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Accelerated Approval (FDA)
The United States Food and Drug Administration (FDA) initiated the FDA Accelerated Approval Program in 1992 to allow faster approval of drugs for serious conditions that fill an unmet medical need. The faster approval relies on use of surrogate endpoints. Drug approval typically requires clinical trials with endpoints that demonstrate a clinical benefit, such as increased survival for cancer patients. Drugs with accelerated approval can initially be tested in clinical trials that use a surrogate endpoint, or something that is thought to predict clinical benefit. Surrogate endpoints typically require less time, and in the case of a cancer patient, it is much faster to measure a reduction in tumor size, for example, than overall patient survival. Drugs approved under the FDA Accelerated Approval Program still need to be tested in clinical trials using endpoints that demonstrate clinical benefit, and those trials are known as phase 4 confirmatory trials. If the drug later proves unabl ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Surrogate Endpoints
In clinical trials, a surrogate endpoint (or surrogate marker) is a measure of effect of a specific treatment that may correlate with a ''real'' clinical endpoint but does not necessarily have a guaranteed relationship. The National Institutes of Health (USA) defines surrogate endpoint as "a biomarker intended to substitute for a clinical endpoint". Surrogate markers are used when the primary endpoint is undesired (e.g., death), or when the number of events is very small, thus making it impractical to conduct a clinical trial to gather a statistically significant number of endpoints. The FDA and other regulatory agencies will often accept evidence from clinical trials that show a direct clinical benefit to surrogate markers. Surrogate endpoints can be obtained from different modalities, such as, behavioural or cognitive scores, or biomarkers from Electroencephalography ( qEEG), MRI, PET, or biochemical biomarkers. A correlate does not make a surrogate. It is a common misconception ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
United States Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Phases Of Clinical Research
The phases of clinical research are the stages in which scientists conduct experiments with a health intervention to obtain sufficient evidence for a process considered effective as a medical treatment. For drug development, the clinical phases start with testing for drug safety in a few human subjects, then expand to many study participants (potentially tens of thousands) to determine if the treatment is effective. Clinical research is conducted on drug candidates, vaccine candidates, new medical devices, and new diagnostic assays. Description Clinical trials testing potential medical products are commonly classified into four phases. The drug development process will normally proceed through all four phases over many years. When expressed specifically, a clinical trial phase is capitalized both in name and Roman numeral, such as "Phase I" clinical trial. If the drug successfully passes through Phases I, II, and III, it will usually be approved by the national regulatory aut ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Confirmatory Trial
A confirmatory trial is an adequately controlled trial where hypotheses are stated in advance and evaluated according to a protocol. This type of trial may be implemented when it is necessary to provide additional or firm evidence of efficacy or safety. The mechanism of the trial implements a key hypothesis of interest which is rigorously tested at the end of the confirmatory trial and directly follows the predefined primary objective of the trial. Of importance in a confirmatory trial is the process of estimating with due precision potential effects attributable to the treatment, the quantity of effects and relating these effects to their clinical significance. According to the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use: "Confirmatory Trials are intended to provide firm evidence in support of claims and hence adherence to protocols and standard operating procedures A standard operating procedure (SOP) is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
JAMA
''JAMA'' (''The Journal of the American Medical Association'') is a peer-reviewed medical journal published 48 times a year by the American Medical Association. It publishes original research, reviews, and editorials covering all aspects of biomedicine. The journal was established in 1883 with Nathan Smith Davis as the founding editor. Kirsten Bibbins-Domingo of the University of California San Francisco became the journal editor-in-chief on July 1, 2022, succeeding Howard Bauchner of Boston University. According to ''Journal Citation Reports'', the journal's 2024 impact factor is 63.1, ranking it 4th out of 168 journals in the category "Medicine, General & Internal". History The journal was established in 1883 by the American Medical Association and superseded the ''Transactions of the American Medical Association''. ''Councilor's Bulletin'' was renamed the ''Bulletin of the American Medical Association'', which later was absorbed by the ''Journal of the American Medical ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Aduhelm
