Medication
Cimetidine was the first drug ever to earn more than one billion dollars in a single year, and with it came a new word: blockbuster. A blockbuster drug is one that generates more than one billion dollars in revenue for a pharmaceutical company in a single year. Medication, also called medicine, pharmaceutical drug, or simply drug, is a drug used to diagnose, cure, treat, or prevent disease. Behind that plain definition sits an enormous machine of discovery, regulation, and money. How did a substance go from honey smeared on an infection to a product that costs roughly 1.8 billion dollars to bring to market? Why do governments fight in court over who can copy a single pill? And why are medications now described as ubiquitous in nearly every lake, river, and stream on the planet? The answers run from a Bronze Age papyrus to the effluent of modern wastewater plants.
Encyclopaedia Britannica calls medication "a substance used in treating a disease or relieving pain", but the legal definitions are far stricter and they disagree across borders. In Europe the term is "medicinal product", defined by EU law as any substance presented as having properties for treating or preventing disease in human beings, or one used to restore, correct, or modify physiological functions by pharmacological, immunological, or metabolic action. The United States writes its definition differently. There a drug is a substance, other than food, intended to affect the structure or any function of the body, or intended for the diagnosis, cure, mitigation, treatment, or prevention of disease. The US definition also folds in any substance recognized by an official pharmacopeia or formulary. Biological products sit inside that same legal umbrella, though they are made by a biological process rather than a chemical one. The National Cancer Institute catalogs the forms a medication can take: tablets, capsules, liquids, creams, and patches. A medication with no active ingredient, used only in research studies, has its own name. It is called a placebo.
The Anatomical Therapeutic Chemical Classification System, known as the ATC system, is one of the most elaborate ways the world organizes its medicines, and the World Health Organization keeps its own list of essential medicines alongside it. One of the key divisions separates traditional small molecule drugs, usually made by chemical synthesis, from biological medical products. That biological group includes recombinant proteins, vaccines, blood products such as IVIG, gene therapy, and cell therapies like stem cell treatments. Drugs can also be grouped by therapeutic function, the role they play in the body. Analgesics relieve pain, antibiotics kill or inhibit bacteria, antipyretics reduce fever, and statins lower blood cholesterol. The list runs long: anticoagulants, antidepressants, bronchodilators, diuretics, beta blockers, mood stabilizers, and vaccines each occupy their own category. Some drugs earn a label that ignores all of this. A "specialty" drug is an ill-defined class that may be hard to administer, require special handling or patient monitoring, carry particular regulatory restrictions, and tends to be expensive relative to other drugs.
Nitroglycerin placed under the tongue absorbs into the bloodstream within moments, a route called sublingual that bypasses the slow path through the stomach. The way a medication enters the body shapes how fast and how well it works. Oral administration, by tablet, capsule, or syrup, is the most common route of all. Other routes target a single spot: ophthalmic drops treat the eyes, otic drops the ear canal, and topical creams the skin. Transdermal patches push a drug through the skin for systemic effect, used for substances like fentanyl and nicotine. Then come the injections, the parenteral routes, each defined by exactly where the needle goes. Intramuscular shots into the deltoid or gluteus carry vaccines and fast-acting drugs. Subcutaneous injections into the fatty tissue deliver insulin and biologics. Intravenous lines feed straight into a vein for emergencies and chemotherapy. The map gets finer still. Intrathecal injection reaches the cerebrospinal fluid in the spinal canal, epidural injection the space around the spinal cord during childbirth, and intraosseous injection the bone marrow itself, an emergency route used in pediatrics and trauma when a vein cannot be found.
In 2010, the research and development cost of each new molecular entity was approximately 1.8 billion dollars, a figure that captures how lengthy, expensive, and inefficient drug discovery remains. Historically, drugs were found by pulling the active ingredient out of traditional remedies or by simple luck, a method known as classical pharmacology. The sequencing of the human genome changed the approach. Scientists could clone and synthesize purified proteins, then run high throughput screening of huge compound libraries against isolated biological targets, an inversion called reverse pharmacology. Now researchers can even see the shape of biological molecules at the atomic level and design candidates to fit. The economics are brutal. The "final product" of discovery is a patent, after which the drug faces very expensive Phase I, II, and III clinical trials, and most candidates fail. Small companies often play a critical role early, then sell their rights to larger firms with the resources to run those trials. Drug discovery and drug development are not the same thing; discovery identifies a new medicine, while development carries that molecule all the way into clinical practice and toward commercial launch. Partnering between companies has become a standard way to move candidates down the pipeline.
A "behind-the-counter" drug needs no prescription, yet it cannot sit on an open shelf; it must stay in the dispensary, out of public view, sold only by a pharmacist or pharmacy technician. This is one of several tiers regulators use to control risk. Over-the-counter drugs face no special restrictions because they are considered safe enough that most people will not accidentally harm themselves. Prescription drugs require a licensed practitioner because of adverse effects and contraindications, and doctors may even prescribe them off-label, for purposes the drug was never originally approved to treat. The United Kingdom adds a category of "pharmacy medicines" sold only in registered pharmacies. Above all this sits the International Narcotics Control Board of the United Nations, which imposes a world law of prohibition and publishes a long list of forbidden chemicals and plants. Pricing is regulated very differently from place to place. In the United Kingdom, the Pharmaceutical Price Regulation Scheme negotiates prices so the National Health Service can buy at reasonable rates, with a 2017 payment percentage set at 4.75 percent. Canada's Patented Medicine Prices Review Board judges whether a price is excessive, comparing it against countries including France, Germany, Italy, Sweden, Switzerland, the United Kingdom, and the United States. In the United States, drug costs are partly unregulated, set instead by negotiations between drug companies and insurance companies. There a troubling pattern holds: the number of new blockbuster drugs approved per billion dollars spent has halved every nine years since 1950.
