The following

outline Outline or outlining may refer to: * Outline (list), a document summary, in hierarchical list format * Code folding, a method of hiding or collapsing code or text to see content in outline form * Outline drawing, a sketch depicting the outer edge ...

is provided as an overview of and topical guide to clinical research:

Clinical research Clinical research is a branch of healthcare science that determines the safety and effectiveness ( efficacy) of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatm ...

is the aspect of

biomedical research Medical research (or biomedical research), also known as experimental medicine, encompasses a wide array of research, extending from "basic research" (also called ''bench science'' or ''bench research''), – involving fundamental scientif ...

that addresses the assessment of new

pharmaceutical A medication (also called medicament, medicine, pharmaceutical drug, 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 re ...

and

biological Biology is the scientific study of life. It is a natural science with a broad scope but has several unifying themes that tie it together as a single, coherent field. For instance, all organisms are made up of cells that process hereditary in ...

drugs,

medical device A medical device is any device intended to be used for medical purposes. Significant potential for hazards are inherent when using a device for medical purposes and thus medical devices must be proved safe and effective with reasonable assura ...

s and

vaccine A vaccine is a biological Dosage form, preparation that provides active acquired immunity to a particular infectious disease, infectious or cancer, malignant disease. The safety and effectiveness of vaccines has been widely studied and verifie ...

s in humans.

General topics

Clinical significance In medicine and psychology, clinical significance is the practical importance of a treatment effect—whether it has a real genuine, palpable, noticeable effect on daily life. Types of significance Statistical significance Statistical significance ...

– a conclusion that an intervention has an effect that is of practical meaning to patients *

Drug discovery In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which new candidate medications are discovered. Historically, drugs were discovered by identifying the active ingredient from traditional remedies or by ...

– the identification of candidates, synthesis, characterization, screening, and assays for therapeutic efficacy *

Drug development Drug development is the process of bringing a new pharmaceutical drug to the market once a lead compound has been identified through the process of drug discovery. It includes preclinical research on microorganisms and animals, filing for re ...

– the process of taking a new chemical through the stages necessary to allow testing in clinical trials *

Biotechnology Biotechnology is the integration of natural sciences and engineering sciences in order to achieve the application of organisms, cells, parts thereof and molecular analogues for products and services. The term ''biotechnology'' was first used b ...

– the technological application that uses biological systems, living organisms to make or modify products or processes for specific use **

Biopharmaceutical A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, t ...

– a drug produced using biotechnology *

Clinical trial Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, dietar ...

– an experiment with human subjects to assess safety and efficacy of drugs **

Academic clinical trials An academic clinical trial is a clinical trial not funded by pharmaceutical or biotechnology company for commercial ends but by public-good agencies (usually universities or medical trusts) to advance medicine. These trials are a valuable compon ...

– clinical trials run at academic centers (e.g., medical schools, academic hospitals, and universities) **

Clinical trials unit Clinical trials units (CTU) are specialised biomedical research units which design, centrally coordinate and analyse clinical trials and other studies. Some CTUs specialise in different methodologies, such as randomised controlled trials, cluste ...

– biomedical research units dedicated to conducting clinical trials *

Epidemiology Epidemiology is the study and analysis of the distribution (who, when, and where), patterns and determinants of health and disease conditions in a defined population. It is a cornerstone of public health, and shapes policy decisions and evidenc ...

– the study of factors affecting the health and illness of populations **

Epidemiological methods The science of epidemiology has matured significantly from the times of Hippocrates, Semmelweis and John Snow. The techniques for gathering and analyzing epidemiological data vary depending on the type of disease being monitored but each study wi ...

– statistical techniques used in epidemiology *

Evidence-based medicine Evidence-based medicine (EBM) is "the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients". The aim of EBM is to integrate the experience of the clinician, the values of t ...

– the assessment of the quality of evidence relevant to the risks and benefits of medical treatments *

Pharmacology Pharmacology is a branch of medicine, biology and pharmaceutical sciences concerned with drug or medication action, where a drug may be defined as any artificial, natural, or endogenous (from within the body) molecule which exerts a biochemica ...

– the study of the interactions that occur between a living organism and drugs that alter normal biochemical function **

Biopharmacology A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesis, semisynthesized from biology, biological sources. Different from total synthesis, ...

– the pharmacology of biopharmaceuticals **

Clinical pharmacology Clinical pharmacology has been defined as "that discipline that teaches, does research, frames policy, gives information and advice about the actions and proper uses of medicines in humans and implements that knowledge in clinical practice". Clinic ...

– the scientific discipline focused on rational drug development and utilization in therapeutics **

Pharmacokinetics Pharmacokinetics (from Ancient Greek ''pharmakon'' "drug" and ''kinetikos'' "moving, putting in motion"; see chemical kinetics), sometimes abbreviated as PK, is a branch of pharmacology dedicated to determining the fate of substances administered ...

