Examination arrangement

Course content

The course consists of three parts, 3 + 2 + 2 days, usually in the beginning of September, October and November.

The course is focusing on Randomized Controlled Trials (RCTs). This course will cover all aspects of RCTs, from design to reporting. Particular emphasis will be placed upon practical aspects. Recent advances in RCT methodology will be covered, including data monitoring committees (DMCs), stopping rules, Independent ethics committee procedures (IECs), good clinical practice (GCP is increasingly demanded as a standard by the European Union), and reporting of trials (Revised CONSORT Statement).

• Background, including non-randomized studies and historical controls. Controlled trials; placebos; double blind; multicentre trials; phase I, II, III IV trials; cross-over trials; equivalence trials.

• Randomization, and how to implement it; cases of failed randomization; is randomization ethical? The uncertainty principle. Patient consent.
• Protocol design: Background, eligibility criteria, randomization, sample size, outcome measures, procedures for participants, monitoring, reporting and analysis, publication policy.
• Form design and layout (CRF, Case Report Form).
• Basic statistics: Significance tests, confidence intervals and p-values.
• Sample size estimation: Methods and application.
• Approval and procedures: Writing committees, protocol review procedures, ethical approval (IRB), Data Monitoring Committees (DMC).
• Statistical aspects of data monitoring: Data monitoring - role and methods, recruitment charts, interim analyses, stopping rules and examples of trials which were stopped prematurely. Bayesian monitoring.
• Organizational aspects: Multicentre trials; structure of trials offices; training of staff; launch of trials; data entry; data management; monitoring procedures; data checking - double data entry.
• Analysis: Patient exclusions, intention to treat analysis, pragmatic or exploratory analyses, data checking, significance tests, equivalence trials and multiple endpoints.
• Conflicts of interest. Fraud, bias and confounding.
• Reporting: Eligibility, patient exclusions, standard journal requirements, reporting randomization procedures, interpreting significance, clinically significant effects, confirmatory trials, BMJ checklist.

The course also covers the use of clinical measurements and studies on diagnostic tests, including precision, accuracy, validity, specificity, sensitivity, reproducibility, feasibility, appropriateness and how to interpret the results in a clinical setting. How to design, use, analyse and interpret the results from cross-sectional-, case-control-, and cohort studies; and the strengths and limits of these designs in a clinical setting. How to establish, maintain and use clinical registers and databases and to differentiate such registers from epidemiological/population based surveys and data in medical records.

Further, the course will focus on Patient Reported Outcomes (PRO). Worldwide there is an increasing demand to always consider measurements of PRO in new clinical trials. Measurements of PRO and subjective symptoms are generally important, but particularly important in studies of palliative care. The results of such measurements are gaining interest as a secondary endpoint in studies where the aim is to decrease morbidity or mortality in severe and/or chronic illnesses.
The course will cover several aspects of these measurements in randomized studies and clinical trials; choice of questionnaire, methods, study design, problem solving, analysis and comprehension of the data, report of results and conclusions.

Learning outcome

After completing the course, the student is:
- able to assess the expediency and application of the different designs in clinical research including more sophisticated designs
- able to contribute to the improvement of designs and methods in clinical research
- by a given hypothesis able to formulate research questions in accordance with the different types of design
- with the correct methods able to design, implement, analyze and publish clinical research on an international level
- able to identify research questions related to design and carry out research with a methodical integrity
- able to communicate clinical research and development through acknowledged national and international channels
- able to select and use methods of patient reported outcome
- able to use methods for relevant literature search
- able to evaluate the quality of clinical research papers according to research methods used

Learning methods and activities

Lectures, group work, home assignment.
To be allowed to take the examination all homework must be delivered on time and at least 85% attendance of the lectures is mandatory.

Compulsory assignments

• Paper/excercise before written exam
• All lectures

Further on evaluation

If retake of the examination this can be either by a written or an oral test. This is to be deceided by the person responsible for the course.

Specific conditions

Recommended previous knowledge

Master's degree knowledge within the field of health sciences or medicine. If you do not have a medical background you are recommended to attend a course on clinical science, epidemiology or statistics on a master degree level.

Required previous knowledge

Master degree or similar. Medical students at The Student Research Programme. Candidates with a lower degree will be assessed individually.

Course materials

Stephen B Hulley et al. Designing Clinical Research (fourth edition)
Forlag: Lippincott Williams & Wilkins: 2013. ISBN: 978-1-60831-804-9.

David Machin & Peter Fayers. Randomized controlled trials. Design, practice and reporting. Forlag: Wiley-Blackwell 2009/2010. ISBN-10: 0471498122. ISBN-13: 978-0471498124.

Credit reductions