Welcome and Announcements
The NIAMS held its ninth NIAMS Coalition Outreach and Education Meeting on September 19, 2023, on the National Institutes of Health (NIH) campus in Bethesda, MD. The NIAMS Coalition is an independent group of more than 95 professional and voluntary organizations interested in the research and mission areas of NIAMS. Approximately 50 representatives from more than 40 Coalition organizations joined NIAMS staff and speakers to total 80 attendees at this meeting.
Jennifer Morgan Gray, Chief of the NIAMS Science Communications and Outreach Branch (SCOB) and Colleen Dundas, NIAMS Public Liaison Officer, opened the meeting. Dundas introduced NIAMS Coalition co-chairs, Tricha Shivas, M.Be., Chief Strategy Officer at the Foundation for Sarcoidosis Research, and Lee Grossman, M.B.A., Chief Executive Officer of the Orthopaedic Research and Education Foundation, who welcomed everyone and introduced NIAMS Director Lindsey A. Criswell, M.D., M.P.H, D.Sc.
The State of the Institute and NIH Leadership Remarks
In her State of the Institute remarks, Dr. Criswell briefly discussed the NIH budget and grant application process. She also spoke about the NIAMS budget for the 2024 fiscal year (FY24), noting the proposed FY24 budget is $688 million, which is the same as FY23.
Dr. Criswell also discussed partnerships that advance NIAMS research, including the Accelerating Medicines Partnership® Autoimmune and Immune-Mediated Diseases (AMP® AIM) Program, which brings together multi-disciplinary researchers to help develop new and effective therapies. The AMP program has also led to the development of the Arthritis and Autoimmune and Related Diseases Knowledge (ARK) Portal, a virtual resource that houses data sets from the AMP® Rheumatoid Arthritis and Systemic Lupus Erythematosus (AMP® RA/SLE) program and will facilitate rapid and efficient data sharing by the broader research community. Another program Dr. Criswell discussed is the AMP® Bespoke Gene Therapy Consortium (AMP® BGTC), which aims to streamline the gene therapy development process and make gene therapy more generalizable and accessible.
Dr. Criswell discussed the Advanced Research Projects Agency for Health (ARPA-H) and its intersections with the NIAMS portfolio. ARPA-H’s first program, the Novel Innovations for Tissue Regeneration in Osteoarthritis (NITRO), will explore technologies in three areas: injectable bone regeneration; injectable cartilage regeneration; and replacement joints built from human cells.
Dr. Criswell spent time discussing NIAMS’ role and support for several collaborative activities to advance research. She described the Institute’s active role in the Muscular Dystrophy Coordinating Center (MDCC) and recent efforts to renew the MDCC Action Plan. Additionally, Dr. Criswell introduced the new NIH Office of Autoimmune Disease Research (OADR), within the NIH Office of Research on Women’s Health (ORWH), which will help to coordinate autoimmune disease research across the NIH.
Dr. Criswell then highlighted NIAMS’ leadership of several programs. She mentioned the Molecular Transducers of Physical Activity Consortium (MoTrPAC), an NIH Common Fund program, which aims to understand how physical activity improves and preserves health. Dr. Criswell also shared details about the NIAMS-led Back Pain Consortium (BACPAC) Research Program and the Restoring Joint Health and Function to Reduce Pain (RE-JOIN) Consortium, both part of the NIH Helping to End Addiction Long-term (HEAL) Initiative.
Dr. Criswell underscored NIAMS’ commitment to ending structural racism and focus on empowering and supporting staff with opportunities to learn about diversity, equity, inclusion, and accessibility. NIAMS created a Culture Committee in 2020 that advises NIAMS leadership on recruiting, retaining, and promoting employees from underrepresented backgrounds. The NIAMS Culture Committee has worked to update the Institute’s core values, designed to foster an inclusive working environment. NIAMS has named Dr. Robert Walker, Jr., as its first Chief Diversity Officer.
Dr. Criswell concluded by providing updates on the forthcoming 2025-2029 NIAMS Strategic Plan. After thanking those who participated in the recent public meeting to gather initial ideas on cross-cutting topics of interest, she encouraged the audience to submit additional input via an upcoming request for information (RFI).
After a short Q&A session, Dr. Criswell introduced NIH Principal Deputy Director, Lawrence Tabak, D.D.S., Ph.D.