– the study of the fate of drugs administered to the body ***

Bioequivalence Bioequivalence is a term in pharmacokinetics used to assess the expected in vivo biological equivalence of two proprietary preparations of a drug. If two products are said to be bioequivalent it means that they would be expected to be, for all i ...

– the biological equivalence of two preparations of a drug **

Pharmacodynamics Pharmacodynamics (PD) is the study of the biochemical and physiologic effects of drugs (especially pharmaceutical drugs). The effects can include those manifested within animals (including humans), microorganisms, or combinations of organisms (fo ...

– the study of the biochemical and physiological effects of drugs on the body **

Pharmacometrics Pharmacometrics is a field of study of the methodology and application of models for disease and pharmacological measurement. It uses mathematical models of biology, pharmacology, disease, and physiology to describe and quantify interactions between ...

– the science of interpreting and describing pharmacology in a quantitative fashion *

Pharmacovigilance Term Given By Tushar Sharma (UPES Batch 2025) Pharmacovigilance (PV, or PhV), also known as drug safety, is the pharmaceutical science relating to the "collection, detection, assessment, monitoring, and prevention" of adverse effects with pharma ...

– the detection, assessment, understanding and prevention of adverse effects of medicines

Drug terminology

Active ingredient An active ingredient is any ingredient that provides biologically active or other direct effect in the diagnosis, cure, mitigation, treatment, or prevention of disease or to affect the structure or any function of the body of humans or animals. T ...

– the substance in a drug that is pharmaceutically active *

Approved drug An approved drug is a medicinal preparation that has been validated for a therapeutic use by a ruling authority of a government. This process is usually specific by country, unless specified otherwise. Process by country United States In the ...

– a drug that has been approved for marketing by a regulatory body such as the

U.S. Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food s ...

or the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...

Excipient An excipient is a substance formulated alongside the active ingredient of a medication, included for the purpose of long-term stabilization, bulking up solid formulations that contain potent active ingredients in small amounts (thus often referred ...

– an inactive substance used as a carrier for the active ingredients of a drug *

Medicinal product A medication (also called medicament, medicine, pharmaceutical drug, 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 rel ...

– any substance or combination of substances used for treating or preventing disease in humans *

Off-label use Off-label use is the use of pharmaceutical drugs for an unapproved indication (medicine), indication or in an unapproved age group, dose (biochemistry), dosage, or route of administration. Both prescription drugs and over-the-counter drugs (OTCs) ...

– the practice of prescribing a drug for an indication for which the drug has not been approved *

Orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

– a drug used to treat a rare medical condition, or

orphan disease A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discove ...

Placebo A placebo ( ) is a substance or treatment which is designed to have no therapeutic value. Common placebos include inert tablets (like sugar pills), inert injections (like Saline (medicine), saline), sham surgery, and other procedures. In general ...

– a sham treatment given to a control group in a clinical study *

Prescription drug A prescription drug (also prescription medication or prescription medicine) is a pharmaceutical drug that legally requires a medical prescription to be dispensed. In contrast, over-the-counter drugs can be obtained without a prescription. The rea ...

– a licensed medicine that can only be obtained by prescription from a doctor *

Standard treatment The standard treatment, also known as the standard of care, is the medical treatment that is normally provided to people with a given condition. In many scientific studies, the control group receives the standard treatment rather than a placebo wh ...

– a currently available drug used in an active control clinical study

Types of study design

Clinical study design Clinical study design is the formulation of trials and experiments, as well as observational studies in medical, clinical and other types of research (e.g., epidemiological) involving human beings. The goal of a clinical study is to assess the saf ...

Blind experiment In a blind or blinded experiment, information which may influence the participants of the experiment is withheld until after the experiment is complete. Good blinding can reduce or eliminate experimental biases that arise from a participants' expec ...

Case report In medicine, a case report is a detailed report of the symptoms, signs, diagnosis, treatment, and follow-up of an individual patient. Case reports may contain a demographic profile of the patient, but usually describe an unusual or novel occurrence ...

Case series A case series (also known as a clinical series) is a type of medical research study that tracks subjects with a known exposure, such as patients who have received a similar treatment, or examines their medical records for exposure and outcome. Ca ...

Case study A case study is an in-depth, detailed examination of a particular case (or cases) within a real-world context. For example, case studies in medicine may focus on an individual patient or ailment; case studies in business might cover a particular fi ...

* Case-control study * Clinical control group *

Cohort study A cohort study is a particular form of longitudinal study that samples a cohort (a group of people who share a defining characteristic, typically those who experienced a common event in a selected period, such as birth or graduation), performing ...

Cross-sectional study In medical research, social science, and biology, a cross-sectional study (also known as a cross-sectional analysis, transverse study, prevalence study) is a type of observational study that analyzes data from a population, or a representative s ...

Crossover study In medicine, a crossover study or crossover trial is a longitudinal study in which subjects receive a sequence of different treatments (or exposures). While crossover studies can be observational studies, many important crossover studies are cont ...

