A study examining the rate and extent at which a drug, when administered in a pharmaceutical dosage form, becomes available, either at the site of pharmacological effect or systemically within the body.
A study aiming to show that the bioavailability of one formulation of a drug is equivalent to another formulation of the same drug.
Clinical trial/interventional trial
Any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate effects on health outcomes. Interventions include but are not restricted to drugs, cells and other biological products, surgical procedures, radiologic procedures, devices, behavioural treatments, process-of-care changes and preventive care. This definition includes phase I–phase IV trials.
Describes research conducted principally for the benefit of the manufacturer or distributor of the medicine or item being trialled.
Conflict of interest
A situation in which professional judgement concerning one interest, such as a person’s health or the validity of research, could be influenced by another interest, such as meeting recruitment targets, financial gain or impact on future career.
Custodian or kaitiaki
An individual or group responsible for protecting, monitoring the use of or managing Māori research data or samples; the term is not limited to a research projects (e.g., a custodian may be responsible for a databank or biobank)
Data and safety monitoring committee (DSMC)
A body that advises the study team and study sponsor, and is responsible for monitoring emerging data during the course of a study. The purpose of these roles is to ensure both that the participants are safe and that the study is conducted to a high quality, so that it generates reliable answers to its study questions. The DSMC may be independent, or may be constituted from those conducting the study. Another term for a DSMC is ‘data and safety monitoring board’.
A pre-specified outcome variable of interest to a study. The primary end-point is the most important outcome, and should reflect clinically relevant effects and the principal objective of the study. Researchers use data on secondary outcomes (secondary end-points) to evaluate additional effects of the intervention.
An intervention study meets the equipoise standard if the evidence is ‘equally poised’ as to the overall balance of risks and benefits of each of the interventions offered in the study.
An ethics committee is any committee that is responsible for ethically reviewing health and disability research proposals. Such a committee may be accredited or otherwise; for more information see ‘Approved ethics committees’ above.
Ethics Committee on Assisted Reproductive Technology (ECART)
A ministerial committee that reviews, determines, and monitors applications for assisted reproductive procedures and human reproductive research.
An individual, company, institution or organisation that provides funding for a study. In some cases the funder may delegate responsibility for initiating or managing a research project to investigators.
See also ‘Sponsor’.
In distinguishing between funders and sponsors, relevant considerations include arrangements of access to study data, control over publications, and conflicts of interest.
A condition for which the use of a certain intervention (eg, a certain medicine) is indicated or appropriate.
A planned deviation from the currently accepted practice of a New Zealand body of health professionals involving an untested or unproven clinical intervention intended to be used on an ongoing basis.
A study in which an investigator controls and studies an intervention(s) provided to participants for the purpose of adding to knowledge of the health effects of that intervention(s). The term ‘intervention study’ is often used interchangeably with ‘experimental study’. Many intervention studies are clinical trials.
Any investigator on a study who is not the coordinating investigator. This includes investigators who are responsible for the conduct of a study at a given location. A study may have any number of investigators.
A document summarising the clinical and other data relating to a new medicine that is relevant to the study of the product in human participants.
A locality is an organisation responsible for a hospital, health centre, surgery or other establishment or facility in New Zealand at or from which the procedures outlined in the protocol of a study are to be conducted.
A study that examines interventions that do not hold the prospect of direct diagnostic, therapeutic or preventive benefit to the individual study participant. Types of non-therapeutic studies include some phase I trials, bioequivalence studies and bioavailability studies.
Research in which (in contrast to intervention or experimental studies) no intervention other than the recording, classifying, counting and analysing of data takes place. In observational studies the investigator has no control over study variables and merely observes outcomes.
A person who is enrolled in a study. In some studies, participants are grouped in communities (eg, geographical communities, or organisations such as schools). Some studies may use participants’ data or tissue. Participants may be patients, consumers, or family members and whānau.