Dr. Tabak reflected on his time at NIH, appreciating the work of the voluntary and professional organizations in the audience, and commenting on the power of the different voices represented in the room coming together. Dr. Tabak also spoke about how NIH is trying to be more responsive to patient advocacy groups, especially those that represent people with rare diseases.
Dr. Tabak spoke about the AMP program and how it has forged a meaningful relationship among industry, patient groups, non-profits, and the NIH. He recognized that NIAMS Deputy Director Robert Carter, M.D., has played a leading role in multiple AMP programs.
He ended his remarks with a request for attendees to respond to an RFI on updating NIH’s mission statement, which is accepting comments until November 24, 2023, emphasizing the importance of input from organizations such as those present.
Following Dr. Tabak’s remarks and a short Q&A session, Criswell returned to the podium to recognize the service of previous NIAMS Coalition co-chair Eryn Marchiolo, M.P.H., Vice President of Mission at the Rheumatology Research Foundation, and out-going co-chair Tricha Shivas.
NIAMS Coalition Overview
NIAMS Coalition co-chairs Tricha Shivas and Lee Grossman opened their session by encouraging participants to network and share their experiences. Shivas and Grossman provided the history and evolution of the NIAMS Coalition. It was founded more than 20 years ago with just a few organizations, and has grown to be an independent consortium of over 95 professional and voluntary organizations. The goal of the Coalition is to advocate for and serve as the voice of patients and professionals who rely on NIAMS-focused research.
Shivas and Grossman wrapped up their talk by announcing that a co-chair position would be opening up soon because Shivas is rotating off. They asked attendees to contact them if interested in learning more about the volunteer position.
FNIH - Bridges to Breakthroughs
Julie Louise Gerberding, M.D., M.P.H., Chief Executive Officer of Foundation for the National Institutes of Health (FNIH), introduced the FNIH and its goal to build bridges between the public and private sectors. FNIH strives to accelerate and amplify the value and impact of NIH and its research.
Over the past 25 years, FNIH has supported nearly 25,000 scientists and has helped to manage the AMP program since it was launched. FNIH also plans to launch at least four more AMP-related programs in 2024. Dr. Gerberding then spoke about the AMP BGTC, which aims to create a regulatory framework for the manufacturing of gene therapy products.
FNIH is working to become more patient-centric. A new position that focuses on patient engagement has been created, and FNIH has established a Patient Engagement Council and a Patient Ambassadors Program. FNIH also participates in the Coalition for Trust in Health & Science, a non-profit organization dedicated to building trust in health care, public health, and science across all segments of society.
This plenary talk was recorded on the NIH Videocast and begins at 1:26:50
Breakout Sessions - Morning
Gayle Lester, Ph.D., Director of NIAMS’ Division of Extramural Research, provided a brief overview of the NIAMS Extramural Program. She described its structure and the responsibilities of the program directors, and then provided guidance for the three breakout sessions.
Systemic Rheumatic Diseases and Skin Diseases
Speakers: Ricardo Cibotti, Ph.D., Director of the NIAMS Skin Immunology and Diseases, Skin Microbiome Program, and Su-Yau Mao, Ph.D., Director of the NIAMS Arthritis Biology Program.
In his opening remarks, Dr. Cibotti described the Institute’s skin diseases portfolio. He also said that NIAMS needs to focus more on establishing repositories for data.
In her opening remarks, Dr. Mao discussed her background and described the NIAMS systemic rheumatic diseases portfolio. She also discussed NIAMS’ role in the AMP program.
Drs. Cibotti and Mao then took questions from Coalition members. They discussed productive ways to interact with NIAMS Program Directors (PDs) and fielded funding questions related to data repositories and R13 programs. Participants discussed the lack of researchers in certain underrepresented disease spaces and challenges to getting more funding for these areas. Dr. Mao noted that researchers often move away from these diseases after they finish their training, but that NIAMS could look to publish notices of special interest (NOSIs) to promote awareness and get researchers into these areas.
Muscle Diseases and Bone Diseases
Speakers: Emily Carifi, Ph.D., Director of the NIAMS Muscle Disorders and Therapies Program, and Faye Chen, Ph.D., Director of the NIAMS Clinical, Integrative Physiology, and Rare Diseases of Bone Program.