First-in-man study The phases of clinical research are the stages in which scientists conduct experiments with a health intervention to obtain sufficient evidence for a process considered effective as a medical treatment. For drug development, the clinical phases ...

Longitudinal study A longitudinal study (or longitudinal survey, or panel study) is a research design that involves repeated observations of the same variables (e.g., people) over short or long periods of time (i.e., uses longitudinal data). It is often a type of obs ...

* Minimisation *

Multicenter trial A multicenter research trial is a clinical trial conducted at more than one medical center or clinic. Most large clinical trials, particularly Clinical trial#Phase III, Phase III trials, are conducted at several clinical research centers. Benefit ...

Nested case-control study ''Nested'' is the seventh studio album by Bronx-born singer, songwriter and pianist Laura Nyro, released in 1978 on Columbia Records. Following on from her extensive tour to promote 1976's ''Smile'', which resulted in the 1977 live album '' Seas ...

Observational study In fields such as epidemiology, social sciences, psychology and statistics, an observational study draws inferences from a sample (statistics), sample to a statistical population, population where the dependent and independent variables, independ ...

Open-label trial An open-label trial, or open trial, is a type of clinical trial in which information is not withheld from trial participants. In particular, both the researchers and participants know which treatment is being administered. This contrasts with a do ...

Placebo-controlled studies Placebo-controlled studies are a way of testing a medical therapy in which, in addition to a group of subjects that receives the treatment to be evaluated, a separate control group receives a sham "placebo" treatment which is specifically designed ...

Prospective cohort study A prospective cohort study is a longitudinal cohort study that follows over time a group of similar individuals (cohorts) who differ with respect to certain factors under study, to determine how these factors affect rates of a certain outcome. F ...

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 te ...

Retrospective cohort study A retrospective cohort study, also called a historic cohort study, is a longitudinal cohort study used in medical and psychological research. A cohort of individuals that share a common exposure factor is compared with another group of equivalen ...

* Run-in period *

Seeding trial A seeding trial or marketing trial is a form of marketing, conducted in the name of research, designed to target product sampling towards selected consumers. In the marketing research field, seeding is the process of allocating marketing to specific ...

Vaccine trial A vaccine trial is a clinical trial that aims at establishing the safety and efficacy of a vaccine prior to it being licensed. A vaccine candidate drug is first identified through preclinical evaluations that could involve high throughput sc ...

Study participant confidentiality and safety

Human subject research *

Adverse drug reaction An adverse drug reaction (ADR) is a harmful, unintended result caused by taking medication. ADRs may occur following a single dose or prolonged administration of a drug or result from the combination of two or more drugs. The meaning of this term ...

Adverse event An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An adverse event can ther ...

Council for International Organizations of Medical Sciences The Council for International Organizations of Medical Sciences (CIOMS) is an international non-governmental organization of 40 international, national, and associate member groups representing the biomedical science community. It was jointly est ...

* Data confidentiality in clinical trials * Data monitoring committees *

Ethics Committee (European Union) The ethics committee, according to Directive 2001/20/EC, is an independent body in a member state of the European Union, consisting of healthcare professionals and non-medical members, whose responsibility is to protect the rights, safety and well b ...

EudraVigilance EudraVigilance (European Union Drug Regulating Authorities Pharmacovigilance) is the European data processing network and management system for reporting and evaluation of suspected adverse reactions to medicines which have been authorised or bein ...

Exclusion criteria In a clinical trial, the investigators must specify inclusion and exclusion criteria for participation in the study. Inclusion and exclusion criteria define the characteristics that prospective subjects must have if they are to be included in a st ...

Great ape research ban This is a list of countries banning non-human ape experimentation. The term ''non-human ape'' here refers to all members of the superfamily Hominoidea, excluding ''Homo sapiens''. ''Banning'' in this case refers to the enactment of formal decrees p ...

* Inclusion criteria *

Institutional review board An institutional review board (IRB), also known as an independent ethics committee (IEC), ethical review board (ERB), or research ethics board (REB), is a committee that applies research ethics by reviewing the methods proposed for research to ens ...

* MedWatch *

Safety monitoring Clinical monitoring is the oversight and administrative efforts that monitor a participant's health and efficacy of the treatment during a clinical trial. Both independent and government-run grant-funding agencies, such as the National Institutes o ...

Serious adverse event A serious adverse event (SAE) in human drug trials is defined as any untoward medical occurrence that at any dose #Results in death #Is life-threatening #Requires inpatient hospitalization or causes prolongation of existing hospitalization #Results ...

* Suspected Adverse Reaction Surveillance Scheme

Clinical study management

* Clinical monitoring * Clinical Trial Management System *

Good clinical practice Good clinical practice (GCP) is an international quality standard, which governments can then transpose into regulations for clinical trials involving human subjects. GCP follows the International Council for Harmonisation of Technical Requirements ...

