Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Figures & data

Table 1. Components of the Hemophilia Treatment Center and its comprehensive care team [20–23].

Core HTC team
	Hematologist
	Nurse coordinator
	Physical therapist
	Social worker
	Laboratory personnel
	Pharmacist
	Data manager
Extended HTC team*
	Orthopedic surgeon
	Dentist or dental hygienist
	Psychologist
	Genetic counselor
	Nutritionist
	Obstetrician/gynecologist
	Infectious disease specialist
	Hepatologist
HTC activities and services^†
	Integrated and comprehensive diagnosis and treatment of PWIBD
	Education for PWIBD and their families
	Counseling (e.g. genetic, behavioral health, and financial)
	Surveillance
	Outreach clinics (physical and virtual)
	Outpatient pharmacy services
	Standardized specialized coagulation laboratory
	Standardized specialized blood bank
	Advocacy
	HCP education and training
	Research

*The extended HTC team may include any combination of these professionals, and possibly others

^†Different HTCs feature different subsets of these activities and services

HCP: health care professional, HTC: Hemophilia Treatment Center, PWIBD: person with an inherited bleeding disorder

Table 2. Six categories of social determinants of health that may impact health outcomes [57,58].

Category	Examples
Economic stability
	Employment and job characteristics
	Income
	Expenses
	Debt
	Medical bills
	Support
Neighborhood and physical environment
	Housing
	Transportation
	Safety
	Parks
	Playgrounds
	Walkability
	Zip code/geography
Education
	Literacy
	Language
	Early childhood education
	Vocational training
	Higher education
Food
	Hunger
	Access to health options
Community and social context
	Social integration
	Support systems
	Community engagement
	Discrimination stress
Health care system
	Health coverage
	Provider availability
	Provider linguistic and cultural competency
	Quality of care

Figure 1. Working Group 5 Diversity, Equity, and Inclusion, Health Services Research, and Implementation Science schematic of community-identified areas for priority research framework *Marginalized and minoritized populations include people who have been traditionally underserved, excluded, and/or oppressed based on a given social standing or some characteristic including but not limited to race, ethnicity, sex, gender identity, sexuality, age, income, disability status, language, culture, faith, geographic location, and country of birth. ^†[82] BD: bleeding disorder, CDC: (United States) Centers for Disease Control and Prevention, PROM: patient-reported outcome measure, QoL: quality of life, SOC: standard of care, VWD: von Willebrand disease.

Table 3. Members of Working Group 5.

Member	Community Role	Affiliation
Judith R. Baker, DrPH, MHSA (cochair)	Public Health researcher; US Hemophilia Treatment Center Network Regional Administrator	Center for Inherited Blood Disorders; Western States Regional Hemophilia Network, Orange, California
Tyler W. Buckner, MD, MSc (cochair)	Hematologist	Department of Medicine, Hematology; Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, Colorado
Vanessa R. Byams, DrPH, MPH (cochair)	Health scientist; Federal agency partner	Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, Georgia
Cindy Bailey PT, DPT, OCS, SCS, ATC	Physical therapist	Los Angeles Orthopedic Hemophilia Treatment Center, Los Angeles, California
Nathan T. Connell, MD, MPH	Hematologist	Hematology, Boston Hemophilia Center, Brigham and Women’s Hospital; Harvard Medical School, Boston, Massachusetts
Melissa S. Creary, PhD, MPH	LEE; Senior Director, Office of Public Health Initiatives, ATHN; Health, equity, inclusion, and diversity researcher	American Thrombosis and Hemostasis Network (ATHN); University of Michigan School of Public Health, Ann Arbor, Michigan
Randall G. Curtis, MBA	LEE; Researcher	Hematology Utilization Group Study (HUGS), University of Southern California, Los Angeles, California; Patient Reported Outcomes, Burdens and Experiences (PROBE), Washington, DC
Alexis Dinno, ScD, MPH, MEM	Social epidemiology and gender minority representation researcher	Oregon Health & Science University–Portland State University School of Public Health, Portland, Oregon
Christine J. Guelcher, RN-BC, MS, PPCNP-BC	Advanced practice provider	Children’s National Hospital, Washington, DC
Michelle Kim, Esq.	LEE; NHF Chapter Executive Director	Hemophilia Foundation of Southern California, Pasadena, California
Roshni Kulkarni, MD	Pediatric Hematologist	Center for Bleeding and Clotting Disorders, College of Human Medicine, Michigan State University, East Lansing, Michigan
Susan Lattimore, RN, GCPH	Regional HTC Director, Nurse Researcher	Mountain States Regional Hemophilia Network; Oregon Health & Science University, Portland, Oregon
Kathryn M. McLaughlin^†, MPH	Federal agency partner	Division of Services for Children with Special Health Needs, Maternal and Child Health Bureau, Health Resources and Services Administration, Washington, DC
Keri L. Norris, PhD, JM, MPH, MCHES	NHF Vice President of Health Equity, Diversity, and Inclusion	NHF, New York, NY
Lucy Ramirez, MSW, LCSW	Social worker	Rush Hemophilia and Thrombophilia Treatment Center, Rush University Medical Center, Chicago, Illinois
Mark W. Skinner, JD	LEE; researcher	Institute for Policy Advancement, Washington, DC; Health Sciences, McMaster University, Hamilton, Ontario, Canada
Susan Symington, MPAS, PA-C, DFAAPA	Industry	Genentech, Phoenix, Arizona
Patricia Tobase, PT, DPT, OCS	Physical therapist	University of California San Francisco Hemophilia Treatment Center, San Francisco, California
Esmeralda Vázquez^‡	LEE	NHF, Chicago, Illinois
Beth B. Warren, MD, MSCS	Pediatric Hematologist	Department of Pediatrics, Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora, Colorado
Emily Wheat, PhD	Psychologist	Department of Pediatrics, Hematology and Oncology, Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, Colorado

