Priority autoimmune projects set for the NCAPG: A Report from the AARDA/NCAPG Autoimmune Diseases Summit
--From a summary provided by Natasha Leskovsek
March madness is excitement that is not limited to the basketball courts; and while the March 2010 "Autoimmune Diseases Summit: the Global State of Autoimmunity Today" sponsored by the American Autoimmune Related Diseases Association (AARDA) in collaboration with the National Coalition of Autoimmune Patient Groups (NCAPG) was not "madness," it certainly was "excitement." The Summit included a day-long program, held on March 3, that was open to the public as well as researchers and other medical and healthcare professionals. It was the first time for an AARDA/NCAPG event to be offered to members and others via Web cast, which still can be seen on AARDA's Web site (www.aarda.org).
Sessions were moderated by Noel R. Rose, M.D., Ph.D., Johns Hopkins University, and Chairman Emeritus of AARDA's Scientific Advisory Board; Donna Jackson Nakazawa, author, The Autoimmune Connection; Virginia T. Ladd, AARDA President and Executive Director; Rita Baron-Faust, MPH, CHES, author, The Autoimmune Connection; and Lawrence D. Tobias, MSc, MBA, author, A Briefing Report on Autoimmune Diseases. Closing remarks were given by M. Eric Gershwin, M.D., University of California Davis and member, AARDA's Scientific Advisory Board. Keynote speaker was actress Melissa Joan Hart. Featured videos were "A Patient's Story: My Wish for the Future of Research" and "A Patients Story: Two Patients and Their Journey of Misdiagnosis and Improper Treatment."
Presenters and discussants emphasized that all of us will be winners if ultimately the diagnosis, treatment and prevention of autoimmune diseases is advanced through the efforts of AARDA and other NCAPG members.
On the following day, a research round table was attended by scientists, clinicians, representatives and researchers from NCAPG member groups,and patient representatives who discussed the crosscutting priorities to advance autoimmune disease research. Moderators were the aforementioned Dr. Rose and John A. Hardin, M.D., Chief Scientific Officer, Arthritis Foundation.
As doing more with less is a universal need among NCAPG members, the following issues were voted for near-term prioritization to derive the most crosscutting benefit:
1. The patient registry problem. Participants lamented that there seem to be too many short-term smaller data sets that are under-utilized by researchers in the autoimmune disease field. Some registries are funded for only a short period of time, before their true longitudinal value can be realized. Patients have provided personal health history data and biological specimens to myriad researchers and groups in the hopes that such data will help to identify clinically relevant autoimmune disease risk factors. The Multiple Autoimmune Diseases Genetics Consortium is one such resource that deserves better attention (www.madgc.org).
In order to get more benefit from existing resources, a master "registry of registries" relating to autoimmune disease is needed. Such a master list might obviate the need for other registries to be planned, or it could inform how to optimally design and carry out such registries.
AARDA hopes to sponsor a meeting of representatives of the registries and epidemiologists to determine what data is available and how that data can be publicized and used by researchers. Please contact AARDA if you manage, participate in, or are aware of an existing autoimmune disease registry.
2. Post-marketing safety of drugs and biological therapies. A need was identified to approach the U.S. Food and Drug Administration (FDA) regarding post-marketing safety surveillance for drugs and biologics. The broad off-label use of products to treat autoimmune diseases not only testifies to the interrelatedness of these diseases but also bespeaks the risk of safety signals and adverse reactions. In order for patients and healthcare providers to understand truly the potential risks and benefits of a product and class of product, FDA needs to aggregate data from among sponsors/manufacturers instead of looking at each sponsor's or manufacturer's products separately. In particular, data is needed on how many people received a product for a particular purpose in order to determine the significance of risks to specific populations, whether on-label or off-label. Please contact AARDA if you are interested in assisting the NCAPG in this effort.
3. Make the NIH grant process more autoimmune-disease friendly. Researchers identified several shortcomings of the NIH grant application and administration process that erect bias against much needed crosscutting autoimmune disease research. NIH leadership will be approached regarding (i) revitalization of the Autoimmune Diseases Coordinating Committee (ADCC); (ii) implementation of the ADCC's road map; (iii) the creation of a study section dedicated to autoimmune disease, engaging experienced physician investigators that can think broadly and can favor patient research over animal research; (iv) institution of a requirement that the eight NIAID-funded Autoimmunity Centers of Excellence engage in patient care, not just research.
4. Standardize, standardize, standardize. Existing diagnostic criteria, disease definitions and treatments for autoimmune diseases are woefully disparate. Better diagnostic information may lead to better incidence data and better estimation of the true cost of autoimmune diseases. AARDA and other NCAPG members present at the Summit resolved to press forward for standardized risk assessment, emphasizing the need to know more about the mechanism of human autoimmune disease. Mechanistic studies are difficult to implement in humans because they need to be large studies--animal models can be used to screen mechanisms. Risk factors for autoimmune disease, especially shared ones, need to be better identified and measured, a particularly difficult problem given the potentially lengthy lag between, for instance, environmental exposure and development of disease, and interaction with multiple genes. A common barrier to the development of standardized assessment tools, diagnostic criteria, and care plans is that development of such infrastructure is not as well funded as research itself.
All of us can help to sustain the excitement of the March National Autoimmune Diseases Summit. Do your part to foster an interdisciplinary approach by connecting disease-specific researchers and clinicians with other autoimmune disease researchers, clinical specialists, and scientists working in autoimmunity generally. Local, regional, and national meetings all can be used for such facilitation. Invite environmental researchers to your scientific gatherings. Contact other NCAPG members to leverage your research resources, not just dollars, but data.
As a result of the enthusiasm stimulated by the Summit panelists and discussions, several committees were formed to aid in the further exploration and implementation of the various recommendations.
The Summit was made possible in part through an educational grant from Genentech; patron sponsorship from FoxKiser; and support, both financial and in-kind, from the following NCAPG member groups: APS Foundation of America, Lupus Alliance of America, Lupus Foundation of Mid and Northern New York, The Myositis Foundation, National Adrenal Diseases Foundation, National Alopecia Areata Foundation, Platelet Disorder Support Association, Sjögren's Syndrome Foundation, and Vasculitis Foundation
