Take-home messages from the 2nd day of the joint #HMA-European Medicines Agency workshop on #PatientRegistries
Vicky Valla, Ph.D.
Regulatory Affairs & Intelligence | Market Access | Strategic Planning for MedTech & Pharma
?? Linkage of registries with other #datasources at a country and international level is important to ensure better population representativeness but also in terms of outcomes. Linkage with other types of #realworlddata can increase data quality;
?? The iterative nature of registries must be acknowledged during the design phase: as data will be collected and analysed, research questions and outcomes may need to be revisited. The flexibility and the way to do so must be described. Acc. to a survey, the key elements to consider when assessing the suitability of a registry to answer a research question revolves around #datagovernance #datarepresentativeness, processes in place for #datacollection and #datamanagement and #datasharing arrangements;
?? Registries need to be fit-for-purpose to address specific questions, either of clinical or regulatory nature. For this, it is necessary to establish a Data Quality Framework #DQF to evaluate fit-for-use within the context of a multi-stakeholder dialogue. DQF’s chapter on #RWD needs optimization in terms of applicability but is a useful tool that can enhance interoperability. The clarity about informed consent and consideration of #GDPR requirements will largely determine the usefulness of the tool;
?? Registry-based data must be based on a continued dialogue with patients and #HCPs to establish whether the specific aspects of the disease’s evolution remain meaningful and/or relevant from a clinical POV. The planning of a registry should include all stakeholders – #academia, #clinicians, regulators and patients in early stage of development. Attention should be paid so that registry patients are not “lost’’ when/if they enter a #clinicaltrial;
?? There is an underestimated burden related to the complexity of describing and interpreting cohorts when there is variability in subgroups and populations. Lack of coherence, completeness and #datareliability often result in suboptimal outcomes with limited usability that have an indirect impact to the community’s willingness to engage with patient registries ;
?? ?Involvement of patients is crucial. Patient-reported registries have shown to be of comparable quality with clinician-reported registries. However, #patientreportedoutcomes must be carefully assessed before their incorporation to the Registry’s protocol to avoid underuse or misinterpretations. PROs must be contextualized before they can translate into tangible contributors to regulatory assessments and decision-making;
?? The sufficiency of a registry’s quality is defined, among others, by the extent of data missingness. Dynamics of a registry (introducing new parameters) often generates a 'high' level of missingness among the data collected prior to introduction of the parameter. External data quality audits may be a solution;
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?? Funding of a registry may also determine #dataaccessibility and #datatransparency; variability of roles (patient advocates, industry, academia) in the Board of a Registry may be able to overcome this obstacle. Provisions for the logistical challenges of accessing registries must be accounted for at the very early stages of their development
?? #Informedconsent is of crucial importance and often determines the dynamics of the Registry. A template for informed consent on Registries should be put forward. Sometimes lack of IC blocks accessibility to data completely;
?? ?#Datadiscoverability is EMA’s priority to facilitate regulatory decision-making. The HMA-EMA catalogues of #realworlddata sources and studies will describe them to
?? help #pharmaindustry and researchers to identify and use such data when investigating the use, safety and effectiveness of #medicines.
???support the assessment of study protocols and study results by providing quick access to information on the suitability of data source(s) proposed to be used in the study protocol or referred to in the study report
?? ?boost #datatransparency of studies and data sources
The catalogues will go live on Feb 15 and a multistakeholder webinar is scheduled for March 4th;
?? #Datainteroperability: The HARMONY Alliance | Public-Private Partnership for Big Data in Hematology is a good example of how multistakeholder public-private collaboration with processes in place to receive data from organizations worldwide can seize the advantages of #datafederation. #DataAnonymization, identification of data sources, #datavisualization and dissemination remain the major challenges;
?? Many registries lack education on regulatory requirements and therefore cannot be used for regulatory decision-making. Registry holders must take into account the required burden/resources and adapt to regulatory requirements to ensure the usability of their data.