Summary Minutes – May 6-7, 2024

National Advisory Board on Medical Rehabilitation Research (NABMRR)
National Center for Medical Rehabilitation Research (NCMRR)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Virtual/In-Person Hybrid Meeting Format

 

Board Members Present:

Board Members Absent:

Steven C. Cramer

Ex-Officio Members Present:

Ex-Officio Members Absent:

Day 1: May 6, 2024

Access the VideoCast recording of the first day of the May 2024 NABMRR meeting. Use the time stamps (in parentheses) in this report to navigate the recording.

NCMRR Deputy Director and NABMRR Executive Secretary Ralph Nitkin, Ph.D., opened the virtual meeting at 10 a.m. ET.

Welcome, Approval of Minutes, and Future Meetings (00:26)

Barbara S. Bregman, Ph.D., PT, Chair, NABMRR

Dr. Bregman welcomed participants and called the roll. The board voted to approve the minutes of the December 4–5, 2023, meeting as written. Future meeting dates are:

• December 2–3, 2024
• May 5–6, 2025
• December 1–2, 2025

NICHD Director’s Report (06:52)

Diana W. Bianchi, M.D., Director, NICHD

In her report, Dr. Bianchi shared fiscal year (FY) 2024 appropriations, recent congressional interactions, information on the White House Women’s Health Research Initiative (WHRI), the new NIH Director’s principles and priorities, and an update to the NIH mission statement. She then described pediatric research at NIH, provided an update on the NICHD strategic planning process, and announced NIH leadership updates.

FY 2024 Appropriations and Congressional Interactions (8:22)

NIH received a total of $47.1 billion for FY 2024 (excluding Advanced Research Projects Agency for Health [ARPA-H]), which was a $300 million increase over FY 2023. NICHD’s total budget of $1.76 billion included $90 million for the INvestigation of Co-occurring conditions across the Lifespan to Understand Down syndromE (INCLUDE) project (the same as FY 2023); $53.4 million for the Implementing a Maternal health and PRegnancy Outcomes Vision for Everyone (IMPROVE) initiative (an increase of $10 million); $12.6 million for the Gabriella Miller Kids First Program (the same as FY 2023); and $1.5 million for a National Academies of Science, Engineering, and Medicine (NASEM) pediatric research assessment. Funding was reduced for the All of Us Research Program, the Brain Research Through Advancing Innovative Neurotechnologies^® (BRAIN) Initiative, and the Undiagnosed Diseases Network. Previous set-aside funds for digital media and technology effects on children, COVID-19 and pregnancy, and COVID-19 and children were discontinued.

Dr. Bianchi recently met with several members of Congress to discuss NICHD initiatives. Those interactions were with U.S. Rep. Lauren Underwood (D-IL) to discuss menopause research, U.S. Rep. Julia Letlow (R-LA) to discuss maternal health and women’s health research, and the Women’s Congressional Policy Institute to discuss the IMPROVE initiative and maternal mortality. Additionally, ACT for NIH: Advancing Cures Today held a 10th-anniversary event that was attended by about 25 members of Congress and provided ample networking opportunities. ACT for NIH is a 501(c)(4) nonprofit advocacy organization that works with Congress to make increasing biomedical research funding a national priority. NICHD staff met with congressional staff from the offices of U.S. Rep. Juan Ciscomani (R-AZ) to discuss gynecologic health and disease research, NICHD’s major clinical trial networks, and the use of pharmaceuticals during pregnancy; U.S. Rep. Katherine Clark (D-MA) to discuss women’s health research; U.S. Rep. Anna Eshoo (D-CA) to discuss challenges in pediatric drug development; and U.S. Sen. Tim Scott (R-SC) to discuss neonatal opioid withdrawal, maternal health, the IMPROVE initiative, and the Rapid Acceleration of Diagnostics Technology (RADx^® Tech) for Maternal Health Challenge.

White House Women’s Health Research Initiative (WHRI) (14:24)

On March 18, 2024, President Biden issued an Executive Order on Advancing Women’s Health Research and Innovation. This cross-government initiative was designed to promote collaborative, interdisciplinary research; to assess unmet needs in women’s health research; and to develop a research agenda and common data elements related to menopause. To date, NIH actions related to the executive order have included:

• Issuing a Notice of Special Interest on women’s health research (NOT-OD-24-079)
• Developing a list of all NIH grant opportunities related to women’s health research
• Proposing an FY 2025 investment of $200 million in NIH funding for women’s health research

NIH Director’s Principles and Priorities (17:26)

Monica Bertagnolli, M.D., became the 17th director of NIH on November 9, 2023. She is a clinician–scientist and surgical oncologist who maintains clinical privileges. Dr. Bertagnolli has articulated the following guiding principles for the work at NIH:

• Our work is not finished when we deliver scientific discoveries; our work is finished when all people are living long and healthy lives.
• NIH research encompasses the laboratory, the clinic, and the community. Patients are partners in discovery.
• To tackle the most persistent and complex problems, NIH aims to bring more members of the public into the research enterprise as our partners in discovery.
• Income, age, race, ethnicity, geographic location, and disability status should not be barriers to participating in research or to benefiting from research advances.
• Progress is accelerated when advanced scientific methods, such as new data analytics, are applied to data that include everyone and when new discoveries are rapidly and equitably adopted in clinical care.
• NIH is committed to harnessing the power of artificial intelligence (AI) and machine learning (ML) to advance research across diverse fields, diseases, and scientific communities.
• Advanced scientific methods, new data analytics, and new technologies are unlocking possibilities to harness data in ways that achieve faster and more definitive results.
• NIH has launched and will continue to launch innovative and ambitious initiatives to propel the fusion of biomedicine and AI/ML.

One of Dr. Bertagnolli’s highest priorities is to use the NIH Common Fund program to support clinical research in primary care settings. Because the health of the U.S. population is declining—and the decline is steepest among the underserved and underrepresented—the goals of primary care clinical research are to:

• Establish a primary care–focused clinical research network that is disease-agnostic, facilitating clinical research in mission areas across all NIH institutes and centers (ICs)
• Integrate innovative research with routine clinical care in real-world settings
• Create a foundation for sustained engagement with communities underrepresented in clinical research

The anticipated budget for this initiative is $5 million from the Office of the Director (OD) in FY 2024, which would be increased to $25 million in FY 2025 and then increased again to $50 million to $100 million per year after assessing feasibility and budget requirements. After gathering feedback in spring 2024, the program will be launched later in 2024 and then expand in 2025 and beyond.

NIH Mission Statement Update (23:02)

Last fall, NIH requested public feedback on a proposed update to its mission statement (NOT-OD-23-163). The proposed update is intended to remove ableist language from the mission statement. There was a robust response to the request for information (RFI), which was due on November 24, 2023. An analysis of the responses will be presented to the NIH Advisory Committee to the Director during its publicly broadcast June 13–14, 2024, meeting.

Increase in FY 2024 Pre- and Postdoctoral Stipends (24:04)

In FY 2024, stipends for extramural trainees will be raised by approximately 4% for predoctoral trainees and by approximately 8% for postdoctoral scholars as compared with last year. This is the most substantial year-over-year increase since FY 2017. NIH selected the current plan to allow for an immediate pay increase without drastic cuts to the number of available National Research Service Awards, although a small reduction in the number of positions is expected. As appropriations and budget realities allow, the NIH goal is to reach the NIH Advisory Committee to the Director’s recommended stipend levels in the coming years (around $70,000 per year for postdoctoral scholars). Additionally, the child care subsidy will be increased by $500 (from $2,500 to $3,000 per scholar) in FY 2024.

Pediatric Research at NIH (26:10)

There is a crisis in pediatric research: In 2024, a substantial number of pediatric residency programs did not fill their slots. New medical school graduates are not applying for pediatric residency programs (possibly because pediatric residents are paid less than other medical specialties). Therefore, NIH is seeking to help make pediatric research a sustainable career. In FY 2023, NICHD spent 56.8% of its budget on pediatric research. Across NIH, NICHD’s pediatric research spending constitutes 16% of all dollars spent in that domain (more than $4 billion), with all other ICs combining to spend the remaining 84%. Pediatric researchers should be searching for funding opportunities from a variety of the ICs; conducting pediatric research is truly an NIH-wide effort.

A NASEM study conducted in FY 2022 analyzed the pediatric subspecialty workforce and its impact on child health and well-being. It strove to identify actions to ensure an adequate pediatric subspecialty workforce that can support both broad access to high-quality care as well as a robust research portfolio to advance the care of all children and youth. The working group’s recommendations included:

• Promoting collaboration and the effective integration of services between pediatric primary care clinicians and subspecialty physicians
• Reducing financial and payment disincentives
• Enhancing education, training, recruitment, and retention
• Supporting the pediatric physician-scientist pathway by:
  • Creating a public repository for data on pediatric physician-scientists’ funding and success throughout their careers, including measures to understand initial success and retention
  • Increasing the number of career development grants in pediatrics
  • Ensuring that pediatric training support at NIH occurs across ICs

A summer 2024 NASEM study on NIH pediatric research will seek to examine the current NIH pediatric research portfolio and structure, analyzing how projects are categorized as pediatrics (i.e., using the Research, Condition, and Disease Categorization system [RCDC]); how pediatric components have been included in or excluded from larger NIH initiatives; whether there are structural or process impediments to pediatric research applicants; how pediatric research priorities are established; and how pediatric research activity is coordinated across ICs. The working group will make recommendations to improve NIH’s overall support of child health research.

