Prescription drug list prices in the United States continually are among the highest in the world. The high cost of prescription drugs became a major topic of discussion in the 21st century, leading up to the American health care reform debate of 2009, and received renewed attention in 2015. One major reason for high prescription drug prices in the United States relative to other countries is the inability of government-granted monopolies in the American health care sector to use their bargaining power to negotiate lower prices, and the American payer ends up subsidizing the world's R&D spending on drugs.
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 Merck Group, branded and commonly known as Merck, is a German multinational science and technology company headquartered in Darmstadt, with about 60,000 employees and a presence in 66 countries. The group includes around 250 companies; the main company is Merck KGaA in Germany. The company is divided into three business lines: Healthcare, Life Sciences and Electronics. Merck was founded in 1668 and is the world's oldest operating chemical and pharmaceutical company, as well as one of the largest pharmaceutical companies globally.
Gilead Sciences, Inc. is an American biopharmaceutical company headquartered in Foster City, California that focuses on researching and developing antiviral drugs used in the treatment of HIV/AIDS, hepatitis B, hepatitis C, influenza, and COVID-19, including ledipasvir/sofosbuvir and sofosbuvir. Gilead is a member of the NASDAQ Biotechnology Index and the S&P 500.
Servier Laboratories is an international pharmaceutical company governed by a non-profit foundation, with its headquarters in France (Suresnes).
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis is the assessment that a particular medical condition is present while an indication is a reason for use. The opposite of an indication is a contraindication, a reason to withhold a certain medical treatment because the risks of treatment clearly outweigh the benefits.
Amifampridine is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and Lambert–Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ad hoc forms of the drug, since there was no marketed form.
The following outline is provided as an overview of and topical guide to clinical research:
The National Organization for Rare Disorders (NORD) is an American non-profit organization aiming to provide support for individuals with rare diseases by advocating and funding research, education, and networking among service providers. It was founded in 1983 by Abbey Meyers, along with individuals and rare diseases leaders of rare disease support groups, and it is a 501(c)(3) tax exempt organization.
Medicines for Malaria Venture (MMV), a not-for-profit public-private partnership, was established as a foundation in Switzerland in 1999. Its mission is to reduce the burden of malaria in disease-endemic countries by developing and facilitating the delivery of antimalarial drugs. Its vision is a world in which these innovative medicines will cure and protect the vulnerable and under-served populations at risk of malaria, and help to ultimately eradicate the disease.
Claudio Cavazza was an Italian entrepreneur, Chairman and founder of the Sigma-Tau pharmaceutical company.
Actelion is a pharmaceuticals and biotechnology company established in December 1997, headquartered in Allschwil near Basel, Switzerland.
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome or muscular dystrophy which affect small numbers of individuals residing in the United States.
Marathon Pharmaceuticals LLC was a privately held biopharmaceuticals company focused on drugs for people with rare diseases. The Illinois-based company developed and manufactured therapeutics and brought them to market. It employed 100 people in four global locations. In 2017, PTC Therapeutics acquired rights to Marathon Pharmaceuticals' drug Emflaza (deflazacort) for $140 million after criticism about their plan to sell the drug at a list price of $89,000 per year to sufferers despite the fact that the same drug was available in Canada and the UK for around $1,000 per year.
Trevor Mervyn Jones, CBE PhD DSc (Hon) FRCP FMedSci FBPhS FRSM FRSC FLSW is a visiting professor at King's College London, and a former Head of R&D, at Wellcome. He continues to have a distinguished career in the pharmaceutical and biotech industry as well as in academia.
Chiesi Farmaceutici S.p.A. is an Italian family controlled global pharmaceutical company based in Parma, Emilia-Romagna. Chiesi has 31 affiliates in the world, nearly 6,500 total employees and provides medicines to patients in 100 nations. Chiesi currently has revenues of 2.749 billion euros. According to 2020 official data from the European Patent Office (EPO), Chiesi Group, with 42 patents filed, is confirmed as the first Italian Pharmaceutical Company and third among Italian Companies across all sectors for filing the highest number of patents.
Henri A. Termeer was a Dutch biotechnology executive and entrepreneur who is considered a pioneer in corporate strategy in the biotechnology industry for his tenure as CEO at Genzyme. Termeer created a business model adopted by many others in the biotech industry by garnering steep prices— mainly from insurers and government payers— for therapies for rare genetic disorders known as orphan diseases that mainly affect children. Genzyme uses biological processes to manufacture drugs that are not easily copied by generic-drug makers. The drugs are also protected by orphan drug acts in various countries which provides extensive protection from competition and ensures coverage by publicly funded insurers. As CEO of Genzyme from 1981 to 2011, he developed corporate strategies for growth including optimizing institutional embeddedness nurturing vast networks of influential groups and clusters: doctors, private equity, patient-groups, insurance, healthcare umbrella organizations, state and local government, and alumni. Termeer was "connected to 311 board members in 17 different organizations across 20 different industries" He has the legacy of being the "longest-serving CEO in the biotechnology industry.
Specialty drugs or specialty pharmaceuticals are a recent designation of pharmaceuticals classified as high-cost, high complexity and/or high touch. Specialty drugs are often biologics—"drugs derived from living cells" that are injectable or infused. They are used to treat complex or rare chronic conditions such as cancer, rheumatoid arthritis, hemophilia, H.I.V. psoriasis, inflammatory bowel disease and hepatitis C. In 1990 there were 10 specialty drugs on the market, around five years later nearly 30, by 2008 200, and by 2015 300.
The US carries out 46% of global research and development (R&D) in the life sciences, making it the world leader in medical research.
The Pharmaceutical Accountability Foundation was established in Amsterdam in July 2018 to deal with pharmaceutical companies that demand excessive prices for medicines in the Netherlands. This followed a report by the Raad voor de Volksgezondheid en Zorg in 2017. Wilbert Bannenberg, an epidemiologist, is the chairman. The group plans to deploy both health and pharmaceutical expertise and lawyers.
