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

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