WORLD FEDERATION OF HEMOPHILIA Mark Brooker, WFH Senior Public - - PowerPoint PPT Presentation

WORLD FEDERATION OF HEMOPHILIA

Mark Brooker, WFH Senior Public Policy Officer London – July 2, 2015

PATIEN TS’ PER SPEC TIVE

The World Federation of Hemophilia (WFH), an international not-for-profit organization, was established in 1963. It is a global network of patient organizations in 127 countries and has

• fficial recognition from the World Health

Organization

PATIEN TS’ PER SPEC TIVE

Global significance of decisions made by FDA/EMA

• Humanitarian aid
• Product withdrawals/recalls
• Indications
• How products are used in different parts of the world

ID EN TIFIED PATIEN TS – R EG ION

50,000 100,000 150,000 200,000 250,000 300,000 350,000 400,000 expected identified

IN C R EA SE IN FA C TO R U SE PER C A PITA

ADDITIONAL TEXT EXAMPLE

0.01 0.01 0.01 0.02 0.02 0.02 0.02 0.02 0.03 0.04 0.06 0.07 0.07 0.64 0.53 0.56 0.54 0.73 0.95 0.97 1.00 1.01 1.22 1.25 1.35 1.45 0.89 0.80 0.84 0.92 0.95 1.26 1.31 1.32 1.41 1.46 1.51 1.76 1.91

0.00 0.25 0.50 0.75 1.00 1.25 1.50 1.75 2.00

2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013

FVIII IU per capita World FVIII use over time

Lower income - less than $2000 Middle income- $2000-$10,000 World

Source: REPORT ON THE ANNUAL GLOBAL SURVEY 2013

PATIEN TS’ PER SPEC TIVE

2013 Global Survey: Reported data source

• 107 countries reporting
• Country registry: 31
• Registry of HTCs: 24

PATIEN TS’ PER SPEC TIVE

55% of new cases – no family history or spontaneous mutation

• Families with a history of hemophilia
• Parents with no experience/knowledge of hemophilia

PATIEN TS’ PER SPEC TIVE

Transparency vs. data protection laws

• WFH would like to see more post-marketing surveillance
• Education on the importance of PMS
• PUPs (and other patients) enrolled in clinical studies, not in

registries?

• Unique, anonymous identifier for all patients?

TH AN K YOU