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Introduction to clinical trials. Definition of a clinical trial. A research activity that involves administration of a test treatment to some experimental unit in order to evaluate the treatment. Key words. Treament. Pharmaceutical, diet, procedure, diagnostic, device, program, placebo.

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definition of a clinical trial
Definition of a clinical trial

A research activity that involves administration of a test treatment to some experimental unit in order to evaluate the treatment.

key words
Key words


Pharmaceutical, diet, procedure, diagnostic, device, program, placebo.

Experimental unit

Subject from a target population.


Assessment of (clinical) effect, but also adverse events, lab variables, vital signs, quality of life, health economy.

the first study
The first study

I have found two treats that makes a letcure seem more interesting.

How do I find out which one is best?

the wheel of science
The Wheel of Science






drug discovery development process
Drug discovery/development process

discovery; refinement; chemical & biological characterisation

safety & toxicity in animals; formulation development

volunteer studies; patient studies

regulatory process


Lessons &


post registration


Discovery=find new active structure : Development=convert it to a useful drug

the path to a new medicine
The path to a new medicine


















First patent


Clinical trial


Product licenceapplication

Drug Discovery

Drug Development

Target and leadidentification


Developmentfor launch

Product lifecycle support

Concept testing


Clinical Development

Phase I12-150people

Phase II50-1000people

Phase III500-5,000people

Phase IV studies continue

Toxicology and pharmacokinetic studies

(absorption, distribution, metabolism, excretion)

Pharmaceutical and analytical development

Process chemistry and manufacturing

Registration and regulatory affairs

Sales and marketing (preparation, promotion, advertising and selling)

No. of compounds

Up to 10,000





phase i
Phase I

The drug seems reasonably safe in animal study, but has never been tested on humans

What do we want to know?

How can we find that out?

phase i trials
Phase I trials

Phase I50-150people

Phase III500-15000people

Phase II100-200people

Phase IV studies continue

  • Focused on tolerability and safety
  • 12-30 (150) healthy people (often males)
  • Efficacy on biomarkers if possible
  • Single and repeated doses
  • Increase dose levels
  • Interaction with other drugs
  • Pharmacokinetics
  • Explorative’
  • ADME (Absorption, Distribution, Metabolism Excretion)
  • Through QT study
  • Bridging , PK in other populations
phase ii
Phase II

The drug seems to be resonably safe in humans and there is some sign of an effect on something.

What do we want to know next?

How can we find that out?

phase 2 trials
Phase 2 trials

Phase II50-1000people

Phase I50-150people

Phase III500-5,000people

Phase IV studies continue

  • 50-1000 patients
  • Extensive monitoring
  • Safety and tolerability in patients
  • Often complicated design, explorative
  • Selection of optimal dose
  • Pharmacokinetics in patients
  • Effect in special populations
  • Explorative
phase iii
Phase III

The drug seems resonable safe to give to patients and we have and idea of which dose to use.

What do we want to know now?

How can we find that out?

phase 3 trials
Phase 3 trials

Phase III500-15000people

Phase I50-150people

Phase II100-200people

Phase IV studies continue

  • 500-20000 patients
  • Effect is verified in the target population
  • Forms the basis of the NDA, New Drug Application
  • Interactions between drugs start to become measurable in the larger population
  • sub-groups start to be established
  • special features and problems show up
  • Confirmative
phase iv
Phase IV

Our drug is approved for use on patients.

What’s next?

phase iv trials
Phase IV trials

Phase I50-150people

Phase II100-200people

Phase III500-5,000people

Phase IV studies continue

  • Often large 500-30000 patients
  • Further investigation of efficacy and safety post approval
  • Special populations
  • New indications
  • Marketing
observational studies

data collection





Observational studies

Data is collected for a set of patients without any randomisation

Prospective: Data is collected after the objectives are set

data collection





Retrospective: Data is collected before the objectives are set

where to look for information
Where to look for information

ICH (international Conference on Harmonisation)

FDA (Food and Drug Agency)

EMEA (European Medicines Agency)

Cochrane Collaboration

  • Quality
  • Efficacy
  • Safety
  • Multidiciplinary
ich quality guidelines
ICH Quality guidelines

Harmonisation achievements in the Quality area include pivotal milestones such as the conduct of stability studies, defining relevant thresholds for impurities testing and a more flexible approach to pharmaceutical quality based on Good Manufacturing Practice (GMP) risk management.

ich safety guidelines
ICH safety guidelines

ICH has produced a comprehensive set of safety Guidelines to uncover potential risks like carcinogenicity, genotoxicity and reprotoxicity. A recent breakthrough has been a non-clinical testing strategy for assessing the QT interval prolongation liability: the single most important cause of drug withdrawals in recent years.

  • S7A: Safety Pharmacology Studies for Human Pharmaceutical
  • S7B: The Non-Clinical Evaluation of the Potential for Delayed Ventricular Repolarization (QT prolongation) by Human Pharmaceutical
ich efficacy guidelines
ICH efficacy guidelines

The work carried out by ICH under the Efficacy heading is concerned with the design, conduct, safety and reporting of clinical trials. It also covers novel types of medicines derived from biotechnological processes and the use of pharmacogenetics/genomics techniques to produce better targeted medicines.

ich efficacy guidelines1
ICH Efficacy Guidelines
  • Clinical Safety E1-E2F
  • Clinical Study Reports E3
  • Dose Reponse Studies E4
  • Ethnic Factors E5
  • Good Clinical Practise E6
  • Clinical Trials E7-E11
  • Clincal Evaluation of a Therapeutic Category E12
  • Clincal Evaluation E14
  • Pharmacogenomics E15-E16
  • Cross Cutting Topics E17
ich efficacy guidelines2
ICH Efficacy Guidelines
  • E7: Studies in Support of Special Populations, Geriatrics
  • E8: General Considerations for Clinical Trials
  • E9: Statistical Principles for Clincal Trials
  • E10: Choice of Control Group and Related Issues
  • E11: Clinical Investigation of Medicinal Products in the Pediatric Population
the clinical study process
The Clinical Study Process

Preparation of statisticalanalysis



Clinical StudyProtocol


Clinical Study Report


Data Capture


StatisticalAnalysis Plan

Clean File

Data base lock

T i m e

chapter 1 reading instructions
Chapter 1 Reading instructions
  • 1.1 What are clinical trials: Read
  • 1.2 History of clinical trials: Less important
  • 1.3 Regulatory process and requirements: Read page 14
  • 1.4 Investigational new drug application: Read page 17–20
  • 1.5 New drug application: Less important
  • 1.6 Clinical development plan and practise: Read
  • 1.7 Aims and structure of this book: Skip