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ORPHAN DRUGS
Presented by:Sakhare Leena
Ist M.Pharm.Dept. Of Pharmacology
K.L.E.S’s College of Pharmacy, Belgaum.
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CONTENTS DEFINITION
ORPHAN DISEASE
CLASSIFICATION OF ORPHAN DRUGS
ORPHAN DRUG ACT
DEVELOPMENT OF ORPHAN DRUGS
EXAMPLES OF ORPHAN DRUGS
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Definition:-
“Orphan drugs are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare.”
Why these drugs are called Orphan?
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A disease which has not been "adopted" by
the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it.
Orphan disease
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ORPHAN DISEASES:-
Many orphan diseases are lesser known, like Juberg Marsidi syndrome (a genetic disorder of childhood that leads to severe
mental retardation, abnormal bone growth resulting in the disfiguring of the head and body and loss of hearing)
Hermansky-Pudlak syndrome (a genetic disorder)
Lambert-Eaton myasthenic syndrome (muscle weakness due to impaired acetylcholine release)
And many more like Aarskog syndrome, Adams Nance syndrome, Bagatelle Cassidy syndrome, Bamforth syndrome, Ballard syndrome and Bahemuka Brown syndrome .
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There are some familiar orphan diseases:-
like Alzheimer's disease, leukemia, multiple sclerosis, Huntington's disease, sickle cell disease, muscular dystrophy, myasthenia gravis and spinal cord injury.
ORPHAN DISEASES CONTI…
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ORPHAN DRUG ACT:-
In 1983, a very important step was taken by Congress when it passed the Orphan Drug Act.
Tax credit
Patent like protection
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PATENTS AND GENERICS:- Patents
Branded drugs
Generic drugs
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Are orphan products approved faster than other drugs?
The approval time for orphan products as a group has been considerably shorter than the approval time for all drugs. This is due to the fact that many orphan products receive expedited review because they are for serious or life-threatening disease.
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HOW MUCH DO ORPHAN DRUGS COST?
The cost of orphan products is determined by the sponsor of the drug and is not controlled by the FDA. The costs of orphan products vary greatly. Generally, health insurance will pay the cost of orphan products that have been approved for marketing.
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Development of orphan drugs
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical treatment.
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The intervention by government can take a variety of forms: Tax
patent laws. Subsidizing and funding clinical research by
universities and industry sponsors to develop medical products) for rare diseases.
Government-run company
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EXAMPLES:
DRUGS Hydroxyurea Lepirudin Modafenil Rifapentine
Valrubicin Aldesleukin
(PROLEUKIN) Artesunate injection
ORPHAN INDICATIONSickle cell anaemiaThrombocytopenia
NarcolepsyPulmonary tuberculosis
Carcinoma of bladder
Renal cell carcinoma
chloroquine resistant malaria
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References:
Essentials of Medical Pharmacology- K.D. Tripathi 5th edition p.no.-5&6
Hand book of Experimental Pharmacology – S.K. Kulkarni
3rd edition p.no.-27 www.google.com.
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