2 Patient registries

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Methodological guidelines » 2 Patient registries

2.1 Definition of a patient registry
2.2 Types of Patient Registries
2.2.1 Disease or Condition Registries
2.2.2 Product Registries
2.2.3 Health Services Registries
2.3 Diversity in Use of Patient Registries
2.4 Overview of European Registries
2.4.1 Member State level registries overview
2.5 Key issues arising within registries

Patient registries collect, analyze and disseminate data and information on a group of people defined by a particular disease, condition, exposure or health-related service.

Key principles:

  • Registries serve a predetermined scientific, clinical or/and public health (policy) purpose - the improvement of patient care and healthcare planning as well as social, economic and quality of life outcomes and other health indicators.
  • According to how their populations are defined, they can focus on a disease/condition, medical product or health service.
  • European registry landscape is a collection of divergent registries often built for a single purpose and with a limited user profile operating under different legal frameworks and with little standardization in interoperability and governance rules.
  • European registries face the issues of:
    • unstable funding,
    • legal ambiguity,
    • unclear stakeholder roles,
    • predominantly paper-based data collection,
    • lack of awareness of existing standards and standard processes,
    • compromised data quality,
    • lack of registry transparency and openness that support data access for research purposes
    • insufficient data dissemination

2.1 Definition of a patient registry

In the field of health, several definitions of the term registry or register[note 1] have been provided. In 1949, Bellows [1] defined register as “system of recording frequently used in the general field of public health which serves as a device for the administration of programs concerned with the long-term care, follow-up or observation of individual cases.” In 1974, the WHO [2] defined a register as a “file of documents containing uniform information about individual persons, collected in a systematic and comprehensive way, in order to serve a predetermined purpose.” Another definition was provided by Solomon et al. [3] who defined a registry as a “database of identifiable persons containing a clearly defined set of health and demographic data collected for a specific public health purpose.” A slightly different definition of a registry is proposed by ISPOR [4], which describes a registry as a “prospective observational study of subjects with certain shared characteristics, which collects ongoing and supporting data over time on well-defined outcomes of interest for analysis and reporting.” A more specific definition is provided by the US National Committee on Vital and Health Statistics [5], which defines a registry as “an organized system for the collection, storage, retrieval, analysis, and dissemination of information on individual persons who have either a particular disease, a condition (e.g., a risk factor) that predisposes (them) to the occurrence of a health-related event, or prior exposure to substances (or circumstances) known or suspected to cause adverse health effects.” Despite variations in definition, it is clear that a registry involves a long-term, systematic and organized process of collecting data, which is driven by specific, predefined aims.

Nowadays the term “patient registry” is often used in the health domain. The use of the term “patient” in combination with “registry” (i.e. patient registry) is mainly used to distinguish the focus of the dataset on health information [6]. The AHRQ [7] provides the definition of the patient registry, which is “an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes”.

For the purpose of these guidelines, patient registry is defined as…

... an organized system that collects, analyses, and disseminates the data and information on a group of people defined by a particular disease, condition, exposure, or health-related service, and that serves a predetermined scientific, clinical or/and public health (policy) purposes.

2.2 Types of Patient Registries

Registries should be designed and evaluated with respect to their intended purpose(s) which can be broadly described in terms of patient outcomes. Some of the major general purposes for establishing and running a patient registry are to describe the natural history of disease, to determine clinical and/or cost-effectiveness, to assess safety or harm, and to measure quality of care, as well as to serve public health surveillance and disease control. In broad terms, patient registries should contribute to the improvement of patient care and healthcare planning as well as social, economical and quality of life outcomes and other health indicators (e.g. access to healthcare, health status, subjective and objective quality, health financing etc.). By following patients in terms of time and location, medium- and long-term outcomes can be observed. A fine differentiation of the types, sub-types, main and secondary purposes of each patient registry is essential. For example, a diabetes registry compared to a surgical procedure registry shares many common datasets that are achieving completely different purposes. Although the logic of tracking how patients progress over time and factors that contribute to outcomes apply to both, there is a clear difference between the two registries used as an example here, as eligibility is characterised by a diagnosis and by an intervention, respectively.

