Day 1: NORD Centres of Excellence - Pamela Gavin
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CORD Rare Drug Conference: June 8-9, 2022 Global, International, and National Rare Disease Networks NORD Centres of Excellence - Pamela Gavin, NORD
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Day 1: NORD Centres of Excellence - Pamela Gavin
- 1. National Organization for Rare Disorders | rarediseases.org NORD RARE DISEASE CENTERS OF EXCELLENCE PROGRAM CORD Conference June 8, 2022 Pam Gavin, Executive Vice-President Marybeth McAfee, MA, GC Associate Director NORD RD CoE Program
- 2. NORD, a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 330 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. NORD® MISSION STATEMENT rarediseases.org 2
- 3. 3 rarediseases.org NORD Rare Disease Centers of Excellence On Nov 4th 2021, NORD announced its designation of 31 medical institutions across the United States with exceptional programs for patients with rare diseases as NORD Rare Disease Centers of Excellence (NORD RD CoE’s).
- 4. 4 rarediseases.org NORD RD CoE Program Vision All persons living with a rare disease, regardless of disease, socioeconomic level, or demographics, have access to timely diagnosis, quality, compassionate clinical care, research opportunities, and supportive resources. • Policy: Advocating for policies that increase affordable access to diagnosis and care. • Professional/Patient Education: Fostering a pipeline of rare disease clinical providers and researchers; Empowering patients and families to take an active role in their healthcare decisions. • Research: educating and advancing broader research collaboration and inclusion across the rare disease community. • Care: Cultivating collaboration across the network and beyond to facilitate quality care, holistic experiences, and sharing of guidelines for the dx and tx of rare and undiagnosed diseases.
- 5. 5 rarediseases.org Building the Application • A framework for the application was developed • Committee refined the application • Scoring rubric developed
- 6. 6 rarediseases.org Application Overview – Multiple Choice Questions • Slightly over 100 Multiple Choice Questions • Qualitatively assessing each site’s “Onsite” or “Local” access to specific staffing, facilities, or services • Quantitative collection of “clinical fulltime equivalents” (cFTE’s) in key specialties very often involved in rare disease diagnosis and care. • Examples: • Liver transplant service • Pediatric heart surgery • Disease patient “navigators” and/or case managers • Specialty laboratories for esoteric rare disease diagnostic or monitoring tests, etc. • # of Genetic Counselors, Clinical Geneticists, Biochemical or Molecular Geneticists, Pediatric Neurologists
- 7. 7 rarediseases.org Application Overview – Essay Questions & LOS 10 Essay Questions • 750-word limit • Answers to focus on rare disease • Rubric for scoring was included Letters of Support • Ranged from 1-4 • Examples: • Multidisciplinary clinics • Types of rare disease certified clinical training programs • Rare disease patient resources • Programs to reach underserved • Pediatric to adult transition • Patient satisfaction scores
- 8. 8 rarediseases.org Applications Process Invitations • Invitations were sent to the 40 plus Institutions • Directed to head of clinical genetics programs • Had ACGME certified clinical training programs Technical Assessment Call • Program information • Application information • Explanation of rubrics • Q&A
- 9. 9 rarediseases.org Analyzing the Applications Multiple choice questions: • Pulled from application system • Did little to differentiate between applicants Essay questions: • Scored using rubric by 3 different NORD staff • Each staff had 8 or so applicants essay answers to review • Letters of Support were read but were not scored or included in scoring rubric
- 10. 10 rarediseases.org 31 NORD RD CoE • University of Alabama at Birmingham Medicine/Children’s of Alabama • Children’s Hospital of Orange County / UC Irvine • University of California, San Francisco & UCSF Benioff Children's Hospitals • University of Colorado Anschutz Medical Campus/Children’s Hospital Colorado • University of Miami Miller School of Medicine • Emory Division of Medical Genetics • Indiana University School of Medicine • University of Iowa Health Care • Johns Hopkins Medicine/Kennedy Krieger Institute • Harvard Medical School Affiliated Hospitals – NORD Center of Excellence for Rare Disorders • Mayo Clinic • M Health Fairview-University of Minnesota Masonic Children's Hospital • Washington University/BJC Healthcare • UNMC Munroe-Meyer Institute, Omaha Children's Hospital and Nebraska Medicine • Columbia University Irving Medical Center • Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai • Duke University Medical Center • UNC Children's - North Carolina Children's Hospital • Cincinnati Children's Hospital Medical Center • Nationwide Children's Hospital/Ohio State University • Oklahoma Children’s Hospital OU Health/University of Oklahoma Health Sciences Center • Penn Medicine/Children’s Hospital of Philadelphia • UPMC Center for Rare Disease Therapy • Vanderbilt University Medical Center • Baylor College of Medicine/Baylor St. Luke’s Medical Center/Texas Children’s Hospital • McGovern Medical School/UTHeath • UT Southwestern Medical Center • University of Utah Medical Genetics • Rare Disease Institute at Children’s National Hospital • Children's Wisconsin/Medical College of Wisconsin • University of Wisconsin Center for Rare Diseases
- 11. 11 rarediseases.org Geographic Distribution vs. Population Density
- 12. 12 rarediseases.org Revising the Application Multiple Choice Questions • Retain most of the questions • Consolidate some of specialist questions • Remove redundancies • Increase clearness of questions Essay Question • Analyzing reviewers’ comments (2021 cycle) • Reconsider 750-word limit • Refine rubric • Modify weighting
- 13. 13 rarediseases.org A Few Thoughts to Consider • Try to envision situations where worthy applicants will apply that won’t meet the designation criteria • Independent children’s hospital • Independent specialty hospitals • Come up with strategies to mitigate loss of quality applicants • Support working with a hospital that treats adults to put in a joint application • Support working with larger university hospital to put in a joint application • Know ahead of time what you want to analyze to distinguish your vision of a rare disease center of excellence • Consider supporting co-director structure rather than single director at each Center
- 14. 14 rarediseases.org Addressing Unmet Needs for Rare Diseases CONFIDENTIAL
- 15. 15 rarediseases.org NORD RD CoE Working Groups CARE: • Tx Guideline Curation & Development Workgroup • Diagnostic Approaches / Access to Testing Workgroup • Clinical Application of Newly Approved Treatments Workgroup RESEARCH: • Facilitating Multi-site Research Studies & Clinical Trials Workgroup • Shared Data Collection & Analysis Workgroup EDUCATION: • Patient Education Workgroup • Training, CME, and Career Development Workgroup POLICY: • Telemedicine / Inter-site Consultation Workgroup • Diversity, Equity, and Inclusion Workgroup • Public Policy & Collective Advocacy Workgroup
- 16. 16 rarediseases.org Progress Since Launch • Onboarding with all 31 sites • Initial meetings with Center Directors • Establishment of Working Groups • Initial meetings with Working Groups have begun with over 200 attendees Next Steps: • Working group assessments and plans • Network Directors meet to review and confirm work plans CONFIDENTIAL
- 17. 17 rarediseases.org Marybeth McAfee, MA, GC mmcafee@raredisease.org Thank you.