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    Orphan Drugs

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    sakumar5678
    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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    Orphan Drugs

    Uploaded by

    sakumar5678
    220 views16 pages
    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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    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.

    Copyright:

    © All Rights Reserved
    Download as PPT, PDF, TXT or read online from Scribd
    Download as ppt, pdf, or txt
    220 views16 pages

    Orphan Drugs

    Uploaded by

    sakumar5678
    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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    © All Rights Reserved
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    of 16

    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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