^†NHF SOSRS Advisory Committee, ^‡NHF SOSRS Steering Committee ATHN: American Thrombosis and Hemostasis Network, LEE: lived experience expert, NHF: National Hemophilia Foundation, SOSRS: State of the Science Research Summit, US: United States

Table 4. Summary of expert presentations and discussion themes for each WG5 subject work block.

Health services research
Speaker	Michael Nichol, PhD
Affiliation	Professor, Health PolicyUniversity of Southern California, Sol Price School of Public PolicyPI, Hematology Utilization Group Studies www.hugsresearch.net
Fields of expertise	Health policy, health care cost and resource utilization, health state preference assessment, cost effectiveness
Presentation focus	Health care access, costs, quality, outcomes as they pertain to patients, providers, institutions, insurers, policies
Discussion prompts	Access, cost effectiveness, outcomes, health care delivery models, global perspectives, pain, mental and behavioral health, etc.
# Questions generated	Approx. 85
Diversity, equity, and inclusion
Speaker	Melissa Creary, PhD, MPH
Affiliation	Senior Director, Office of Public Health InitiativesAmerican Thrombosis and Hemostasis Network Assistant Professor, University of Michigan School of Public Health
Fields of expertise	Intersection of public health, science and technology studies, medical anthropology
Presentation focus	Centering DEI, health equity, and antiracism in BDs research
Discussion prompts	Application of antiracist lens: How do racism, sexism, phobias operate in data collection, clinical care, community engagement, policies/systems? What must be challenged? What structural forces contribute to inequities?
# Questions generated	13
Implementation science
Speaker	Brian Mittman, PhD
Affiliation	Research Scientist, Kaiser Permanente Division of Health Services Research and Implementation Science U.S. Department of Veterans Affairs, University of Southern California, and the University of California, Los Angeles
Fields of expertise	Implementation science, innovative approaches to health care delivery and improvement
Presentation focus	Applying implementation science in BDs research
Discussion prompts	How to apply IS methods to HSR? How does IS intersect with DEI research? How to apply IS to other WG topics and NHF SOSRS patient input?
# Questions generated	27

BD: bleeding disorder, DEI: diversity, equity, and inclusion, HSR: health services research, IS: implementation science, NHF: National Hemophilia Foundation, PI: principal investigator, SOSRS: State of the Science Research Summit, WG: working group

Table 5. Priority research questions in the areas of health services research; diversity, equity, and inclusion; and implementation science.