The NIH Pediatric Research Consortium (N-PeRC) was established in 2018 to increase collaboration and coordination of pediatric research across NIH. Its focus areas thus far include:

• Pediatric pain research
• Pediatric medical devices (PMDs)
• The transition from adolescent to adult health care
• COVID-19 funding for pediatric research
• Pediatric clinical trials
• The pediatric research workforce

A PMD public-private partnership (PPP) is now in the design phase. The PMD PPP seeks to address the problem that PMD approval lags behind approval for adult devices. Challenges for PMDs include a lack of validated endpoints in very young populations (e.g., difficulty in assessing benefit); the potential for long-term device use (e.g., device longevity, changing sizes, exposure to implanted materials); and a relatively low return on investment (e.g., small market, sample size) from a business perspective. NIH is partnering with the Foundation for the National Institutes of Health (FNIH) to lead the design of a full-scale PPP. More information can be found on the FNIH website’s Pediatric Medical Devices Design Phase page . A public engagement session on the final workstream outputs will be held June 17–18, 2024.

NICHD Strategic Planning (38:51)

NICHD is working on updating its 2020 Strategic Plan, which must be updated every 5 years as required by the 21st Century Cures Act. Strategic planning goals address research, stewardship, management, and accountability. NICHD has been documenting and tracking activities and achievements toward its current objectives and will begin refreshing the strategic plan over the next year. An RFI to solicit feedback from the public should be issued in August 2024, and the expectation is that the strategic plan update will be released in spring 2025.

NIH Leadership Updates (39:55)

Dr. Bianchi noted the following director updates:

• W. Kimryn Rathmell, M.D., Ph.D., is the new director of the National Cancer Institute (NCI)
• Sean Mooney, Ph.D., is the new director of the NIH Center for Information Technology
• Kathryn Neuzil, M.D., M.P.H., is the new director of the Fogarty International Center
• Joshua A. Gordon, M.D., Ph.D., director of the National Institute of Mental Health (NIMH), recently announced his resignation and plans to return to Columbia University as the chair of the Department of Psychiatry
• The National Library of Medicine posted to fill its director vacancy

NICHD currently has job openings for extramural branch chiefs, program officers, extramural policy officers, and intramural laboratory postdoctoral fellows and trainees.

Discussion (42:17)

Regarding pre- and postdoctoral salaries, a board member asked whether NIH fellows were moving toward unionization. Dr. Bianchi said that trainees cannot live on previous salary levels and gave an example of Stanford research fellows living in tents. NIH’s intramural program trainees have voted to unionize, so negotiations will likely be forthcoming. The bottom line is that the number of trainees will be reduced, that sponsoring institutions will need to provide supplements, or that other creative solutions will need to be implemented.

The board member asked whether new funding opportunites would be announced for the PMD PPP. Dr. Bianchi said that the PMD PPP program would be prioritized in NICHD’s 2025 Strategic Plan, and well-scored applications that address the aims of the strategic plan are usually funded. Theresa H. Cruz, Ph.D., reminded the group that the PMD PPP program was on the agenda for Day 2 of this meeting.

Another board member said that the National Multiple Sclerosis Society (NMSS) was following NICHD’s lead in raising salaries for its postdoctoral researchers. NMSS also has a network of pediatric multiple sclerosis centers that can collect data and promote collaboration among physicians who treat children with multiple sclerosis. The board member offered this network to help with NICHD research efforts. Dr. Bianchi said that she would help connect NMSS with N-PeRC.

A board member said that he and his colleagues were in favor of raising salaries for pre- and postdoctoral research fellows. He asked whether consideration would be given to cost-of-living differences for various locations across the United States. Dr. Bianchi said that she would inquire about cost-of-living differences and noted that geographic location is taken into consideration when offering compensation for other NIH positions.

NCMRR Director’s Report (52:51)

Theresa H. Cruz, Ph.D., Director, NCMRR

Dr. Cruz thanked the board members for their time and participation in this group and acknowledged the passing of Kenneth J. Ottenbacher, Ph.D., OTR, professor emeritus and former director of the Rehabilitation Sciences Ph.D. Program at The University of Texas Medical Branch at Galveston, who served on the NABMRR and supported health services research as a mentor and collaborator. In her report, she presented NCMRR staff updates, activities, collaborations, training and career developments, and the latest science advances.

NCMRR Staff Updates (55:41)

Joe Bonner, Ph.D., was selected for the highly completitive NIH Senior Leadership Program, and Toyin Ajisafe, Ph.D., was selected to serve on the NICHD Office of Health Equity’s internal advisory committee.

The National Institute of Neurological Disorders and Stroke (NINDS) is hiring a program director in neurorehabilitation. Interested candidates should send a current curriculum vitae and a letter of introduction describing their experience and interest in the position to Ling Wong, Ph.D.

Activities (57:18)

RFA-HD-24-007: Understanding and Mitigating Health Disparities experienced by People with Disabilities caused by Ableism (R01 Clinical Trial Optional), issued in August 2023 and co-sponsored by NICHD, NCI, the National Eye Institute, and the National Institute of Biomedical Imaging and Bioengineering (NIBIB), was designed to encourage research on understanding how ableism contributes to health disparities for people with disabilities (PWD) and/or developing system-level interventions to combat the negative health impacts of ableism. The request for applications (RFA) also required a plan to include the lived experience. Applications were due on November 30, 2023, and reviewed in March 2024; funding plans will be presented at the June National Advisory Child Health and Human Development Council. Dr. Cruz will announce the funded awards at the December NABMRR meeting.

RFA-HD-25-001: Medical Rehabilitation Research Resource (P50 Clinical Trial Optional), a revamped infrastructure RFA co-sponsored by NICHD, NINDS, NIBIB, the National Institute on Deafness and Other Communication Disorders, and the National Center for Complementary and Integrative Health, was published on April 3, 2024. It changes the funding mechanism to the P50, places more emphasis on research than the previous RFA, and now requires the inclusion of the lived experience. An applicant webinar will be held on May 15, 2024, at 2 p.m. ET. The due date for applications is July 30, 2024.

Build UP Trust , a $1.25 million NIH community engagement challenge, is a prize program designed to solicit solutions that increase research participation and the adoption of medical care by building trust and improving engagement with historically underserved communities, which includes PWDs. Phase I winners will be announced in May 2024. With the Build UP Trust Challenge, NIH seeks to become more accessible and create alternative ways to engage with new people who have not traditionally applied for funding. The process for interaction has been simplified, and this program does not require the long applications that are required for some NIH funding opportunities.

Between January and April 2024, NCMRR staff attended the following conferences and in-person outreach events:

• Training in Grantsmanship for Rehabilitation Research (TIGRR)
• Association of Academic Physiatrists
• American Academy of Orthotists and Prosthetists
• Symposium on Regenerative Rehabilitation
• Reeve Summit
• American Society of Neurorehabilitation
• University of Texas at Austin Research Day
• American Speech-Language-Hearing Association
• Society for the Neural Control of Movement
• Consortium of Research-Intensive Schools of Health Professions Meeting
• Neurorehabilitation and Restorative Neuroscience Training Network Scholar Day

The NCMRR budget has grown steadily, from $67 million in FY 2014 to $88 million in FY 2023. NCMRR is an NICHD extramural program. Research is getting more expensive, so “buying power” is not quite keeping pace with the budget growth.

Training and Career Development (TCD) Updates (1:06:33)

In May 2023, an NABMRR TCD Working Group began deliberations on how NCMRR has been supporting TCD efforts; they presented their finding and recommendations at the December 2023 NABMRR meeting. NCMRR has begun to act on these findings. In addition, the following activites relate to broader NICHD and NIH support of TCD:

• An NIH-wide notice (NOT-OD-24-084) has been issued regarding changes coming to the fellowship grant application review process in 2025. The NIH Center for Scientific Review is changing the review framework and the weighting of each application section. Additional information could be presented at a future NABMRR meeting if board members would like this topic to be added to the agenda.
• NICHD has issued an RFI (NOT-HD-24-011) to invite comments and suggestions on its support of extramural research training and early-career development.
• NICHD announced in NOT-HD-24-015 that it is participating in PAR-23-222: Advancing Research Careers (ARC) Predoctoral to Postdoctoral Transition Award to Promote Diversity (F99/K00 - Clinical Trial Not Allowed). By the time of award, the candidate must be a citizen or a noncitizen national of the United States or have been lawfully admitted for permanent residence (i.e., must possess a currently valid Permanent Resident Card [USCIS Form I-551] or other legal verification of such status).
• To obtain better metrics and outcomes data on its training programs, NCMRR has begun using Scopus publications to determine the research engagement of its previous 1,500 trainees (e.g., about half of them have published in the past year).
• Later in this meeting, NCMRR staff will present a career development concept clearance.
• For trainees who are not interested in academic positions, NIH OD issued NOT-OD-24-051: the BIO-Entrepreneurship Capstone, a program that aims to enhance early-career scientists’ understanding of the potential value and health care impact of their life science research activities, and to encourage consideration of an entrepreneurial career within the life science industry.
• Newly released NIH-wide Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs are providing Small Business Transition Grants to support early-career scientists and health professionals who are interested in transitioning to work in a small business. The funding allows recipients to gain industry experience, receive entrepreneurial training and mentorship, and advance biomedical or public health technology with commercial potential. A webinar will be held on June 11, 2024, to introduce the Small Business Transition Grant for New Entrepreneurs.
• The NIH Entrepreneurship Bootcamp is a 7-week program to provide early-stage (pre-SBIR) innovators with an introduction to customer discovery and business model validation. Each team can have up to three members, and at least one member must have previously been supported on at least one NIH-funded research project or training grant. Support from a principal investigator (PI) or key personnel is not required. Innovators with an active SBIR/STTR award or those who plan to submit a Phase I application during the program are ineligible.
• The Concept to Clinic: Commercializing Innovation (C3i) Program provides medical device innovators with specialized business frameworks and essential tools for successful translation of biomedical technology from laboratory (concept) to market (clinic). It is offered to active NIH-funded research projects and has two discrete components:
  • The eC3i course is a virtual 10-week-long summer entrepreneurial education and training program that gives researchers the tools to answer the question “Does my technology have commercial potential?” It features 20 hours of interaction with business advisors, project managers, and stakeholders. The most highly recommended teams include graduate students or postdoctoral candidates.
  • The C3i 24-week bootcamp uses a mix of in-person meetings and telephone calls. It is focused on validation and execution. Phase I focuses on characterizing the unmet market need, identifying the customer and stakeholder segments, conducting customer and stakeholder interviews, and validating the business opportunity. Phase II provides commercialization planning support, covering intellectual property strategy, regulatory and reimbursement requirements, risk assessment, milestone creation and valuation, and pitching to investors and/or strategic partners.
• The University of Southern California’s Reproducible Rehabilitation (ReproRehab) Research Program offers a free 6-month program for rehabilitation researchers to learn data science and computer programming, best practices for reproducibility, and open science principles. Its fellows receive hands-on training from teaching assistants. Applications for the 2024–25 cycle were due on May 1, but there are free YouTube tutorials on the ReproRehab course website , along with a searchable database of open-access datasets and courses for rehabilitation researchers.

