What is it?
Patient registries are organised systems with a ’predetermined scientific, clinical or public health purpose’ (PARENT definition). They are used to prospectively collect, analyse and disseminate data on a group of patients with specific characteristics in common, for example people with a particular condition, having a certain treatment or using a health-related service. They are cohort studies that are developed with a predetermined health-related aim. Patient registries evolved from paper-based records in a doctor’s office to data collected electronically, usually in databases. They often containing large amounts of data, such as clinical information or biological samples stored in biobanks.
Patient registries may be classified as:
- disease or condition registries, based on populations with a particular disease or group of diseases (ISPOR definition)
- product-specific registries, based on populations using specific products (often developed by manufacturers to assess long-term safety and adverse effects).
Examples of patient registries
- The European Cystic Fibrosis Society registry is used to collect data on cystic fibrosis and its treatment, both to improve treatment and provide data for epidemiological research.
- The Cross-border Patient Registries Initiative (PARENT) has developed a ‘registry of registries’ that lists over 200 patient registries across Europe.
Why is it useful?
- Informing routine clinical practice and disease management: especially in post-marketing surveillance.
- Estimating effectiveness and/or relative effectiveness: by collecting data on:
- the natural history of disease
- clinical and/or comparative effectiveness outcomes
- safety or harm from exposure to health technologies
- quality of care and quality of life.
Registries are being used to:
- support the European Medicines Agency’s (EMA’s) initiatives for early access to medicines, such as the adaptive pathways approach, which strongly builds on real-world data
- monitor orphan drugs, personalised medicines, advanced therapies and biosimilars
- provide insight into the use of medicines under real-life conditions, which inform health technology assessment discussions
- improve healthcare systems, through disease registries that are part of health systems and daily practice and through product-specific registries that provide information on the safety and effectiveness of treatments used in practice.
What are its limitations?
- Quality of data and methods: patient registries are only as good as the underlying data collection and methods. It is critical that the research design is robust and the relevant outcomes are defined appropriately.
- Incorrect or missing data: problems with remembering information or recalling information correctly can be an issue with patient-reported data.
- Confounding and bias: there may be issues such as channeling bias to newer treatments for sicker patients, information bias and selection bias.
- Heavy investment in time and resources: commitment and engagement are needed from participating patients. It can be difficult to recruit patients and it can take time to accumulate data, resulting in delays between data collection and reporting (sometimes years). Such data can become out of date in fast-changing disease areas.
- Lack of standards and uniformity: lack of standardised data collection across hospitals, regions and countries prevents the pooling of data across registries.
- Lack of comparator: in product-specific registries having no comparator prevents assessment of relative effectiveness.
What do stakeholders say?
Acceptability to stakeholders is dependent on the registry methods and robustness of design.
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) has published guidance on reliability and validity of data sources, including registries. Most healthcare agencies value single cohort studies (in other words, those without a comparator), which can be especially useful for rare outcomes and for examining the safety of orphan drugs (ISPOR presentation).
Drug exposure and safety measurements through registries are well accepted by regulatory bodies and health authorities, and are often required in post-marketing surveillance.
Advantages of patient registries for patients include:
- outcomes that are relevant to patients but might be missed or not appropriately captured in trial data can be considered, such as health-related quality of life (see the report from the Agency for Healthcare Research and Quality)
- patients can be actively involved in clinical research and have input into drug development.
However, as registries contain sensitive health information, data security is a higher priority for patients than other issues.
Findings related to the accessibility of patient registries from GetReal case studies are reported here.
Denise Umuhire, Janssen EMEA