ORPHAN DRUGS

Presented by:Sakhare Leena

Ist M.Pharm.Dept. Of Pharmacology

K.L.E.S’s 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?

  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

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 .

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…

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 INDICATIONSickle cell anaemiaThrombocytopenia

NarcolepsyPulmonary 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.