Clinical Trials II - Study Design, Bias Minimisation, and Endpoints

Vocabulary flashcards covering core clinical trial concepts including study ethics, informed consent, bias minimisation, randomisation techniques, blinding, statistics, hypothesis testing, and trial endpoints.

Clinical Trial

A research study in which one or more human subjects are prospectively assigned to one or more interventions to evaluate the effects of those interventions on health-related biomedical or behavioural outcomes.

Ethics

Moral principles that govern a person's behaviour or the conducting of an activity.

Informed Consent

A founding principle of research ethics ensuring human participants enter research freely (voluntarily) with full information about what participation entails before entering the research.

Bias

Systematic errors that encourage one outcome over others, potentially causing investigators to draw wrong conclusions about the beneficial and harmful effects of interventions.

Control Group

The standard to which comparisons are made in an experiment, composed of participants who do not receive the experimental treatment.

Randomisation

The process by which treatments are assigned to participants by chance rather than by choice to avoid bias in assigning volunteers to different treatments.

Simple Randomisation

Randomisation based on a single sequence of random assignments (such as flipping a coin) that maintains complete randomness of group assignment.

Block Randomisation

A technique designed to randomise subjects into groups that result in equal sample sizes by calculating all possible balanced combinations within a set block size and randomly selecting blocks.

Stratified Randomisation

A randomisation method that addresses the need to control and balance the influence of covariates by generating a separate block for each combination of covariates and randomly assigning subjects within each block.

Allocation Concealment

A method ensuring the treatment assignment remains hidden from the person recruiting participants until after the participant has entered the trial.

Blinding

Study designs intended to prevent research team members and study participants from influencing results, facilitating the collection of scientifically accurate data.

Single-blinded Study

A clinical trial in which only the researcher conducting the study knows which treatment or intervention the participant receives until the trial concludes.

Double-blinded Study

A clinical trial in which neither the participants nor the researcher knows which treatment or intervention participants receive until the clinical trial concludes.

Placebo

A treatment designed to look and taste identical to the real intervention but containing no active ingredient.

Placebo Effect

When a person's physical or mental health appears to improve after receiving a placebo or 'dummy' treatment.

Sampling Variability

The chance fluctuation where individual samples differ from the true population value (for instance, obtaining a spread other than an exact 50/50 distribution in small samples).

Hypothesis

A proposed assumption for a phenomenon that may or may not be true.

Hypothesis Testing

The evaluation performed by a researcher to confirm or disprove a hypothesis.

Null Hypothesis ($H_0$)

A hypothesis formulated to capture the current situation (e.g., that a new medicine is no better than the current treatment), assumed true until proven otherwise.

Alternative Hypothesis ($H_1$)

A hypothesis formulated to capture what researchers aim to demonstrate by conducting the trial (e.g., that a new medicine is better than the current treatment).

True Endpoint

A clinically meaningful endpoint that directly measures how a patient feels, functions, or survives to determine whether a treatment worked.

Surrogate Endpoint

A measurement of a specific outcome (such as a shrinking tumour or lower biomarker levels) used in place of another as a predictor to indicate whether a treatment works.

Dissemination of Results

The process of getting research findings to people who can make use of them in order to maximise the benefit of the research.

Principles of good dissemination (5)

• stakeholder engagement

• Format/lagnuage

• Utilise opportunities

• Context

• Timing

Stakeholder engagement

work out who your primary audience is, engage with them throughout the project before/during the trial

How is format/ language imporatn in dissemination of results

Need to produce outputs that are in appropriate format for the other user. Use plain English which is accessible to all audience

How can opportunities be utilised in dissemination of results

Build partnerships with established networks, use existing conferences and events to exchange knowledge and raise awareness of your work

How is context important in dissemination of results

Understand the service connect of your research

Things to consider with dissemination fo results (5)

• objective → what fo you wnat to achieve

• Audience → identify them- who do you need to influence to maximise the uptake of your research

• Timeline → when will dissemination activtiy occur

• Resources → think abotu the expertise in your team and whether you need additioanl help with dissemination

• Strategy → partners/ influence, messaging, channels, coverage/ frequency , potential risks/ sensitivity