Collaborations (1:22:30)

NCMRR is continuing its collaboration with NIBIB on the Design by Biomedical Undergraduate Teams (DEBUT) Challenge, wherein undergraduate student teams are challenged to develop technological solutions to meet unmet needs in any area of health care. For the challenge, NCMRR offers a $15,000 prize for the best project that develops rehabilitative and assistive technologies for the functional and health care needs of people with physical disabilities. The current round of applications is due on May 31, 2024.

The Limb Loss and Preservation Registry (LLPR) , a collaboration between NIH and the U.S. Department of Defense, is an electronic health record–based quality registry that collects limb loss experience and procedural data from more than 200 enrollment locations. The registry contains data from more than 400,000 patients. To ensure long-term sustainability, NIH is transferring ownership of the LLPR to a nonprofit entity, where it will be operated in partnership with the Amputee Coalition of America.

The hybrid (in-person and virtual) NIH Rehabilitation Research Conference will be held March 10–11, 2025.

The NIH 2025 Research Plan will be discussed later in this meeting. NCMRR issued an RFI to solicit feedback on what purpose the research plan should serve, and those responses will be discussed.

Science Advances (1:26:36)

Darcy Reisman, Ph.D., PT (R01 HD086362), is studying capacity versus performance. Her research team found that only individuals with chronic stroke who completed a step activity monitoring behavioral intervention with skilled coaching and goal progression demonstrated improvements in physical activity in the real world (i.e., steps per day). (PMID: 38134254)

Time magazine featured NIH-funded research conducted by Tommaso Lenzi, Ph.D. (R01 HD098154), in its Best Inventions of 2023 issue. Dr. Lenzi and his team at the University of Utah invented the Utah Bionic Leg, which is capable of more natural leg and knee movement, making it easier for the user to move and climb stairs. The Utah group is partnering with Ottobock to commercialize the invention.

In a small clinical trial for six patients with moderate to severe traumatic brain injury (msTBI), Nicholas D. Schiff, M.D., and his team tested the safety and efficacy of applying deep brain stimulation (DBS) to the central lateral (CL) nucleus of the thalamus and its associated medial dorsal tegmental (CL/DTTm) tract. After 3 months, the participants showed an average improvement of 30.7% on a test of cognitive function. These results suggest that CL/DTTm DBS could be safely applied and may improve executive control in patients with msTBI, paving the way for larger clinical trials. Importantly, however, Dr. Schiff entered an NIH-funded neuroethics collaboration that revealed the challenge of adaptation to improvements in cognitive function and emotional regulation as well as altered (and restored) relationships and family dynamics. The team found that most subjects and families identified the need for supportive counseling to adapt to the new trajectory of their lives. (PMIDs: 38049620 and 37850455)

Discussion (1:32:11)

Dr. Cruz reminded attendees to stay in touch by joining the NCMRR Rehabilitation Research listserv.

A board member said that they were pleased to hear that NICHD is participating in PAR-23-222 and asked whether students should apply before or after they have defended their doctoral thesis. Dr. Cruz said that the grants support the completion of doctoral dissertation projects. Dr. Cruz added that the National Institute on Disability, Independent Living, and Rehabilitation Research recently announced an RFA to support dissertation-level graduate students.

A board member asked whether the Build UP Trust Challenge would continue in future years. Dr. Cruz said that she did not know but hoped that additional challenges would present opportunities.

A board member asked whether NIH planned to develop public-private industry partnerships that could provide students with industry experience while they were still in school. Dr. Cruz said that C3i does not place students in industry, but it allows students to interact with entreprenuers. It is not the same as an internship. Another board member said they received an R25 grant for their Neuromotor Skill Advancement for Post-baccalaureates program , which gives students experience working in industry. Dr. Nitkin added that there is an R25 funding mechanism to connect engineers with rehabilitation patients so that the engineers can learn more about lived experiences and unmet medical needs from the patients. A board member asked whether the engineering programs were open to people outside the award insitution. Dr. Nitkin said that not all of them are, but perhaps that factor should be taken into consideration for future announcements. The board member said that it would be nice to allow cross-institutional participation.

A board member suggested including presentations on both RepoRehab and the use of AI at a future NABMRR meeting. Dr. Cruz agreed and said that she would also send out information she had received about an upcoming AI in rehabilitation medicine meeting.

Sports Medicine for the Para and Adaptive Athlete: Making the Medal Platforms Fully Accessible (1:49:08)

Cheri Blauwet, M.D., Director, Kelley Adaptive Sports Research Institute, Brigham and Women’s Hospital, and Associate Professor, Harvard Medical School

In her presentation, Dr. Blauwet provided some professonal and personal background on the Paralympic movement, unique patterns of injury and illness in Paralympic and adaptive athletes, how the science is informing clinical care, and future directions in injury- and illness-prevention research in elite athletes and its translation to community-based adaptive sports. Dr. Blauwet’s personal journey and lived experiences as an athlete, physician, and leader inspires her to continue expanding the field and advocating for change.

Participation in the Paralympics continues to grow rapidly. At the Stoke Mandeville Games, which were held in 1948, 16 athletes with spinal cord injury participated to advocate for the use of sport for rehabilitation. At the 2020 Summer Paralympic Games in Tokyo, which were postponed until 2021 because of the COVID-19 pandemic, 4,403 athletes from 162 countries participated in 22 elite competition sports, with 1,200 hours of television coverage in the United States and 4.25 billion viewers on digital media. Six countries (Ethiopia, Pakistan, Ecuador, Sri Lanka, Costa Rica, and the United Arab Emirates) won their first Paralympic gold medals in Tokyo. At the 2022 Winter Paralympic Games, held in Beijing, 564 athletes from 46 countries competed. Team USA won 20 medals, including its fourth straight gold medal for ice hockey. The 2024 Summer Paralympic Games will be held in Paris.

All athletes, including Paralympic athetes, accept the risks involved in competing. Because of the success of the Paralympic program, adaptive sports centers, national membership organizations, and outdoor access initiatives are popping up all over the world. Clinicians and researchers must work to promote physical activity, health, chronic disease prevention, and safe participation in sports for all athletes. The benefits of physical activity and participation in sports include reduced anxiety and depression, improved social integration, enhanced life satisfaction, and an increased likelihood of employment. Adaptive sports are critical for expanding opportunities for PWDs and allowing them to be fully engaged in the community alongside their peers.

Paralympic and adaptive athletes have unique injury and illness patterns. These patterns have been discovered through the use of research frameworks and longitudinal injury surveillance studies that began at the 2012 Paralympic games in London, where data were captured on 3,329 female and male athletes and 46,606 athlete-days. A web-based injury surveillance system with greater than 85% compliance has provided valuable insights, including:

• Injury patterns vary between Olympic and Paralympic athletes. Male and female Paralympic athletes have similar injury rates.
• Of 633 injuries, 258 involved the upper limb: the shoulder, wrist/hand, and elbow are most commonly and disproportionately involved.
• Shoulder injury incidence increases with age in Paralympic athletes, and such injuries were more likely to be reported as chronic or overuse injuries.
• Injury incidence varies by sport:
  • Surveillance research data on football five-a-side players helped change policy, rules, equipment, and concussion protocols to increase safety.
  • In track and field, ambulant athletes had lower extremity injuries, whereas wheelchair or seated athletes experienced upper extremity injuries. Further analysis revealed that the seated throwers had more injuries when compared with wheelchair racers, leading to the hypothesis that the explosive nature of the throwing mechanism coupled with less leg and core strength to generate force placed the upper extremities at high risk.
• Because the incidence of osteoarthritis goes up 20 to 30 years after an injury, young athletes must take the long-term health implications of upper extremity injuries into consideration.
• Age and gender are not independent predictors of illness in Paralympic and adaptive athletes. Although respiratory and gastrointestinal (GI) illnesses are most common, the rates of nonrespiratory and non-GI illness (e.g., skin and genitourinary) are much higher in Paralympians than in Olympians. Most illnesses in a games time environment are infectious, and Paralympic athletes are late in reporting illnesses to clinicians.
• The skin/subcutaneous and genitourinary systems are uniquely affected in Paralympic athletes, especially those with spinal cord injuries and amputations.

