Introduction to Clinical Trials Flashcards
What is the definition of a ‘fair test’?
A fair test is the controlled investigation carried out to answer a scientific question.
What is a clinical trial?
Clinical Trial is any research study which prospectively assigns human participants or groups of humans to one or more health - related interventions to evaluate the effects on health outcomes.
*HEALTH RELATED INTERVENTIONS = Drugs, Cells, Surgical procedures and Radiological procedures.
How many phases are there in clinical trials?
Phase 0 / Phase 1 / Phase 2 / Phase 3 / Phase 4
Explain phase 1 of the clinical trials.
Often has about 50 - 200 healthy volunteers.
Main questions needed to be answered are:
- Is the IMP safe in humans?
- Pharmacokinetics
- Pharmacodynamics
Explain phase 2 of the clinical trials.
Often has about 100 - 400 patients with the targeted disease.
Main questions needed to be answered are:
same as phase 1
Would it work on patients?
Explain phase 3 of the clinical trials.
Often has 100 - 5000 patients : (depending on how common the disease is).
Main questions needed to be answered are:
- As mentioned in Phases 1 & 2 : but has less pharmacokinetics and pharmacodynamics.
- Would it work? (efficacy)
Explain phase 4 of the clinical trials.
Often 1000 to 1 million patients. Main questions needed to be answered are: - Safety in clinical practice. - Efficacy in clinical practice. - Comparison to existing treatment.
Explain phase 0 of the clinical trials.
Often for cancer medicine. Known as ‘Microdose’ study.
Main questions needed to be answered are:
- Does the drug bind the target protein?
- Pharmacokinetics.
List the 3 clinical trial regulations.
Under PURPOSE:
- Protect the clinical trail participants. (Rights / Safety / Welfare.)
- Quality of assurance of data.
- Harmonise + Simplify administrative procedures.
Who decide whether a clinical trial should be conducted at a national scale?
There are 2 main organisations : MHRA and HRA.
MRHA is the Medicine and Healthcare products Regulatory Agency.
HRA is the Health research authorities. They are known as being part of the ethics committee.
What is a sponsor in drug trials and why are they important?
A sponsor is an individual / company / institution / organisation / group of organisations which takes responsibility for initiation // management // financing of the research.
It’s a legal requirement for any clinical trial to be an investigational medicinal product.
Who is a chief investigator in the clinical trials?
An individual who has overall responsibility for the conduct of the whole project in the UK.
Who is a principal investigator?
An individual who is responsible for the conduct of the research at a research site.
There should be one PI for each research site.
What are the characteristics of a successful clinical trial?
1) Address simple and relevant clinical questions.
2) Results would be useful for everyday clinical practice.
3) Clearly defined endpoint and statistical input and analysis.
4) Robust binding (if appropriate).
How do we eliminate bias from clinical trials?
- Randomised
- Double blinded
- Controlled (placebo // active // historical control)
- Pre-specify data analysis. (avoiding multiplicity and post - hoc analysis)
What is the basic design of clinical trials?
Parallel Group = Independent groups receiving different treatment.
Crossover Group = Participants receive the same treatments in different order or sequence. May involve a washout period in between.
What are the sources of bias in clinical trials?
- Selection Bias
- Performance Bias
- Detection Bias
- Attrition Bias
- Observer Bias
What is the CONSORT statement?
An evidence - based, minimum set of recommendations for reporting randomised trials.
Promotes transparent reporting of trials.
What is selection bias?
The systemic differences between baseline characteristics of the groups that are compared.
NOT EASY TO CONTROL
What is performance bias?
Systematic differences between the care that is provided // in exposure to factors other than interventions of interest.
BIAS TOWARDS NEW TREATMENT // PLACEBO
What is detection bias?
Systematic differences between groups in how outcomes are determined.
What is attrition bias?
Systematic differences between groups in withdrawal from a study.
What is observer bias?
Failure to measure // classify the exposure or outcomes appropriately.
