Problem statement

Regulators may require marketing authorisation applicants/holders (MAAs/MAHs) to establish a registry in order to measure the safety or efficacy of individual products in routine clinical practice. Various challenges with regard to registries currently exist, including a lack of sustainability and a lack of harmonised protocols and data structures. In addition, existing disease registries or other data sources established at national or international levels by physicians’ associations or national agencies are not fully utilised, a situation that may lead to duplication of efforts and inefficiencies. Therefore, the need has been identified to explore opportunities to improve this situation by making better use of existing registries and facilitating the establishment and utility of new registries as a source of high- quality post-authorisation data for regulatory decision-making.

Objective of the initiative

The main objective of the initiative for patient registries is to facilitate the use of existing patient registries and facilitating the establishment and utility of new registries if none are available or adequate, in order to collect and analyse high quality data informing regulatory decisions.
The initiative for patient registries includes two components: a strategy on registries and a pilot phase to test whether this strategy better supports MAAs/MAHs to meet regulators’ (and potentially other stakeholders’) needs for data and information.


Registry – The term registry is used in this context to indicate an organised system that uses observational methods to collect uniform data on a population defined by a particular disease, condition, or exposure, and that is followed over time.
Disease Registry – A disease registry includes patients based on diagnosis whereas product registries include patients based on the treatment they receive. Disease registries therefore gather insights in natural history and clinical aspects of diseases and allow comparison between different treatments prescribed for the same indication.
The distinction between the two approaches is however imprecise as patient populations covered by registries can vary which makes it difficult to circumscribe the concept of registries to being only disease-based or product-based. Therefore, the term patient registry is used in this document, taking into account that the design of a registry (including the definition of the patient population and the outcomes to be measured) should be primarily based on the objectives and the planned analyses as described in a protocol.

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Acknowledgement: Prof David Hutchinson,