Future studies will include the longitudinal characterization of injuries and illness beyond the Paralympic Games (e.g., year-round monitoring by national and international paralympic committees and sports federations), and increased data collection on mental health, concussion, and relative energy deficiency in sport.

Before becoming a physician and administrator, Dr. Blauwet grew up on a Midwestern farm, where she was injured in a farm accident when she was only 16 months old. She has used a wheelchair her entire life and developed a positive self-identity despite not meeting other children who used wheelchairs. She discovered adaptive sports in high school with the help of an encouraging coach and enjoyed the experiences of being on the track team and later participating in an adaptive sports group. These early experiences gave her the confidence to train and compete at an elite level. Dr. Blauwet competed in her first Paralympic Games during college while attending and competing for the University of Arizona, matriculated to the Stanford School of Medicine, competed in two more Paralympic Games for Team USA, and won the Boston Marathon in 2004 and 2005.

Being a doctor with a disability has allowed Dr. Blauwet to identify and address barriers to working in the medical field. She has also helped build a new medical specialty called Paralympic sports medicine, bridged leadership roles, and used her platform to bring enhanced resources and recognition to athletes, including equal medal payouts for Paralympians. Ongoing challenges remain in collegiate and high school athletics.

In summary, there has been exponential growth of the Paralympic movement. Surveillance data and research have improved our understanding of injury and illness patterns in para athletes, and the field of Paralympic sports medicine is burgeoning. Every NABMRR member has influence and should not be afraid to use it to improve athlete health and well-being and provide enhanced resources to athletes.

Discussion (2:29:50)

A board member asked which NIH grants were best for conducting Paralympic sports medicine research. Dr. Blauwet said that NIH and other federal funding opportunities existed for physical activity research in PWDs, but sports medicine societies (e.g., the American College of Sports Medicine, American Medical Society for Sports Medicine, International Olympic Committee) were more likely to fund research specific to Paralympic athlete injuries and illnesses.

Another board member asked about the differences between studying rehabilitation versus wellness in PWDs. Dr. Blauwet said that several studies show how adaptive sports can be used to enhance rehabilitation and bridge the gap from rehabilitation to wellness. Formal rehabilitation progression can move from inpatient to outpatient to physical therapy and occupational therapy, but less has been done to bridge the gap between formal therapy and lifelong wellness. Mentor programs may offer pathways to wellness through the modeling of physical activity behaviors, healthy eating, and mindfulness—especially for people who have experienced a new disability within the past 2 years. The board member said that Paralympic athletes could be good models for how to implement wellness behaviors.

A board member asked about the status of formal adaptive programs at universities. Dr. Blauwet said that there are many adaptive programs throughout the United States. She added that although 60% of the U.S. athletes at the Tokyo Paralympic Games based their training at a university, the National Collegiate Athletics Association varsity ecosystem doesn’t offer any of the sports. Most of the programs are based in disability student services or run as club sports; the two largest adaptive sports programs in the United States—at the University of Illinois Urbana-Champaign and the University of Arizona—are both run by disability student services departments. It has become a separate-but-equal system, which is not the best approach. More progress is needed to make interscholastic sports inclusive.

A board member asked whether performance-enhancing compounds were an issue at the Paralympic Games, given that some PWDs need to use medications that might be banned in competition to treat their conditions. Dr. Blauwet said that doping does occur in Paralympic sports, but the same testing authorities that oversee Olympic sports (i.e., the U.S. Anti-Doping Agency, the World Anti-Doping Agency) also oversee the Paralympics. All athletes are held to the same standards, but therapeutic use exemptions are allowed for athletics with specific medical conditions.

NINDS Traumatic Brain Injury (TBI) Nomenclature Workshop (2:43:14)

Hibah Awwad, Ph.D., Program Director, Repair and Plasticity Cluster, Division of Neuroscience, NINDS

Dr. Awwad introduced the session by saying that an NINDS steering committee is addressing TBI classification and nomenclature. This initiative first arose during a 2007 workshop on the classification of TBI for targeted therapies when subject matter experts (SMEs) identified the need to develop more precise classification of TBI to improve patient care. Researchers have spent more than 15 years gathering evidence from patient databases, standardizing computed tomography (CT) classification, improving standardization and reliability of existing measures, identifying common data elements for TBI, and improving informatics approaches. The steering committee is now prepared to update the classification system and nomenclature for TBI.

The current approach to classifying the clinical severity of head injuries in roughly 2 million Americans each year using the terms “mild,” “moderate,” and “severe” is outdated and imprecise, and treatments based on the use of those terms are leading to increased medical costs and preventable deaths. According to NASEM, these common labels for defining TBI may obscure the severity of head injuries and lead to inadequate treatment and potential harm to the patient. Beyond being imprecise, the current classification system is crude, lacks a continuum, and has limited associations with mechanisms of injury. There are no effective targeted treatments for mild, moderate, or severe TBIs. Finally, the terms lead to treatment bias, and patients and families do not want caregivers to use terms that label patients for life and limit access to and delivery of care.

A February 2022 NASEM consenus study titled Traumatic Brain Injury: A Roadmap for Accelerating Progress identified the following challenges:

• TBI care in the United States often fails to meet the needs of individuals, families, and communities affected by this condition.
• High-quality care for TBI requires that it be managed as a condition with both acute and long-term phases.
• Public and professional misunderstandings are widespread with respect to the frequency, manifestations, long-term consequences, and proper detection, treatment, and rehabilitation of TBI.
• The United States lacks a comprehensive framework for addressing TBI.

After the consensus group recommended creating and implementing an updated classification for TBI, NINDS established a steering committee of SMEs to tackle this task.

On January 22–23, 2024, NINDS held a TBI classification and nomenclature workshop. The primary goal of the workshop was to develop a more precise and validated classification and nomenclature system for TBI patients that is based on early clinical symptoms (e.g., the Glasgow Coma Scale sum score), neuroimaging results from CT and magnetic resonance imaging (MRI), blood-based biomarker measurements, psychosocial and environmental modifiers, implementability of knowledge to practice (i.e., using implementation science), and retrospective classification using validated measures (e.g., having a prior TBI). The new system should also allow TBI classification to change as a person’s condition evolves over time.

Additional workshop goals included identifying current gaps and research topics that may inform, refine, and update the new TBI classification and nomenclature system. The ultimate goal was to develop and implement a global solution for multidimensional TBI characterization and classification. To achieve these goals, the steering committee formed working groups to address each of the previously enumerated areas. Working group members are clinicians, researchers, and people with lived experience with a TBI.
Attendees proposed a “CBI+M” framework for improved characterization and classification of a brain injury within the first 24 hours of diagnosis. The framework contains four critical pillars:

• Clinical symptoms
• Biomarker measurements
• Imaging results
• Modifiers: injury, patient, and social and environmental factors that influence assessment, management, recovery, and outcome

Each pillar is further defined by core and expanded elements. The implementation of the CBI+M framework will depend on:

• Determining whether it is pragmatic for clinical use and adaptable for research applications
• Anticipating and overcoming potential hurdles to implementation and penetration
• Determining whether it becomes policy and offers a pathway to reimbursement
• Securing buy-in from end-users, including a broad array of specialists, professionals, and settings
• Monitoring penetration and impact of the new system on clinical care and research and determining whether it drives performance improvement

Going forward, steering committee and working group members still need to complete the following tasks:

• Refine and operationalize the CBI+M pillars
• Finalize the core and expanded components of the CBI+M framework
• Pilot-test the pillars in existing datasets to provide supporting evidence
• Prepare a consensus document
• Conduct extensive intra- and inter-pillar analyses and cluster analyses to provide further support (e.g., associations of pillar elements with outcome, care paths, and management decisions)
• Develop and execute an implementation plan in partnership with stakeholders
• Validate the CBI+M pillar concept in prospective studies

The goal of this initiative is to develop a simple evidence-informed, pragmatic multimodal and multidimensional TBI classification system that will allow clinicians to provide individualized care and be adaptable beyond hospital trauma care. If these efforts are successful, they will also accelerate research and significantly advance the field to improve patient care and outcomes following TBI. Publications will be forthcoming, and the recordings and working group reports from the workshop are available on the NINDS event page.

Dr. Awwad acknowledged all members of the steering committee, working groups, support staff, and NINDS leadership, especially Nsini Umoh, Ph.D., who leads this initiative for NINDS. She invited NABMRR members to contact her or Dr. Umoh to become involved in these efforts.

Discussion (3:08:00)

Regarding the clinical pillar, a board member asked whether the expanded elements would include other functional domains beyond cognition, vestibulo-occular, and balance. Dr. Awwad said that the current framework is meant to address only the first 24 hours after TBI. Other functional domains were discussed by working group members for various patient populations and settings (e.g., sports, military).

The board member said that it would be useful for the new framework to inform therapies and patient selection for each treatment. Dr. Awwad agreed that it would be ideal if the new system helped optimize individualized care and treatment.

