Dr Panagiotis Kanavos

In addition, Professor Allan Hackshaw (Director, Cancer Research UK and UCL Cancer Trials Centre, UCL) will be teaching on this course.

This course is compulsory on the Executive MSc in Healthcare Decision-Making, in collaboration with NICE. This course is not available as an outside option.

Interventions in clinical medicine and public health are usually evaluated through robust evidence including randomised clinical trials (RCTs). These have been the cornerstone for changing practice for many decades. In recent years, large scale observational studies (such as disease registries from governmental or commercial organisations, drug prescription claims databases, and reimbursement databases) have been used to complement the evidence from RCTs, or to be used with (single arm) clinical trials to show a therapy’s efficacy and safety.

The module will provide students with practical skills in the following key areas:

• Understanding the different types of clinical trials (phase I to III), and how they provide different levels of evidence; how they are designed and analysed; how they form part of the evidence package that ultimately leads to the approval of a new drug or combination of drugs, or other intervention type; the limitations of modern RCTs, especially with the increasing use of molecular biomarkers; and how earlier phase trials can be used to approve therapies for rare/orphan disorders;

• Understanding the principles of real world data (RWD) studies: randomised pragmatic trials and large scale observational studies; the value of RWD for clinical decision making and approving new interventions; different sources of RWD; how RWD studies are designed, analysed and interpreted; strengths and design and analytical limitations of RWD; and how RWD studies have been successfully used to influence decision making (for example, approval of a new drug or extending the licence of a drug already in routine use), and examples where this has failed.

• Examining the efficacy, safety and health-related quality of life of health care interventions (an important part of a complete health economic evaluation of a clinical trial).

• Interpreting research results and conclusions using aspects of epidemiology and medical statistics, and how to communicate study findings.

• Reading and understanding published journal articles or pharmaceutical company reports. And

• Familiarity with systematic reviews (i.e. how several studies are combined, and the importance of looking at the evidence as a whole).

The course will be delivered by interactive workshops and will be 25 hours in total over the teaching week

Students will be given two short exercises before the course begins, via Moodle, to help prepare for the course. The tutor will go over these during the contact week, and address any queries from the students. Detailed written answers are provided to the students, as template responses. The tutor is also available for one-to-one email contact with any student.

• Hackshaw A. A concise guide to clinical trials. BMJ Books, Wiley-Blackwell, first edition 2009.

• Hackshaw A. A concise guide to observational studies in healthcare. BMJ Books, Wiley-Blackwell, first edition 2015.

• Greenhalgh T. How to Read a Paper: The Basics of Evidence-Based Medicine. BMJ Books, Wiley-Blackwell, fourth edition, 2010.

• Students will be given access to essential readings before the course begins through the pre-sessional reading programme on Moodle.

Coursework (50%) and coursework (50%).

There will be two pieces of coursework in the form of a written assignment provided as a PowerPoint slide deck (about 25-30 slides) and a statement of 400 words of further discussion and conclusions, based on:

• a randomised clinical trial (50%)

• a real world evidence study (50%).