Patient registries are where big data meets new and exciting regulatory opportunities in pharma, and regulators are keen to address the gaps between regulation and innovation in this space. The European Medicines Agency (EMA) has published new draft guidelines on registry-based studies (Guidelines) for a three-month public consultation period. The Guidelines make the case for optimising the use of registry-based studies as a source of real-world evidence.
The new Guidelines will be an essential tool for a range of players in pharma and data science, from marketing authorisation applicants and holders, to the data science companies creating and managing registries.
We’ve set out our top 5 takeaways from the draft Guidelines below:
- Prior consultation is key: If your organisation is proposing to use a registry-based study, engage in prior consultation with national competent authorities, where applicable, and with EMA via the procedure for Scientific advice and protocol assistance.
- Is data privacy the elephant in the room? The Guidelines urge compliance with data privacy laws in connection with registry-based studies, but offer little in the way of hard guidance on complex questions like legal bases for processing (including jurisdictional variations across the EU on this issue) and ensuring data minimisation under the GDPR. This is in stark contrast to the Guidelines’ detailed overview of regulatory requirements related to registry-based studies outside the data privacy space.
- The case for using registry-based study for evidence generation: Registry-based studies can be useful throughout the lifecycle of medicines, including:
- supplementing the evidence generated in the pre-authorisation phase: one of the most exciting possibilities for studies based on registries is contextualising the results of uncontrolled trials, and providing comparator groups of patients for a single arm trial where a randomised controlled trial (RCT) is not feasible or unethical. They may even be used to support registry-based randomised controlled trials or patient recruitment (for example to identify patients meeting inclusion/exclusion criteria) and data collection.
- providing data sources or infrastructure for post-authorisation evidence generation: for example, patient registry-based studies can be data sources for RCTs and non-interventional studies, post-authorisation efficacy studies (PAES) or post-authorisation safety studies (PASS).
- Planning a registry-based study: Planning a registry-based study can be complex. Steps can include: identifying one or several suitable registries; obtaining agreement to collaborate from each registry as well as from each individual centre if no central registry coordination exists; possibly identifying a third-party to be involved in the study and setting up a database, a data extraction process and quality control activities. The Guidelines set out detailed tips on how to make this happen as smoothly as possible.
- Next steps? As part of the consultation process, EMA is hosting a virtual workshop on 19 October 2020 to present the draft guideline to key stakeholders. Stakeholders are invited to send their comments to the consultation via an online form by 31 December 2020.