The Medicines Company Developed Nimble Protocols to Scale for Global Trials
The Medicines Company (Now Novartis) Applied CTTI's Quality by Design Recommendations
For the first CTQ factor (recruitment), the study team required sites to create a list of patients they could invite to participate and also used the National Health System's central system (NHS Digital) to identify potential participants. For the second CTQ (retention), the study team developed an Information Technology (IT) system that allowed sites or regional coordinating centers to have a real-time view of missed or late appointments, as well as non-compliance with the medication. They also established a chat function with study coordinators to enable real-time troubleshooting for issues during the trial. The team established a long, two-month run-in period between the first trial visit and the start of the randomized treatment; the rationale was that if patients were still interested in participating after two months, they would more likely stay enrolled for the 5-year duration of the trial. Sites with a good track record of patient retention were subsequently selected for the trial.
To help the team stay focused on what matters to the study, they pressure-tested every suggested component for the protocol with four core questions:
- Why do we need this component (or do we)?
- Can we do this in an easier way?
- What are the drivers of this component?
- Can we get this information elsewhere?
Resisting the urge to heavily formalize the process across an organization can help QbD become a common sense, helpful approach rather than a bureaucratic to-do that may unnecessarily burden teams.