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| | FDA Consumer: Orphan Drug Law.(US law to promote research on ... @ HighBeam Research |
 | | The Orphan Drug Act of 1983 has attempted to aid in the creation for drugs to treat these diseases by giving drug research companies incentives to explore drug therapy for orphan diseases. | | | He took the drug through two years of clinical trials starting in 1995, when he was 15, and continued the regimen after the drug's approval in 1997. | | | Pharmaceutical companies did, however, develop a few drugs of limited commercial potential in the 1960s and 1970s and even provided some at little or no charge. |
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http://www.highbeam.com/library/doc0.asp?DOCID=1G1:54612391&refid=ip_encyclopedia_hf
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| | FDA > CDRH > CFR Title 21 Database Search |
| | When a drug proposed for orphan drug designation is intended to treat a life-threatening or severely debilitating illness, especially where no satisfactory alternative therapy exists, the sponsor may wish voluntarily to provide this information. | | | A sponsor of such a drug may be entitled to expeditious development, evaluation, and marketing under 21 CFR part 312, subpart E. (11) An explanation of how the data summarized and analyzed under paragraphs (b)(9) and (b)(10) of this section support the rationale for use of the drug in the rare disease or condition. | | | (5) Where the sponsor of a drug that is otherwise the same drug as an already-approved orphan drug seeks orphan-drug designation for the subsequent drug for the same rare disease or condition, an explanation of why the proposed variation may be clinically superior to the first drug. |
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http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=316&showFR=1
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| | Providing Treatments for Rare Diseases: Is the Orphan Drug Act Enough |
| | Once a drug receives designation, it is eligible to receive: grants, tax reductions, guidance to facilitate development, accelerated procedures on new drug approval applications, a ten year period of exclusivity of the collected data from practical medical use and a ten year term for re-examination. | | | Surprisingly, the FDA also opposed any new legislation and stated the problem could be fixed by relaxing the approval process specifically for orphan drugs due to their unique and special need. | | | This is also partially due to the lack of sponsorship, or no one to “adopt” the orphan drug, to conduct the necessary testing to obtain the necessary approval from the Food and Drug Administration (“FDA”). |
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http://www.cwru.edu/med/epidbio/mphp439/Orphan_Drug.htm
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| | STOA: Orphan Drugs |
| | The lack of orphan drugs status in Europe lead patients affected by rare diseases to wait that the drugs already sold in the USA or in Japan, are available in their countries, or that a European company can develop such products. | | | The extension of the orphan drug status to tropical disease treatments (in Europe only) will facilitate the emergence of new important medicines, even if it has a marginal impact on drug financing in the developing world. | | | Patient associations have a strong role and are influential in the development of orphan drugs. |
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http://www.europarl.eu.int/stoa/publi/167780/default_en.htm
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| | [No title] |
| | In the context of antibody therapeutics for orphan drugs, under this approach clinical superiority would be the means by which developers of second antibody therapeutics to the same antigen could prove their antibody sufficiently different to warrant approval. | | | The problem is how to provide sufficient protection for pioneering orphan drugs to stimulate the development of new drugs for smaller patient populations, while not so broadly protecting orphan drugs as to preclude additional therapeutics that make new contributions to the treatment of those diseases. | | | The principal and recurrent problem has been determining when a second applicant for the approval of a drug to treat an orphan disease (or “indication”) is seeking approval for the “same” drug as a previously approved orphan drug. |
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http://www.stlr.org/html/volume5/bohrer.txt
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| | [No title] |
| | Moreover, a drug which receives an orphan drug approval for one disease can be prescribed for others, a practice that some companies encourage. | | | Concern about patent protection undoubtedly is a major factor in drug development decisions, and unpatentability was often mentioned as one deterrent to orphan drug development. | | | The 200,000 target for the client population is extremely high when one considers that some drugs with client populations less than 10,000 have several firms competing vigorously to win FDA races for orphan drug approval. |
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http://www.cptech.org/ip/health/orphan/orphan92.html
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| | Cancer-Pain Remedy Wins Orphan-Drug Status |