The majority of patient registries can be divided into three general categories with multiple subcategories and combinations. These categories include observational studies in which the patient has a particular disease or condition, has had exposure to a product or service, or various combinations of these.

The multitude of possible combinations of categories and subcategories can sometimes lead to overlaps in many registries and difficulties in determining the taxonomic position of a particular registry (an example is a registry for treated drug addicts primarily a disease, product or service registry, or a mixture of equally important purposes: disease surveillance, outcomes, natural history of disease, national intervention programmes evaluation). Furthermore, in some countries a very clearly defined chronic disease registry (such as a cancer registry) very often serves many secondary purposes, some of which could eventually become its primary purposes. Therefore, in order to establish an appropriate data exchange (sharing) framework for secondary data use in particular (i.e. research questions), an extensive in-depth context analysis of each registry’s content unit (data set, data element with properties and classes, value domains and property) should be performed. Such analysis would enable a correct interpretation of the results and a transparent disclosure of methodological restrictions.

Related to this, one of the most important quality indicators of patient registries is the amount and frequency of registry-related scientific publishing (meta-analysis and/or systematic review-like approach). See subchapter 4.3, 5 and 8.1.6.

With the help of information gathered through literature review, as well as with the insights gathered through the construction of the questionnaire and subsequent survey of registries for the RoR pilot, and with concern for the above stated complexity of taxonomy of registries, a multi-level classification of patient registries is offered [note 2] (Table 2.1).

Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products, medical devices or diagnostic/therapeutic equipment. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure, or the same group of conditions such as a disability [7].

Table 2.1 Patient registry classification
Category Diseases and conditions Products Services, events
Object type chronic, acute communicable, rare diseases, disabilities, cause of death medicines, devices, equipment diagnostic, curative, preventive, discharges, births, abortions
Purposes / objectives (primary and secondary) disease surveillance, control, natural course of disease post-market surveillance intervention evaluation, quality of care
health outcomes (objective, patient reported)
effectiveness (clinical, comparative, financial)
safety and harm (HTA, vigilance)
intervention (planning, guidelines, reminders)
Coverage (geographical and organizational) health care unit (GP, hospital)
local (counties, districts, insurers, professional associations, NGOs)
national (MS, non-MS)
international (regional, EU, European region, global)
Population definition population (geographically based)[note 3]
population based (exposition dependent)[note 4]
Observation unit patient (user, client, insuree)
person with a characteristic of observation person related device, equipment item person related event (birth, death, service)

2.2.1 Disease or Condition Registries

The main inclusion criterion which disease or condition registries use is the state of a particular disease or condition. That state varies, as the patient may have a lifetime disease (e.g. rare disease such as cystic fibrosis, chronic condition such as disability) or for a more limited amount of time (e.g. short-term infectious disease). The disease registry could be hospital/clinic-based or population based. The former is used for a specific disease irrespective of the location of the case. Alternatively, a population based registry is used to compile information on specified diseases by region, community, and state in which they are diagnosed. The aims of disease or condition registries are most often primarily descriptive, such as describing the typical clinical features of individuals with a disease, variations in phenotype, and the clinical progression of the disease over time (i.e. natural course of the disease). The value of disease registries is increasingly recognized as they are able to provide historically comparable data and long-term evaluation, potentially serving as an addition to randomized clinical trials, and thus providing insights about real-sites outcomes that could not be addressed in the limited controlled studies. These registries become even more important to regulators (and other parties involved) when the disease cases are rare or require highly specialised health intervention. Here registries may be the only means by which data can be obtained.

Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure, or the same group of conditions such as disability [7].

As an example of an EU project/initiative concerning improving disease registries in terms of defining purposes, legal context, semantic and technical aspects, EUBIROD (“European Best Information through Regional Outcomes in Diabetes”) (http://www.eubirod.eu) is mentioned here. The project aims at sharing knowledge about prevention, treatment and patient care. Although there is a large amount of data and reports available, the information on diabetes in Europe is scattered and under-utilized. For this reason, the objective of the EUBIROD project was to improve information supplied to the public and formulate appropriate strategies, policies and actions and targeting appropriate sustainable coordination, in the area of health information, collection of data and information, comparability issues, exchange of data and information within and between Member States, continuing development of databases, analyses, and wider dissemination of information, and in fact to build a common European infrastructure for standardized information exchange in diabetes care. The main outcome of the project is a permanent and sustainable online standardised exchange of data and knowledge between EU countries [8]. Production of information is primarily enabled through the use of a common dataset[note 5], automatically achieving results that can later be harmonised to produce global indicators. Overall, EUBIROD can serve as a good example and model to be re-used for other chronic diseases as well [9].

