So, you think you know everything about RARE DISEASES and how inexorably linked pharmacy, specifically specialty pharmacy, is to treatment development?
Can you pass the QUIZ?
The article below is a great quick read / study guide for anyone (shall we even say….. everyone) in specialty pharmacy today if they are serious about playing in the ‘rare’ sandbox with sufficient knowledge to earn a passing grade.
Here are some of the essential top line FAQs…..
· How many people live with a rare disease today in the US?
· How many people does it take for a drug to qualify for an Orphan Drug designation?
· How many rare diseases and conditions are there?
· What is the ARC Program run by CDER?
· What are the four (4) expedited review programs and how do they differ?
· And, last but not least, what the heck does CDER stand for? And how about CBER too?
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9 Things to Know About CDER’s Efforts on Rare Diseases
CLICK HERE to read the full article from the FDA
The Orphan Drug Act defines a rare disease as any disease or condition that affects less than 200,000 people in the U.S. There are approximately 25 to 30 million Americans living with a rare disease (about 1 in 10 people). Many rare conditions are life-threatening, and most do not have treatments. Accelerating the development of safe and effective drugs is CDER’s core mission, and CDER understands there are unique challenges in demonstrating the safety and effectiveness of drugs that treat rare diseases.