| | The Food and Drug Administration has granted orphan drug status to methylnaltrexone, a medication that blocks the side effects of morphine without interfering with pain relief. | | | UR Labs, a privately held corporation active in drug development, has been working with the University of Chicago to develop methylnaltrexone for use in patients with cancer pain. | | | Roizen and colleague Joseph Foss, M.D., assistant professor of anesthesia and critical care, have done the preliminary animal and clinical testing of the drug. |
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http://www.eurekalert.org/pub_releases/1996-07/UoC-CRWO-150796.php
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| | The Orphan Drug Act (as amended) |
| | (C) if a license for such drug is issued under section 351 of the Public Health Service Act, the approval, certification, or license would be for use for such disease or condition, the Secretary shall designate the drug as a drug for such disease or condition. | | | (7) reorganize the efforts of public and private entities and individuals in seeking the development of drugs for rare diseases or conditions and in developing such drugs. | | | (b) The function of the Board shall be to promote the development of drugs and devices for rare diseases or conditions and the coordination among Federal, other public, and private agencies in carrying out their respective functions relating to the development of such articles for such diseases or conditions. |
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http://www.fda.gov/orphan/oda.htm
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| | Drug Information Journal: Orphan drug programs/policies in Australia, Japan, and Canada |
| | An orphan drug is defined as a medicinal product where there is such a small patient group that there is no reasonable expectation that the cost of researching, registering, and making the drug available would be recovered by potential sales. | | | In developing concepts for the orphan drug program, it was determined that the most important health issues for Australians fell into a number of key areas where, from a public health perspective, there were significant gaps in available therapeutics (Table 1). | | | The concept of an orphan drug program in Australia was first considered in 1991, when a landmark review of the Australian drug evaluation process was undertaken. |
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http://www.findarticles.com/p/articles/mi_qa3899/is_200101/ai_n8949849
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| | Medical Dictionary - Surgical Glossary - O |
| | O-Vax granted orphan drug designation for use as an adjuvant therapy in the treatment of ovarian cancer in September 2000. | | | Product is currently being evaluated in phase III trials for the treatment of intestinal graft-versus-host disease (GVHD) and is a designated orphan drug for (1) the prevention of GVHD and (2) the treatment of intestinal GVHD, e.g., following bone marrow transplant. | | | Injectable, nonresorbable, bone-bonding cement is beginning clinical trials in France for use in vertebroplasty, a newly developed procedure for the treatment of spinal compression fractures that result from osteoporosis or carcinoma. |
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http://www.mtdesk.com/o.shtml
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| | Antigenics' Oncophage (HSPPC-96) Receives Orphan Drug Status in Melanoma |
| | The orphan drug program is intended to encourage research, development and approval of products for diseases that affect fewer than 200,000 patients in the United States. | | | In addition to potential market exclusivity, orphan drug status provides tax incentives for up to 50 percent of a company’s investment in US clinical research, available funding to support clinical trials, and study design assistance. | | | “Beyond the fundamental advantages, what encourages us most about our latest orphan drug designation is the continued recognition that our flagship cancer product has the potential to change the lives of patients facing such a devastating disease,” said Garo H Armen, PhD, chairman and CEO of Antigenics. |
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http://www.antigenics.com/news/2002/0723.phtml
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| | Issues related to the Orphan Drug Act |
| | The Orphan Drug Act has been written so that a very wide range of drugs quality as Orphans, including, for example, all AIDS medicines in the United States, plus countless other severe illnesses. | | | Firms spent $7.7 million on clinical research per orphan drug approval in 1997. | | | CPT Table on USFDA Orphan Drug Approvals 1999. |
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http://www.cptech.org/ip/health/orphan
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| | cannabisnews.com: MAPS Announce Orphan Drug Designation for Cannabis |
| | Orphan Drug designation is merely the first step in a very long process. | | | MAPS is the first organization to use the Orphan Drug program to help facilitate research into any medical use of marijuana. | | | As a non-profit organization, MAPS does not face such pressure and will therefore focus on facilitating research with the marijuana plant. |
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http://www.cannabisnews.com/news/thread3246.shtml
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| | Pancreatic Cancer Drug Awarded Orphan Drug Status |
| | Lorus is a biopharmaceutical company focused on the research and development of cancer therapies. | | | The compound is in development as second line therapy for advanced pancreatic cancer patients who are refractory or intolerant to conventional first line therapies. | | | The drug is made by Lorus Therapeutics Inc. |
