An orphan drug is a 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 of the country.
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency (EMEA).
Vertex Pharmaceuticals is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in South Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.
BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I, by manufacturing laronidase. BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU).
Astellas Pharma Inc. is a Japanese multinational pharmaceutical company, formed on 1 April 2005 from the merger of Yamanouchi Pharmaceutical Co., Ltd. and Fujisawa Pharmaceutical Co., Ltd.. On February 5, 2020, the company announced management changes effective from April 1, 2020.
The Drugs for Neglected Diseases initiative (DNDi) is a collaborative, patients' needs-driven, non-profit drug research and development (R&D) organization that is developing new treatments for neglected diseases, notably leishmaniasis, sleeping sickness, Chagas disease, malaria, filarial diseases, mycetoma, paediatric HIV, cryptococcal meningitis, hepatitis C, and dengue. DNDi's malaria activities were transferred to Medicines for Malaria Venture (MMV) in 2015.
Almirall, S.A. is a Spanish pharmaceutical company, with headquarters in Barcelona, founded in 1943.
Eculizumab, sold under the brand name Soliris among others, is a recombinant humanized monoclonal antibody used to treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis, and neuromyelitis optica. In people with PNH, it reduces both the destruction of red blood cells and need for blood transfusion, but does not appear to affect the risk of death. Eculizumab was the first drug approved for each of its uses, and its approval was granted based on small trials. It is given by intravenous infusion.
The Canadian Agency for Drugs and Technologies in Health, or CADTH, is a Canadian national organisation that provides research and analysis to healthcare decision-makers.
Lieve Fransen is a senior adviser to the European Policy Centre on health, social and migration policies, and published studies on investing in social infrastructure, energy poverty and social investment. Between 2011 and 2015 she was the social policies director in the Directorate for Employment and Social affairs for the European Commissionin charge of social policies, poverty eradication, pensions, health and social protection. Before that she was director for communication and representations in the EC's communication directorate for more than 500 networks across the European Union and from 1987 till 1997 she was head of unit for human development in the European Commissions department for development.
The European Cooperation in Science and Technology is running an EU-funded programme which enables researchers and innovators to set-up their own research networks in a wide range of scientific topics, called COST Actions. While COST does not fund research activities as such, it provides funding for scientific collaboration in the form of conferences, meetings, training schools and scientific exchanges.
Health technology assessment (HTA) is a multidisciplinary process that uses systematic and explicit methods to evaluate the properties and effects of a health technology. Health technology is conceived as any intervention at any point in its lifecycle. HTA aim is to inform "decision-making in order to promote an equitable, efficient, and high-quality health system". It has other definitions including "a method of evidence synthesis that considers evidence regarding clinical effectiveness, safety, cost-effectiveness and, when broadly applied, includes social, ethical, and legal aspects of the use of health technologies. The precise balance of these inputs depends on the purpose of each individual HTA. A major use of HTAs is in informing reimbursement and coverage decisions by insurers and national health systems, in which case HTAs should include benefit-harm assessment and economic evaluation." And "a multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. Its aim is to inform the formulation of safe, effective, health policies that are patient focused and seek to achieve best value. Despite its policy goals, HTA must always be firmly rooted in research and the scientific method".
Christopher Fearne is a Maltese physician and politician. He was appointed Parliamentary Secretary for Health in April 2014 and Minister for Health since April 2016. In July 2017, the Labour Party elected him as Deputy Leader for Parliamentary Affairs, thus assuming the role of Deputy Prime Minister of Malta and Leader of the House.
Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder.
The Health Intervention and Technology Assessment Program (HITAP) is a semi-autonomous research unit under Thailand’s Ministry of Public Health. It was established in 2007 as a non-profit organization in order to take responsibility for appraising a wide range of health technologies and programs, including pharmaceuticals, medical devices, interventions, individual and community health promotion, and disease prevention as well as social health policy to inform policy decisions in Thailand. HITAP assumes an advisory role to health governmental authorities by providing rigorous scientific evidence through professional assessment of health data in support of public decision-making. These assessments cover a range of topics including system design, selection of technologies for assessment, and the actual assessment of those selected and agreed upon by relevant government agencies. In this effort, HITAP publishes research and studies in the following areas: methodological development, databases and guidelines; knowledge transfer and exchange (KTE) and capacity development; technology assessments on drugs, medical devices, medical procedures, disease prevention and health promotion measures; benefit packages of care – mixing screening and treatments; and other public health policies, e.g. evaluation of Thailand’s government compulsory license policy.
Digital therapeutics, a subset of digital health, are evidence-based therapeutic interventions driven by high quality software programs to prevent, manage, or treat a medical disorder or disease. Digital therapeutic companies should publish trial results inclusive of clinically meaningful outcomes in peer-reviewed journals. The treatment relies on behavioral and lifestyle changes usually spurred by a collection of digital impetuses. Because of the digital nature of the methodology, data can be collected and analyzed as both a progress report and a preventative measure. Treatments are being developed for the prevention and management of a wide variety of diseases and conditions, including type 1 & type II diabetes, congestive heart failure, obesity, Alzheimer's disease, dementia, asthma, substance abuse, ADHD, hypertension, anxiety, depression, and several others. Digital therapeutics often employ strategies rooted in cognitive behavioral therapy.
Brexit and arrangements for science and technology refers to arrangements affecting scientific research, experimental development and innovation that are within the scope of the negotiations between the United Kingdom and the European Union on the terms of Britain's withdrawal from the European Union (EU).
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein.
Health Action International (HAI) is a non-profit organization based in The Netherlands. Established in 1981, HAI works to expand access to essential medicines through research, policy analysis and intervention projects. The organization focuses on snakebite envenoming, access to insulin and developing European policies on medicines. HAI is listed by the World Health Organization (WHO) as an official non-state actor.
Horizon scanning (HS) or horizon scan is a method from futures studies, sometimes regarded as a part of foresight. It is the early detection and assessment of emerging technologies or threats for mainly policy makers in a domain of choice. Such domains include agriculture, environmental studies, health care, biosecurity, and food safety.