There is also the European Academy of Allergy and Clinical Immunology (EAACI) (http://www.eaaci.org) as the next example of making efforts in improving disease registries. It is an association of clinicians, researchers and allied health professionals, dedicated to improving the health of people affected by allergic diseases.

The EAACI project also has goals to help standardization of data collection on allergic diseases, diagnosis and treatment and ultimately improve allergic disease and allergen exposure management. EAACI overall project objective is to provide a platform for the establishment of allergic disease registries across EU country borders to develop suitable monitoring tools for use in both clinical practice and research. The initial A-reg project is focused on two national allergic disease registries that are planned to grow into a pan-European registry, namely anaphylaxis and drug allergy. The main advantage of starting a registry in several European reference centres at the same time is that the same methodology (data collection, software use, data analysis and ethics) ensures direct comparability (see chapter 4.2.1, 5 and 10.10.2) from the start.

Since PARENT’s main aim is to support EU MS in developing comparable and coherent patient registries, EAACI recognized this effort and has joined forces with the PARENT project as an official Partner Organization [10][11].

Regarding cancer, EU policymaking institutions of the EU identified cancer control as a major public health priority, and consequently many EU projects/initiative were started. During 2009-2013, the European Partnership for Action Against Cancer (EPAAC) (http://www.epaac.eu) was established. It conceived a framework for identifying and sharing information, capacity and expertise in cancer prevention and control, in order to avoid scattered actions and the duplication of efforts. The main objective was to assist countries in developing National Cancer Control Programmes (NCCPs), but also includes goals on health promotion and prevention regarding cancer, screening and early diagnosis, research support, and mapping the existence of various data and information sources for cancer in Europe as well as checking the availability and quality of these data [12]. Given the importance of cancer registries, much effort has been made to monitor and improve the quality, type and coverage of the information they gather. The European Network of Cancer Registries (ENCR) has the goal to enhance comparability of cancer incidence data, promote cancer registration in the European region, and foster the use of cancer information for research and planning. Today, more than 200 cancer registries are active under ENCR in Europe. Data collection systems in the EU reflect the specific organisation of national health systems, and barriers in data access persist. The move from the national to the European scale is still difficult as not all indicators are comparable across the EU. Registries presently provide most epidemiologic data on cancer, yet they are underfunded, mostly understaffed, struggling with national and European laws on protection data, or launched without proper planning [13].

In the area of cancer control, information and data are precious resources for researchers, health professionals and policymakers alike. Potential advantages in the cross-border exchange of cancer data are numerous, but achieving this goal is by no means simple. Cancer registries, being the main repository of data, vary widely in terms of geographical coverage and data quality.

The EPAAC project gathered insights about these issues and has given attention to the need to create an integrated and comprehensive European Cancer Information System (ECIS). The main tasks of an ECIS should not imply collection of new data, but rather reorganisation and better coordination of existing activities. Five main types of tasks which should be carried out under ECIS, have been identified: data management (each dataset flowing into ECIS must be organised according to a unique and coherent structure); data quality control (continuous improvement of quality and data standardisation as the only way for obtaining reliable data; datasets organisation (a user-friendly pathway should be implemented to structurally connect different datasets) (such cancer incidence and risk factors distribution across populations); data analysis (a plan of analysis for the main outcomes should be systematically and periodically laid down); information sharing (the ECIS would be a key epidemiologic infrastructure for the European Research Area and the results should be disseminated through general and specialised publications, press, leaflets, and web-based tools) [13].

When discussing disease or condition registries, rare disease registries are given a special overview, due to their specificity. By EU definition [14], a disease or disorder is defined as rare when it affects fewer than 5 individuals in every 10.000 citizens. Yet, because there are so many different RDs – between 6.000 and 8.000 – taken together they affect a significant proportion of the population. Between 30 and 40 million people in the EU, many of whom are children, suffer from rare diseases. Most rare diseases have genetic origins while others are the result of infections, allergies and environmental causes. They are usually chronically debilitating or even life-threatening.

