Priority Setting at the NIAMS: A Report to Congress

Reviewed July 9, 2008

Introduction

In June of 2006, the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) publicly posted its Long-Range Research Plan for fiscal years 2006 to 2009. The plan serves as a broad scientific outline for NIAMS, and the investigative and lay communities, by identifying compelling research opportunities that will inform the Institute's priority-setting process. Over time, the plan will help propel research progress related to the understanding, diagnosis, treatment, and, ultimately, prevention of arthritis, musculoskeletal, and skin diseases. With 20 years of scientific accomplishments behind us - and unprecedented research challenges before us - the plan provides a collective perspective on key priority areas. While the plan is not meant to be comprehensive, as the mission of NIAMS is broad-ranging, it will help guide our assessment of emerging scientific opportunities and public health needs that the Institute is positioned to address.

How Was the NIAMS Long-Range Plan Developed and Distributed?

The plan was developed with significant input from extramural investigators, representatives of research societies and health voluntary organizations, and NIAMS scientific staff. To lay the foundation for the plan, the Institute sponsored a series of roundtable discussions with outside scientists who are experts in our key mission areas. The input from these meetings was used to develop a draft plan that included both cross-cutting areas of science - such as behavioral and biopsychosocial research, biomarkers discovery and development, and regenerative medicine - and program-specific sections.

Scientific staff from the Institute analyzed and refined the draft plan before it was presented to the NIAMS Advisory Council for review and comment. The revised draft was then made available on the Institute's website for public comment. Special notices were sent to the Institute's funded investigators, as well as scientific and patient groups with a particular interest in the NIAMS' activities, to ensure that we received broad input on the draft plan. All public comments were reviewed and analyzed, and appropriate changes were made. The final plan was presented to the NIAMS Advisory Council and posted publicly on the website, again with targeted notices to our investigative and lay communities. The Institute Director and other senior scientific staff regularly highlight the plan in conference presentations and at meetings with colleagues from research organizations and patient groups.

How Does the Plan Affect the Institute's Priority-Setting and Decision-Making?

One of the most significant areas of common need identified in our plan relates to research translation, and how the Institute can best stimulate and support it. To this end, the NIAMS has recently launched a new Centers of Research Translation (CORT) program, to help bridge the gap between laboratory bench and patient bedside. CORTs are designed to bring together basic and clinical research in a way that helps translate fundamental discoveries into new diagnostics and treatments. The Institute has funded four new centers as part of this program, and we are currently soliciting applications for a second round. The new centers will focus on the following areas: the first will study the biological basis of fracture healing and the efficacy of a potential new treatment for healing of fragility fractures in the elderly; the second will investigate the role of different cell types in lupus pathogenesis, develop markers of disease activity and severity, and explore new targets for therapies; the third will study the molecular contributors to a genetic form of rickets, and work toward developing new treatments; and the fourth will explore the molecular basis of scleroderma to understand its underlying causes using functional genomics and gene networks.

While the scientific opportunities and research needs that are highlighted in our plan do not necessarily lead to specific new initiatives, they do help guide areas that we emphasize in funding solicitations, including cross-cutting areas. For example, in our most recent announcement for Rheumatic Diseases Research Core Centers - which provide resources for established, currently funded investigators, often from different disciplines, to adopt a multidisciplinary approach to common research problems, and to ensure greater productivity than we would expect from separate research projects - we listed several areas of potential emphasis that are discussed in our plan. These include, but are not limited to: exploring mechanisms of autoimmunity underlying rheumatic diseases, including environmental factors that contribute to the development of these conditions; investigating the genetic basis of rheumatic diseases, including in animal models and defined clinical populations; studying mechanisms underlying organ damage, including inflammatory processes; and identifying new risk factors and developing technological approaches to evaluate them, in order to predict disease susceptibility more reliably.

In addition, the long-range plan helps to inform our decision-making about joining initiatives led by other Institutes and Centers in areas that intersect with our scientific interests. To illustrate this, we recently partnered with the National Institute of Dental and Craniofacial Research and the National Cancer Institute to solicit research projects focusing on basic and translational studies of osteonecrosis of the jaw, an oral complication associated with bisphosphonate use. Bisphosphonates are prescribed to alleviate bone pain in certain cancer patients, and to reduce bone loss in osteoporotic or osteopenic individuals. Although there is a growing knowledge base on the effects of bisphosphonates on bone quality and strength, there is a gap in our understanding of how they may interfere with bone healing and repair at the genetic, molecular, cellular, and tissue levels. These new initiatives are seeking to address that gap, in order to develop knowledge that will serve as the basis for the prediction, prevention, diagnosis, and treatment of this condition.

