refuse to destroy: re-purposing orphan drugs is an economical method to reduce treatment costs and...
TRANSCRIPT
R&D spending by Pharmaceutical Industry
$ bi
lliom
s
0
15
30
45
60
1980 1990 2000 2010
Refuse to destroy: re-purposing orphan drugs is an economical method to reduce treatment costs and open up new revenue streams
117 mil 30 mil
Chronic Disease
Rare Disease
# of patients in U.S.A.
$330 K$26 K
Annual cost of Treatment
HIV
Average of 10 most expensive orphan drugs
The estimated cost to develop a new drug is $5 billion
Rare disease: A disease that affects <200,000 patients (1 in 1,500) (Source: U.S. Orphan Drug Act)
In 2010, Forbes magazine estimated the market for rare disease therapeutics could be worth $1 trillion annually. This assumes an annual cost of $33,000 per patient in the U.S.
Orphan Drug: A drug that treats a rare disease. There are tax incentives and patent protections available to encourage companies to develop these drugs, in the U.S. and Europe.
HOWEVER, treating a rare disease with an orphan drug can cost almost $330,000 annually!
What if an orphan drug could be re-purposed?
Compare disease symptoms
Chronic Disease
Rare Disease
Symptom 1 XSymptom 2 X X
What do current drugs target?
Receptors
Hormones
Genes
Are any of these targets
common?
Chronic Disease
Rare Disease
Receptor 1 X XGene 1 X
Is there evidence that
the common target is abnormal?
NO
YES
RE-PURPOSE
By re-purposing an orphan drug for treatment of another disease, new revenue streams can be realized, and cost of the drug to patients or health insurance companies can be reduced.
Source: Centers for Disease Control and Prevention, 2012 Source: Pharmaceutical Research and Manufacturers of America, 2013 profile
Source: U.S. FDA, IMS Health
Since R&D is so expensive and the patient population for any rare disease is small, the treatment cost reflects an effort to recoup investments.
Created by Firhan Malik ([email protected])