Just to list a few examples, there are registries for: Niemann-Pick disease [15], Fabry disease [16] and organic acidurias and urea cycle defects [17]. A common aim of rare disease registries is to contribute to a better understanding of the natural course/history of rare diseases, through pooling cases of rare diseases, and studying their outcomes. Additional objectives of rare disease registries are to connect affected patients[note 6], families and clinicians, and to support research on various (genetic, molecular, physiological) bases of rare diseases.

In the case of rare disease registries and due to low individual prevalence and the scarcity of information, knowledge and experience related to each rare disease, research is often conducted on the widest geographic scope possible (i.e. multi-nationally and/or across the continent), as the benefits of international collaboration, sharing efficiencies and maximization of limited resources should be most obvious here. Also, when resources are combined, identifying standards (i.e. common data elements) becomes more important to allow data to be compared and shared across registries.

Considering the specific nature of rare disease registries another thing may come to mind – creating a single global registry for each disease (or a certain group of diseases). That however is not always feasible, for a multitude of practical reasons and, most importantly, a single global registry would not always be in the best interests of researchers.

At EU level, much is being done to increase research, funding, and public awareness of RD (rare diseases).

To aid the EC with the preparation and implementation of Community activities in the field of rare diseases, The European Union Committee of Experts on Rare Diseases (EUCERD) (http://www.eucerd.eu) was established. The EUCERD issued Recommendations on national/regional RD patient registration and data collection, which summarize the guiding principles that future actions on RD registration will rely upon and which harmonisation and standardisation procedures should be based across national and regional registries in Europe. [note 7]

Through project initiatives EUCERD is performing additional tasks, divided into five main areas:

  1. The implementation of plans and strategies for rare diseases at national level;
  2. The standardisation of rare disease nomenclature at international level;
  3. Mapping the provision of specialised social services and integration of rare diseases into mainstream social policies and services;
  4. The leveraging of the value of EU networking for improving the quality of care for rare diseases;
  5. The integration of RD initiatives across thematic areas and across Member States [18].

Orphanet (http://www.orpha.net/consor/cgi-bin/index.php) is another initiative related to RD, and is considered here a good practice. It is a reference portal and database for information on rare diseases and orphan drugs, run by a large consortium of European partners, with an aim to help improve the diagnosis, care and treatment of patients with rare diseases. Some of Orphanet’s services include: an inventory of RD and its classification; an encyclopaedia of RD; a list of European RD registries [19]. One of the benefits of the listed services is assistance in identification of potential data sources and collaborators.

EPIRARE (http://www.epirare.eu) (The European Platform for Rare Disease Registries) project is another important action in the RD field on the EU level. Its wide-ranging mission includes several areas such as: to provide RD methods and guides for EU researchers and policy makers, while also aimed at agreeing on a common RD data set, disease-specific data collection and data validation, simultaneously addressing legal and ethical issues associated with the registration of RD projects. In order to accomplish these objectives EPIRARE has, among other things, conceived a central website platform which would share information and resources (data repository function and predefined output production), and hence increase the sustainability, networking and interoperability of registries, promote the use of standards and of registry quality procedures (common data set and quality assurance function) and provide an effective way of disseminating the results.[note 8] EPIRARE has produced guidelines for data sources and quality[note 9] and by working on the existing registries it is attempting to formulate the core data elements, which then might be shared in a useful manner within the registry platform. The types of datasets being studied are: a minimum set of common data elements to be collected by all registries (necessary to interlink registries and to selectively extract basic data), other purpose-specific sets of common data elements (selected depending on the predefined outputs to be achieved by the platform), and project-specific sets of data elements (agreed by registries collaborating in ad hoc studies and/or in research on specific diseases).[note 10]

The PARENT project and its Registry of Registries (RoR) component is, although not RD specific, sharing several common goals with the EPIRARE project and is also implementing a cross border platform to support research in various ways.

