orphan diseases, Indian scenario of Orphan disease management, Drugs for Orphan diseases. Orphan drug act

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