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Orphan Drugs

This document discusses orphan drugs, which are medications intended for rare diseases or conditions affecting a small percentage of the population. It defines orphan drugs and orphan diseases. Orphan diseases lack treatments because they provide little financial incentive for pharmaceutical companies. The Orphan Drug Act of 1983 was passed to encourage orphan drug development by providing tax incentives and market exclusivity. It gives examples of orphan diseases and drugs that have been developed to treat rare conditions like sickle cell anemia and malaria. The document outlines the regulatory process for orphan drug approval and factors influencing their costs.

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460 views16 pages

Orphan Drugs

This document discusses orphan drugs, which are medications intended for rare diseases or conditions affecting a small percentage of the population. It defines orphan drugs and orphan diseases. Orphan diseases lack treatments because they provide little financial incentive for pharmaceutical companies. The Orphan Drug Act of 1983 was passed to encourage orphan drug development by providing tax incentives and market exclusivity. It gives examples of orphan diseases and drugs that have been developed to treat rare conditions like sickle cell anemia and malaria. The document outlines the regulatory process for orphan drug approval and factors influencing their costs.

Uploaded by

sakumar5678
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© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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ORPHAN DRUGS

Presented by:
Sakhare Leena
Ist M.Pharm.
Dept. Of Pharmacology
K.L.E.Ss College of Pharmacy,
Belgaum.

CONTENTS

DEFINITION

ORPHAN DISEASE

CLASSIFICATION OF ORPHAN DRUGS

ORPHAN DRUG ACT

DEVELOPMENT OF ORPHAN DRUGS

EXAMPLES OF ORPHAN DRUGS

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?

Orphan disease
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 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 .

ORPHAN DISEASES CONTI

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 DRUG ACT:

In 1983, a very important step was


taken by Congress when it passed the
Orphan Drug Act.

Tax credit

Patent like protection

PATENTS AND GENERICS:

Patents

Branded drugs

Generic drugs

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.

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.

Development of orphan drugs


Orphan drugs generally follow the same
regulatory development path as any other
pharmaceutical treatment.

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

EXAMPLES:

DRUGS
Hydroxyurea
Lepirudin
Modafenil
Rifapentine
Valrubicin
Aldesleukin
(PROLEUKIN)
Artesunate
injection

ORPHAN INDICATION
Sickle cell anaemia
Thrombocytopenia
Narcolepsy
Pulmonary tuberculosis
Carcinoma of bladder
Renal cell carcinoma
chloroquine resistant
malaria

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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