Aducanumab, sold under the brand name Aduhelm, is an anti-amyloid drug designed to treat Alzheimer's disease. It is a monoclonal antibody that targets aggregated forms (plaque) of amyloid beta (Aβ) found in the brains of people with Alzheimer's disease to reduce its buildup. It was developed by Biogen and Eisai. Aducanumab is given via intravenous infusion. Aducanumab was approved for medical use in the United States by the Food and Drug Administration (FDA) in June 2021, in a controversial decision that led to the resignation of three advisers to the FDA in the absence of evidence that the medication is effective. The FDA stated that it represents a first-of-its-kind treatment approved for Alzheimer's disease and that it is the first new treatment approved for Alzheimer's since 2003. Aducanumab's approval is controversial for numerous reasons including ambiguous clinical trial results regarding efficacy, the high cost of the medication and the very high rate of serious adv ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Biogen
Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, United States specializing in the discovery, development, and delivery of the treatment of neurological diseases to patients worldwide. Biogen operates in Argentina, Brazil, Canada, China, France, Germany, Hungary, India, Italy, Japan, Mexico, Netherlands, Poland, Sweden, and Switzerland. History Biogen was founded in 1978 in Geneva as ''Biotechnology Geneva'' by several prominent biologists, including Kenneth Murray from the University of Edinburgh, Phillip Allen Sharp from the Massachusetts Institute of Technology, Walter Gilbert from Harvard University (Gilbert served as CEO during the start-up phase of Biogen), Heinz Schaller from the University of Heidelberg, and Charles Weissmann from the University of Zurich (Weissmann contributed the first product interferon alpha).Werner Grundlehner''Zürcher Antikörper gegen Alzheimer hat Milliardenpotenzial – und Gegenwind.''Neu ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
The Hill (newspaper)
''The Hill'', formed in 1994, is an American newspaper and digital media company based in Washington, D.C. Focusing on politics, policy, business and international relations, ''The Hill''s coverage includes the U.S. Congress, the presidency and executive branch, and election campaigns. Its stated output is "nonpartisan reporting on the inner workings of Government and the nexus of politics and business". The company's primary outlet is ''TheHill.com''. ''The Hill'' is additionally distributed in print for free around Washington, D.C., and distributed to all congressional offices. It has been owned by Nexstar Media Group since 2021. In 2020, ''The Hill'' was ranked second for online politics readership across all news sites, behind only CNN, remaining ahead of ''Politico'', Fox News, NBCNews.com, and MSNBC. ''The Hill'' had around 32 million monthly viewers in 2023. History Founding and early years The company was formed as a newspaper in 1994 by Democratic power broker ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Fast Track (FDA)
Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need. Fast track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and attempt to make a decision within sixty days. Purpose Fast track is one of five FDA approaches to make new drugs available as rapidly as possible: the others are priority review, breakthrough therapy, accelerated approval and regenerative medicine advanced therapy. Fast track was introduced by the FDA Modernization Act of 1997. Requirements Fast track designation is designed to aid in the development and expedite the review of drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need. Serious condition: det ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug
An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug medical research, research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to en ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Concise Encyclopedia Of Economics
Liberty Fund, Inc. is an American nonprofit foundation headquartered in Carmel, Indiana, that promotes the libertarian views of its founder, Pierre F. Goodrich, through publishing, conferences, and educational resources. The operating mandate of the Liberty Fund was set forth in an unpublished memo written by Goodrich "to encourage the study of the ideal of a society of free and responsible individuals".Robert T. Grimm (ed.), ''Notable American Philanthropists: Biographies of Giving and Volunteering'', Greenwood Publishing Group, 2002, pp. 125–128 History Liberty Fund was founded by entrepreneur Pierre F. Goodrich in 1960. Goodrich, "one of the richest men in Indiana", was involved with coal mines, corn production, telecommunications, and securities. Goodrich was a member of the neoliberal or classically liberal Mont Pelerin Society, an international organization of academics, intellectuals, and business leaders who advocated free market economic policies. Goodrich was al ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
