NATIONAL BLEEDING DISORDERS FOUNDATION

COMMUNITY SERVICES NBDF'S PUBLIC POLICY DEPARTMENT WORKS TO ADVOCATE FOR POLICIES THAT PROMOTE HEALTH, SAFETY, RIGHTS AND ACCESS TO CARE FOR PEOPLE WITH INHERITABLE BLOOD DISORDERS BY EDUCATING FEDERAL AND STATE LAWMAKERS, OTHER GOVERNMENT AGENCIES AND OFFICIALS, AS WELL AS INDUSTRY AND ALLIED HEALTH EDUCATION AND TRAINING ORGANIZATIONS. TWO KEY AREAS OF FOCUS ARE PAYER AND CONSUMER EDUCATION, AND SELF-ADVOCACY.THE PUBLIC POLICY DEPARTMENT PROVIDES TRAINING, TOOLS, AND HANDS-ON SUPPORT TO CONSUMERS TO HELP THEM BECOME EFFECTIVE SELF-ADVOCATES.THROUGH THE STATE-BASED ADVOCACY PROGRAM, NBDF AWARDS GRANTS TO CHAPTERS, COMPETITIVELY, TO SUPPORT THEIR ADVOCACY EFFORTS. SUCCESSFUL GRANT RECIPIENTS ARE REQUIRED TO ATTEND SEVERAL VIRTUAL OR IN-PERSON TRAINING EVENTS ANNUALLY, HOLD REGULAR ADVOCACY COMMITTEE MEETINGS, INCLUDE NBDF STAFF IN STATE ADVOCACY EVENTS, PROVIDE A MIDYEAR PROGRESS REPORT, COLLECT, SUBMIT SPECIFIED PROGRAM METRICS QUARTERLY, HOST A STAKEHOLDER PLANNING MEETING AND SUBMIT A FINAL REPORT AT YEAR'S END. THIS PROGRAM IS NAMED THE STATE BASED ADVOCACY COALITION (SBAC) PROGRAM. GRANTS ARE ISSUED ON AN ANNUAL BASIS, WITH CHAPTERS APPLYING EACH DECEMBER. ALL CHAPTERS ARE ELIGIBLE TO APPLY FOR FINANCIAL AND PROGRAMMATIC SUPPORT.MEMBERS OF THE INHERITABLE BLOOD DISORDERS COMMUNITY THROUGHOUT THE COUNTRY ARE ENCOURAGED TO PARTICIPATE IN NBDF'S ANNUAL WASHINGTON DAYS PROGRAM, WHICH IS A GRASSROOTS ADVOCACY EVENT THAT BRINGS PATIENTS AND THEIR FAMILIES TO WASHINGTON, DC TO MEET WITH THEIR MEMBERS OF CONGRESS AND EDUCATE THEM ABOUT THE DISORDERS IMPACTING THE COMMUNITY. THIS EVENT TOOK PLACE IN EARLY MARCH. CONSUMERS ARE ALSO ENCOURAGED TO ATTEND STATE ADVOCACY DAYS AT THEIR LOCAL STATE CAPITAL. NBDF ALSO PROVIDES EDUCATIONAL OPPORTUNITIES FOR PAYERS TO HELP THEM BETTER UNDERSTAND THE UNIQUE HEALTHCARE NEEDS OF THOSE AFFECTED BY BLOOD DISORDERS, INCLUDING LIVE PRESENTATIONS, WEBINARS, ONLINE EDUCATIONAL MODULES, AND A JOINT COLLABORATIVE BRINGING TOGETHER PAYERS, MEDICAL PROVIDERS AND PATIENTS. NBDF HOSTS ABOUT 25-30 STATE ADVOCACY DAYS PER YEAR.

