Syllabus database for doctoral courses

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Swedish title Pragmatiska randomiserade kontrollerade studier inom sjukvården
English title Pragmatic randomised controlled trials in healthcare
Course number 2917
Credits 4.5
Responsible KI department Institutionen för global folkhälsa
Specific entry requirements Participants should have completed an introductory course in either epidemiology, biostatistics or quantitative research methods.
Grading Passed /Not passed
Established by The Board of Doctoral Education
Established 2016-03-10
Purpose of the course This course will enable students to to learn about the key elements of a randomised controlled trial (RCT), what a pragmatic trial is, why it is relevant, how to develop a protocol for a pragmatic RCT, issues in the conduct of an RCT, from writing up the protocol to trial close out. The student will also learn about ethical issues in trials, reporting trials, and nesting relevant studies in a trial.
The course is particularly useful for those students with an interest in evaluating pragmatic interventions in healthcare using a randomised trial design and those interested in trials from an epidemiological method perspective.
Intended learning outcomes At the end of the course, the student should be able to:
- Understand the continuum from Explanatory to pragmatic randomized trials - the PRECIS tool.
- Understand what makes a trial pragmatic and the relevance of pragmatic trials
- Know the CONSORT criteria for reporting of pragmatic randomized trials
- Describe the elements of a trial protocol and demonstrate this through development of a pragmatic trial protocol
- Have knowledge of PICOT to frame the research question and identify the primary and secondary outcomes
- Explain the principles, methods and relevance of randomization and allocation concealment
- Describe recruitment strategies, trial governance, quality assurance and control, participant retention and adherence
- Dealing with serious adverse events in a pragmatic trial
- Describe the data management process including data and safety monitoring plan, data analysis (predefined and post hoc analyses), different data types, measures of effect
- Explain the handling of serious adverse events in a pragmatic trial
- Explain the ethical principles in conducting RCTs
- Know how to embed qualitative studies and economic assessments into pragmatic trials
- Understand the practicalities of conducting RCTs
- Explain the challenges of conducting a RCT
- Demonstrate the ability to develop a protocol for a pragmatic RCT.

Contents of the course The course is divided into six sections with the following content:

Section 1 This is an introduction to different types of randomized controlled trials (RCTs) with particular emphasis on pragmatic randomized controlled trials (pRCTs). We will explain the reasons why one would do a pragmatic RCT and what makes them a special case of randomized controlled trials. We will introduce you to the frameworks for designing and reporting pRCTs, PRECIS and the CONSORT extension for pRCTS. The student will be be guided on working on the first stages of a trial protocol.

Section 2:The focus of the course this section is the basic elements of the design of a trial: the research question, the population and setting included in the students study; the intervention and the comparison; the outcomes used to measure the effects of the intervention; and the study target. These basic elements of the trial are summarised in the PICOT acronym. PICOT stands for participants, intervention, comparison, outcome, and target. At the end of the section the student should be able to define their research question, using the PICOT structure; be able to clearly and precisely define the participants and study settings of your randomized controlled trial; define inclusion and exclusion criteria; select outcomes, and specify the target, superiority or non-inferiority.

Section 3: The content of the course in this section is randomization and sample size. The student will learn why randomization is important, and what benefits it has for the interpretation of a trial. The student will learn to use tools, such as random number tables and online calculators for randomizing participants. The student will also be introduced to sample size and estimations for simple RCTs and for cluster RCTs. In this section we discuss post recruitment retentions, safety monitoring and data collection. At the end of the section, the student will be guided to write the the randomization and sample size part of their protocol for their planned pRCT.

Section 4: The emphasis of the course in this section is data analysis and interpretation. At the end of this section, the student will be guided to write the data analysis part of their protocol for their planned pRCT.

Section 5: The section has two subsections - ethics, and economic evaluations. While ethics are important to all research projects, there are particular considerations to be taken into account, particularly when the trial is cluster randomized. These issues will be discussed. Economic evaluations are useful for those conducting a trial and for those making a decision on whether an effective intervention is worth implementing widely. The student will be guided to think through and articulate the ethical issues arising in their protocol.

