Partners for a CPAG: Healthier Future Perspective from an RDCRN - - PowerPoint PPT Presentation

CPAG:

Partners for a Healthier Future

Perspective from an RDCRN Consortium PAG as Research Partner

WHAT I S CPAG?

 The Coalit ion of Pat ient Advocacy Groups or ‘CPAG’ is t he

collect ive represent at ion of pat ient groups affiliat ed w it h t he consort ia of t he Rare Diseases Clinical Research Net w ork ( RDCRN) , a program of ORDR/ NCATS.

 CPAG is composed of represent at ives from each pat ient

group in t he net w ork w ho are t asked w it h being FULL PARTNERS in t he research undert aken by t heir RDCRN consort ium

WHAT I S CPAG?

 CPAG members influence t he direct ion of t he Rare Diseases

Clinical Research Net work as a whole. They part icipat e in net w ork-level discussions and meet ings. The CPAG coalit ion, t hrough it s leadership, is a vot ing member of t he RDCRN St eering Commit t ee.

WHAT I S CPAG?

 100+ volunt ary healt h/ pat ient advocacy organizat ions

represent ing a variety of rare disorders

 Ext remely diverse in resources, experience, name

recognit ion and infrast ruct ure ( see “ snapshot ” slide)

 All committ ed t o rare disorders research t hrough t he RDCRN

WHAT CPAG I S NOT

 CPAG is not an independent pat ient represent at ive group  CPAG as an ent it y exist s only in t he cont ext of t he RDCRN  CPAG’s focus and act ivit ies are limit ed t o ( and only t o)

issues relat ed t o t he RDCRN

CHALLENGES FOR CPAG DI VERSI TY CHALLENGE

 The member groups t hat comprise t he ent ity collect ively know n

as “ CPAG” are incredibly diverse and range from “ Kit chen Table”

• rganizat ions w it h no st aff and lit t le revenue t o mult i-million

dollar non-profit corporat ions w it h professional st aff and access t o consist ent funding sources.

 Many of t he PAGs associat ed w it h t he RDCRN are operat ing

w it h ext remely limit ed financial and human resources. H o w

• w ca n

a n w e m e e t t h e n e e d s o f

• f su ch a

a d i v e r se co a l

• a l i t i o n
• n o f
• f

m e m b e r r g r o r o u p s a n d f u l f i l l o u r r co m m i t m e n t t o b e p a r t n e r s s w i t h RD CR CRN i n v e st st i g a t o r s? s?

CHALLENGES FOR CPAG HAT CHALLENGE

• The member groups that comprise the entity collectively

known as “CPAG” are dedicated professionals with a laser focus on their individual diseases As CPAG represent at ives, w e m ust w ear several different hat s and be sure w e are co considering t he needs of t he RDCRN as a w hole, in addit ion t o our r individual consort rt ia and d gr groups ps

ADDRESSI NG THE CHALLENGES

• CPAG leadership

Chair: Chuck Mohan (UMDF/NAMDC) Co-Chairs: Janet Hieshetter (DRF/DC) Joyce Kullman (VF/VCRC) Cynthia LeMons (NUCDF/UCDC)

• Work closely and receive guidance from ORDR staff

OPPORTUNI TI ES FOR CPAG

• 1. Face to Face Meetings
• 2. Monthly Calls
• 3. Working Groups of CPAG
• 4. Committees of the Steering Committee

CPAG MI SSI ON AND VI SI ON

 CPAG Vision

Through collaborat ion, pat ient advocacy groups and researchers can make fast er progress t oward new t reat ment opt ions and cures, which can improve t he lives of all persons and families affect ed by a rare disease.

 CPAG Mission

The Coalit ion for Pat ient Advocacy Groups will promot e collaborat ion bet ween rare disease advocacy organizat ions and t he Rare Diseases Clinical Research Net work in order t o facilit at e bet t er access t o, and earlier benefit from, research conduct ed on rare diseases. As t he pat ient advocacy arm of t he Rare Diseases Clinical Research Net work, CPAG members will use t heir posit ion t o advance t he cause of rare disease research and improved pat ient out com es t hrough t he net work.

1904 2015

1904 1918 1932 1946 1960 1974 1988 2002

1st Case Report

1/1/1904

Kartagener's Triad Described

1/1/1933

EM Defect Described/ICS

1/1/1976

Microscopy/PCD

1/1/1983

Low nNO Described

1/1/1994

First Gene Identified

1/1/1999

1st PCD Family Day Second PCD Gene ID'd PCD & CHD Publication First Cilia & Mucus GRC 1st Genetic for PCD--4 genes 1st PCD/Ciliopathy Mtg BestCilia Formed PCD Clinical Centers Established PCD CC Mtg PCD & Cri du Chat PCD Foundation Incorporated

1/1/2002

RDCRN Formed

1/1/2004

32 Genes

Indicates start of RDCRN/GDMCC

PCD Timeline