RARE DISEASES BIG IMPACT
RARE DISEASES WORLDWIDE
7,000
Source: National Institutes of Health
RARE DISEASES BIG IMPACT
<200,000patients in the United States
BY DEFINITION, A RARE DISEASE AFFECTS
Source: National Institutes of Health
RARE DISEASES BIG IMPACT
30 MILLION
1 IN 10 AMERICANS
IN TOTAL, RARE DISEASE IMPACT
Source: National Institutes of Health
RESEARCH PROGRESS HOPE
RARE DISEASES BIG IMPACT
1983
ORPH
AN DRUG ACT 2,900 ORPHAN DRUG DESIGNATIONS
SINCE 1983, NEARLY
Source: Office of Orphan Products Development, Food and Drug Administration
RARE DISEASES BIG IMPACT
A GOOD START, but
400FDA TREATMENTS APPROVED
<5% RARE DISEASES HAVE A TREATMENT
of
Source: Food and Drug Administration, EveryLife Foundation for Rare Diseases
RARE DISEASES BIG IMPACTRARE DISEASES BIG IMPACT
medicines& vaccines indevelopment
452
Source: PhRMA, 2013 Rare Diseases Report
RARE DISEASES BIG IMPACT
HOPE
PROGRESS
RESEARCH
Source: PhRMA