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http://unisci.com/stories/20011/0221016.htm
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| | Orphan drug program |
| | The orphan drug program and improving community access to effective drugs for rare diseases | | | Options for improving community access to effective drugs for rare diseases | | | Home > Prescription medicines > Orphan drug program review |
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http://www.tga.gov.au/docs/html/orphan.htm
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| | BioPortfolio Limited |
| | Table 3.4 - Rituxan (rituximab) - drug monograph | | | Table 3.6 - Neupogen (filgrastim) - drug monograph | | | Table 3.10 - Taxol (paclitaxel) - drug monograph |
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http://www.bioportfolio.com/cgi-bin/acatalog/info_884.html
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| | BW Online December 13, 2002 Biotechs Adopt the Orphan-Drug Market |
| | But as Genzyme is proving with Cerezyme, there may be gold in niche drugs. | | | Thanks to so-called orphan-drug laws, it may turn out that the rarest diseases could prove to be the most profitable. | | | Still, investors should take note that as competition grows more intense, Genzyme's plan to develop additional orphan drugs becomes riskier. |
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http://www.businessweek.com/technology/content/dec2002/tc20021213_0830.htm
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| | BioPortfolio Limited |
| | Graph 10.1 Medical Reimbursement for Orphan Drugs, 2001-2009 | | | Table 9.3 Growth Rates for the Leading Orphan Drug Therapeutic Categories, 2003-2009 | | | Graph 4.3 Top Five Therapeutic Categories for Orphan Drugs by Number of Orphan Indications, 2003 |
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http://www.bioportfolio.com/cgi-bin/acatalog/info_877.html
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| | Orphan Drug Designation Received |
| | The Office of Orphan Products Development also administers a clinical research grants program, whereby researchers may compete for funding to conduct clinical trials to support the approval of drugs for rare diseases. | | | Terms and conditions, including restrictions on redistribution, apply. | | | Amarillo Biosciences has been granted Orphan Drug status previously for orally-administered interferon alpha for the treatment of Behcet's disease and a meeting with FDA, regarding Behcet's disease, has been requested. |
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http://www.prnewswire.com/cgi-bin/stories.pl?ACCT=104&STORY=/www/story/08-22-2000/0001295497&EDATE=
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| | Orphan Drug Approval |
| | The full text of the Orphan Drug Regulation. | | | Press > Valuable links > Orphan Drug Approval | | | Orphan Drug Implementing Regulation: Definitions of Clinical Superiority and Similarity. |
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http://www.epposi.org/approval.html
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| | PharmaMar - Acerca del cáncer - Orphan Drug |
| | Orphan legislation also exists in Japan and Australia but for practical reasons only EU and the US are discussed. | | | Orphan medicinal products are drugs for diagnosing, preventing or treating life-threatening or very serious conditions that are rare. | | | Orphan medicinal product designation may be obtained at any stage of the development. |
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http://www.pharmamar.com/en/cancer/orphan.cfm
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| | Technical Issues |
| | The FDA would review more resources and the drug companies were promised smaller time periods for review of their drug applications(Meadows). | | | Patients characterized with life-threading illnesses don’t have time to wait. | | | However, since 1983, there have been more than 200 orphan drugs produced to treat rare diseases. |
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http://www.geocities.com/ketchyup/technical.html
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| | Definition: orphan drug designation |
| | The US Food and Drug Administration’s orphan drug program is intended to encourage research, development and approval of products that affect fewer than 200,000 patients in the United States. | | | Oncophage has been granted orphan drug designation in kidney cancer and metastatic melanoma. | | | In addition to seven years of potential market exclusivity following marketing approval, orphan drug designation provides tax incentives for up to 50 percent of a company’s investment in US clinical research, available funding to support clinical trials, and study design assistance. |
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http://www.antigenics.com/glossary/definition.phtml?word=317
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| | Squalamine Orphan Drug Designation - Press Release |
| | There is a very substantial unmet medical need and market opportunity for an effective and well-tolerated treatment of advanced ovarian cancer. | | | Squalamine, an anti-angiogenic drug, is currently in clinical development for the treatment of solid tumors in conjunction with chemotherapy. | | | Orphan Drug designation is granted to applicants when the prevalence of the disease occurs in less than 200,000 patients in the Unites States. |
