Orphan diseases are rare, often genetic conditions that lack effective treatment and are difficult to diagnose, with some patients waiting years for a diagnosis. Approximately 10% of human diseases are classified as rare, and while orphan drugs can be developed for treatment, economic feasibility and limited patient populations hinder research and availability. In India, awareness and knowledge about orphan drugs among pharmacy graduates are low, and medical stores do not stock these drugs due to their limited market value.

1. Orphan Drugs
By Sumaiya Banu
S.Y.B.Pharmacy

2. Orphan disease
• Orphan diseases : rare; have no definite cure;
affects small percentage of population.
• Why is it called orphan disease?
• Because most are genetic they impact children at
a high rate or develop as the body begins to age.
• Diagnosis is difficult.
– 30% of Patients Take 3 to 5 Years to Diagnosis
– 15% of Patients Wait 7 Year or More for Diagnosis
– And those are the lucky ones, some, never get
diagnosed

3. Statistics
• Approx. 10% of the human diseases are
classed as rare.
• Of this, 10 % have genetic origin.
• About 250 new discovered every year

4. Examples

6. A.K.A. The Vampire Syndrome

9. Orphan drug
Developed for treatment of rare disease

10. ODA
• Orphan drugs act
• USA first to propose a legal framework
• Followed by Singapore, US, Japan, Europe.

13. Problems
• In some cases there is not even a medical
specialty that ‘owns’ the disease.
• These diseases affect so few people that it is
not profitable to develop treatments and
therefore they get little or no money or
attention from the medical system.
• Lack of economic feasibility leads to a lack of
even preliminary research into these disorders

14. Disadvantage in OD development
• High prices of OD
• Small patient population
• Delay in diagnosis and deficient diagnostic
system
• Limited treatment availability
• Lack of knowledge and training
• Poor understanding of the disease
• Limited public awareness

15. Indian scenario
• Rare disease day in New Delhi on February
28th, 2010.
• 2nd and 3rd rare disease days in Chennai and
Mumbai in 2011 and 2012

16. Drug store Survey
• All D.Pharmacy graduate.
• Most have no idea what orphan drugs or
orphan disease exactly is.
• “Epilepsy is a rare disease for which people
ask medicine for from us mostly”
• “Medical stores don’t hold orphan drugs as
they do not have much market value.”
• “Orphan drugs are compounded in the
hospital it self and distributed. “

17. THANK-YOU