Regarding the imaging pillar, another board member asked whether neuroimaging domains that captured dynamic changes (e.g., electroencephalography) would be incorporated into the framework. Dr. Awwad said yes and encouraged the board member to review the report from the imaging working group. The timing of each exam was discussed and considered by the working group.

Concept Clearance (3:13:15)

Ralph Nitkin, Ph.D., Deputy Director, NCMRR

Dr. Nitkin asked NABMRR members for feedback on a proposed concept for a rehabilitation clinical scientist K12 research program. The purpose of the program is to establish a national career development network to support a diverse pool of clinically trained rehabilitation researchers at their home institutions through centralized content, individualized mentoring, and annual networking opportunities. The proposal was developed in response to an increasing demand for NCMRR to reinvest in career development and specific recommendations from the NABMRR.

Each K12 network will be responsible for targeting appropriate professional constituencies, developing centralized materials to promote core competencies, recruiting diverse motivated scholars, providing individualized research mentorship and career support, and monitoring outcomes, including productivity and professional advancement. The objectives of this program are to attract and nurture a new cadre of rehabilitation clinical scientist–researchers, encouraging them to seek independent research support, and to empower them to become independent researchers and academic leaders.

Discussion (3:16:08)

A board member asked whether this concept was meant to reinvigorate the K12 program. Dr. Nitkin said that it uses the same mechanism with updated ideas.

Another board member asked whether a K12 was an individual or group award. Dr. Nitkin said that K12s create cadre networks that support several individual scholars through a centralized support structure. Researchers in K12 programs can subsequently apply for individualized career-development (K) awards.

A board member asked whether K12 networks were located only within universities. Dr. Nitkin said that traditionally NIH K12 networks can either be institutionally based or networks to support scholars at their home institutions; this proposal would use the latter model to support a cadre of scholars, keep them connected, and help them network.

A motion to approve the concept carried unanimously.

Recess (3:19:50)

Dr. Bregman thanked the presenters and NABMRR members for an engaging day with lively discussion. She adjourned Day 1 of the meeting at 2:05 p.m. ET.

Day 2: May 7, 2023

Access the VideoCast recording of the second day of the May 2024 NABMRR meeting.

Welcome (00:04)

Ralph Nitkin, Ph.D., Deputy Director, NCMRR
Barbara S. Bregman, Ph.D., PT, Chair, NABMRR

The meeting resumed at 10 a.m. Dr. Nitkin welcomed everyone to Day 2, reviewed the meeting agenda, and noted that the VideoCast recording and meeting minutes would be available online. Dr. Bregman called the roll.

Trajectories in Cerebral Palsy (CP): What Do the National Aeronautics and Space Administration (NASA) and the American Academy for Cerebral Palsy and Developmental Medicine (AACPDM) Have in Common? (03:45)

Wade Shrader, M.D., Freeman Miller Endowed Chair of Cerebral Palsy, Nemours Children’s Health^®

Dr. Shrader shared the trajectories of his life and work that led to his becoming a pediatric orthopedic surgeon with expertise in treating children with CP. His research on pulmonary function, scoliosis, muscular dystrophy, and the measurement of walking activity has led to improved outcomes and increased quality of life for children with CP, including his own children.

While growing up in small town in Mississippi, Dr. Shrader developed a love for NASA. After earning a degree in aerospace engineering, he began working in flight mechanics at NASA’s Marshall Space Flight Center, where he was responsible for developing guidance, navigation, and control for the National Launch System. He grew into other positions at NASA and even won a prestigious award, but when budgets were decreased in 1991, following the end of the Cold War, pathways to a career change seemed to be opening and calling him into medicine.

During medical school, Dr. Shrader’s triplets were born prematurely at 28 weeks. Although all three babies did well in the neonatal intensive care unit (NICU) and the care providers reassured Dr. Shrader and his wife that the triplets would be fine, with no significant disabilities, having them in the NICU was a terrifying experience. The boys had noticeable developmental delays and other symptoms, but a pediatrician insensitively diagnosed the CP by blurting out, “Of course he has CP…what did you expect me to say?” That day marked an important moment in Dr. Shrader’s life: He realized that more needed to be done for families with children who had CP. After completing his orthopedic surgery residency at the Mayo Clinic, a pediatric rotation gave him a new purpose in life: to help other families like his.

Nemours Children’s Health^® is a legacy of the Alfred I. DuPont family in the Delaware River Valley. It is a world leader in pediatric orthopedic surgery and CP research and care. The multidisciplinary CP program at Nemours, which sees more than 2,000 patients with CP from all over the country and performs 300 surgeries annually, was started by Freeman Miller, M.D. Dr. Schrader was hired to lead the CP program at Nemours when Dr. Miller retired in 2018. Available technologies at Nemours’ Gait Laboratory include kinematics, kinetics, oxygen consumption, foot pressure, and walking activity. The institution also developed markerless motion-capture technology (a new frontier in gait assessment).

Dr. Schrader’s research addresses current clinical care gaps in performing pulmonary function tests, neuromuscular diseases, CP and CP-like conditions, skeletal dysplasia, and scoliosis.

• To assess pulmonary function in CP, the Nemours team developed and tested pneuRIP™, a noninvasive respiratory monitor, and measured pulmonary dysfunction in children with muscular dystrophy and CP. A future goal will be to determine how scoliosis treatment affects pulmonary function measurements.
• Because children with CP have increased connective tissue, long sarcomeres, decreased satellite cell number and function, and epigenetic anomalies, Nemours developed a preclinical study to test whether a collagenase could decrease muscle stiffness; it did. If translatable, collagenase could be an effective alternative to botulism toxin. The group is also using magnetic resonance elastography as a novel noninvasive measure of static/dynamic muscle properties and conducting several other musculoskeletal studies as well.
• Commercially available wearable trackers do not work for children with disabilities, so Nemours researchers developed a specialized tool to measure walking activity during postoperative care and intervention assessment. The Modus StepWatch is a U.S. Food and Drug Administration (FDA)–approved device that is worn around the ankle to track walking activity. It can be taken home to measure stride count and frequency and, to ensure accuracy, it is calibrated to follow the pattern and cadence for each participant. Walking activity research is especially useful for improving postoperative rehabilitation; future studies will also include knee surveillance.
• Walking performance, mobility, and activities of daily living (ADLs) deteriorate as much as 25% in adults with CP. Nemours has collected longitudinal data, including walking performance from adolescence to adulthood, on a large cohort of patients. Interestingly, the data show that gait pattern remains stable from the second to third to fourth decade in individuals functioning at Gross Motor Function Classification System levels I, II, and III. Pain studies are showing that pain interference is slightly higher than normal for the Nemours group, and the overall prevalence of moderate to severe pain is 36% (which is quite high and consistent with the literature). Compared with youth with CP, lower walking activity is found in adults, which is similar to the decrease seen in the population without disabilities. Nemours also collects self-reported outcomes on adults with CP. When compared with age-matched control groups, depression levels and college education levels in adults with CP are similar to those for adults without a disability; however, rates of unemployment and disability are higher in adults with CP.

Being a parent of children with CP allows Dr. Shrader to share both positive and negative experiences with the families he treats. His children have had nearly every orthopedic surgical procedure that he performs in this population. Raising children with CP has been the most difficult thing he has ever experienced, despite his being highly trained in this area. Because of this difficulty, Dr. Schrader has begun the process of developing a biopsychosocial model of CP. Biological, social, and psychological factors are all incredibly important to medical and surgical outcomes. Because the family is the child’s main environment and CP impairs family well-being, family-centered care should address its needs. CP poses significant challenges to families, including increased stress and poorer mental health functioning, and the psychosocial needs of families of youth with CP are not systematically assessed. The Psychosocial Assessment Tool (PAT) is a brief caregiver report that can be used to screen for family psychosocial risk. It assesses potential stressors (psychosocial risks) and resources within the family and categorizes families into three levels of risk consistent with the Pediatric Psychosocial Preventative Health Model.

Nemours researchers are now working to develop a psychosocial risk screening tool that can be incorporated into the care of families with children who have CP. The measure, called the PAT-CP, will be tailored to the challenges and stressors for families with a child with CP, provide an effective and inclusive way to screen families, and allow for the systematic identification of families who are most in need and the appropriate allocation of psychosocial supports and resources. The incorporation of stakeholders—caregivers of children with cerebral palsy—is crucial to understanding the lived experiences of patients and families and to ensuring that the voices of patients and caregivers are highlighted during the adaptation process.

Dr. Shrader concluded his remarks by thanking the audience for listening to his journey from NASA to AACPDM. He said that serving on the NABMRR has been one of the greatest privileges of his career and that he is proud to continue investigating ways to improve the lives of children with disabilities and the lives of their families, and to improve the transition to adulthood for children with CP. He shared the following favorite quote from President Franklin Roosevelt: “We have always held to the hope, the belief, the conviction that there is a better life, a better world, beyond the horizon.” Dr. Shrader said he has had a great career “being in the business of hope.”

Discussion (48:44)

A board member asked Dr. Shrader what technological innovations were available to his children. Dr. Shrader said that his sons are now 27 years old, and that technologies were more limited 25 years ago. There were some exoskeletons and robotics back then—as well as gait trainers, reverse walkers, and standers—but they were not as advanced as they are now. Dr. Shrader said that exoskeletons should be made available to all families, because early loss of function is devastating to quality of life and ADLs. Technology-wise, using an iPad has allowed one of his boys to have a job.

