Welcome to Module 5: Clinical Development
The WHO definition of a clinical trial is:
‘“Any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions [i.e. drugs, cells or other biological products, surgical procedures, devices] to evaluate the effects on health outcomes”
Conducting clinical trials in human participants helps determine the safety and efficacy of the intervention for the new disease (indication) that has been repurposed for.
The gold standard in clinical trials is a randomised control trial (RCT), where participants are divided into 2 (or more) groups of equal size. One group take the investigational medical product (IMP) – in this case the repurposed drug – and the other group will either not take the drug, have the standard of care treatment or will take a placebo in its place.
Though this is the gold standard for clinical trials, and the simplest proof of how effective the drug is (efficacy) for regulatory approval, it may not be the most effective or appropriate way to run a trial in all circumstances. Depending on the patient cohort or the stage of development that you are in, alternative trial designs can be considered.
In this module you will:
- Consider what trial design, outcome measures and strategy fit with your end goal
- Understand the key stages and requirements to set up a clinical trial
- Understand the key stakeholders and their roles
The learning objectives of this module are:
- To understand the key procedures and documentation required for setting up a clinical trial
- To understand who act as important contacts within the clinical trial process, their roles and how they can support your trial
- To explore some of the different types of clinical trial and consider which may work for you
- To understand the patient experience of clinical trials and how to design a trial that meets their needs
Click on the topics in the panel on the right to navigate through the content. Do not forget to click ‘mark as complete’ at the bottom of each page to track your progress.
Good luck on your repurposing journey!
Acknowledgements
Primary author: Evelyn Hewitt (Beacon for Rare Diseases)
Reviewers: Christine Kubiak and Sareema Javaid
We’ve worked hard to bring this module to life, aiming to make it comprehensive and valuable for you. Because this is the very first launch of the Digital Academy, we really need your feedback. Please take a moment to complete our 3-minute feedback form at the end—we’d love to hear your thoughts!
Learn More
Should RCTs Remain the Gold Standard?
Article discussing whether the randomised control trial design can keep up with innovations in trial design in clinical research industry.
