|
Ultra-rare Disease
An ultra-rare disease is a disease that affects an extremely small percentage of the population. In some parts of the world, an ''ultra-orphan disease'' is a rare disease whose rarity means there is a lack of a market large enough to have support and resources for discovering treatments for it. Distinct countries define and provide special economic incentives for companies developing drugs that treat ultra-rare diseases. In 2018, the Scottish Government introduced a new definition of 'ultra-orphan medicines' that can treat very rare conditions affecting fewer than 1 in 50,000 people – around 100 people or less in Scotland. The new definition also allows the Scottish Medicines Consortium (SMC) the ability to treat some medicines for rare orphan diseases as ultra-orphan medicines. The changes mean if the medicine meets the new definition of an ultra-orphan medicine and the SMC consider it clinically effective, then it will be made available on the NHS for at least three years while i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Disease
A disease is a particular abnormal condition that adversely affects the structure or function (biology), function of all or part of an organism and is not immediately due to any external injury. Diseases are often known to be medical conditions that are associated with specific signs and symptoms. A disease may be caused by external factors such as pathogens or by internal dysfunctions. For example, internal dysfunctions of the immune system can produce a variety of different diseases, including various forms of immunodeficiency, hypersensitivity, allergy, allergies, and autoimmune disorders. In humans, ''disease'' is often used more broadly to refer to any condition that causes pain, Abnormality (behavior), dysfunction, distress (medicine), distress, social problems, or death to the person affected, or similar problems for those in contact with the person. In this broader sense, it sometimes includes injury in humans, injuries, disability, disabilities, Disorder (medicine) ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Rare Disease
A rare disease is any disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease whose rarity results in little or no funding or research for treatments, without financial incentives from governments or other agencies. Orphan drugs are medications targeting orphan diseases. Most rare diseases are genetic in origin and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthdays. Fields condition is considered the rarest known disease, affecting three known individuals, two of whom are identical twins. With four diagnosed patients in 27 years, ribose-5-phosphate isomerase deficiency is considered the second rarest. While no single number has been agreed upon for which a disease is considered rare, several efforts have been undertaken to ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Scottish Medicines Consortium
Healthcare Improvement Scotland (HIS) () is the national healthcare improvement organisation for Scotland. It is a public body which is part of the Scottish National Health Service, created in April 2011. History NHS Quality Improvement Scotland (NHS QIS) was established on 1 January 2003 as a special health board with a remit to improve the quality of healthcare in Scotland. Healthcare Improvement Scotland (HIS) was established by the Public Services Reform (Scotland) Act 2010, taking over the work of QIS and the regulatory functions, in regard to independent healthcare provision, previously conducted by the Care Commission, now renamed the Care Inspectorate. The first chair of HIS, serving from 2010 to 2018, was Dame Denise Coia. The function of this body is to implement the healthcare priorities of the Scottish Government, in particular the Healthcare Quality Strategy of NHS Scotland. Units Healthcare Improvement Scotland incorporates several organisations: * Healthcare E ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Institute For Clinical And Economic Review
The Institute for Clinical and Economic Review (ICER) is a Boston-based independent nonprofit organization that seeks to place a value on medical care by providing comprehensive clinical and cost-effectiveness analyses of treatments, tests, and procedures. ICER was founded in about 2005 by physician-researcher Steven D. Pearson. Until 2014 it concentrated on assessing health care costs (rather than evaluating drugs). It evaluates the cost-effectiveness of drugs in a similar way to the UK's NICE, and has come under some criticism from the drug industry. ICER has placed a monetary value on several prescription drugs since 2014. Insurers have used those evaluations to justify which drugs are approved or denied. The institution is funded by Arnold Ventures LLC (formerly the Laura and John Arnold Foundation), drug makers, insurers, and government grants. Threshold cost criteria ICER uses a “value assessment framework” to decide prices for select medical treatments. The framework wa ... [...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] |