Another example in the rare disease category is The European Register for Multiple Sclerosis (EUReMS) (http://www.eurems.eu). European MS Platform (EMSP) is developing this tool to assess, compare and enhance the status of people with MS throughout the EU, enabling better data for better outcomes. With regard to persons with multiple sclerosis, across European countries there is currently a widely recognized lack of data at EU and national level on treatment and care for people with multiple sclerosis. A comprehensive approach to data collection in MS is hence needed in addressing these issues, building on existing systems for MS data collection, but bridging their gaps and limitations by incorporating additional purposes, standardized methodological strategies and ensuring its validity across different European populations. Such an effort to provide a cross-border infrastructure for data collection, analysis, interpretation and dissemination of results in the MS field should be built on existing national/regional data collections, registries or cohorts and using the expertise of clinicians, researchers and patient organizations involved. As is the case with other similar cross-border initiatives, interoperability (presented and discussed in chapter 3) is the key enabler here and the prerequisite for completing such objectives.

The OSSE project (Open Source-Registersystem für Seltene Erkrankungen in der EU / Open Source Registry System for Rare Diseases in the EU) (http://www.unimedizin-mainz.de/imbei/informatik/ag-verbundforschung/osse.html), funded by the German Federal Ministry of Health, provides a reusable software for RD registries. The aim of the project is to provide patient organizations, physicians, scientists and other parties with open-source software for the creation of patient registries. As a result, the national registry landscape would be improved to comply with European principles regarding minimum data sets, data quality etc. (summarized in the EUCERD Recommendation on RD registries mentioned above) along with achieving necessary interoperability to allow a federation of registries on a national and international level (e.g. distributed searches designed to comply with data protection requirements and preserve data sovereignty).
OSSE’s backbone is a registry toolkit that enables scientists to build a registry for a specific rare disease even without special IT knowledge. A registry editor allows for the definition of forms for longitudinal and basic medical data and of the corresponding data schema, including an ID management/ pseudonymization service. ID Registry fields (including, inter alia, data type, ranges, measurement units and value sets) are defined within the metadata repository (MDR) which is another integral part of the OSSE architecture, providing semantic interoperability and data quality. It is envisaged that all harmonized data sets for rare diseases would be available through the MDR. Also, each user of the OSSE registry toolkit should register with a registry of registries (RoR). Exchanging data among (national and regional) rare disease registries on the OSSE architecture is achieved taking into account data ownership and privacy aspects, through a search function with specified search queries based on the existing MDR. Depending on the search exposé which contains the description of the research question along with contact information, the data owner decides if and what to reply. Also, the OSSE architecture is not restricted to a single registry software solution but also enables integration of registries built on different software.

Another initiative which aims to develop a global registry or registries for a certain rare disease or diseases is TREAT-NMD (http://www.treat-nmd.eu). It is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. When a clinical trial is being planned, it is very important that patients suitable for that trial can be found and contacted quickly and the best way of ensuring this can happen is to make sure that patients' details are all collected together in a single database or "registry". That registry then contains all the information that researchers will need, including each patient's particular genetic defect and other key information about their disease.

The TREAT-NMD network is creating this kind of registry in countries across Europe and is also linking with other national registry efforts worldwide. The national registries all feed into a single global registry which combines the information from each of the national registries (with a pre-agreed internationally mandatory dataset), and this ensures that patients who register in their national registry can be contacted if their profile fits a clinical trial. In addition, these registries can help researchers to answer questions such as how common the individual diseases are across the world and will support other activities to improve patient care, such as the assessment of care standards in different countries. The network has also, issued a registries tool kit as a useful concise guide for creating a registry (be it general or NMD-specific)[note 11]. Some benefits of the TREAT-NMD registries include [20]: one single entry point for access to patient data worldwide; registries contain accurate, verified genetic diagnosis together with key clinical data items including medication use and ambulation status; patient data are updated at least once a year; it is a powerful feasibility tool as it can filter patients by precise mutation, age, ambulation status, medication type and location; and finally it is a powerful recruitment tool since patients have consented to being contacted about trials for which they may be eligible.