Phase I study
A study involving the initial administration of a new investigational intervention into humans. Although human pharmacology studies are typically identified as phase I, they may also be later phase studies. Phase I studies usually have non-therapeutic objectives, and may be conducted in healthy volunteer subjects, or in patients with a specific disease (particularly in the case of studies of cytotoxic drugs). Studies in this phase can be open or baseline controlled, or may use randomisation with blinding to improve the validity of observations. Studies conducted in phase I typically involve one or a combination of estimation of initial safety and tolerability, pharmacokinetics, assessment of pharmacodynamics, and early measurement of drug activity.
Phase II study
A study usually considered to start exploring the therapeutic efficacy of an intervention in patients. Initial therapeutic exploratory studies use a variety of study designs, including concurrent controls and comparisons with baseline status. Subsequent phase II studies are usually randomised and use concurrent controls to evaluate the efficacy of an intervention and its safety for a particular therapeutic indication.
Studies in phase II are usually conducted in a group of patients who are selected by relatively narrow criteria, leading to a relatively homogeneous population that is closely monitored. One important goal for this phase is to determine the dose(s) and regimen for phase III studies.
Additional objectives may include evaluation of potential study end-points, therapeutic regimens (including concomitant medications) and target populations (eg, mild versus severe disease) for further study in phase II or III. Phase II studies are sometimes further categorised as phase IIa studies (where the focus is on assessing dose requirements) or phase IIb studies (which are designed to evaluate efficacy).
Phase III study
A study with the primary objective of demonstrating or confirming therapeutic benefit.
Phase III studies are designed to confirm the preliminary evidence accumulated in phase II that an intervention is safe and effective for the intended indication and recipient population.
Studies in phase III may also further explore the dose–response relationship, or investigate the intervention’s use in wider populations, in different stages of disease or in combination with another intervention.
For interventions intended to be administered for long periods, studies involving extended exposure to the intervention are usually conducted in phase III, although they may be started in phase II.
Phase IV study
A study (other than routine surveillance) performed after an intervention’s approval, related to the approved indication. Phase IV studies are studies that were not considered necessary for approval but can be important for optimising the intervention’s use. They may be of any type of study design, but should have valid scientific objectives.
Studies in this phase commonly examine additional drug–drug interaction or the dose–response relationship or safety, or investigate use under the approved indication, such as mortality/morbidity studies and epidemiological studies.
A description of a study’s objectives, design, methodology, statistical considerations and organisation.
The protocol often gives the background and rationale for the trial, but other documents referenced by the protocol may provide these.
Research involving the studied use of empirical materials such as case studies, personal experience, life stories, interviews, observations and cultural texts.
Research involving systematic empirical investigation via statistical, mathematical, or computational techniques.
Randomised controlled trial
The general term for a study in which participants are randomly assigned to intervention and control groups to receive or not receive a diagnostic, preventive or therapeutic intervention. Findings in such a study are assessed by comparing rates of disease, death, recovery or other appropriate end-points in the intervention and control groups.
Increasingly, health research and quality improvement involve responsibilities that are broader, extending to institutions and organisations. The Standards primarily use the term ‘Researcher’ throughout when referring to corresponding responsibilities, however it should be understood that these Standards use the term Researcher broadly, intending to address all those responsible for the conduct of health and disability research, quality improvement activities, data and tissue governance, and any other activity described in these Standards.
Researcher-initiated research is research proposed by researchers and without a company or commercial entity taking the role of sponsor. Such research can be conducted by an individual researcher, an institution, a group of institutions, a collaborative study group or a cooperative group.
An individual, company, institution or organisation that is responsible for initiating, managing and/or financing a study. This excludes an individual company, institution or organisation that has been requested to provide money for a trial and does not benefit in any way from the results of the trial. See also funder.
Standing Committee on Therapeutic Trials (SCOTT)
A standing committee of the HRC whose function is to make recommendations to Medsafe regarding the approval of clinical trials of new medicines under section 30 of the Medicines Act 1981(external link).
A study that examines interventions that hold the prospect of direct diagnostic, therapeutic or preventive benefit.
Any type of intervention that may be studied, including medicines, tests, methods of health care delivery and other health or disability support interventions.