Clinical research documents

Clinical trial protocol In natural and social science research, a protocol is most commonly a predefined procedural method in the design and implementation of an experiment. Protocols are written whenever it is desirable to standardize a laboratory method to ensure succe ...

Informed consent Informed consent is a principle in medical ethics and medical law, that a patient must have sufficient information and understanding before making decisions about their medical care. Pertinent information may include risks and benefits of treatme ...

* Investigator's brochure *

Source document A source document is a document in which data collected for a clinical trial is first recorded. This data is usually later entered in the case report form. The International Conference on Harmonisation of Technical Requirements for Registration of ...

Standing operating procedure A standard operating procedure (SOP) is a set of step-by-step instructions compiled by an organization to help workers carry out routine operations. SOPs aim to achieve efficiency, quality output, and uniformity of performance, while reducing misc ...

Clinical research personnel

Clinical investigator A clinical investigator involved in a clinical trial is responsible for ensuring that an investigation is conducted according to the signed investigator statement, the investigational plan, and applicable regulations; for protecting the rights, safe ...

Clinical research associate A clinical research associate (CRA), also called a clinical monitor or trial monitor, is a health-care professional who performs many activities related to medical research, particularly clinical trials. Clinical research associates work in vario ...

Clinical research coordinator A Clinical Research Coordinator (CRC) is a person responsible for conducting clinical trials using good clinical practice (GCP) under the auspices of a Principal Investigator (PI). ''Good clinical practices principles'' have been defined by Madelen ...

Contract research organizations

Contract research organization In the life sciences, a contract research organization (CRO) is a company that provides support to the pharmaceutical, biotechnology, and medical device industries in the form of research services outsourced on a contract basis. A CRO may provid ...

* PPD *

Covance Labcorp Drug Development is a contract research organization (CRO) headquartered in Burlington, North Carolina, providing nonclinical, preclinical, clinical and commercialization services to pharmaceutical and biotechnology industries. Formerly ...

* Parexel *

Quintiles IQVIA, formerly Quintiles and IMS Health, Inc., is an American multinational company serving the combined industries of health information technology and clinical research. IQVIA is a provider of biopharmaceutical development and commercial outs ...

Westat Westat is an employee-owned professional services corporation located in Rockville, Maryland, USA. It provides research services to agencies of the U.S. Government, as well as businesses, foundations, and state and local governments. The corpo ...

Data collection and management

Clinical data acquisition Acquisition or collection of clinical trial data can be achieved through various methods that may include, but are not limited to, any of the following: paper or electronic medical records, paper forms completed at a site, interactive voice respons ...

Case report form A case report form (or CRF) is a paper or electronic questionnaire specifically used in clinical trial research. The case report form is the tool used by the sponsor of the clinical trial to collect data from each participating patient. All data on ...

Clinical data management system A clinical data management system or CDMS is a tool used in clinical research to manage the data of a clinical trial. The clinical trial data gathered at the investigator site in the case report form are stored in the CDMS. To reduce the possibili ...

* Clinical data repository *

Data clarification form A data clarification form (DCF) or data query form is a questionnaire specifically used in clinical research. The DCF is the primary data clarification tool from the trial sponsor or contract research organization (CRO) towards the investigator to ...

Electronic data capture An electronic data capture (EDC) system is a computerized system designed for the collection of clinical data in electronic format for use mainly in human clinical trials. EDC replaces the traditional paper-based data collection methodology to stre ...

Good clinical data management practice Good clinical data management practice (GCDMP) is the current industry standards for clinical data management that consist of best business practice and acceptable regulatory standards. In all phases of clinical trials, clinical and laboratory in ...

* Patient diary *

Patient-reported outcome A patient-reported outcome (PRO) is a health outcome directly reported by the patient who experienced it. It stands in contrast to an outcome reported by someone else, such as a physician-reported outcome, a nurse-reported outcome, and so on. PRO m ...

* Remote data entry

Medical term coding dictionaries

Medical classification A medical classification is used to transform descriptions of medical diagnoses or procedures into standardized statistical code in a process known as clinical coding. Diagnosis classifications list diagnosis codes, which are used to track diseases ...

Uppsala Monitoring Centre Uppsala Monitoring Centre (UMC), located in Uppsala, Sweden, is the field name for the World Health Organization Collaborating Centre for International Drug Monitoring. UMC works by collecting, assessing and communicating information from member c ...

COSTART The Coding Symbols for a Thesaurus of Adverse Reaction Terms (COSTART) was developed by the United States Food and Drug Administration (FDA) for the coding, filing and retrieving of post-marketing adverse reaction reports. COSTART provides a metho ...

MedDRA A subscription-based product of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), MedDRA or Medical Dictionary for Regulatory Activities is a clinically validated international medical te ...

