Clinical Trials

Fill in the phases of clinical development of a drug

Scientific method of clinical trials

What does PICO stand for

Population

Intervention

Control/Comparison

Outcome

What is PICO used for

A framework to formulate specific, answerable clinical or research questions and guide literature searches for evidence-based practice and systematic reviews.

Which of these is not a characteristic of a phase 3 trial

• Randomization to groups

• Placebo controlled

• Compared against current best treatment

• Most funded by pharmaceutical industry

• Well-defined protocol

• Results submitted to Regulatory Authorities

Trick question - they are all characteristics of a phase 3 trial

Name 3 Sources of Funding

Pharmaceutical companies: responsible for organizing the great majority of clinical trials.

National Health Organizations: MRC (UK), NIH (USA): Only justified for trials of major treatment issues.

Locally based trials: No external backing. The best of such studies can provide important source of new therapeutic ideas, but many are poorly organized

IND and NDA are 2 important stages of regulation for new drugs. What do they stand for & what is their purpose

Investigational New Drug (IND) for first in human studies.

New drug Application (NDA) for launching on to the market

What is the regulatory authority in Ireland for new drugs

Health Products Regulatory Authority (HPRA)

What is the regulatory authority in the EU for new drugs

European Medicines Evaluation Agency (EMEA)

What kind of problems can arise with historical trials

Potential incompatibility with patient selection and patient environment

Who is blind in double blind trials

Neither the patient nor those responsible for care and evaluation know which treatment they are receiving

Belmont Report principles

Respect for persons, the requirement to treat individuals as autonomous agents, and the requirement to protect those with diminished autonomy.

Beneficence, maximizing possible benefits and minimizing possible harms.

Justice, as demonstrated by fairness in distribution of the opportunity to participate in research

What are some advantages & disadvantages to multi-centre trials

In a publication of findings (trial report) what steps are missing?

• Title

• Introduction: Why did you start?

• Methods: What did you do?

• Results: What did you find?

• Title

• Summary

• Introduction: Why did you start?

• Methods: What did you do?

• Results: What did you find?

• Discussion: What does it mean?

If a trial has negative results, are they posted

Yes - all results add to medical knowledge

Fill in the Evidence based medicine pyramid

Clinical opinion: based on experience/expertise

Case reports: a finding from a single patient

Case series: a number of cases

Case-control studies: a retrospective study that compares subjects who have a condition (cases) with patients who do not (controls)

Randomized controlled trials (RCTs): prospective investigations of effects where groups are assigned before treatment begins

Meta-analysis

A statistical technique that combines numerical data from multiple studies

When are meta-analyses used

It is an optional part of a systematic review.

Used if data from multiple studies are suitable to combine.

(Know difference between this and a systemic review - Systematic Review: A structured, comprehensive review of all relevant studies on a specific research question. Meta-Analysis: A statistical technique that combines numerical data from multiple studies.)

What is a Forest Plot used for

It combines data from systematic reviews and shows their potential to improve healthcare

What does each arrow point to in this Forest Plot and Cochrane logo