A board member asked whether the medical community within orthopedics was ready to incorporate biopsychosocial factors into patient care. They wondered whether implementation of the PAT-CP would require a lot of education and cultural change in the provider community. Dr. Shrader said that the field is not likely ready for this type of change, but it is truly needed because perfect surgical outcomes cannot be obtained when families are struggling. Dr. Shrader said that he intends to help educate the pediatric orthopedic community about the importance of the measures and the effects of bias and social determinants of health (SDOH) on outcomes.

Dr. Nitkin asked how clinicians can motivate protective parents to push children with CP out of their comfort zones to promote rehabilitation, and how siblings could help or be involved. Dr. Shrader said that it is fascinating to see how different families approach disability: Some plow forward while others bury their heads in the sand. Most need to learn to advocate for their children for therapy, specialist consults, and technological advances. The PAT-CP will help identify which families need which type of assistance. Siblings can also be critical to positive family dynamics and often endure sacrifices by going to medical visits with a sibling who has CP. The PAT-CP includes a sibling assessment.

NICHD Office of Health Equity (OHE) (1:00:15)

Amanda Alise Price, Ph.D., Director, OHE, NICHD

Dr. Price presented an overview of the NICHD OHE, covering its mission, priorities, and activities. She highlighted several of NICHD’s diversity, equity, inclusion, and accessibility (DEIA) efforts and shared recent outreach and engagement efforts.

OHE’s mission is to serve as a catalyst to strengthen the institute’s commitment to ensuring the health and well-being of all children, adults, families, and communities. Its three main priorities are advancing DEIA goals, improving the number of underrepresented individuals in the scientific workforce, and addressing health disparities and promoting health equity. The governance structure for OHE includes extramural and intramural liaisons, a program analyst, a staff assistant, trainees, fellows, students, and targeted contract support. NICHD’s STrategies to enRich Inclusion and achieVe Equity (STRIVE) initiative outlined the eight committee goals with associated immediate, intermediate, and long-term actions that inform OHE’s ongoing work.

OHE activities fall into three broad categories, each with the shared priority of outreach and engagement. The categories are extramural division, intramural division, and internal workforce collaboration.

• In the extramural division, the Division of Extramural Research and the Division of Extramural Activities hold regular meetings with extramural leadership. They are developing a process for NICHD sign-on and management of notices of funding opportunities that focus on health equity, scientific workforce diversity, and research infrastructure.
• The Diversity Supplement Working Group and Office of Extramural Policy are creating an onboarding workshop for diversity supplement recipients and streamlining policies and procedures for reviewers.
• The Office of Clinical Research is developing resources, best practices, and guidance on enhancing engagement, inclusion, and representation in research.
• In the intramural division, the Division of Intramural Research is holding regular meetings with intramural leadership, funding summer research on health disparities and health equity, sponsoring an intramural health disparities and health equity seminar series, and providing short trainings and OHE presentations at PI meetings.
• The Office of Education is increasing diversity and inclusion in its intramural research, outreach/recruitment, training, and education efforts. It also presented a talk titled “What is Your Role in Health Equity?” at a retreat for intramural fellows.
• The intramural division collaborated with the extramural division to hold a workshop for intramural fellows on “Understanding the Extramural Grant Process” and facilitated information sessions for program officers, scientific review officers, and intramural trainees.
• Internal workforce collaborations with the OD (including the Executive Office and the Office of Administrative Management) include establishing a workplace culture that emphasizes sharing opportunities for advancement and professional development, providing resources for employees and supervisors, and fostering a welcoming and inclusive workplace.
  • Recruitment, retention, and hiring efforts focus on increasing recruitment efforts to support diversity in the internal workforce, using inclusive and equitable hiring practices, and prioritizing retention.
  • Trainings have covered microaggressions, bystander training, and DEIA topics.

NICHD needs diverse scientists at every level to expand its talent pool, provide a wider range of perspectives to inform research questions, avoid blind spots, address the cultural experiences of research participants, foster inclusive and successful teams, and improve external validity to provide research that is applicable to all. Recent DEIA efforts include the following:

• In 2023, NIH designated PWDs as a health disparity population. NICHD is working to add a new “disability” RCDC system category to improve identification of research related to disability.
• To support inclusion and engagement in NICHD research, OHE is collaborating with the NICHD Office of Clinical Research to gather and disseminate best practices and guidance on how to enhance inclusion and representation in clinical trials and increase engagement with communities and people with lived experience throughout the entire research process.
• To support scientific workforce diversity at NICHD, OHE is collaborating with the NICHD Extramural Training and Career Development Working Group to enhance the institute’s support of scientists from backgrounds underrepresented in biomedical science.

OHE’s recent outreach and engagement efforts include giving presentations, attending conferences, conducting a listening tour, distributing newsletters and communications updates, and participating in community service events. OHE participates on the Chief Officer for Scientific Workforce Diversity’s Diversity Catalyst Committee and Conference Planning Subcommittee to garner NIH-wide participation in DEIA conferences. Additional plans include the launch of a summer intern program and seminar series, the development of a diversity supplement reviewer recruitment and orientation process, and the completion of diversity supplement onboarding workshops. OHE staff also attends and presents at several external meetings, sharing outreach resources at a wide range of conferences, meetings, and panels.

Discussion (1:23:20)

Dr. Price asked NABMRR members to share their thoughts on how to support inclusion of PWDs in research and engagement with communities by sending an email to NICHD_OHE@nih.gov.

Dr. Price also asked NABMRR members to share information about any rehabiliation meetings that OHE staff should attend to improve its diversity efforts. Her email address is amanda.alise.price@nih.gov.

A board member suggested attending the American Society of Neurorehabilitation meeting. They also asked whether the Office of Clinical Research could provide any resources on increasing diversity in the populations recruited for human subjects research. Dr. Price said that half of her team was in the process of gathering best practices, strategies, and emerging resources for clinical research recruitment. She will disseminate the information throughout NICHD when it becomes available, including NCMRR.

The board member asked whether OHE had developed any resources for engaging with elementary school-aged children to stimulate early interest in engineering. Dr. Price acknowledged the importance of early engagement with children and said that OHE planned to partner with the Intramural Office of Education to participate in science fairs, provide prizes, and develop additional ideas. She added that some NIH grant programs are currently available to high school students.

Another board member noted that their institution has been partnering with museums and performing arts groups to share information about science and research, including with 1,500 fifth graders. Regarding scientific workforce diversity, the board member asked whether OHE had considered developing F awards to help students apply to Ph.D. programs. Dr. Price said that the Training Working Group recommendations, which are being implemented now, were led by Dr. Cruz. The mechanism and avenues of support will now be developed as part of the implementation process. Dr. Price added that the intramural division now has both pre- and postbaccalaureate training programs, and insights will be taken from those programs to inform the Training Working Group’s efforts on the extramural side.

A board member shared concerns about recent movements to disband DEIA efforts. Dr. Price said that NIH continues to have a broad definition of diversity, beyond race and ethnicity. NIH is planning to repackage some programs and initiatives to preserve the resilience of its efforts in this area, because the goal is to improve health for all. NICHD’s commitment remains strong and will be broadened with the addition of a new website promoting “Research for All.”

PMD PPP Program (1:36:00)

Antonello Pileggi, M.D., Ph.D., Program Officer, Obstetric and Pediatric Pharmacology and Therapeutics Branch, NICHD

Dr. Pileggi provided background information and discussed plans for the launch of NIH’s PMD PPP program. He shared information about the partners involved in the project as well as the project’s timeline for 2024.

According to the FDA, PMDs are medical devices manufactured for use by people from birth through age 21. The following list describes several of the many challenges inherent in designing PMDs:

• Children are not small adults; they are growing and continually going through many physiological and biological changes.
• There are unique ethical considerations when conducting research and clinical trials with children.
• There is a lack of validated endpoints in very young populations (i.e., it is difficult to assess benefit).
• Children may be long-term device users, so device longevity and exposure to implanted materials must be taken into consideration.
• There is a relatively low return on investment because of the small market size or sample size; companies often avoid taking on the risk of developing devices for this population.

Development of medical devices for children continues to lag compared to development for adults. Some products are designed specifically for children while others are “borrowed” from adult applications or produced for more general use. From 2008 to 2017, the number of medical devices approved for adults grew at a rate that was three to four times higher per year than that for PMDs. Most importantly, there was no significant overall change in the number of PMDs or the percentage of PMDs during that 10-year period. The number of FDA-approved medical devices with pediatric labeling remained stagnant through 2020, with the lowest percentage of approvals affecting the youngest patients.

Since 2005, the following federal efforts aimed to increase the number of approved PMDs:

• 2005: Publication of the Institute of Medicine’s Safe Medical Devices for Children
• 2007: PMD Safety and Improvement Act (in the FDA Amendments Act of 2007)
• 2012: FDA Safety and Innovation Act
• 2016: 21st Century Cures Act
• 2017: Research to Accelerate Cures and Equity (RACE) for Children Act
• 2018: FDA meeting on PMDs
• 2018: Launch of N-PeRC
• 2019: Critical Path Institute System of Hospitals for Innovation in Pediatrics–Medical Devices (SHIP-MD) workshop
• 2021: SHIP-MD public meeting
• 2021: Formation of the N-PeRC PMD Subgroup
• 2022: FDA Reauthorization Act

Despite these efforts, however, the lack of PMDs remains a problem. This issue led NIH to begin focusing on the development of PMDs. Leveraging SHIP-MD to more effectively focus NIH opportunities to promote innovation and PMD product development (and to create a PMD ecosystem) seemed to be a viable option. At a PMD stakeholder meeting in early 2022, Dr. Bianchi outlined the following points:

• The lack of medical devices specifically designed or studied to address the needs of pediatric patients represents a persistent and major public health problem.
• The underlying challenges impose the need to establish transdisciplinary, multistakeholder collaborative platforms for the development of reasonable solutions.
• Coordination of national multistakeholder efforts through a PPP could be a significant change in the pediatric medical device arena.