Systematized Nomenclature of Medicine The Systematized Nomenclature of Medicine (SNOMED) is a systematic, computer-processable collection of medical terms, in human and veterinary medicine, to provide codes, terms, synonyms and definitions which cover anatomy, diseases, findings, pro ...

(SNOMED) *

WHOART The WHO Adverse Reactions Terminology (WHOART) is a dictionary meant to serve as a basis for rational coding of adverse reaction terms. The system is maintained by the Uppsala Monitoring Centre (UMC), the World Health Organization Collaborating Cent ...

Common Terminology Criteria for Adverse Events The Common Terminology Criteria for Adverse Events (CTCAE), formerly called the Common Toxicity Criteria (CTC or NCI-CTC), are a set of criteria for the standardized classification of adverse effects of drugs used in cancer therapy. The CTCAE syst ...

Clinical Data Interchange Standards Consortium

Clinical Data Interchange Standards Consortium The Clinical Data Interchange Standards Consortium (CDISC) is a standards developing organization (SDO) dealing with medical research data linked with healthcare, to "enable information system interoperability to improve medical research and rela ...

* Study Data Tabulation Model (

SDTM SDTM (Study Data Tabulation Model) defines a standard structure for human clinical trial (study) data tabulations and for nonclinical study data tabulations that are to be submitted as part of a product application to a regulatory authority such a ...

) *

Standard for Exchange of Non-clinical Data (SEND) The Standard for Exchange of Nonclinical Data (SEND) is an implementation of the CDISC Standard Data Tabulation Model (SDTM) for nonclinical studies, which specifies a way to present nonclinical data in a consistent format. These types of studies ...

JANUS clinical trial data repository Janus clinical trial data repository is a clinical trial data repository (or data warehouse) standard as sanctioned by the U.S. Food and Drug Administration (FDA). It was named for the Roman god Janus (mythology), who had two faces, one that could ...

Data analysis

Analysis of clinical trials Clinical trials are medical research studies conducted on human subjects. The human subjects are assigned to one or more interventions, and the investigators evaluate the effects of those interventions. The progress and results of clinical trials ...

Censoring (clinical trials) In statistics, censoring is a condition in which the value of a measurement or observation is only partially known. For example, suppose a study is conducted to measure the impact of a drug on mortality rate. In such a study, it may be known ...

Effect size In statistics, an effect size is a value measuring the strength of the relationship between two variables in a population, or a sample-based estimate of that quantity. It can refer to the value of a statistic calculated from a sample of data, the ...

End point of clinical trials Clinical endpoints or clinical outcomes are Outcome measure, outcome measures referring to occurrence of disease, symptom, Medical sign, sign or laboratory abnormality constituting a target outcome in clinical trial, clinical research trials. The t ...

Hazard ratio In survival analysis, the hazard ratio (HR) is the ratio of the hazard rates corresponding to the conditions characterised by two distinct levels of a treatment variable of interest. For example, in a clinical study of a drug, the treated populati ...

Meta-analysis A meta-analysis is a statistical analysis that combines the results of multiple scientific studies. Meta-analyses can be performed when there are multiple scientific studies addressing the same question, with each individual study reporting me ...

Number needed to harm In medicine, the number needed to harm (NNH) is an epidemiological measure that indicates how many persons on average need to be exposed to a risk factor over a specific period to cause harm in an average of one person who would not otherwise have ...

Number needed to treat The number needed to treat (NNT) or number needed to treat for an additional beneficial outcome (NNTB) is an epidemiological measure used in communicating the effectiveness of a health-care intervention, typically a treatment with medication. The ...

Odds ratio An odds ratio (OR) is a statistic that quantifies the strength of the association between two events, A and B. The odds ratio is defined as the ratio of the odds of A in the presence of B and the odds of A in the absence of B, or equivalently (due ...

Intention to treat analysis In medicine an intention-to-treat (ITT) analysis of the results of a randomized controlled trial is based on the initial treatment assignment and not on the treatment eventually received. ITT analysis is intended to avoid various misleading artifac ...

Post-hoc analysis In a scientific study, post hoc analysis (from Latin '' post hoc'', "after this") consists of statistical analyses that were specified after the data were seen. They are usually used to uncover specific differences between three or more group mea ...

Relative risk The relative risk (RR) or risk ratio is the ratio of the probability of an outcome in an exposed group to the probability of an outcome in an unexposed group. Together with risk difference and odds ratio, relative risk measures the association bet ...

* Risk–benefit analysis *

Sensitivity and specificity ''Sensitivity'' and ''specificity'' mathematically describe the accuracy of a test which reports the presence or absence of a condition. Individuals for which the condition is satisfied are considered "positive" and those for which it is not are ...

* Subgroup analysis *

Substantial equivalence In food safety, the concept of substantial equivalence holds that the safety of a new food, particularly one that has been genetically modified (GM), may be assessed by comparing it with a similar traditional food that has proven safe in normal use ...

