In the U.S, an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ("orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States or low prevalence is taken as prevalence of less than 5 per 10,000 in the community. The United States of America —commonly referred to as the Medication, also referred to as medicine, can be loosely defined as any substance intended for use in the diagnosis cure mitigation treatment or prevention of disease Federal law is the body of Law created by the Federal government of a country A rare disease (sometimes known as an orphan disease has such a low Prevalence in a population that a doctor in a busy General practice would not This has been adopted as a subclause of the Food and Drug Administration (FDA) regulations. The granting of orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive/un-profitable to develop under normal circumstances. Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity (a "monopoly") on that drug for an extended time (seven years post-approval). Biomedical research (or experimental medicine) in general simply known as medical research, is the Basic research or Applied research conducted Medication, also referred to as medicine, can be loosely defined as any substance intended for use in the diagnosis cure mitigation treatment or prevention of disease In Economics, a monopoly (from Greek monos, alone or single + polein, to sell exists when a specific individual or enterprise has sufficient The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research. Under the act many drugs have been developed, including drugs to treat glioma, multiple myeloma, cystic fibrosis, phenylketonuria and snake venom. A glioma is a type of Cancer that starts in the brain or spine Multiple myeloma (also known as MM, myeloma, plasma cell myeloma, or as Kahler's disease after Otto Kahler) is a type of Cystic fibrosis (also known as CF, mucoviscoidosis, or mucoviscidosis) is a hereditary disease affecting the exocrine (mucus glands of the lungs Phenylketonuria ( PKU) is an Autosomal recessive Genetic disorder characterized by a deficiency in the enzyme Phenylalanine hydroxylase Snake venom is highly modified saliva that is produced by special glands of certain species of Snakes. In the US, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization in the US. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.
A similar status exists in the European Union, administered by the Committee on Orphan Medicinal Products of the European Medicines Agency (EMEA). The European Union ( EU) is a political and economic union of twenty-seven member states, located primarily in The European Medicines Agency ( EMEA) is a European agency for the evaluation of medicinal products. The European Medicines Agency ( EMEA) is a European agency for the evaluation of medicinal products. In late 2007, the FDA and EMEA had come to an agreement whereby the same application could be used for both agencies, thereby reducing the time and finances required of companies to apply for orphan drug status. However, the two agencies still maintained separate approval processes. [1]
In 2003, the leading orphan drug by worldwide sales revenue was Amgen’s Erythropoietin (Epogen), with sales of $2. Amgen Inc () is an international Biotechnology company headquartered in Thousand Oaks California. Erythropoietin (ɨˌɹɪθɹoʊˈpɔɪɨtɨn /ɨˌɹɪθɹoʊˈpɔɪtən/ or /ɨˌɹiːθɹoʊ-/ or EPO is a Glycoprotein Hormone that controls 4bn.
Contents |
Development of orphan drugs
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical treatment. Testing focuses on the characterization of the molecule(s), stability, safety, and efficacy. However, some statistical burdens are reduced. For example, the regulations generally reflect the fact that it is not possible to test 1,000 patients in a Phase III clinical trial when fewer than 1,000 patients have the disease in question. In health care clinical trials are conducted to allow safety and Efficacy data to be collected for new drugs or devices
Since few markets would naturally exist to create these goods, as the costs of developing, researching and producing this drug would likely exceed any revenues, government intervention is required, usually to establish such a market or to produce the goods itself. Sao Paulo Stock Exchangejpg|thumb| Virtual market arena where buyer and seller are not present and trade via intemediates and electronical information Critics of the free market often cite this as a market failure in free market economic systems. Market failure is a concept within economic theory wherein the allocation of goods and services by a Free market is not efficient. A free market is a Market in which property rights are voluntarily exchanged at a price arranged completely by the mutual consent of sellers and buyers Economics is the social science that studies the production distribution, and consumption of goods and services. Free market advocates often respond that, without government intervention in the form of extensive safety and efficacy testing, the development costs would be considerably lower.
The intervention by government can take a variety of forms:
- Tax benefits to companies who produce or research these drugs
- Granting of additional rights above and beyond those granted by the regular patent laws. A patent is a set of Exclusive rights granted by a State to an inventor or his assignee for a fixed period of time in exchange for a disclosure of an
- Subsidizing and funding clinical research by universities and industry sponsors to develop medical products (including drugs, biological products, devices, and medical foods) for rare diseases. A university is an institution of Higher education and Research, which grants Academic degrees in a variety of subjects
- Creating a government-run company to research and produce drugs (see Crown corporation for an example of this type of solution). In the Commonwealth realms a Crown corporation is a State-controlled company or enterprise
See also
References
- ^ Donna Young. In European Union member countries a supplementary protection certificate (SPC is a Sui generis, Patent -like Intellectual property "U.S., EU Will Use Same Orphan Drug Application.", BioWorld News, 2007-11-28. Year 2007 ( MMVII) was a Common year starting on Monday of the Gregorian calendar in the 21st century. For the town in Argentina, see 28 de Noviembre. Events Retrieved on 2008-01-06. 2008 ( MMVIII) is the current year in accordance with the Gregorian calendar, a Leap year that started on Tuesday of the Common Events 1066 - Harold Godwinson is crowned King of England. 1205 - Philip of Swabia becomes King Archived from the original on 2007-12-11. Year 2007 ( MMVII) was a Common year starting on Monday of the Gregorian calendar in the 21st century. Events 359 - Honoratus, the first known Prefect of the City of Constantinople, takes office "In an attempt to simplify the process for obtaining orphan status for medications targeting rare diseases, the FDA and the European Medicines Agency (EMEA) have created a common application. . . . U. S. and European regulators still will conduct independent reviews of application submissions to ensure the data submitted meet the legal and scientific requirements of their respective jurisdictions, the agencies said. "
External links
- European Commission - The Orphan drugs strategy
- List of European Orphan Drugs
- USA Food and Drug Administration: The Orphan Drug Act (as amended)
- US FDA List of Orphan Designations and Approvals
- Orphan drugs regulation and market figures
Dapyx Software network: MP3 Explorer | Ebook Manager | Zenithic