Outline of clinical research

General topics

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

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

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

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

Biopharmaceutical – a drug produced using biotechnology

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

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

Clinical trials unit – biomedical research units dedicated to conducting clinical trials

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

Epidemiological methods – statistical techniques used in epidemiology

Evidence-based medicine – the assessment of the quality of evidence relevant to the risks and benefits of medical treatments

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

Biopharmacology – the pharmacology of biopharmaceuticals

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

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

Bioequivalence – the biological equivalence of two preparations of a drug

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

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

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

Drug terminology

Active ingredient – the substance in a drug that is pharmaceutically active

Approved drug – a drug that has been approved for marketing by a regulatory body such as the U.S. Food and Drug Administration or the European Medicines Agency

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

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

Off-label use – the practice of prescribing a drug for an indication for which the drug has not been approved

Orphan drug – a drug used to treat a rare medical condition, or orphan disease

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

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

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

Types of study design

Blind experiment

Case report

Case series

Case study

Case-control study

Clinical control group

Cohort study

Cross-sectional study

Crossover study

First-in-man study

Longitudinal study

Minimisation

Multicenter trial

Nested case-control study

Observational study

Open-label trial

Placebo-controlled studies

Prospective cohort study

Randomized controlled trial

Retrospective cohort study

Run-in period

Seeding trial

Vaccine trial

Study participant confidentiality and safety

Adverse drug reaction

Adverse event

Council for International Organizations of Medical Sciences

Data confidentiality in clinical trials

Data monitoring committees

Ethics Committee (European Union)

EudraVigilance

Exclusion criteria

Great ape research ban

Inclusion criteria

Institutional review board

MedWatch

Safety monitoring

Serious adverse event

Suspected Adverse Reaction Surveillance Scheme

Clinical study management

Clinical monitoring

Clinical Trial Management System

Good clinical practice

Clinical research documents

Clinical trial protocol

Informed consent

Investigator's brochure

Source document

Standing operating procedure

Clinical research personnel

Clinical investigator

Clinical research associate

Clinical research coordinator

Contract research organizations

PPD

Covance

Parexel

Quintiles

Westat

Data collection and management

Case report form

Clinical data management system

Clinical data repository

Data clarification form

Electronic data capture

Good clinical data management practice

Patient diary

Patient-reported outcome

Remote data entry

Medical term coding dictionaries

Uppsala Monitoring Centre

COSTART

MedDRA

Systematized Nomenclature of Medicine (SNOMED)

WHOART

Common Terminology Criteria for Adverse Events

Clinical Data Interchange Standards Consortium

Study Data Tabulation Model (SDTM)

Standard for Exchange of Non-clinical Data (SEND)

JANUS clinical trial data repository

Data analysis

Censoring (clinical trials)

Effect size

End point of clinical trials

Hazard ratio

Meta-analysis

Number needed to harm

Number needed to treat

Odds ratio

Intention to treat analysis

Post-hoc analysis

Relative risk

Risk–benefit analysis

Sensitivity and specificity

Subgroup analysis

Substantial equivalence

Surrogate endpoint

Systematic review

Therapeutic effect

Results reporting

Medical writing

Clinical trials publication

Common Technical Document

Consolidated Standards of Reporting Trials (CONSORT)

Electronic Common Technical Document

Preferred reporting items for systematic reviews and meta-analyses (PRISMA)

Strengthening the reporting of observational studies in epidemiology (STROBE)

Uniform Requirements for Manuscripts Submitted to Biomedical Journals (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 – 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 largest study to investigate the use of statins in the prevention of cardiovascular disease.

International Studies of Infarct Survival – 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 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 – a study of over 6,000 men infected with HIV that has been ongoing for over 25 years

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

European Union

Directive 2001/20/EC

Directive 2001/83/EC

Directive 2005/28/EC

Directive 65/65/EEC

Directive 93/41/EEC

Directive 95/46/EC on the protection of personal data

United States

Federal Food, Drug, and Cosmetic Act (FD&C) – gives authority to the Food and Drug Administration (FDA) to oversee the safety of food, drugs, and cosmetics.

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

Prescription Drug User Fee Act – 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 – the section of Federal regulations that interprets and enforces FD&C.

Title 21 CFR Part 11 – 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 (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 (United Nations)

Food and Drugs Act (Canada)

International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (European Union, Japan, and United States)

Government agencies

Australian Drug Evaluation Committee

European Medicines Agency

Committee for Medicinal Products for Human Use

Federal Agency for Medicines and Health Products (Belgium)

Medicines and Healthcare products Regulatory Agency (United Kingdom)

Ministry of Health, Labour and Welfare (Japan)

Norwegian Medicines Agency

State Food and Drug Administration (China)

Swedish Medical Products Agency

Therapeutic Products Directorate (Canada)

Therapeutic Goods Administration (Australia)

Departments

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

Center for Devices and Radiological Health – 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 – responsible for review and approval of all drugs

Office of Regulatory Affairs – 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 – a submission to the FDA review and approval of a generic drug

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