Table 1.

Factors facilitating registry use for supporting regulatory assessments

Factor	Value in supporting registry use for regulatory assessments
Use of common core data sets	Collecting a common core set of data items with agreed definitions and data dictionaries increases the capacity to combine or pool data across patients or registries for regulatory assessments. Ideally, data items match regulatory needs. Capacity to collect additional data elements, even for a limited period, may be beneficial.
Common data coding terminologies	The availability of coding terminologies such as the Medical Dictionary for Regulatory Activities (MedDRA®) that could be used by all registries helps in facilitating the conduct of studies using data from multiple registries [21].
Complete information collection	Complete information on critical disease variables is necessary. Medication information is often limited; primary disorder medication information is essential and should include the start and stop (where applicable) dates. Most registries do not record other medications, but some information is desirable. PROs are of increasing interest to stakeholders, but are not collected in most registries.
Data access and sharing	Clear consent specifications on data use facilitate sharing of registry data with third parties including regulators and MAAs/MAHs. Data sharing and access are further determined by relevant national and European data protection legislation.
Data linkage capacity	Linkages to external databases, for example, prescription dispensing, employment, or death registries add to the value of registry data, but linkages may be variable across member state.
Registry reporting, and quality assurance processes and governance	Most registries have processes in place for annual reporting and for quality assurance including source data verification. While these are heterogeneous currently, they represent good baselines for further development in individual registries.
Timeliness of consideration	Consideration of registry data in the authorisation process generally occurs when risk management plans and post-authorisation data needs are being discussed. Planning early in the authorisation process for registry use facilitates data access by reducing timelines for data upload from treating centres and for registry quality assurance processes.
Direct communication	To best fulfil regulator-requested or regulator-imposed studies, regulators, MAAs/MAHs and registry holders need to communicate directly.
Sustainability	Registry funding and support may be limited, causing difficulties in maintaining database systems, reliable quality assurance processes, and staff training. Data entry is often done on a voluntary basis and manually by clinical staff either directly or by importing information from electronic health records. Registry sustainability is crucial for long-term development.
Availability of a regulatory framework	EMA guidelines and procedures for PASS and PAES provide a structure for stakeholder dialogue on registry use [22]. EMA scientific advice can support CHMP qualification opinions or advice on the suitability of a registry for undertaking pharmacoepidemiological studies [23].

CHMP Committee for Medicinal Products for Human Use, EMA European Medicines Agency, MAAs/MAHs marketing authorisation applicants/holders, PAES post-authorisation efficacy studies, PASS post-authorisation safety studies, PROs patient reported outcomes