From 2021 to 2023, NICHD collaborated with FNIH to gather public feedback and plan the program. In September 2023, NICHD launched the design phase of the PMD PPP . The project seeks to streamline processes that may bolster investments in PMD innovation through multistakeholder interactions and establishment of a self-sustaining entity to manage PMD in the public trust. Its six workstreams are navigation, hospitals, decentralized clinical trials, reimbursement, regulatory, and finance. The objective is to collaborate within the FNIH framework to design and implement sustainable PMD partnerships.

The PMD PPP partners that FNIH is convening for the design phase of the project include:

• Public: NIH (NICHD; NIBIB; NCI; NIMH; the National Center for Advancing Translational Sciences; the National Heart, Lung, and Blood Institute; the National Institute of Arthritis and Musculoskeletal and Skin Diseases; the Biomedical Advanced Research and Development Authority (BARDA); the Division of Research, Innovation, and Ventures (DRIVe); and the FDA Center for Devices and Radiological Health (CDRH)
• Private: AdvaMed, the American Academy of Pediatrics, Berlin Heart, Children’s National Hospital, Children’s Hospital of Orange County, CobiCure, the Charles H. Hood Foundation, Johnson & Johnson, Lurie Children’s Hospital, MedStar Research Institute, OrthoPediatrics, Philips Healthcare, Texas A&M, and Veeva MedTech
• FNIH: Harvard-MIT Center for Regulatory Science

In terms of the current project timeline, plans for 2024 include a virtual stakeholder meeting in April, an in-person design phase meeting in June, and the publication of an operational plan white paper in the third quarter.

Overall, the PMD PPP is a multistakeholder venue to advance the national PMD ecosystem.

Discussion (2:00:03)

A board member said they were excited about the PMD PPP. They asked why the National Science Foundation (NSF) was not included as a partner, noting that NSF has a new director for the Technology, Innovation and Partnerships program and that NSF is a partner in the Building Reliable Advances and Innovations in Neurotechnology (BRAIN) industry/university cooperative research center. The board member noted the need to educate people in regulatory science, including the development of tools for pediatric regulatory science, to avoid having a shortage of specialists. They asked about synergies between PMD PPP and the NIH Blueprint MedTech.

Regarding NSF, Dr. Pileggi said that PMD PPP is hoping to add more public and private partners, and interest in the program is growing as it builds momentum. Dr. Pileggi said that he agrees that more focus is needed for pediatric regulatory sciences; he is working with FDA to increase the workforce (including consultants) and add expertise for the pediatric patient population. Blueprint MedTech has developed many valuable resources, Dr. Pileggi said, so PMD PPP will use rather than duplicate those assets. The program will also continue integrating other existing resources, including mentoring, consulting, and marketing assistance, to move ideas to patients as quickly as possible.

A board member suggested adding FDA class (I, II, III) to Dr. Pileggi’s regulatory slides to help identify opportunities between the different classes of devices. Dr. Pileggi agreed, noting that there are a broad range of ages and a broad range of devices (e.g., algorithms to implantable devices) across the FDA device classification system. He added that FDA expertise, access, and guidance are needed to help innovators working on PMDs.

The board member asked whether PMD PPP would intersect with the SBIR program in future phases. Dr. Pileggi said that the type of infrastructure developed for PMD PPP was still being discussed and designed, but it should eventually be able to help gather evidence and provide resources to shorten the time to FDA approval and time to market for SBIR grantees. The goal is to help innovators at all stages of product development.

A board member asked for clarification on whether there was a lack of PMD innovation or if newly invented devices were not making it through the FDA approval pipeline. Dr. Pileggi said that the problem is multifold: Some innovators do not include regulatory pathway and reimbursement planning in the early stages of the product development process; FDA device pathways are not as advanced as drug approval pathways, so a shift in culture is needed; and the device ecosystem has not been united to share resources and build collective strength.

The board member then asked how the PMD PPP white paper would be disseminated. Dr. Pileggi said that the white paper will be an internal document used to develop a blueprint for building the PMD PPP infrastructure.

The board member said that the Children’s Hospital of Philadelphia has an innovation center and asked how that type of institute could become involved in the PPP. Dr. Pileggi said that such institutes could engage with FNIH now or compete for the PMD PPP’s eventual infrastructure grants.

Updates from ARPA-H (2:16:02)

Calvin Roberts, M.D., Program Manager, Health Science Futures, ARPA-H

Dr. Roberts provided an overview of ARPA-H, shared some of its current programs and projects, and described one of the “hard problem” programs that he is managing: whole eye transplantation. The creation of ARPA-H was inspired by the success of the Defense Advanced Research Projects Agency. The mission of ARPA-H is to accelerate better health outcomes for everyone.

ARPA-H is a funding agency. It is an independent component of the U.S. Department of Health and Human Services (HHS) within NIH; it is not one of the ICs. The ARPA-H director reports directly to the HHS secretary. ARPA-H has no internal research laboratories and is disease-agnostic. It is independently funded by $2.5 billion in appropriations. ARPA-H funds are awarded via outcomes-based contracts (not grants) with accelerated timelines. The agency is more concerned with the development side of research and development (as opposed to NIH, which is more concerned with the research side). The key to ARPA-H is in its bottom-up, problem-centric approach to address the toughest challenges in health.

ARPA-H has a lean, nimble management structure with an autonomic decision-making process. Term limits of 3 to 6 years for all program managers bring urgency and idea flow. Flexibility in hiring allows ARPA-H to recruit at levels competitive with industry. “Bottom-up decision-making” means that visionary program managers with management and research experience have autonomy to make decisions quickly and inspire radical change through high-risk, high-payoff projects. ARPA-H is a problem-focused organization. Its success is measured by real-world impact.

Program managers identify difficult health-related challenges that are ripe for solving, then seek and oversee several groups of performers working  to solve the same problem in unique ways. Performers compete to carry out their potential innovative solutions and win the challenge. Beyond the contracts that have well-defined endpoints, performers are provided with additional support from program managers, partners, and ARPA-H offices to ensure the best chance of success throughout the process. Performers who do not meet their milestones are eliminated from the challenge, and resources are reallocated. Lessons learned from each project are collected and shared.

ARPA-H is divided into four offices:

• The Health Science Futures Office seeks to expand what is technically possible. Its goal is to accelerate advances across research areas and remove limitations that stymie progress toward solutions. Its tools and platforms apply to a broad range of diseases.
• The Scalable Solutions Office seeks to reach everyone quickly. Its goal is to address health challenges—including geography, distribution, manufacturing, data and information, and economies of scale—to create programs that result in impactful, timely, and equitable solutions.
• The Proactive Health Office seeks to keep people from being patients. Its goal is to develop preventive programs that create new capabilities to detect and characterize disease risk and promote treatments and behaviors to anticipate threats to Americans’ health, whether those are viral, bacterial, chemical, physical, or psychological.
• The Resilient Systems Office seeks to build integrated health care systems. Its goal is to create capabilities, business models, and integrations to weather crises such as pandemics, social disruption, climate change, and economic instability. Its systems are sustained between crises—from the molecular to the societal—to achieve better health outcomes.

Program managers develop the programs within ARPA-H, and external groups can propose projects to receive ARPA-H funding. The following programs are currently underway:

• Novel Innovations for Tissue Regeneration in Osteoarthritis (NITRO) seeks to eradicate osteoarthritis through targeted regenerative therapeutics that aim to revolutionize care, prevent pain, lessen economic burden, and eliminate the need for repeat joint surgery.
• Precision Surgical Interventions (PSI) seeks to develop technologies that will be used by surgeons during operations to visualize tumors’ edges to increase chances of complete removal, and to develop devices that surgeons can use to clearly see critical anatomy in 3D while operating.
• Antigens Predicted for Broad Viral Efficacy through Computational Experimentation (APECx) seeks to transform vaccine antigen discovery, first by developing toolkits that successfully design broadly effective antigens, targeting entire viral families, and then by demonstrating the toolkits’ accuracy by evaluating candidate vaccine antigens in clinical trials.
• Personalized Regenerative Immunocompetent Nanotechnology Tissue (PRINT) seeks to use state-of-the-art bioprinting technology to 3D-print personalized-on-demand organs, such as kidneys, hearts, and livers, which do not require immunosuppressive drugs.
• Lymphatic Imaging, Genomics, and pHenotyping Technologies (LIGHT) seeks to create a scalable, accessible, accurate, and clinically useful diagnostic toolkit to detect lymphatic structure and function in health and disease.
• Transplantation of Human Eye Allografts (THEA) seeks to enable whole eye transplantation to restore vision for the blind and visually impaired.

THEA is the program Dr. Roberts manages. It proposes that curing blindness could advance health solutions for all. Transplanting an eye would mean transplanting the optic nerve, which would provide a cure for blindness and could also lead to therapies for other cranial nerve problems. Corneal transplants are now common, with more than 70,000 people donating their eyes for transplantation each year. THEA proposes a surgical solution, with unique hurdles. The three technical areas for THEA are tissue harvest and preservation, optic nerve reattachment and repair, and surgery, postoperative care, and assessment. THEA performers will be selected in July 2024, and the program’s kickoff will be in September.