2.2.2 Product Registries

Once a drug or device passes the stage where it is approved for use by a regulatory authority (depending on the national state legislation) the user base becomes much bigger and from a more diverse population than the one in the stage of clinical trials, when the population is narrowly defined and only a small segment of the overall population. To address a need for quality assessment during this important post approval phase is where using a registry for identifying and enhanced understanding of product safety (acute as well as chronic use) should, as one of the available tools, come into consideration. Registries that aim to assess safety or harm associated with the use of various products (drugs) or devices need to anticipate and assess the need for adverse event (AE) detection, processing, and reporting and registry sponsors are encouraged to discuss plans for AE collection and processing with local health authorities when planning a registry.

It is important to note that medical devices are significantly different from pharmaceuticals in the manner in which AEs and product problems present themselves, in the aetiology of their occurrence, and in the regulation governing the defining and reporting of these occurrences, as well as post approval study requirements.[note 12]

Also, compared with drugs, device technologies change more rapidly over a shorter time span, requiring device registries to adapt accordingly to the changes. In addition, healthcare providers may have different levels of experience with the device, which then may influence patient outcomes (especially with devices considered as implants). Medical device registries should attempt to classify all parts of a device with as much identifying information as possible. All of the abovementioned special characteristics of medical devices should be thus taken into consideration when developing a device registry.

Product registries include patients who have been exposed to biopharmaceutical products, medical devices or diagnostic/therapeutic equipment [7].

Device registries can be designed for a variety of purposes, such as providing helpful information on the long-term effectiveness of devices and their safety, combined with keeping track of the impact of factors such as type of surgical technique, surgeon, hospital, and patient characteristics. Proper analysis from medical device registries, with control for the most relevant confounding variables, can often provide important information for decision making by clinicians, patients and policymakers.[note 13]

Post marketing vigilance of medical devices and drugs is needed as too much is unknown about the safety of the product when it is approved, and spontaneous AE reporting is a traditional (and legally binding) method through which this need is addressed. In comparison with spontaneous reporting of AEs, safety/harm registries provide certain advantages, which are here considered. There are two main characteristics of these registries that are extremely important. Firstly, we know from other science fields that any choice data architecture that demands an active and non-systematic effort by the clinician to report an adverse event is inferior (in terms of under-reporting, rather than the quality of reporting) to a systematic follow-up of those events. Secondly, and related to this, in a non-systematic reporting of adverse events we usually do not know the denominator (the exposed population) and are therefore not able to provide any epidemiological measures of disease occurrence. In a structured safety/harm registry with a defined population we can calculate the incidence of adverse events and these registries are becoming increasingly more common in the area of medical products and medical devices [note 14].

Thus, depending on the need to comply with a post-marketing requirement or out of a desire to complement spontaneous AE reporting, the proposed product and disease registries should also be considered as a resource. The registries could be used for examining unresolved safety issues and/or as a tool for proactive risk assessment in the post approval stage. Once again, the advantage of registries is that their observational method and non-restrictive design may allow for surveillance of a diverse patient population that can include sensitive subgroups and other groups not typically included in initial clinical trials (such as children or patients with multiple co-morbidities). In contrast to clinical trials, registry populations are generally more representative of the population actually using a product or undergoing a procedure. To list just a few advantages that those registry features provide: data collection may lead to insights about provider prescribing, and also any follow-up duration can take a long time to identify the consequences of long-term use [21].

Legislation on the EU level regarding pharmacovigilance for medicines marketed within the EU is provided for in: Regulation (EC) No 726/2004[note 15] with respect to centrally authorised medicinal products and in Directive 2001/83/EC[note 16] with respect to nationally authorised medicinal products (including those authorised through the mutual recognition and decentralised systems). There is also a central European medicine agency (EMA) (http://www.ema.europa.eu/ema), which could be roughly compared to the U.S. Food and Drug Administration (FDA), although not centralized and with a lesser level of authority. EMA has issued Guideline on good pharmacovigilance practices[note 17] (GVP) in order to facilitate the performance of pharmacovigilance activities. Finally, EMA is responsible for the management of EudraVigilance (https://eudravigilance.ema.europa.eu/human/index.asp) – an EU data processing network and management system for reporting and evaluating suspected adverse reactions during the development and after the market approval of medicinal products in the European Economic area (EEA).
The current system for medical devices is defined by European Medical Device Directive 93/42/EC[note 18], which sets and describes harmonized standards[note 19] for device manufacturing, labelling, and expected performance and safety profiles to be met. Any medical device placed on the European market must comply with the relevant legislation, where there are three types of medical devices outlined: general medical devices, active implantable medical devices, and In-vitro diagnostic medical devices.

