Community Networks for Children and Families with Rare Diseases and Chronic Conditions

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Community Networks for Children and Families with
Rare Diseases and Chronic Conditions

• Bill McKellin, PhD
• Emilie Gladstone
• Dept. of Anthropology
• Milan Patel, MD
• Rosemarie Rupps, MSc
• Dept. of Medical Genetics
• UBC

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The Objectives

• Identify resources available to families of
  children who have rare diseases and chronic
  conditions
• Develop local, collaborative, multidisciplinary
  networks of
• Parents
• Support groups
• Health and social service professionals
• Expertise across existing disease-specific
  organizational boundaries.

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Assumptions

• Differentiation among
• Disease pathophysiology
• Illness personal phenomenological experience
• Sickness social ecology
• Illness experience and sickness are shaped by
• Service providers' patterns of practice
• Health care
• Education
• Social services etc.
• Child and family encounters with services
• Patterns of practice reflect local social
  ecologies

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Rare and Chronic Conditions

• Rare or "orphan" disease is one that affects
  fewer than 1 in 2,000
• There are more than 7,000 rare disorders
• Chronic conditions affect approximately 3 million
• 1 in 10 Canadians
• An estimated total of 30.3 of Canadian children
  aged 6 to 11 had one or more chronic physical
  health conditions/impairments

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Rare to Ultra- rare

• Cystic fibrosis
• 1/2500
• Muscular dystrophy
• 1 in 6,000
• Achondroplasia
• 1 in 26,000
• Fibrodysplasia ossificans progressive
• 1 in 2 million
• N1 Name?

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OverviewOrganizations for Rare Diseases

• Disease Specific
• Generic Rare Diseases
• Generic Support

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Disease Specific Organizations

• Organizations defined by disease or a medical
  condition
• Canadian Cystic Fibrosis Foundation
• Muscular Dystrophy Canada
• Mucopolysacharidosis Society of Canada
• Roles
• Research
• Support
• Advocacy

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Organization of National, Provincial, and Local
Chapters

• National and Provincial
• Canadian Cystic Fibrosis Foundation
• Muscular Dystrophy Canada
• Mucopolysaccharidosis  Society of Canada
• Foundation for Prader Willi Research Canada
• International
• 5P- Society - Cri du Chat Syndrome

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SupportDisease Specific Information

• Knowing what to expect is really important.
  Talking to people on the internet really helped
  us to know what it was like., even if our
  daughters experience was different..
• Father of a child with Fibrodysplasia ossificans
  progressive

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Support

• Fill in gaps in the health care system
• comprehensive care
• Mental health and emotional support
• parent to parent
• organization to parent
• Navigating Systems and referrals
• The most important in my mind is the
  networking and knowing what is available.

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• When do families need support?
• Transition times
• Medical, social, educational, age
• Transition out of high school is very big
  because thats when everything ends. The
  government recognizes that as a big important
  milestone but for the individual, their support
  needs may not have changed very much at all. Its
  a very artificial barrier that is created for
  families.
• Sometimes families are reluctant to be involved
  with service organizations when they are not in
  times of crisis or transition.

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Research

• Few organizations actually perform research but
  many give money to research projects
• Organizations often provide access to potential
  research participants for researchers
• Would like to be more involved in research and
  have assistance from researchers

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Research as Social Support

• Some organizations felt that involvement in
  research was often a positive experience for
  families
• For families who manage rare and chronic
  conditions, research becomes individual care
• Members find participation very valuable. They
  are doing something. They are also learning
  something.
• How do researchers conceptualize the individuals
  health care needs in a research setting?

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Advocacy

• Awareness and education of community and medical
  professionals
• The number one problem is knowledge. Not only in
  the public, but in the professional realm there
  is a lack of information and misinformation.
• Correcting misconceptions

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Fundraising

• Fundraising is one of the most time consuming and
  least enjoyable parts of running an organization!
• All organizations identified funding as a barrier
  to providing services
• Many organizations had particular boards or
  committees to manage fundraising
• Issues of funding are linked to issues of
  awareness

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Local Variation

• The Social Ecology of Disease-specific
  Organizational Support

• Every district varies toothats another problem
  when you are provincial and you are trying to
  serve the different districts-- especially
  education. There are different paths in every
  district.

