Definition of terms

WHAT ARE THE PHASES OF CLINICAL TRIALS?

Clinical trials are conducted in phases. The trials at each phase have a different purpose and help scientists answer different questions:

In Phase I trials, researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.

In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

WHAT ARE THE DIFFERENT TYPES OF CLINICAL TRIALS?

Treatment trials - test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

Prevention trials - look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vaccines, vitamins, minerals or lifestyle changes.

Diagnostic trials - are conducted to find better tests or procedures for diagnosing a particular disease or condition.

Screening trials - test the best way to detect certain diseases or health conditions.

Quality of Life trials (or Supportive Care trials) - explore ways to improve comfort and the quality of life for individuals with a chronic illness.

TERMS

ADVERSE EVENT (AE)
Any untoward medical occurrence affecting a trial participant during the course of a clinical trial.

ADVERSE REACTION (AR)
An adverse event when there is at least a possibility that it is causally linked to a trial drug or intervention.

AUDIT
An independent and systematic review of study data, associated records, protocol procedures, and study conduct to determine whether the information is accurate and whether the study has been carried out in compliance with the protocol, SOPs, GCP, and other applicable regulations.

BLINDING (allocation concealment)
Manufacturing trial treatment packs as products that appear identical in size, shape, colour, flavor, and other attributes to make it very difficult to determine which treatment arm is being administered.

CASE REPORT FORM (CRF)
A printed or electronic document used to record protocol-required information for each subject in a study.

CHIEF INVESTIGATOR
(a) in relation to a clinical trial conducted at a single trial site, the investigator for that site;
(b) in relation to a clinical trial conducted at more than one trial site, the authorised health care professional, whether or not he is an investigator at any particular site, who takes primary responsibility for the conduct of the trial; that is: a doctor, a dentist, a nurse, a pharmacist.

CODE OF FEDERAL REGULATIONS (CFR)
US Federal rules and regulations that govern research with human subjects. Title 21 includes most of the regulations affecting the discovery, development, approval, and marketing of drugs and devices.

CONSENT (Informed consent)
A process by which a subject voluntarily confirms his or her willingness to participate in a clinical trial after having been informed of all aspects relevant to the subject's decision to participate.

CONTRACT RESEARCH ORGANISATION (CRO)
An organisation contracted by the sponsor to perform one or more of the sponsor's study-related responsibilities.

CLINICAL INVESTIGATOR (Principal investigator, PI, Investigator)
An individual who conducts a clinical study and directs the use, administration, and distribution of the investigational product to the subject.

DRUG ACCOUNTABILITY
Records of the receipt and distribution of investigational product. Also records of disposal of investigational product at the end of the study.

ELIGIBILITY CRITERIA
Rules for selecting subjects to participate in a clinical trial. Participants must meet all inclusion and no exclusion criteria to be eligible.

ESSENTIAL DOCUMENTS
Documents that individually and collectively permit evaluation of the conduct of a trial and the quality of the data produced.

EXPLANATORY TRIAL
Test whether an intervention is efficacious; that is, whether it can have a beneficial effect in an ideal situation. The explanatory trial seeks to maximise the internal validity by assuring rigorous control of all variables other than the intervention.

GOOD CLINICAL PRACTICE (GCP)
The standards for the design, conduct, performance, monitoring, auditing, recording, analysing and reporting of clinical trials.

INDEMNITY
An indemnity is a sum paid by way of compensation for a particular loss suffered. The indemnifying party may or may not be responsible for the loss suffered by the indemnified party. An indemnity policy provides protection from loss and damage claims filed by another person.

INSTITUTIONAL REVIEW BOARD (IRB)/INDEPENDENT ETHICS COMMITTEE (IEC)
A board or committee that reviews and approves clinical studies at an investigative site.

INTERNATIONAL CONFERENCE ON HARMONIZATION (ICH)
A committee established to develop a unified standard for the EU, Japan and the US to facilitate mutual acceptance of clinical data by regulatory authorities of these jurisdictions.

INVESTIGATIONAL NEW DRUG APPLICATION (IND)
An application that sponsors must submit to the appropriate drug regulatory agency before beginning studies of an investigational drug in humans.

INVESTIGATIONAL PRODUCT (IP)
Drug, biologic, or device being studied for approval.

INVESTIGATOR'S BROCHURE (IB)
A brochure compiled by the sponsor providing all known information about the investigational product.

INVESTIGATIVE SITE
The location where the study is being conducted.

MONITORING
Overseeing the progress of a clinical trial to ensure that it is conducted, recorded and reported according to the protocol, SOPs, GCP, and applicable regulations.

NEW DRUG APPLICATION (NDA)
An application submitted to the FDA requesting approval to market a new drug (or a new use for an established drug) for human use.

PLACEBO
An inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested.

PRAGMATIC TRIALS
Measure effectiveness; they measure the degree of beneficial effect in real clinical practice. In pragmatic trials, a balance between external validity (generalisability of the results) and internal validity (reliability or accuracy of the results) needs to be achieved. The pragmatic trial seeks to maximise external validity to ensure that the results can be generalised.

PROTOCOL
A document that identifies the plan or set of rules for conducting a specific clinical trial, and states the objectives, design, methodology, statistical considerations, and overall organisation of a trial.

RANDOMISATION
Participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively.

REGULATORY AUTHORITIES
Entities with the authority to enforce the laws applicable to clinical studies of investigational products (e.g., FDA, IRB, OHRP).

SECTION J CHECKLIST
Information that the concerned EU Member State's Competent Authority and Ethics Committee (CA and EC) require for submission

SERIOUS ADVERSE EVENT (SAE)
An adverse event where the death of the participant resulted or was otherwise threatened or where the participant required hospitalisation or prolonged hospital stay; or resulted in persistent or significant disability or incapacity; or was a congenital anomaly/birth defect.

SERIOUS ADVERSE REACTION (SAR)
A SAE that is thought to be causally linked to a trial drug or intervention.

SERIOUS UNEXPECTED SUSPECTED ADVERSE REACTION (SUSAR)
An unexpected occurrence of a SAR; there need only be an index of suspicion that the event is a previously unreported reaction to a trial drug or a previously reported but exaggerated or unexpectedly frequent adverse drug reaction.

SOURCE DOCUMENT(s)
Original documents, data and records from which case report forms (CRF) are compiled.

SPONSOR
An individual, company, institution or organisation that has overall responsibility for the conduct of the clinical investigation.

STANDARD OPERATING PROCEDURE (SOP)
Detailed written instructions that provide a structure to ensure that activities are performed in a consistent manner.

SUBJECT (participant)
An individual who participates in clinical research, either as a recipient of investigational product, or as a control.

SUB-INVESTIGATOR (co-investigator)
An individual member of a clinical research team to whom trial related responsibilities have been delegated by the investigator.

SUMMARY OF PRODUCT CHARACTERISTICS (SPC)
The summary of product characteristics contains a description of a medicinal product's properties and the conditions attached to its use. It sets out the agreed position of the medicinal product as distilled during the course of the assessment process by relevant regulatory agency(ies). As such the content cannot be changed except with the approval of the originating regulatory agency authority. The SPC is the basis of information for health professionals on how to use the medicinal product safely and effectively.