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Efficacy
Efficacy is the ability to perform a task to a satisfactory or expected degree. The word comes from the same roots as '' effectiveness'', and it has often been used synonymously, although in pharmacology a distinction is now often made between efficacy and effectiveness. The word ''efficacy'' is used in pharmacology and medicine to refer both to the maximum response achievable from a pharmaceutical drug in research settings, and to the capacity for sufficient therapeutic effect or beneficial change in clinical settings. Pharmacology In pharmacology, efficacy () is the maximum response achievable from an applied or dosed agent, for instance, a small molecule drug. Intrinsic activity is a relative term for a drug's efficacy relative to a drug with the highest observed efficacy. It is a purely descriptive term that has little or no mechanistic interpretation. In order for a drug to have an effect, it needs to bind to its target, and then to affect the function of this tar ...
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Intrinsic Activity
Intrinsic activity (IA) and efficacy (Emax) refer to the relative ability of a drug- receptor complex to produce a maximum functional response. This must be distinguished from the affinity, which is a measure of the ability of the drug to bind to its molecular target, and the EC50, which is a measure of the potency of the drug and which is proportional to both efficacy and affinity. This use of the word "efficacy" was introduced by Stephenson (1956) to describe the way in which agonist An agonist is a chemical that activates a Receptor (biochemistry), receptor to produce a biological response. Receptors are Cell (biology), cellular proteins whose activation causes the cell to modify what it is currently doing. In contrast, an R ...s vary in the response they produce, even when they occupy the same number of receptors. High efficacy agonists can produce the maximal response of the receptor system while occupying a relatively low proportion of the receptors in that system. Th ...
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Pragmatic Clinical Trial
A pragmatic clinical trial (PCT), sometimes called a practical clinical trial (PCT), is a clinical trial that focuses on correlation between treatments and outcomes in real-world health system practice rather than focusing on proving causative explanations for outcomes, which requires extensive deconfounding with inclusion and exclusion criteria so strict that they risk rendering the trial results irrelevant to much of real-world practice. Examples A typical example is that an anti-diabetic medication in the real world will often be used in people with (latent or apparent) diabetes-induced kidney problems, but if a study of its efficacy and safety excluded some subsets of people with kidney problems (to escape confounding), the study's results may not reflect well what will actually happen in broad practice. PCTs thus contrast with explanatory clinical trials, which focus more on causation through deconfounding. The pragmatic versus explanatory distinction is a spectrum or ...
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Receptor (biochemistry)
In biochemistry and pharmacology, receptors are chemical structures, composed of protein, that receive and Signal_transduction, transduce signals that may be integrated into biological systems. These signals are typically chemical messengers which bind to a receptor and produce physiological responses, such as a change in the electrophysiology, electrical activity of a cell. For example, GABA, an inhibitory neurotransmitter, inhibits electrical activity of neurons by binding to GABAA receptor, GABA receptors. There are three main ways the action of the receptor can be classified: relay of signal, amplification, or integration. Relaying sends the signal onward, amplification increases the effect of a single ligand (biochemistry), ligand, and integration allows the signal to be incorporated into another biochemical pathway. Receptor proteins can be classified by their location. Cell surface receptors, also known as transmembrane receptors, include ligand-gated ion channels, G prote ...
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Affinity (pharmacology)
In biochemistry and pharmacology, a ligand is a substance that forms a complex with a biomolecule to serve a biological purpose. The etymology stems from Latin ''ligare'', which means 'to bind'. In protein-ligand binding, the ligand is usually a molecule which produces a signal by binding to a site on a target protein. The binding typically results in a change of conformational isomerism (conformation) of the target protein. In DNA-ligand binding studies, the ligand can be a small molecule, ion, or protein which binds to the DNA double helix. The relationship between ligand and binding partner is a function of charge, hydrophobicity, and molecular structure. Binding occurs by intermolecular forces, such as ionic bonds, hydrogen bonds and Van der Waals forces. The association or docking is actually reversible through dissociation. Measurably irreversible covalent bonding between a ligand and target molecule is atypical in biological systems. In contrast to the definition o ...
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Pharmacology
Pharmacology is the science of drugs and medications, including a substance's origin, composition, pharmacokinetics, pharmacodynamics, therapeutic use, and toxicology. More specifically, it is the study of the interactions that occur between a living organism and chemicals that affect normal or abnormal biochemical function. If substances have medicinal properties, they are considered pharmaceuticals. The field encompasses drug composition and properties, functions, sources, synthesis and drug design, molecular and cellular mechanisms, organ/systems mechanisms, signal transduction/cellular communication, molecular diagnostics, interactions, chemical biology, therapy, and medical applications and antipathogenic capabilities. The two main areas of pharmacology are pharmacodynamics and pharmacokinetics. Pharmacodynamics studies the effects of a drug on biological systems, and pharmacokinetics studies the effects of biological systems on a drug. In broad terms, pharmacod ...
