is likely to correlate with the extent to which it owns and controls resources.
From page 428...
... Based on these reviews, the Committee concludes that the Office of AIDS Research can provide a highly desirable model for a new Office of Autoimmune Diseases/ Autoimmunity Research. This organizational model has provided such important elements for coordinating HIV and HIV-related research through: • Advice provided to the Director of NIH, HHS and other entities on HIV and HIV-related research; • Establishment of research priorities/goals; • Development and annual evaluation of strategic plan for HIV and HIV-related research throughout NIH; • Assurance that funds are invested in the areas of scientific priority and allocates such funding; • Oversight, coordination and management of HIV and HIV-related research (scientific, budgetary, legislative, and policy)
From page 429...
... . Using the OAR as a model, the committee believes that a well-configured OAD/AR could be an essential ingredient in further stimulating promising and impactful autoimmune disease and autoimmunity research at NIH that can be translated into clinical practices that will prevent autoimmune diseases from occurring or that will improve the lives of those living with autoimmune disease.
From page 430...
... 5. Address autoimmune disease research workforce and training issues, including diversity of the workforce.
From page 431...
... 7. Coordinating autoimmune disease research across other HHS agencies and other federal agencies (e.g., AHRQ, CDC, FDA)
From page 432...
... First, though more modest than those required by a new Institute, creating a new Office would require significant resources for infrastructure, robust staffing, and the capacity to function nationally and internationally as a convener, broker, and communication hub. Second, it would likely require new funding to stimulate new crosscutting and collaborative autoimmune disease research and enhance workforce development.
From page 433...
... The committee found a lack of long-term (20 years or more) population-based epidemiology studies on autoimmune disease.  Such studies would allow for assessing trends, identifying differences among population subgroups, and in determining the prevalence and incidence of under-researched autoimmune diseases and conditions, such as celiac disease. The National Cancer Institute's Surveillance, Epidemiology, and End Results Program, which provides information on cancer incidence and survival in the United States is a model for such studies already exists at NIH.
From page 434...
... Early life exposures and timing of these exposures influence health at different life stages at a population and individual patient level. Autoimmune diseases are long-lasting, and heterogeneous conditions for which manifestations and coexisting morbidities change over time.
From page 435...
... Heterogeneity in autoimmune diseases takes many forms, which could range from differences in symptom presentation and disease progression, as seen in persons with systemic lupus erythematosus, to severity of disease, as observed in myocarditis in men compared with women, to varying responses to the same therapy. Dissect heterogeneity across and within autoimmune diseases to decipher common and disease-specific pathogenic mechanisms.
From page 436...
... influence autoimmune disease, examining the effect of puberty, pregnancy, and menopause to understand sex differences in autoimmune diseases • Identify autoantigens across autoimmune diseases using new technologies that allow T cell receptors identified by single cell RNA sequencing from tissue to be interrogated without bias transfecting mRNA libraries into reporter antigen presenting cells • Dissect molecular mechanisms associated with poor outcomes • Design research that bridges animal models and human studies to better understand common mechanisms in inflammation for specific autoimmune diseases Researching less common autoimmune diseases can be difficult owing to smaller pools of persons affected. At the same time, identifying genetic variants, for example, could reveal insights that might be applied to more common diseases.
From page 437...
... influence autoantibody levels/types and/or immune complex formation that are diagnostic or pathological for autoimmune diseases • Explore the pipeline for development and validation of autoanti bodies as disease biomarkers and surrogate endpoints of disease • Identify novel biomarkers and complex "signatures" that predict and diagnose autoimmune diseases including cytokines, microR NAs and other markers As discussed in Chapter 2 and Chapter 4, genetics, and particularly gene variants, environmental exposures, and timing interact to increase or blunt an individual's susceptibility to autoimmune diseases. Identifying the biological functions of gene variants and the effects of environmental exposures on their functions over time could reveal new avenues for care.
From page 438...
... and social determinants of health in children and adults, including stress, for individual and/or multiple autoimmune diseases, including disease flares • Determine the effect of environmental exposures on immune pathways in patients and animal models of autoimmune disease • Utilize a life-course approach along with advanced methodolo gies (e.g., computational science) to identify and examine the potential effect of interacting co-exposures that may increase sus ceptibility to disease, and to understand how these factors affect onset, progression, and severity of disease • Conduct a systematic investigation of the effects of nutrients, dietary antigens, exercise, aging, and microbiome in normal tis sue development and homeostasis in the pre-clinical phase of disease and during disease and recovery • Explore novel methodologies to identify distinct and interacting biological and biopsychosocial factors contributing to observed sex/gender differences in autoimmune diseases, including the PREPUBLICATION COPY -- Uncorrected Proofs
From page 439...
... Evaluating the epidemiology and risks for developing other morbidities, identifying those at greatest risk, assessing the impacts of coexisting morbidities, and interventions to prevent or ameliorate these could greatly improve patient care. Determine the impact of coexisting morbidities, including co-occurring autoimmune diseases and complications of autoimmune diseases, across the lifespan, and develop and evaluate interventions to improve patient outcomes.
From page 440...
... Foster research to advance health equity for all autoimmune disease patients. • Investigate genetic, biological, social determinants, and environ mental mechanisms of autoimmune diseases in different racial, ethnic, and other underrepresented groups, as well as across sexes and over the lifespan • Identify individuals at high risk of autoimmune diseases and implement prevention strategies • Investigate disparities and determinants of disparities in disease stage at presentation and progression of disease, including com plications, coexisting morbidities, functional impairments, and disability • Explore novel methodologies to identify distinct and interacting biological and biopsychosocial factors contributing to observed sex differences in autoimmune diseases • Collaborate with consumers, community-based organizations, and other partners to develop and evaluate health care delivery and/or policy interventions to address identified disparities and generate evidence to support their feasibility, sustainability, and dissemination • Build long-term partnerships between patients, communities, cli nicians, and scientists to increase participation of underserved populations in interventional clinical trials and other clinical and translational research studies • Conduct dissemination/implementation research to determine best practices and to improve outcomes for all • Conduct research on how to increase recruitment of underserved populations in clinical research, and in interventional clinical tri als in particular • Conduct research on how best to target interventions for patients with worse outcomes and/or underserved populations Chapter 2 noted the rates of many autoimmune diseases are rising.
From page 441...
... 2005. Progress in Autoimmune Disease Research, Autoim mune Disease Coordinating Committee Report to Congress.
From page 442...
... PREPUBLICATION COPY -- Uncorrected Proofs


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