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http://www.genaera.com/pressreleases/2001_may30.htm
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| | OHSU News Release: FDA Orphan Drug Program |
| | "Pre-clinical animal studies are being completed in anticipation of submitting an Investigational New Drug application to the FDA." | | | The clinical effectiveness of these drugs is marginal and patients often have significant side effects. | | | Multiple sclerosis drug developed by Virogenomics and OHSU/VA scientists |
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http://www.ohsu.edu/news/2003/051303fda.html
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| | AllRefer.com - orphan drug (Pharmacology) - Encyclopedia |
| | You are here : AllRefer.com > Reference > Encyclopedia > Pharmacology > orphan drug | | | orphan drug, drug developed under the U.S. Orphan Drug Act (1983) to treat a disease that affects fewer than 200,000 people in the United States. | | | Since the 1983 act went into effect, many orphan drugs have been approved, including those for the treatment of such conditions as AIDS, cystic fibrosis, blepharospasm (uncontrolled rapid blinking), and snake bite. |
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http://reference.allrefer.com/encyclopedia/O/orphandru.html
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| | List of Orphan Drug and Orphan Device |
| | Either there is no alternative drug and other intervention, or it is expected to have much higher efficacy or safety compared to existing drugs and interventions. | | | Orphan products, defined as drugs and medical devices for rare diseases, are hard to develop because of their low profitability. | | | Vernal keratoconjonctivis (in those patients whose symptoms can not be adequately controlled by anti-allergic drugs) |
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http://orphanet.infobiogen.fr/docs/japan.html
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| | Definition of Orphan Drug |
| | Orphan drug status gives the manufacturer specific financial incentives to provide the drug. | | | --> Definition: A category created by the Food and Drug Administration for medications used to treat diseases that occur rarely (less than 200,000 cases) or where is no hope for recovery of development costs, so there is little financial incentive for industry to develop them. | | | Orphan drugs are controlled by the FDA's Office of Orphan Products Development (OOPD). |
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http://www.phoenix5.org/glossary/Orphan_Drug.html
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| | Orphan Drug Status |
| | For more information about the Orphan Drug program and the Orphan Drug Act (ODA), go to the Food and Drug Administration's | | | We have completed the review of this application and have determined that beta alethine qualifies for orphan drug designation for the treatment of metastatic melanoma. | | | Reference is made to your orphan drug application of January 20, 1997 submitted pursuant to section 526 of the Federal Food, Drug, and Cosmetic Act for the designation of Beta LT™ (beta alethine) as an orphan drug (application 97-1033). |
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http://www.ltpharma.com/Orphan.html
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| | Genmab's HuMax-CD4 Receives U.S. Orphan Drug Designation newratings.com |
| | expectation that the cost of developing and making available a drug for such | | | may also receive orphan drug designation if it is used to treat a disease | | | Lymphomas (CTCL), has been designated an orphan drug by the U.S. Food and |
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http://www.newratings.com/analyst_news/article_491198.html
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| | Ophthalmology Times - Microplasmin receives orphan drug protection |
| | Please send any technical comments or questions to our webmaster. | | | The drug is injected to induce posterior vitreous detachment and facilitate vitrectomy in children, in whom the vitreous is particularly adherent, which makes surgery rife with complications. | | | In addition to use in vitrectomies, the drug may help prevent the progression of diabetic retinopathy and protect against diabetic macular edema. |
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http://www.ophthalmologytimes.com/ophthalmologytimes/article/articleDetail.jsp?id=94038
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| | Orphan Drug Production |
| | Each section has access to the searchable database of rare diseases, which contains an article written by professionals, information about specialised outpatient clinics, diagnostic laboratories, current research projects and support groups (currently the database only includes data on French outpatient clinics and French clinical and research laboratories). | | | Professionals can also gain information about orphan drugs, which "are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions." Support groups and professional organisations are given the opprtunity to create and maintain their own Web site from this site. | | | ORPHANET is a relational database of 1150 rare diseases and orphan drugs. |
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http://omni.ac.uk/browse/mesh/D009965.html
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| | YM BioSciences' TheraCIM Granted US Orphan Drug Status- EGFr.. |