Surrogate endpoint In clinical trials, a surrogate endpoint (or surrogate marker) is a measure of effect of a specific treatment that may correlate with a ''real'' clinical endpoint but does not necessarily have a guaranteed relationship. The National Institutes of He ...

Systematic review A systematic review is a Literature review, scholarly synthesis of the evidence on a clearly presented topic using critical methods to identify, define and assess research on the topic. A systematic review extracts and interprets data from publ ...

Therapeutic effect Therapeutic effect refers to the response(s) after a treatment of any kind, the results of which are judged to be useful or favorable. This is true whether the result was expected, unexpected, or even an unintended consequence. An adverse effect (i ...

Results reporting

Medical writing A medical writer, also referred to as medical communicator, is a person who applies the principles of clinical research in developing clinical trial documents that effectively and clearly describe research results, product use, and other medical i ...

Clinical trials publication Clinical trials publication is having research published in a peer reviewed journal following clinical trials. Most investigators will want to have such a publication but the nature of clinical trials may create special considerations and obstacle ...

Common Technical Document The Common Technical Document (CTD) is a set of specifications for an application dossier for the registration of Medicines and designed to be used across Europe, Japan and the United States and beyond. Synopsis The CTD is an internationally agre ...

Consolidated Standards of Reporting Trials CONSORT (Consolidated Standards Of Reporting Trials) encompasses various initiatives developed by the CONSORT Group to alleviate the problems arising from inadequate reporting of randomized controlled trials. It is part of the larger EQUATOR Network ...

(CONSORT) *

Electronic Common Technical Document The electronic common technical document (eCTD) is an interface and international specification for the pharmaceutical industry to agency transfer of regulatory information. The specification is based on the Common Technical Document (CTD) format a ...

Preferred Reporting Items for Systematic Reviews and Meta-Analyses PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) is an evidence-based minimum set of items aimed at helping scientific authors to report a wide array of systematic reviews and meta-analyses, primarily used to assess the b ...

(PRISMA) *

Strengthening the reporting of observational studies in epidemiology The STROBE ''(STrengthening the Reporting of OBservational studies in Epidemiology)'' Statement is a reporting guideline including a checklist of 22 items that are considered essential for good reporting of observational studies. It was published si ...

(STROBE) *

Uniform Requirements for Manuscripts Submitted to Biomedical Journals The ICMJE recommendations (full title, "Recommendations for the Conduct, Reporting, Editing, and Publication of Scholarly Work in Medical Journals") are a set of guidelines produced by the International Committee of Medical Journal Editors for stan ...

(URM)

Notable clinical studies

* British Doctors Study – in 1956 provided convincing statistical proof that tobacco smoking increased the risk of lung cancer. *

Framingham Heart Study The Framingham Heart Study is a long-term, ongoing cardiovascular cohort study of residents of the city of Framingham, Massachusetts. The study began in 1948 with 5,209 adult subjects from Framingham, and is now on its third generation of participa ...

– a cardiovascular study based in Framingham, Massachusetts, which began in 1948 with 5,209, and is now on its third generation of participants. *

Heart Protection Study The Heart Protection Study was a randomized controlled trial run by the Clinical Trial Service Unit, and funded by the Medical Research Council (UK), Medical Research Council (MRC) and the British Heart Foundation (BHF) in the United Kingdom. It st ...

– the largest study to investigate the use of statins in the prevention of cardiovascular disease. *

International Studies of Infarct Survival The International Studies of Infarct Survival (ISIS) were four randomized controlled trials of several drugs for treating suspected acute myocardial infarction ("heart attack"). More than 134,000 patients from over 20 countries took part in four la ...

– four randomized controlled trials of several drugs for treating suspected acute myocardial infarction. * Intersalt study – a landmark observational study that showed a strong association between dietary salt and risk of cardiovascular disease. *

JUPITER trial The JUPITER trial (Justification for the Use of Statins in Primary Prevention: An Intervention Trial Evaluating Rosuvastatin trial) was a clinical trial aimed at evaluating whether statins reduce heart attacks and strokes in people with normal chole ...

– the first clinical trial to demonstrate that statin therapy may provide benefit to patients with low-to-normal LDL levels and no known cardiovascular disease. *

Multicenter AIDS Cohort Study The Multicenter AIDS Cohort Study (MACS) is an ongoing cohort study involving over 6,000 men, including both those infected with HIV, as well as HIV-negative men. The MACS has four main sites: Baltimore, Pittsburgh, Los Angeles, and Chicago. The Los ...

– a study of over 6,000 men infected with HIV that has been ongoing for over 25 years *

Stateville Penitentiary Malaria Study The Stateville Penitentiary malaria study was a controlled but ethically questionable study of the effects of malaria on prisoners of Stateville Penitentiary near Joliet, Illinois, in the 1940s, conducted by the Department of Medicine at the Univer ...