A new ARPA-H project is the Sprint for Women’s Health. ARPA-H is committing $100 million to galvanize the innovator, investor, researcher, and patient advocate communities to spur innovation in women’s health. The Sprint for Women’s Health’s new request for solutions funding opportunity seeks unconventional approaches and innovative new avenues to push high-impact biomedical research forward to improve women’s health outcomes. The six topic areas that have been identified can be found on the ARPA-H website .

Dr. Roberts closed with a quote from Alicia Eggert’s art installation at the Renwick Gallery in Washington, DC: “This present moment used to be the unimaginable future.”

Discussion (2:46:05)

A board member said that it was exciting to learn about ARPA-H and its definition of radical change. They asked whether the length of a project proposal should be 3 years to match the length of a program manager’s term. Dr. Roberts said no, because the lengths of time between programs and program managers are not synchronous. Dr. Roberts’ programs will not end when his time is up; they will be passed on to another program manager. Each program has a different timeline and different project lengths.

Another board member asked whether each ARPA-H program was created within a certain budget. Dr. Roberts said that each program manager proposes the budget for their program. There is no budget limit for any proposed idea; the ARPA-H director sets the final budget when accepting a proposal.

A board member asked whether team science was important to ARPA-H. Dr. Roberts said the key to ARPA-H is that each problem is too big for any one investigator or institution. Therefore, the only means to success is to form a team. Proposers’ Day provides a venue for teams to form. Teams are critical in ARPA-H.

The board member then asked how ARPA-H handled data sharing. Dr. Roberts said that data sharing is another critical area of ARPA-H, so the agency does have platforms for that.

NIH Research Plan on Rehabilitation RFI Results (2:52:14)

Theresa H. Cruz, Ph.D., Director, NCMRR

On February 12, 2024, on behalf of the NIH Medical Rehabilitation Coordinating Committee, NCMRR released an RFI inviting comments and suggestions to guide the drafting of the next NIH Research Plan on Rehabilitation. The RFI requested input on the community’s perception of progress toward achieving the priorities specified in the previous research plans, potential priorities or areas of research to consider in the next research plan, current priorities that should be retained and continued, emerging areas of science that will affect the research plan on rehabilitation, and potential focus areas for concentrated work in medical rehabilitation research.

Of the 24 responses received, 16 were from individuals, 5 were from special interest groups, and 3 were from research institutions. One notable finding was that the second of five prompts, “Potential priorities or areas of research to consider in the next research plan,” was most referenced by respondents. The following key themes emerged when analyzing responses:

• K12 funding
• The importance of genetic studies
• Health disparities and SDOH
• The role of basic science and experimental studies involving humans to inform treatment
• Technology, sensors, telehealth, technology transfer
• Location (low- and middle-income countries, acute care, intensive care units, skilled nursing facilities)
• Collaboration/knowledge translation (World Health Organization; National Institute on Disability, Independent Living, and Rehabilitation Research)
• Secondary conditions (cardiovascular health, physical activity, medication adherence, pressure injuries, functional cognition)
• Disability Research Office

Next steps for the NIH Research Plan on Rehabilitation include drafting the plan this summer, with target draft completion set for August or September; engaging with the working group for feedback in September or October; and reporting out to the full board at the December NABMRR meeting.

NCMRR will hold its next rehabilitation conference on March 10–11, 2025, at the NIH Natcher Conference Center. The meeting will be a hybrid event with a maximum in-person capacity of 500. The planning committee (an NIH internal working group) is accepting suggestions for speakers, topics, and formats. External working group members include Drs. Dorn, Ehde, Ehrlich-Jones,  and Wheaton.

Discussion (3:03:11)

Dr. Cruz asked NABMRR members to share their reactions to the RFI, noting whether they were surprised by any of the responses, pointing out anything that was missing, sharing what purpose the plan should serve, and so forth.

A board member said they were encourged to see the location suggestions, because they are passionate about providing rehabilitation in nonhospital settings. They added that “Rehabilitation for All” includes low-resource settings.

The board member also said they were surprised to see the Office of Disability Research suggestion and wondered how it might differ from the current ICs. Dr. Cruz said that an Office of Disability Research would be located within the OD. If created, its mission would be to coordinate NIH-wide efforts for disability research.

A board member said that 24 responses may not serve as a representative sample. Dr. Cruz said that she will solicit additional responses at road show presentations and conferences.

Another board member said they were happy to see “translation from basic research” on the list but surprised to see that some basic science and experimental studies involved humans. They added that translation from animal models can often be controlled all the way into the clinic, and they are important for mechanistic studies (especially in neurorehabilitation). Keeping up with technologies relies on understanding the underlying mechanisms. Dr. Cruz said that translational research is one of the themes in the current plan, so it might be good to retain it.

A board member appreciated the list of nine key themes and suggested adding “learning and applying knowledge” from one area to another area on the list (i.e., developing a method to share and implement rehabilitation learnings with other areas of medicine). Dr. Cruz agreed that implementation of knowledge is an important area. Another board member agreed, saying that implementation science was a hot topic at their clinic. A board member added another vote for implementation science and said that there are synergies between implementation science and engaging with and gathering lived experiences from research participants. Another board member said that implementation science is important, but an educational component or continuing medical education (CME) is what is needed in rehabilitation. Dr. Cruz said that engaging with professional societies that provide CME could be considered.

A board member said they were pleased to see cardiovascular health on the list, because the level of disability associated with cardiopulmonary diseases is quite high. They commended Dr. Cruz for taking on this project.

A board member said they would add “understanding and addressing the impact of comorbidities” to the list of secondary conditions. They also advocated for providing rehabilitation earlier. Another board member added that they work with HIV and stroke as comorbidities of rehabilitation.

A board member asked for clarification on the timing of the NIH Plan for Rehabilitation Research. Dr. Cruz said that the plan is published every 5 years. It was last published in 2021 (and before that in 2016), so the next version will go out in 2026.

A board member said they supported SDOH and the issue of reproducibility of interventions across populations.

A board member said that the plan could articulate the “Rehabilitation for All” visionary theme with practical strategies for realizing that vision as sections of the plan. The plan would then essentially mirror the approach that NCMRR takes for most of its programming.

Dr. Cruz encouraged NABMRR members to submit additional comments to her via email.

Comments From Retiring Board Members (3:33:16)

Barbara S. Bregman, Ph.D., PT, Chair

Dr. Bregman thanked Dr. Cruz and Dr. Nitkin for planning such a great 2-day board meeting. She added her gratitude for the sign-language interpreters and other support staff. After thanking each of them for their friendship and service on the NABMRR, Dr. Bregman invited Dr. Hammond, Dr. Blauwet, and Dr. Shrader to share their parting comments.

Flora Hammond, M.D. (3:35:00)

I appreciate the honor and opportunity to serve on the board. I’m leaving the board having gained so much awareness. It will be useful for me as a researcher and department chair. I hope I have also been able to give back to the board. I leave incredibly inspired by the colleagues I have met on the board, as well as by Dr. Nitkin and Dr. Cruz. Thank you so much.

Cheri Blauwet, M.D. (3:35:52)

Thank you for this opportunity. I have easily learned and gained more than I gave over the last several years. I left every meeting a little bit smarter and a little more informed, and was inspired by the knowledge and depth of experience and commitment to rehab that has been shown across the entirety of the board. It has been an honor to be involved in the critically important process of advising on the development of research priorities and programs that impact so many Americans with disabilities. It has been wonderful to see our progress over the last several years as it relates to thinking about concepts of ableism and ensuring an anti-ableist approach to our research that is supported through NIH. We saw significant milestones, like the designation of people with disabilities as a health disparities population in 2023. Those changes and that progress is going to have a huge impact into the future. It has been a real honor, and I also wanted to specifically thank Dr. Cruz and Dr. Nitkin for your partnership and collaboration. Thank you, everyone.

Wade Shrader, M.D. (3:37:27)

Thank you all so much. Dr. Bregman, thank you for being our chair this year. Thank you, Dr. Nitkin and Dr. Cruz, for your amazing leadership. It truly has been an honor. I have learned so much, and I have made so many connections. I have learned more about NIH and all of the different organizations. I feel honored and privileged to have had input into our research priorities and research trajectories. Being on the Board has helped me grow organizations like AACPDM. It has been one of the highlights of my career, and I thank you all from the bottom of my heart.

Final Summary and Planning for the Next Meeting (3:38:20)

Barbara S. Bregman, Ph.D., PT, Chair

Dr. Bregman asked NABMRR members to share any potential topics for future NABMRR meetings. Board members made the following suggestions:

• Working group readouts
• More information on ReproRehab
• Implementation science
• Scientific presentations from outgoing NABMRR members
• Update on the implementation of NIH’s new grant review process
• AI in rehabilitation, large language models and their applications
• Return to work
• Decentralized trials

Adjournment (3:41:35)

Barbara S. Bregman, Ph.D., PT, Chair
Theresa H. Cruz, Ph.D., Director, NCMRR
Ralph Nitkin, Ph.D., Deputy Director, NCMRR

Dr. Bregman thanked the members of NABMRR for their thoughtful participation. Dr. Cruz thanked Dr. Bregman for leading the meeting. She also thanked Dr. Nitkin, the board members, speakers, and support staff. Dr. Nitkin said that the larger audience that watches the meetings includes families, researchers, and other interested parties. He welcomed input from any interested party.

Dr. Nitkin adjourned the meeting at 2:13 p.m. ET.