Moderator: Paul Melmeyer, Muscular Dystrophy Association
Drs. Carifi and Chen both discussed their research portfolios, and their positive experiences attending Coalition member events. They encouraged Coalition members to publicize relevant NIAMS advances and announcements.
Drs. Carifi and Chen answered questions related to funding opportunities and how best to interact with NIAMS PDs. They reiterated that they are always willing to help and answer questions. They also discussed the best way for Coalition members to find and engage with early-stage investigators, reminding participants that the NIAMS RePORTER was a great tool. Drs. Carifi and Chen also addressed concerns regarding the funding of clinical trials.
Osteoarthritis and Orthopaedic Diseases and Disorders
Speakers: Xincheng (Ted) Zheng, M.D., Ph.D., Director of both the NIAMS Clinical Osteoarthritis and Diagnostic Imaging Program and the Cartilage and Connective Tissue Program, and Rebecca Lenzi, Ph.D., a Health Science Administrator who manages the NIAMS Institutional Training (T32) Program, and is the co-lead of the Back Pain Consortium (BACPAC) Research Program.
Moderator: Lynne Jones, Orthopaedic Research Society
Both Drs. Zheng and Lenzi gave brief overviews of their program portfolios. Dr. Lenzi also spoke about the Biomarkers for Evaluating Spine Treatments (BEST) Trial. BEST, a collaborative trial developed by the BACPAC program, uses a stakeholder board composed of patients, providers, professional societies, and payors, as well as industry, federal, and government-sponsored organizations. This board provides ongoing feedback about the trial and is a great example of how to engage with stakeholders.
Drs. Zhang and Lenzi took questions from Coalition members. They discussed how to better get the word out about research, including through RFIs, social media, and newsletters. They also talked about the importance of engaging with people of diverse backgrounds. They noted that NIAMS includes patients as part of the review panel for clinical trial recruitment materials.
ARPA-H: Accelerating Better Health Outcomes for All Americans
Renee Wegrzyn, Ph.D., Director of ARPA-H, gave a presentation that focused on ARPA‑H’s mission and business model, the NITRO project to eradicate osteoarthritis (OA), and strategies ARPA‑H is using to address challenges associated with disseminating research results. President Biden established ARPA-H in March 2022 to support research that accelerates health outcomes for everyone. Initial projects will fit under one of four focus areas.
Program managers at ARPA-H work closely with researchers, who are supported through cooperative agreements instead of grants. ARPA-H’s business model relies on the swift transition of research finding to commercialization. The agency is taking steps to create marketplaces that will be receptive to the solutions that emerge from the projects it supports.
NITRO focuses on finding new ways to help the human body repair itself by investing in injectable substances that regenerate bone and cartilage, and replacement joints built from human cells. Metrics of success include accelerated recovery time for patients and a reduced need for repeat surgeries. The program manager is assembling project teams from researchers who applied to join NITRO.
Dr. Wegrzyn also discussed how dissemination of healthcare solutions is hindered by geography —many patients do not live near major healthcare centers and biotech centers. ARPA-H is addressing these issues by creating “a hub-and-spoke health innovation network” called ARPANET-H, which aims to establish a presence in all 50 states and 16 U.S. territories.
This plenary talk was recorded on the NIH Videocast and begins at 2:29:30
Breakout Sessions - Afternoon
Strength of Patient Voices: Advancing Research with Patient-focused Initiatives
Dr. Gokani spoke about the need to integrate patient voices into research and strengthen patient voices to raise awareness. She discussed some of the difficulties that patients face in getting their voices heard. Dr. Gokani also spoke about how ACPA encourages patients to share their experiences. She noted that ACPA facilitates a number of support groups, a quarterly publication, and many other resources for patients. Dr. Gokani also highlighted ACPA’s robust public awareness program.
Gondo talked about the history of the NPF and discussed some of the organization’s patient-focused initiatives. NPF engages with federal regulators and also trains patients to engage with researchers. NPF also hosts a Patient Navigation Center that provides mentorship support and educational resources for patients. Gondo also talked about NPF’s annual surveys which helps the organization to understand disease status, healthcare utilization, and health outcomes of patients with psoriatic disease.
Dr. Gokani and Gondo also stressed the need to have well-trained patient advocates who are impacted by the disease work alongside health care providers to ensure that patient populations are represented.