– a controlled study of the effects of malaria on the inmates of Stateville Penitentiary near Joliet, Illinois. *

Tuskegee Study of Untreated Syphilis in the Negro Male The Tuskegee Study of Untreated Syphilis in the Negro Male (informally referred to as the Tuskegee Experiment or Tuskegee Syphilis Study) was a study conducted between 1932 and 1972 by the United States Public Health Service (PHS) and the Cente ...

– a clinical study, conducted between 1932 and 1972 in Tuskegee, Alabama studied the natural progression of the disease if left untreated.

Legislation, regulations and guidances

European Union

Directive 2001/20/EC The Clinical Trials Directive (Officially Directive 2001/20/EC of 4 April 2001, of the European Parliament and of the Council on the approximation of the laws, regulations and administrative provisions of the Member States relating to implementati ...

Directive 2001/83/EC Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relates to medicinal products for human use in mainly countries that are part of the European Union. The Directive dealt with the disparitie ...

Directive 2005/28/EC {{Use dmy dates, date=July 2018 The Good Clinical Practice Directive (Directive 2005/28/EC of 8 April 2005 of the European Parliament and of the Council) lays down principles and detailed guidelines for good clinical practice as regards conducting ...

* Directive 65/65/EEC *

Directive 93/41/EEC Council of the European Communities Directive 93/41/EEC of 14 June 1993 repealed Directive 87/22/EEC on the approximation of national measures relating to the marketing of high-technology medicinal products, as this directive had been superseded by ...

Directive 95/46/EC on the protection of personal data The Data Protection Directive, officially Directive 95/46/EC, enacted in October 1995, is a European Union directive which regulates the processing of personal data within the European Union (EU) and the free movement of such data. The Data Pr ...

United States

Federal Food, Drug, and Cosmetic Act The United States Federal Food, Drug, and Cosmetic Act (abbreviated as FFDCA, FDCA, or FD&C) is a set of laws passed by the United States Congress in 1938 giving authority to the U.S. Food and Drug Administration (FDA) to oversee the safety of f ...

(FD&C) – gives authority to the

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...

(FDA) to oversee the safety of food, drugs, and cosmetics. **

Kefauver Harris Amendment Carey Estes Kefauver (; July 26, 1903 – August 10, 1963) was an American politician from Tennessee. A member of the Democratic Party, he served in the U.S. House of Representatives from 1939 to 1949 and in the Senate from 1949 until his de ...

– requires drug manufacturers to provide proof of the effectiveness and safety of drugs before approval. *

Prescription Drug User Fee Act The ''Prescription Drug User Fee Act'' (PDUFA) was a law passed by the United States Congress in 1992 which allowed the Food and Drug Administration (FDA) to collect fees from Pharmaceutical company, drug manufacturers to fund the new medication, ...

– allows the Food and Drug Administration (FDA) to collect fees from drug manufacturers to fund the new drug approval process. *

Title 21 of the Code of Federal Regulations Title 21 is the portion of the Code of Federal Regulations that governs food and drugs within the United States for the Food and Drug Administration (FDA), the Drug Enforcement Administration (DEA), and the Office of National Drug Control Policy (O ...

– the section of Federal regulations that interprets and enforces FD&C. *

Title 21 CFR Part 11 Title 21 CFR Part 11 is the part of Title 21 of the Code of Federal Regulations that establishes the United States Food and Drug Administration (FDA) regulations on electronic records and electronic signatures (ERES). Part 11, as it is commonly cal ...

– defines the criteria under which electronic records and electronic signatures are considered to be trustworthy, reliable and equivalent to paper records. *

Health Insurance Portability and Accountability Act The Health Insurance Portability and Accountability Act of 1996 (HIPAA or the Kennedy– Kassebaum Act) is a United States Act of Congress enacted by the 104th United States Congress and signed into law by President Bill Clinton on August 21, 1 ...

(HIPAA) – Title II of HIPAA addresses the security and privacy of health data, including data collected from subjects in clinical research.

Other

Declaration of Helsinki The Declaration of Helsinki (DoH, fi, Helsingin julistus, sv, Helsingforsdeklarationen) is a set of ethical principles regarding human experimentation developed originally in 1964 for the medical community by the World Medical Association (WMA) ...

(United Nations) *

Food and Drugs Act The ''Food and Drugs Act'' (the ''Act'') (formal title ''An Act respecting food, drugs, cosmetics and therapeutic devices'') is an act of the Parliament of Canada regarding the production, import, export, transport across provinces and sale of f ...

(Canada) *

International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) is an initiative that brings together regulatory authorities and pharmaceutical industry to discuss scientific and technical aspects of pha ...

(European Union, Japan, and United States)

Government agencies

Australian Drug Evaluation Committee {{Use Australian English, date=October 2016 The Australian Drug Evaluation Committee or ADEC, was a committee that provided independent scientific advice to the Australian Government regarding therapeutic drugs. The committee was originally formed ...