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Randomized Controlled Trial
A randomized controlled trial (or randomized control trial; RCT) is a form of scientific experiment used to control factors not under direct experimental control. Examples of RCTs are clinical trials that compare the effects of drugs, surgical techniques, medical devices, diagnostic procedures, diets or other medical treatments. Participants who enroll in RCTs differ from one another in known and unknown ways that can influence study outcomes, and yet cannot be directly controlled. By randomly allocating participants among compared treatments, an RCT enables ''statistical control'' over these influences. Provided it is designed well, conducted properly, and enrolls enough participants, an RCT may achieve sufficient control over these confounding factors to deliver a useful comparison of the treatments studied. Definition and examples An RCT in clinical research typically compares a proposed new treatment against an existing standard of care; these are then termed the 'expe ...
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Pharmaceutical Drug
Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy ( pharmacotherapy) is an important part of the medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management. Drugs are classified in many ways. One of the key divisions is by level of control, which distinguishes prescription drugs (those that a pharmacist dispenses only on the medical prescription) from over-the-counter drugs (those that consumers can order for themselves). Medicines may be classified by mode of action, route of administration, biological system affected, or therapeutic effects. The World Health Organization keeps a list of essential medicines. Drug discovery and drug development are complex and expensive endeavors undertaken by pharmaceutical companies, academic scientists, and governments. As ...
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Effectiveness
Effectiveness or effectivity is the capability of producing a desired result or the ability to produce desired output. When something is deemed effective, it means it has an intended or expected outcome, or produces a deep, vivid impression. Etymology The origin of the word ''effective'' stems from the Latin word , which means "creative, productive, or effective". It surfaced in Middle English between 1300 and 1400 AD. Usage Science and technology Mathematics and logic In mathematics and logic, ''effective'' is used to describe metalogical methods that fit the criteria of an effective procedure. In group theory, a group element acts ''effectively'' (or ''faithfully'') on a point, if that point is not fixed by the action. Physics In physics, an effective theory is, similar to a phenomenological theory, a framework intended to explain certain (observed) effects without the claim that the theory correctly models the underlying (unobserved) processes. In heat ...
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Sola Scriptura
(Latin for 'by scripture alone') is a Christian theological doctrine held by most Protestant Christian denominations, in particular the Lutheran and Reformed traditions, that posits the Bible as the sole infallible source of authority for Christian faith and practice. The Catholic Church considers it heresy and generally the Orthodox churches consider it to be contrary to the phronema of the Church. While the scriptures' meaning is mediated through many kinds of subordinate authority—such as the ordinary teaching offices of a church, the ecumenical creeds, councils of the Catholic Church, or even personal special revelation— in contrast rejects any infallible authority other than the Bible. In this view, all non-scriptural authority is derived from the authority of the scriptures or is independent of the scriptures, and is, therefore, subject to reform when compared to the teaching of the Bible. is a formal principle of many Protestant Christian denominations, a ...
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Means Of Grace
The means of grace in Christian theology are those things (the ''means'') through which God gives grace. Just what this grace entails is interpreted in various ways: generally speaking, some see it as God blessing humankind so as to sustain and empower the Christian life; others see it as forgiveness, life, and salvation. Catholic theology According to the Catholic Church, the means of grace that Christ entrusted to the Church are many.Catholic Bishops' Conferences of England & Wales, Ireland and Scotland, ''One Bread One Body''
, p. 7
They include the entirety of revealed truth, the and the hierarchical ministry.
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Medicine
Medicine is the science and Praxis (process), practice of caring for patients, managing the Medical diagnosis, diagnosis, prognosis, Preventive medicine, prevention, therapy, treatment, Palliative care, palliation of their injury or disease, and Health promotion, promoting their health. Medicine encompasses a variety of health care practices evolved to maintain and restore health by the prevention (medical), prevention and treatment of illness. Contemporary medicine applies biomedical sciences, biomedical research, medical genetics, genetics, and medical technology to diagnosis (medical), diagnose, treat, and prevent injury and disease, typically through pharmaceuticals or surgery, but also through therapies as diverse as psychotherapy, splint (medicine), external splints and traction, medical devices, biologic medical product, biologics, and Radiation (medicine), ionizing radiation, amongst others. Medicine has been practiced since Prehistoric medicine, prehistoric times, and ...
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Placebo (origins Of Technical Term)
A placebo ( ) can be roughly defined as a sham medical treatment. Common placebos include inert tablets (like sugar pills), inert injections (like saline), sham surgery, and other procedures. Placebos are used in randomized clinical trials to test the efficacy of medical treatments. In a placebo-controlled trial, any change in the control group is known as the ''placebo response'', and the difference between this and the result of no treatment is the ''placebo effect''. Placebos in clinical trials should ideally be indistinguishable from so-called verum treatments under investigation, except for the latter's particular hypothesized medicinal effect. This is to shield test participants (with their consent) from knowing who is getting the placebo and who is getting the treatment under test, as patients' and clinicians' expectations of efficacy can influence results. The idea of a placebo effect was discussed in 18th century psychology, but became more prominent in the 20th c ...
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