The Kahun Gynaecological Papyrus, dating to about 1800 BC, is the oldest known medical text of any kind and records the first documented use of a drug. Egyptian medical papyri describe using honey to treat infections and the legs of bee-eaters to treat neck pains. Ancient Babylonian medicine used medicinal creams and pills in the first half of the second millennium BC. On the Indian subcontinent, the Atharvaveda described plant-based drugs, and the student of Ayurveda was expected to master ten arts, from distillation and metallurgy to sugar manufacture and the preparation of alkalis. The Greek physician Pedanius Dioscorides wrote his pharmacopoeia De materia medica between 50 and 70 CE, and it was read for more than 1,500 years. Medieval Western Europe had few truly effective drugs beyond opium and quinine, leaning instead on folklore cures and poisonous metal-based compounds. In 1842 Oliver Wendell Holmes Sr. delivered a famous verdict on the era's medicines: "if all medicines in the world were thrown into the sea, it would be all the better for mankind and all the worse for the fishes". Then the wonder drugs arrived. Antibiotics entered the medical scene in 1932 thanks to Gerhard Domagk, and sulfa drugs dropped the US pneumonia mortality rate from 0.2 percent a year to 0.05 percent by 1939. Streptomycin, found in 1942, became the first drug effective against tuberculosis.
Three months after Librium was approved, it had become the most prescribed tranquilizer in the nation, the first of a new chemical class called the benzodiazepines. Three years later Valium arrived, ten times more effective as a muscle relaxant and anti-convulsant, and the most versatile of the minor tranquilizers. The pattern of a single drug reshaping daily medicine repeats again and again. Meprobamate, the first minor tranquilizer, became the country's largest-selling prescription drug only fourteen months after release, and by 1957 the fastest-growing drug in history. Premarin, conjugated estrogens introduced in 1942, dominated hormone replacement therapy, and between 1991 and 1999 it ranked as the most popular prescription and best-selling drug in America. The first oral contraceptive, Enovid, won FDA approval in 1960, more effective than the condom or the diaphragm, and by 1982 a biphasic version appeared, followed by a triphasic pill by 1985. Ritalin, or methylphenidate, was first marketed in 1955 for narcolepsy among the middle-aged and elderly, but only in the 1980s did it reach children for hyperactivity. Today 85 percent of the world's methylphenidate is consumed in America. Mevacor became the first influential statin in the American market, joined by Pravachol in 1991 and Zocor. In 1998, Viagra was released as a treatment for erectile dysfunction.
Pharmaceutical medications are now described as ubiquitous in nearly every type of environmental medium worldwide, present in lakes, rivers, streams, estuaries, seawater, and soil at low concentrations measured in nanograms to micrograms per liter. The main route there runs through the effluent of wastewater treatment plants, both from industrial production and from municipal plants after people consume the drugs. Agricultural pollution adds another large stream, fed by the heavy use of antibiotics in livestock. Scientists judge a chemical's environmental impact by three measures: persistence, bioaccumulation, and toxicity. Most medications are bio-active and naturally degradable, yet they are called "pseudopersistent" because their sources constantly replenish them. These Environmentally Persistent Pharmaceutical Pollutants rarely reach toxic concentrations, but they have been observed to bioaccumulate in some species and compound gradually across food webs rather than striking acutely. That slow, climbing effect earned them a stark label from the US Geological Survey, which classifies them as "Ecological Disrupting Compounds".
Common questions
What is a medication and what is it used for?
A medication is a drug used to diagnose, cure, treat, or prevent disease. It is also called a medicine, pharmaceutical drug, medicinal product, or simply a drug. A medication with no active ingredient, used only in research studies, is called a placebo.
What is a blockbuster drug and which was the first one?
A blockbuster drug is a drug that generates more than one billion dollars in revenue for a pharmaceutical company in a single year. Cimetidine was the first drug ever to reach more than one billion dollars a year in sales, making it the first blockbuster drug.
How much does it cost to discover a new drug?
In 2010, the research and development cost of each new molecular entity was approximately 1.8 billion dollars. Drug discovery is a lengthy, expensive, and inefficient process, and most candidates fail during the expensive Phase I, II, and III clinical trials.
What are the main ways medications are administered?
Medications can be taken orally as tablets, capsules, or syrups, which is the most common route. Other routes include sublingual under the tongue, topical on the skin, transdermal patches, inhalation, and injections such as intramuscular, subcutaneous, intravenous, intrathecal, and epidural.
When did antibiotics first arrive and what effect did they have?
Antibiotics first arrived on the medical scene in 1932 thanks to Gerhard Domagk and were called the wonder drugs. The introduction of sulfa drugs dropped the US pneumonia mortality rate from 0.2 percent each year to 0.05 percent by 1939, and streptomycin, found in 1942, was the first drug effective against tuberculosis.
How do medications affect the environment?
Pharmaceutical medications are described as ubiquitous in lakes, rivers, streams, estuaries, seawater, and soil at concentrations in the nanogram to microgram per liter range. They reach the environment mainly through wastewater treatment plant effluent and agricultural antibiotic use, and the US Geological Survey classifies these Environmentally Persistent Pharmaceutical Pollutants as Ecological Disrupting Compounds.