Committee for Medicinal Products for Human Use The Committee for Medicinal Products for Human Use (CHMP), formerly known as Committee for Proprietary Medicinal Products (CPMP), is the European Medicines Agency's committee responsible for elaborating the agency's opinions on all issues regarding ...

Federal Agency for Medicines and Health Products The Federal Agency for Medicines and Health Products (FAMHP) of Belgium is responsible for the approval and registration of new medication in Belgium and for pharmacovigilance. Xavier De Cuyper is General Administrator of the agency. The Dutch na ...

(Belgium) *

Medicines and Healthcare products Regulatory Agency The Medicines and Healthcare products Regulatory Agency (MHRA) is an executive agency of the Department of Health and Social Care in the United Kingdom which is responsible for ensuring that medicines and medical devices work and are acceptably ...

(United Kingdom) *

Ministry of Health, Labour and Welfare (Japan) The is a cabinet level ministry of the Japanese government. It is commonly known as in Japan. The ministry provides services on health, labour and welfare. It was formed with the merger of the former Ministry of Health and Welfare or and the ...

Norwegian Medicines Agency The Norwegian Medicines Agency (''Statens legemiddelverk'', ''Legemiddelverket'') is the national, regulatory authority for new and existing medicines and the supply chain. The Agency is responsible for supervising the production, trials and market ...

State Food and Drug Administration The National Medical Products Administration (NMPA, , formerly the China Food and Drug Administration, or CFDA) was founded on the basis of the former State Food and Drug Administration (SFDA). In March 2013, the former regulatory body was rebra ...

(China) *

Swedish Medical Products Agency The Medical Products Agency (MPA; sv, Läkemedelsverket) is the government agency in Sweden responsible for regulation and surveillance of the development, manufacturing and sale of medicinal drugs, medical devices and cosmetics. Its task is als ...

Therapeutic Products Directorate Pharmaceutical Drugs Directorate (PDD) is a Canadian federal authority that regulates small molecule pharmaceutical drugs for human use. Prior to being given market authorization, a manufacturer must present substantive scientific evidence of a pro ...

(Canada) *

Therapeutic Goods Administration The Therapeutic Goods Administration (TGA) is the medicine and therapeutic regulatory agency of the Australian Government. As part of the Department of Health and Aged Care, the TGA regulates the quality, supply and advertising of medicines, pa ...

(Australia)

United States Food and Drug Administration

Departments

Commissioner of Food and Drugs The United States Commissioner of Food and Drugs is the head of the Food and Drug Administration (FDA), an agency of the United States Department of Health and Human Services. The commissioner is appointed by the president of the United States an ...

– as head of the Food and Drug Administration, the commissioner reports to the Secretary of the Department of Health and Human Services *

Center for Biologics Evaluation and Research The Center for Biologics Evaluation and Research (CBER) is one of six main centers for the Food and Drug Administration, U.S. Food and Drug Administration (FDA), which is a part of the U.S. Department of Health and Human Services. The current Dir ...

– responsible for review and approval of biologic products, including vaccines, blood products, gene therapy and human cloning *

Center for Devices and Radiological Health The Center for Devices and Radiological Health (CDRH) is the branch of the United States Food and Drug Administration (FDA) responsible for the premarket approval of all medical devices, as well as overseeing the manufacturing, performance and safe ...

– responsible for review and approval of medical devices and safety of non-medical equipment that emit certain types of radiation *

Center for Drug Evaluation and Research The Center for Drug Evaluation and Research (CDER, pronounced "see'-der") is a division of the U.S. Food and Drug Administration (FDA) that monitors most drugs as defined in the Food, Drug, and Cosmetic Act. Some biological products are also leg ...

– responsible for review and approval of all drugs *

Office of Regulatory Affairs The Office of Global Regulatory Operations and Policy (GO), also known as the Office of Regulatory Affairs (ORA), is the part of the U.S. Food and Drug Administration (FDA) enforcing the federal laws governing biologics, cosmetics, dietary supplem ...

– enforces FDA laws and regulations

Review and approval programs

* Investigational Device Exemption – allows an investigational device to be used in a clinical study in order to collect safety and effectiveness data * Investigational New Drug – allows an investigational drug to be used in a clinical study in order to collect safety and effectiveness data * New Drug Application – a submission to the FDA by a pharmaceutical company for review and approval of a new drug **

Abbreviated New Drug Application An Abbreviated New Drug Application (ANDA) is an application for a U.S. generic drug approval for an existing licensed medication or approved drug. The ANDA is submitted to Food and Drug Administration, FDA's Center for Drug Evaluation and Resea ...

– a submission to the FDA review and approval of a

generic drug A generic drug is a pharmaceutical drug that contains the same chemical substance as a drug that was originally protected by chemical patents. Generic drugs are allowed for sale after the patents on the original drugs expire. Because the active ch ...

FDA Fast Track Development Program Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical ne ...

– a designation given to an NDA by the FDA that accelerates review and approval of new drugs