Priority#	Question
Health services research
1	Among PWIBD and their caregivers, do those who use telehealth visits have the same quality of care, satisfaction, adherence to treatment, and clinical and HRQoL outcomes as patients who are primarily treated in person?
2	Do behavioral health support services* beyond psychosocial assessments completed as a part of annual comprehensive care visits improve the patient experience and clinical and HRQoL outcomes? Do these services affect direct and/or indirect costs for inherited BD diagnosis and illness management?
3	How do the type and characteristics of insurance coverage affect patient access to HTC care, treatment, bleeding, and other clinical and HRQoL outcomes?
4	In PWIBD, how do care, treatment, and clinical and HRQoL outcomes of those receiving specialized/integrated care at HTCs compare to those outside the HTCs?
5	How do provision of care components (e.g. access to care, patient satisfaction, and treatment) and clinical and HRQoL outcomes of PWIBD from marginalized and minoritized populations** compare to PWIBD who do not identify as part of these populations?
Diversity, equity, and inclusion
1	What conscious and unconscious biases are present within HTCs and among the multidisciplinary care team? What interventions can be implemented to mitigate identified biases?
2	What policies/processes/practices can local and national patient organizations and other stakeholders implement to ensure active partnership with marginalized and minoritized populations in the development and implementation of data collection (e.g. registries, surveillance, clinical trials, research, repositories) and educational initiatives?
3	What data are viewed as important and meaningful to members of marginalized and minoritized populations that are not currently being collected by clinical trials/surveillance studies?
4	What are inequities in experiences of care between non-marginalized and marginalized populations in the HTC setting? (Conduct needs assessment to identify the problem.)
5	What are barriers and facilitators for marginalized and minoritized populations to substantively engage in the conceptualization, design, and conduct of research and to participate in research?
6	What programs and/or pilot projects should be implemented to mitigate individual provider behaviors, clinical and organizational structures, and other systems in place that hinder equitable delivery of care and services?
Implementation science
1	For any given evidence-based treatment/intervention, what elements need to be adapted to enhance fit and cultural relevance to a specific sub-population (e.g. gender, language, marginalized and minoritized populations, etc.) or setting?
2	In HTCs caring for people with severe hemophilia, what practices, programs, or center characteristics lead to adherence to the prescribed treatment plan? What are factors that lead to concordance between PWIBD and HCP?
3	What data-driven benchmarks and requirements for implementation of, and adherence to, VWD and hemophilia guidelines in health systems (starting with the USHTCN) could be developed and monitored, which also include markers of outcomes specific to marginalized and minoritized populations?
4	In HTCs caring for PWIBD, what characteristics increase the adoption of evidence-based behavioral health support services and interventions for PWIBD?
5	What organizational factors, within HTCs or the institutions with which they are affiliated, promote or hinder health equity and antiracism within an organization/institution (e.g. organizational climate and culture; internal policies supporting equity; employee and leader attitudes and motivations regarding equity)?

*Behavioral health support services are any service that aims to support mental health, well-being/QoL, pain management, and healthy lifestyles**Marginalized and minoritized populations include people who have been traditionally underserved, excluded, and/or oppressed based on a given social standing or some characteristic including but not limited to race, ethnicity, sex, gender identity, sexuality, age, income, disability status, language, culture, faith, geographic location, and country of birth

BD: bleeding disorder, HCP: health care professional, HRQoL: health-related quality of life, HTC: Hemophilia Treatment Center, PWIBD: person with an inherited bleeding disorder, QoL: quality of life, USHTCN: United States Hemophilia Treatment Center Network, VWD: von Willebrand disease

Figure 2. Priority research themes identified by WG5 in the area of health services research. *Marginalized and minoritized populations include people who have been traditionally underserved, excluded, and/or oppressed based on a given social standing or some characteristic including but not limited to race, ethnicity, sex, gender identity, sexuality, age, income, disability status, language, culture, faith, geographic location, and country of birth. **Behavioral health support services are any service that aims to support mental health, well-being/QoL, pain management, and healthy lifestyles. HSR: health services research, HTC: Hemophilia Treatment Center, QoL: quality of life, WG: working group.

Figure 3. Priority research themes identified by WG5 in the area of diversity, equity, and inclusion. *Marginalized and minoritized populations include people who have been traditionally underserved, excluded, and/or oppressed based on a given social standing or some characteristic including but not limited to race, ethnicity, sex, gender identity, sexuality, age, income, disability status, language, culture, faith, geographic location, and country of birth. DEI: diversity, equity, and inclusion, HTC: Hemophilia Treatment Center, WG: working group.

Figure 4. Priority research themes identified by WG5 in the area of implementation science. HTC: Hemophilia Treatment Center, IS: implementation science, WG: working group.

Table

AMA	American Medical Association
APHA	American Public Health Association
ASH	American Society of Hematology
ATHN	American Thrombosis and Hemostasis Network
BD	Bleeding disorder
CDC	U.S. Centers for Disease Control and Prevention
CMMI	Centers for Medicare and Medicaid Services Innovation Center
DEI	Diversity, equity, and inclusion
F	Factor
HCP	Health care professional
HIV	Human immunodeficiency virus
HRQoL	Health-related quality of life
HRSA	Health Resources and Services Administration
HSR	Health services research
HTC	Hemophilia Treatment Center
HUGS	Hematology Utilization Group Study
IS	Implementation science
ISTH	International Society on Thrombosis and Haemostasis
LEE	Lived experience expert
MASAC	Medical and Scientific Advisory Council
NHF	National Hemophilia Foundation
NIH	National Institutes of Health
PI	Principal investigator
PICOTS	Population/patient problem, intervention, control/comparison, outcome, timing, setting
PPM	Person who has or had the potential to menstruate
PROBE	Patient Reported Outcomes, Burdens and Experiences
PROM	Patient-reported outcome measure
PWIBD	Person with an inherited bleeding disorder
QoL	Quality of life
SC	Steering Committee
SDoH	Social determinants of health
SOC	Standard of care
SOSRS	State of the Science Research Summit
US	United States
USHTCN	US Hemophilia Treatment Center Network
VWD	Von Willebrand disease
WFH	World Federation of Hemophilia
WG	Working Group

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