One of the main problems with ICH-GCP is that it has made randomised trials much more complex and costly than they need to be. The result has been an ever increasing cost of doing trials with reports of single phase-3 trials now costing as much as a billion US dollars. This not only prevents the development of many potential new treatments, but makes it much more difficult to properly evaluate many existing treatments and other interventions. Therefore, there is an urgent need to reduce the cost of randomised trials.
The vast majority of health research is conducted with the full informed consent of the people who participate, but in many countries there are specific laws or regulation in place to allow research to be undertaken without consent. Examples include the so called “Common Rule” in the US (CFR 46.116) and “Section 251” support in the UK.
The National Academies of Sciences, Engineering, and Medicine are hosting a series of workshops looking at how real-world data can be converted to reliable real-world evidence to improve patient outcomes. Professor Sir Rory Collins spoke at the recent workshop and argued that rather than placing greater reliance on observational data, it would be much better to make it much easier to do randomised trials properly. You can watch the talk here.
Professor Rob Califf, in a recent talk at the National Academies of Sciences, Engineering, and Medicine workshop on “real-world evidence” highlighted the failures of the current healthcare evidence generation system and presented a new approach to conducting randomised trials. You can watch the talk here.