| | The U.S. Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. | | | Head and neck cancer Phase II trial results, reported in the Journal of Clinical Oncology, showed that the drug doubles a patient's response to radiation treatment. | | | YM BioSciences Inc. is a cancer drug development company. |
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http://www.amex.com/newsDetails/CmnNewsDet.jsp?id=XpressFeed_NewsDetails_1101816200179.html
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| | Canadian Organization for Rare Disorders - Annual General Meeting Invitation |
| | information on diagnoses, treatment, medications and drug trials. | | | Canada requires a national Orphan Drug Policy (ODP) to ensure patients with orphan diseases are treated equally to those with more common illnesses. | | | Today there are drugs and treatments available even for those suffering from an ultra orphan disease such as Fabry and MPS1, however Canadian patients can not access these life saving drugs because individuals afflicted with these rare diseases are not covered under the Canada Health Act. |
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http://www.cord.ca/odp.asp
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| | orphan disease /drug |
| | rare diseases and the drugs and other products related to them, so called because development is not profitable; note that this term has nothing to do with orphan children. |
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http://crisp.cit.nih.gov/Thesaurus/00005865.htm
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| | Orphan drug finds home |
| | The current cost of treatment per patient is US$700. | | | Earlier this year, WHO established a Human African Trypanosomiasis Network which is to "monitor drug resistance and find and recommend solutions for the treatment of sleeping sickness". | | | One of the Network's working groups – Drugs Availability and Affordability – is chaired by a nongovernmental organization Médecins sans Frontières (MSF). |
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http://www.who.int/inf-pr-1999/en/pr99-74.html
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| | Medicare Slashes Orphan Drug Reimbursements |
| | Since treatment may cost thousands of dollars per year for just one drug, people in need of orphan drugs are concerned that reduced reimbursement might limit access to those who can afford to pay some or all of the cost. | | | Also, health care providers may stop treating people with orphan drugs if the difference between actual cost and insurance reimbursement is too great. | | | As an example, Medicare states that Allergan, the maker of Botox, has found a very lucrative market for its drug beyond relieving the symptoms of dystonia, its original purpose, so Botox no longer needs special price treatment. |
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http://www.malattiemetaboliche.it/articoli/medicare_slashes_orphan.htm
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| | Office of Orphan Products Development |
| | Office of Orphan Products Development (OOPD) has been dedicated to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions since it was created in 1982. | | | OOPD interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups. | | | The ODA has been very successful - more than 200 drugs and biological products for rare diseases have been brought to market since 1983. |
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http://www.fda.gov/orphan
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| | FDA Grants Encysive Orphan Drug Designation For Thelin |
| | The U.S. Orphan Drug Act of 1983 provides incentives for companies that are developing and marketing therapies for diseases and disorders that affect fewer than 200,000 people in the United States. | | | Encysive Pharmaceuticals Inc., a biopharmaceutical company focused on the discovery, development and commercialization of novel drugs, is recognized for our expertise in small molecule drug development and vascular biology. | | | Following FDA approval, the Orphan Drug Act provides a drug market exclusivity for a particular indication for a period of seven years. |
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http://www.primezone.com/newsroom/news_releases.mhtml?d=67353
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| | BehaveNet® Clinical Capsule: orphan drug |
| | According to the FDA, "The term "orphan drug" refers to a product that treats a rare disease affecting fewer than 200,000 Americans." | | | The Orphan Drug Act and Related Law and Regulation |
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http://www.behavenet.com/capsules/pharm/orphan.htm
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| | Myogen's Ambrisentan granted orphan drug designation by Europeans |
| | Arginox Receives Orphan Drug Designation For Cardiogenic Shock Drug | | | Myogen today announced that the Commission of the European Communities, with a favorable opinion of the Committee for Orphan Medicinal Products of the European Medicines Agency (EMEA), has granted orphan drug designation to ambrisentan for the treatment of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hyperten... | | | CytRx Corporation today announced that the U.S. Food and Drug Administration (FDA) has granted "Fast Track" designation for the company's leading drug candidate arimoclomol for the treatment of... |
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http://www.medical-buzz.com/v66492.html
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