RESEARCHNATIONAL RESEARCH BLUEPRINT (NRB): A COMMUNITYWIDE PROJECT TO REDEFINE THE WAY RESEARCH IN THE BLEEDING DISORDERS COMMUNITY WAS LAUNCHED IN 2019. THE GOALS OF THE NATIONAL RESEARCH BLUEPRINT (NRB) ARE: (1) DEVELOP A MULTIDISCIPLINARY INTEGRATED RESEARCH ENTERPRISE CENTERED IN A NETWORK OF BOTH SPECIALTY AND COMMUNITY-BASED CARE; DRIVEN BY PATIENTS WITH INHERITED BLEEDING DISORDERS (PWIBD) AS LIVED EXPERIENCE EXPERTS (LEES); EMBEDDED IN THE PRINCIPLES OF SOCIAL JUSTICE; TO STEADILY ADVANCE THE STANDARD OF CARE FOR PWIBDS THROUGH IMPACTFUL BASIC, TRANSLATIONAL, CLINICAL, HEALTH OUTCOMES, AND IMPLEMENTATION RESEARCH, ADVOCACY AND EDUCATION; (2) EXPAND THE NATIONAL RESEARCH INFRASTRUCTURE TO SUPPORT THE ENVISIONED RESEARCH ENTERPRISE THROUGH FACILITATION COLLABORATION AND INCLUSION, AND (3) REINVIGORATE A SUSTAINABLE WORKFORCE ACROSS COMPREHENSIVE CARE AND SCIENTIFIC DISCIPLINES THAT INCORPORATES THE LEE PERSPECTIVE TO ADVANCE HEALTH THROUGH THE SEAMLESS INTEGRATION OF CARE AND RESEARCH AS WELL AS ACTIVE COMMUNITY ENGAGEMENT TO FOSTER A RESEARCH CULTURE. IN 2024, NBDF CONVENED KEY STAKEHOLDERS IN A NRB SUMMIT TO PRESENT THE FRAMEWORK AS DEFINED BY ALL WORKING GROUPS. AS A RESULT, FIVE MANUSCRIPTS DESCRIBING THE PROCESS ARE IN PROGRESS, WITH AN ESTIMATED SUBMISSION DATE DURING THE SUMMER OF 2025. THE RESEARCH TEAM PRESENTED IN NUMEROUS SPEAKING ENGAGEMENTS NATIONALLY TO CREATE AWARENESS AND ESTABLISH PARTNERSHIPS. COMMUNITY VOICES IN RESEARCH (CVR): CVR IS A COMMUNITY-POWERED REGISTRY INTENDED TO CAPTURE THE EXPERIENCE OF LIVING WITH A BLEEDING DISORDER DIRECTLY FROM THOSE AFFECTED AND THEIR IMMEDIATE RELATIVES. IT PROVIDES AN INTIMATE LOOK OF THE LIVED EXPERIENCE AND AIMS TO UNDERSTAND AND IMPROVE KEY ASPECTS OF HEALTH-RELATED QUALITY OF LIFE (QOL), IDENTIFY RESEARCH QUESTIONS IMPORTANT TO COMMUNITY MEMBERS, AND PROVIDE VETTED RESOURCES AND OTHER RESEARCH OPPORTUNITIES TO PARTICIPANTS. AN IMPROVED VERSION OF THE REGISTRY WAS LAUNCHED IN SEPTEMBER OF 2023. NUMEROUS PRESENTATIONS AND ABSTRACTS WERE PRESENTED AND A MANUSCRIPT DETAILING ITS DEVELOPMENT WAS PUBLISHED IN A PEER REVIEWED JOURNAL.2024 CVR HIGHLIGHTS: - NBDF HAS A MULTIPRONG COMPREHENSIVE ENROLLMENT PLAN. CURRENT ENROLLMENT IS OVER 1,000 PARTICIPANTS. - THE ENROLLMENT, BASELINE SURVEY AND PARTICIPANT DASHBOARD ARE NOW IN BOTH ENGLISH AND SPANISH. VIRTUAL ADVISORY PANELS (VAPS) AND IN PERSON