refuse to destroy: re-purposing orphan drugs is an economical method to reduce treatment costs and...

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 (firhan.malik@gmail.com)