HP4F6E Half Unit
Critical Appraisals of Clinical Trials and Real-World Evidence in Decision Making
This information is for the 2023/24 session.
Teacher responsible
Dr Panagiotis Kanavos
In addition, Professor Allan Hackshaw (Director, Cancer Research UK and UCL Cancer Trials Centre, UCL) will be teaching on this course.
Availability
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.
Course content
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 pu