ADVISORY PANELS (PAPS): VAPS AND PAPS ARE VIRTUAL AND IN-PERSON FOCUS GROUPS. FOUR VAPS WERE FACILITATED IN 2024. A TOTAL OF 24 ADVISORS PARTICIPATED. INFORMATION GATHERED PROVIDED PATIENT/CAREGIVER INSIGHTS ON EDUCATIONAL RESOURCES, HEALTH LITERACY, CLINICAL TRIAL DESIGN, IDENTIFYING DISPARITIES AND GAPS IN CARE. RESEARCH JOURNAL CLUB: THE VIRTUAL RESEARCH JOURNAL CLUB WAS OPEN TO THE COMMUNITY, RESEARCHERS, CLINICIANS, AND CHAPTERS. IN 2024 NBDF HELD TWO SESSIONS.2024 NBDF RESEARCH GRANTS AWARDS: JUDITH GRAHAM POOL POSTDOCTORAL RESEARCH (JGP) - DR. HUONG CHAU AT STANFORD: ELUCIDATING INNATE IMMUNE RESPONSE TO FACTOR IX THROUGH HEMOPHILIA B MOUSE MODEL - DR. MARISSA BRAKE, BETH ISRAEL MEDICAL CENTER: THE ROLE OF TISSUE FACTOR IN BLOOD COAGULATION ACTIVATION AND BLEEDING RISK IN MICE AND HUMANSCAREER DEVELOPMENT AWARD: (CDA) FUNDED BY SANOFI - DR. BHAVYA DOSHI- CHOP TRANSFERRING TO EMORY 2025: UNRAVELING THE CYTOKINE AND CELLULAR IMMUNE RESPONSES UNDERLYING FVIII IMMUNOGENICITYEXCELLENCE AWARDS FOR NURSING, SOCIAL WORK, & PHYSICAL THERAPY: - NURSING: LOUISE BACA/MAINE HEALTH: OUTREACH IN RURAL MAINE: IDENTIFYING NEW PATIENTS AND ESTABLISHING SATELLITE SITES. - PHYSICAL THERAPY: JOHN DELOACH/ U OF FLORIDA: ASSESSING EFFECTS OF EXERCISE ON VASCULAR ACCESS, TRYPANOPHOBIA, AND KNESIOPHOBIA IN HEMOPHILIA PATIENTS. - SOCIAL WORK: KYELIN COOK/INTERMOUNTAIN HEALTHCARE FOUNDATION: (CONTRACTING PENDING) ADDRESSING GAPS IN CARE AMONG MENSTRUATING INDIVIDUALS IN UTAHNBDF-TAKEDA CLINICAL FELLOW DR. CALLIE BERKOWITZ, MD, UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL WAS SELECTED TO RECEIVE MENTORED-TRAINING IN 2024-2026COREHEM MENTAL HEALTH TOOL: DEVELOPED THROUGH AN INITIATIVE CO-LED BY NBDF, COREHEM MENTAL HEALTH OUTLOOK QUESTIONNAIRE (COREHEM-MHO) IS A PATIENT-REPORTED OUTCOME MEASUREMENT (PROM) INSTRUMENT THAT ASSESSES MENTAL HEALTH OUTLOOK (INCLUDING PSYCHOLOGICAL STATUS AND EMOTIONAL FUNCTIONING) ASSOCIATED WITH RECEIVING GENE THERAPY OR ANY DURABLE TREATMENT FOR HEMOPHILIA. THE COREHEM-MHO INSTRUMENT IS OWNED BY NBDF AND AVAILABLE FOR USE IN RESEARCH AND WITHIN CLINICAL TRIAL SETTINGS THROUGH A LICENSE AGREEMENT. THE PROJECT HAS 12 PEER-REVIEWED PUBLICATIONS TO DATE AND HAS BEEN PRESENTED AT TENS OF NATIONAL AND INTERNATIONAL MEDICAL CONFERENCES. WE ARE CURRENTLY COLLABORATING WITH 4 PARTNERS WHO ARE USING THE TOOL IN CLINICAL TRIALS AND PRACTICE.