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