Rare Disease Groups: Making Connections to Raise Awareness
Sullivan highlighted key goals of the organization, which include the elimination of the diagnostic odyssey for rare disease patients, and the advancement of regulatory science for and the facilitation of patient access to rare disease therapies. She described the power of patient-centric rare disease communications and provided tips for building partnerships with federal organizations and industry. Sullivan outlined specific programs and resources that her organization has developed keyed to rare disease advocacy.
Mason discussed NORD’s mission and explained that in the United States, a disease or condition is considered rare if it affects fewer than 200,000 Americans. She offered multiple examples of how patients can become involved in community engagement and advocacy, including making public comments, sending letters to media and publication outlets, drafting op eds, attending legislative meetings and hearings, and participating in Capitol Hill days. She highlighted some examples of partnerships with government (e.g., FDA Patient Listening Sessions) and industry.
Data and New Technology: How Organizations Can Leverage New Tools to Advance Their Mission
Speakers: Belinda Seto, Ph.D., Deputy Director of the NIH Office of Data Science Strategy, Lauren Isbell, M.N.M., Executive Director of Nevus Outreach, and Tricha Shivas, M.Be., Chief Strategy Officer at the Foundation for Sarcoidosis Research.
Dr. Seto discussed the artificial intelligence (AI) technology landscape and how NIH has increased funding and support for AI in health research. She highlighted how AI has been used for several things in the healthcare industry, including predictive machine model learning and techniques to improve imaging. While AI is a promising tool to use in healthcare and research, she noted that it is not perfect and should not solely be relied upon. Dr. Seto closed by briefly discussing common data elements (CDEs), or ways to enable sharing and comparing data systematically across different platforms. There is a mandate from Congress to develop CDEs for autoimmune diseases.
Isbell spoke about the evolving landscape of technology and using AI to help automate processes, unlock insights, and predict future trends, in service of your organization’s mission. She pointed out several AI platforms that she has used in helping to manage her organization. She noted that having a project management application and an application to manage donor profiles and revenue were keys to success.
Shivas discussed software that collects health insurance claims that are processed in the United States. This data can be used to get a snapshot of a patient’s journey. It can assess tests or medications being used and look at where people are being treated for sarcoidosis. She noted that this patient journey-mapping can help identify trends in misdiagnosis. However, the technology is limited because it only captured data from insured patients and lacked strong diversity data.
The NIAMS Strategic Plan: Updates, Next Steps, and Your Role
Stephanie Burrows, Ph.D., Chief of the NIAMS Scientific Planning, Policy and Analysis Branch (SPPAB) provided an overview of the Institute’s work on its FY 2025-2029 Strategic Plan, including progress to date and opportunities for Coalition member organizations to provide input. The plan will focus on cross-cutting thematic research opportunities where the Institute can be best positioned to make a difference in the lives of all Americans, and gathering public input is a crucial step in its development.
In September 2023, NIAMS held a meeting to crowdsource ideas for cross-cutting themes that would complement the suggestions submitted in response to an earlier RFI. Participants included researchers, NIAMS Advisory Council members, individuals from other agencies and NIH components, and representatives of professional and voluntary organizations. In contrast to the Institute’s traditional listening sessions conducted in the past, this meeting was based on an approach called “Open Space” where participants name the topics they want to discuss, pitch ideas, and attend breakout sessions.
Participants expressed considerable interest in a number of themes, including:
- environmental influences on the development of diseases;
- how lifestyle interventions could help prevent or treat diseases;
- the importance of a robust data science infrastructure;
- health disparities and health equity; and
- pain research.
The draft plan will likely be published on the NIAMS website in the late summer of 2024 for public comment and released in its final form later in the year.
Summary and Closing
At the end of the meeting, Dr. Criswell thanked participants for attending and noted that she greatly enjoyed her first in-person Outreach and Education Day.
Coalition co-chairs Shivas and Grossman provided closing remarks. They highlighted themes, takeaways, and potential action items from the meeting, thanking everyone for their contributions. Shivas and Grossman encouraged Coalition members to reach out if they are interested in taking a leadership role within the Coalition, especially given Shivas’ upcoming completion of co-chair service. The co-chairs urged participants to continue engaging with the Coalition and the Institute, and to keep sharing updates via the NIAMS Coalition-led newsletter, the Monthly Round Up.
Portions of the meeting were recorded and can be watched on the NIH Videocast.