Finally, the long-range plan also influences choices about topics to address at our annual roundtable discussions and scientific retreat, held as part of our overall planning process. It affects Institute decisions about select pay of research proposals beyond our payline, and acceptance of unsolicited applications over $500,000 in annual direct costs. Further, priority areas in the plan help inform activities of trans-NIH working groups that involve the Institute. For example, at the most recent meeting of the Lupus Federal Working Group - which NIAMS leads on behalf of the Department of Health and Human Services - our plan was highlighted as a document of interest to other agencies, as well as research societies and health voluntary organizations. Similarly, the discussion at the last meeting of the Federal Working Group on Bone Diseases, held in October 2006, included a session on potential collaborations across NIH Institutes and Centers. Relevant sections of the NIAMS plan were shared with Working Group members to help stimulate ideas for possible future efforts.

How Does the Plan Help Facilitate Collaborations and Partnerships?

There are a number of ways that the long-range plan facilitates collaborations and partnerships in fields of high priority for the Institute. To the extent that areas of shared scientific interest are highlighted, the plan provides a platform for pursuing joint efforts with other Institutes and Centers. For example, we have a strong partnership with the National Institute of Neurological Disorders and Stroke and the Eunice Kennedy Shriver National Institute of Child Health and Human Development in muscular dystrophy research and training. This includes support for Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, which promote side-by-side basic, translational, and clinical research; provide resources that can be used by the national muscular dystrophy and neuromuscular communities; and offer training and advice about muscle diseases for researchers and clinicians. We are also pursuing collective efforts to encourage translational research in all forms of muscular dystrophy, and to stimulate career development opportunities for muscle disease researchers. Taken together, these activities are designed to facilitate the development of new and more effective treatments for muscular dystrophy, and to increase the number and quality of investigators in basic, translational, and clinical research focused on this disease.

We are also building on insights from the plan to nurture emerging areas of science that may need stimulation by the Institute. For example, we have recently created a new extramural research program that is focused on musculoskeletal development, tissue engineering, and regenerative medicine, and we are exploring potential initiatives in these areas with other Institutes and Centers. While these initiatives are still under development, their overall aim would be to better understand the mechanisms by which connective tissues are originally generated. These insights could then be translated into tissue engineering approaches for the repair and regeneration of connective tissues in clinical applications. In addition, work would be needed to integrate developmental mechanisms with the remodeling processes that occur normally in mature tissues. Such efforts would require considerable collaboration between basic biologists and tissue engineering specialists. Ultimately, functional tissue restoration and regeneration have the potential to reduce major sources of disability in society, as millions of Americans are affected by conditions that could benefit from such advances, including osteoarthritis, degenerative disc disease, and sports injuries to the musculoskeletal system.

With respect to research training, the long-range plan articulates a broad need to develop a cadre of clinician scientists who are positioned to pursue epidemiology, clinical trials, and health outcomes research across our mission areas. To facilitate this, the Institute has recently partnered with the Orthopaedic Research and Education Foundation and the American Skin Association to encourage applications for individual National Research Service Award post-doctoral fellowships. These fellowships would support up to two years of advanced training in the relevant methodologies to obtain a Master's of Public Health and/or a doctoral degree in epidemiology to qualify the fellows to pursue careers in these areas as they relate to musculoskeletal or skin diseases. The overall goals of these initiatives are to increase the number of clinically-trained orthopaedic surgeons and dermatologists who are prepared to make advances in epidemiology, clinical trials, and health outcomes research, and to enhance the visibility of these disciplines at U.S. academic health centers.

Conclusion

The NIAMS mission covers a broad and diverse spectrum of research and training responsibilities, as bones, muscles, joints, and skin are central components of the body. The Institute's Long-Range Research Plan for fiscal years 2006 to 2009 is one of many tools we use to guide our planning and priority-setting in times of tremendous scientific opportunity. Other cross-cutting documents - such as our Strategic Plan for Reducing Health Disparities, the NIH Muscular Dystrophy Research and Education Plan, and the NIH Autoimmune Diseases Research Plan, to name a few - also help shape our decision-making and resource-allocation choices, as well as our collaborative efforts. Collectively, they provide critical guideposts to ensure that we are taking full account of the needs and gaps that are most challenging for our research communities. Our ability to address these effectively will, over time, benefit the millions of Americans who are affected by both the common and rare diseases we study.