Section 6: The content of the course in this final section is qualitative evaluation alongside pragmatic randomized trials. Qualitative evaluations are an important addition to pRCTs; this is because they can be a valuable part of a process evaluation. Process evaluations are necessary to find out how, and why an intervention works; whereas the pRCT is aimed at finding out if an intervention works; and what its effects are.
Teaching and learning activities This course is a blended learning course i.e a combination of classroom and online teaching/learning. The course runs for 6 weeks at 50% pace (equivalent to 3 weeks of full time work). There will be one contact session at the beginning of each week for 2.5 hours. This lecture will outline the learning for the week (pace the sessions), discuss some concepts in more detail, clarify issues raised by the students and explain the assignment progress expected at the end of the week. The students will have access to a tutor on one other day of the week to discuss/ clarify any other issues the have arisen during the self learning/ assignment work up phase. The online part of the course is hosted on an Edx platform which each student will have access to. The platform contains lectures, reading material, and exercise as formative assessment. In addition students will have access to a discussion forum to interact and learn from each other - by posting questions and having discussions around topics of relevance as they work through the course. The tutor may also interact on some threads. The final assessment will be based on a complete trial protocol developed in stages as the course progresses.
Compulsory elements None of the lectures in the classroom or the tutor interaction sessions are mandatory. However students will be expected to develop the protocol in steps, as prescribed over the duration of the course. This protocol will be the final assessment.
Examination Student achievement of the learning objectives will be assessed through a written protocol that is developed in stages over the course submitted at the end of the course. This will be marked by a tutor. Grades given will be pass/not pass.
Literature and other teaching material Books:
- David. Machin Peter Fayers. Randomized Clinical Trials Design, Practice and Reporting. Chicester : Wiley 2010. ISBN 9780471498124(available as an e book in the KI Library)
- Pocock S. Clinical Trials: A Practical Approach. Wiley-Blackwell. 2013 ISBN 9780471968832 (available as an e book in the KI Library)

- Treweek S, Zwarenstein M. Making trials matter: pragmatic and explanatory trials and the problem of applicability. Trials. 2009 Jun 3;10:37. Review. PubMed PMID: 19493350;
- Schulz KF, Grimes DA. Generation of allocation sequences in randomised trials: chance, not choice. Lancet 2002;359(9305):515-9
- Schulz KF, Grimes DA. Allocation concealment in randomised trials: defending against deciphering. Lancet 2002;359(9306):614-8
- Schulz KF, Grimes DA. Blinding in randomised trials: hiding who got what. Lancet 2002;359(9307):696-700
- Loudon K, Zwarenstein M, Sullivan F, Donnan P, Treweek S. Making clinical trials more relevant: improving and validating the PRECIS tool for matching trial design decisions to trial purpose. Trials. 2013 Apr 27;14:115. doi: 10.1186/1745-6215-14-115.
- Zwarenstein M, Treweek S, Gagnier JJ, Altman DG, Tunis S, Haynes B, Oxman AD, Moher D; CONSORT group;Pragmatic Trials in Healthcare (Practihc) group. Improving the reporting of pragmatic trials: an extension of the
- CONSORT statement. BMJ. 2008 Nov 11;337:a2390. doi: 10.1136/bmj.a2390. PubMed PMID:19001484.
- Karanicolas PJ, Montori VM, Devereaux PJ, Schünemann H, Guyatt GH. A new ""mechanistic-practical"" framework for designing and interpreting randomized trials. J Clin Epidemiol. 2009 May;62(5):479-84. PMID:18468856.
- Oxman AD, Lombard C, Treweek S, Gagnier JJ, Maclure M, Zwarenstein M. Why we will remain pragmatists: four problems with the impractical mechanistic framework and a better solution. J Clin Epidemiol. 2009 May;62(5):485-8. PMID:19348973.
- Against pragmatism: on efficacy, effectiveness and the real world. Trials. 2009 Jul 6;10:48. doi: 10.1186/1745-6215-10-48.
- Rosenthal GE. The role of pragmatic clinical trials in the evolution of learning health systems. Trans Am Clin Climatol Assoc. 2014;125:204-16
Course responsible Carina King
Institutionen för global folkhälsa

Contact person Mariano Salazar
Institutionen för global folkhälsa