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Relationships Among Organizations

• Little coordination, communication or
  collaboration among disease-specific groups
• Non-profit sector as competitive because of donor
  competition
• We would be swallowed up by bigger fish.The
  bottom line is that we need to stay alive in
  order to ensure the entire message is given out
  to people
• Others frequently wish to collaborate but have
  not yet done so because of logistical and time
  constraints

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Organizations as Cultural Groups

• Groups maintain distinct cultural identities for
  children and families
• Organizations must be seen as unique to maintain
  their cultural groups identities and to draw the
  attention of funders
• Segmentary opposition maintains boundaries
  between groups
• Limited interaction between cultural groups helps
  to explain the lack of collaboration between
  groups

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Generic Rare Disease Organizations

• International - 25 organizations
• Canada - 3
• CORD
• Canadian Gene Cure Foundation
• Rare Disease Foundation
• United States - 14
• European Union (including the UK) - 8

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Relationships among organizations
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Generic Rare Disease Organizations

• 25 Organizations
• Activities
• 22 are engaged in family and peer support
  activities
• 12 undertake advocacy
• 13 support or conduct research
• 13 were founded by patients and/or family
  members, 5 by researchers

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Generic Rare Disease Organizations

• CORD
• Orphan drug policy
• Rare Disease Foundation
• Research
• Generalizable tools for genetic research
• Micro-grants for low incident small scale
  research
• Support
• For n1 diseases
• Address local generic needs defined by the family
  interaction with health, educational, and social
  services

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Generic Support Organizations

• Services based on functional needs of recipients
  not diagnosis
• Mobility
• Visual impairment
• Developmental delay
• Services to caregivers
• Respite
• Social support
• Training
• Advocacy
• Family Support Institute
• Western Society for Children

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Children's and Parents' Experience

• Uncertainty of disease
• Multiple clinical encounters
• Multiple Special education encounters
• Social services involvement
• Financial demands

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Parents Experience
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N1 Community of Many

• Until I met other parents, I didnt realize how
  similar our issues were.
• Father who has a child with a unique condition

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Care across stove pipes

• Every child and their family comprises an N1

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Shared Issues

• Disease
• Access to pharmaceuticals / treatments
• Care coordination
• Information sharing among physicians
• Lack of needs-based assessment

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Shared Issues

• Personal
• Social support from others with similar diagnosis
  
• Respite for care givers
• Financial advice
• Difficulty attending disease specific
  organization events

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Shared Issues

• Social Ecology
• Health care
• Timely access to health records
• Coordinated scheduling
• Transition
• Schools
• IDP provincial mandate - local implementation
• Advocacy
• Transition
• Social services
• Transition

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Generic Support Organizations

• Functional need of child
• Locally based
• Services fill the gaps in institutional /
  systemic care
• Link among disease specific groups whose children
  have needs for similar services

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Emerging Rare Disease Network

• Workshop Representatives of 10 organizations
• Disease specific organizations
• Generic service providers
• Generic rare disease organization

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Rare Disease Network

• Disease specific groups
• Share generalizable resources developed for
  specific groups
• Dont re-invent the wheel collaborate in new
  initiatives
• Generic support services
• Extend services geographically
• Extend services generically
• Advocacy
• Building communities
• Virtual
• Face to face

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Network Contributions

• Parents support
• Family Support Institute - Online registry (Nov.)
  Supportworkercentral.com
• Local social networking across diseases RDF
  Ning site
• Health services
• Information sheet for emergencies
• MPS Society Binder for regular visits
• Advocacy
• Schools
• On-line advocacy video training
• CF - Parent mentors for school districts
• Social services
• Collaborate on transition programs

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Needs based local services

• Many personal and social needs of children and
  their families are not disease-specific
• Local social ecology shapes patterns of practice
• the interaction among children, parents, and
  services
• Disease-specific and generic rare disease
  organizations can collaborate to serve the local
  personal and social needs of children and their
  families.

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Thanks to

• The organizations an families who participated
  in this study
• University of British Columbia
• BC Childrens Hospital
• Rare Disease Foundation
• Child and Youth Health Research Network