Other Models That Work: Disclosure

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Other Models That Work Disclosure

• Due to time, have selected 2 non-hematologic
  pediatric disorders
• No implications to the superb work of
• Cooleys Anemia Foundation
• DBA Foundation
• Fanconi Anemia Research Fund
• Jeffrey Modell Foundation
• and others

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Other Models That Work Cystic Fibrosis

• 80 Chapters
• Pharmacy services with a Patient Assistance
  Program
• Resource filled website

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Key Program Elements

• Research and Development program
• - 10 programs
• - Lead by variety of physiologists, gene
  therapists and pulmonologists
• CF Foundation accredited care centers
• - 115 care centers, 95 adult care programs, 50
  affiliates
• - Regular review and some funding
• Clinical research website
• Drug development pipeline

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Other Models That Work CF The Bell Curve
The Bell Curve What happens when patients find
out how good their doctors really are? by Atul
Gawande December 6, 2004
The one thing that the clinicians failed to tell
them, however, was that Cincinnati Childrens was
not, as the Pages supposed, among the countrys
best centers for children with cystic fibrosis.
What makes the situation especially puzzling is
that our system for CF care is far more
sophisticated than that for most diseases. The
hundred and seventeen CF centers across the
country are all ultra-specialized, undergo a
rigorous certification process, and have lots of
experience in caring for people with CF. They all
follow the same detailed guidelines for CF
treatment. They all participate in research
trials to figure out new and better treatments.
You would think, therefore, that their results
would be much the same. Yet the differences are
enormous. Patients have not known this. So what
happens when they find out?
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Other Models That Work Muscular Dystrophy

• 235 hospital-affiliated clinics
• 290 support groups
• Research grants program (45K/year, 1-3 years)
• Translational research program focused on drug
  development
• Clinical trials development website

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Sponsorship ? A Key To Success
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Continuing The Timeline
2001 Senators Wellstone and Collins introduce
MD CARE Act, signed into law, establishing
an interagency coordinating council,
and authorizing Centers of Excellence
at the NIH and CDC surveillance and
epidemiology 2003 3 Centers of Excellence
funded by NIH 2005 3 new Centers of Excellence
funded by NIH CDC develops partnership
with PPMD Current activities
continue
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Take Home Points

• Cause
• Organization
• Sponsorship
• Federal connections

Opportunity