Also, the EC has issued guidelines which aim at promoting a common approach by manufacturers and Notified Bodies[note 20] involved.[note 21] EC Directives also describe the basic standards for manufacturing quality-control systems and responsibilities for AE reporting.

Guidance documents also contain templates for data collection and reports, including ‘‘clinical evaluation reports,’’ which are intended to provide an outline of the technology underlying a specific device and current clinical data supporting its use, ideally in reference to established standards or similar devices. In practice, each country variously interprets the requirements for quality assurance and AE reporting.

Competent Authorities, which oversee NBs in each MS, submit AE and recall data to the European Databank on Medical Devices (EUDAMED)[note 22] (http://ec.europa.eu/idabc/en/document/2256/5637.html), a central database run by the EC. Since the database is non-public the basis for device approval and any post marketing commitments are largely unknown and EU-wide adverse event data are not accessible, though some MSs post market surveillance events in a non-systematic manner. There are numerous product/device registries in the EU, differing in objectives, scope, field of medical expertise etc.

EU-ADR (http://www.euadr-project.org) was an EC funded project (FP7 program) with an objective to design, develop and validate a computerized system that exploits data from electronic healthcare records and biomedical databases for the early detection of adverse drug reactions (ADRs). In this project, electronic health records (EHRs) comprising demographics, drug use and clinical data of over 30 million patients from several European countries were available. EHR databases also form the foundation of the project, insofar as they supply the patient data on top of which the system is built. The EU-ADR system then intended to generate signals (drug-event pairs of pharmacovigilance interest) through the use of data mining, and epidemiological, computational and text mining techniques. Finally, an ultimate objective of the project was to demonstrate that an earlier detection of ADRs is possible through using EHRs [21].

EuraHS (European Registry for Abdominal Wall Hernias) (http://www.eurahs.eu) is a registry which observes hernia operations and not patients. Its mission is to develop and provide for all members of the EHS (European Hernia Society): an international platform for registration and outcome measurement; an online platform for reporting early or late mesh complications (as a survey of implant materials); a set of definitions and classifications for use in clinical research on abdominal wall hernias; a uniform method of presenting outcome results in clinical studies of its repair. It is also trying to convince existing European hernia databases to join the EuraHS, in order to collect their data on the same Internet platform, and to fulfil the goal of the registry as being a good instrument to acquire data for post marketing surveillance, increasing quality and quantity of outcome reports in hernia device- related surgeries [22].

EAR (The European Arthroplasty Register) (www.ear.efort.org) is a major activity of the European Federation on National Associations of Orthopaedics and Traumatology (EFORT). It is organized as a scientific non-profit making society located in Austria. It was founded in 2001 as a voluntary network of national arthroplasty registries. The main aims of the EAR project are: to support national orthopaedic societies to establish national arthroplasty registries based on the EU-level standardization and harmonization of processes, to conduct basic research on comprehensive patient registries, to give support for scientific activities related to arthroplasty registries and cooperation with other stakeholders using arthroplasty registry data. Currently, 30 projects in 26 countries in Europe, Israel and Saudi Arabia are linked to the EAR-network. The projects are in different stages of development. The work is organized at national level as a cooperation of orthopaedic societies and national public health authorities. EAR’s main focus of activities is on outcome research and methodological research in the context of arthroplasty registries. Arthroplasty Registries are considered as a powerful instrument to assess the performance of arthroplasty procedures, and a major source for scientific discussion. EAR supports the development of Arthroplasty Registries and registry documentation, and aims to enhance the comparability of reports by standardization. EAR also produces minimal datasets which are included in all national arthroplasty registries upon which EAR’s evaluations are based [23].

As already discussed above, a product/device registry may possess great potential and effectiveness in areas of post market surveillance, adverse effect reporting, assessing safety and harm but also in improving quality of care, depending on the registry's objectives.

The Swedish Hip Arthroplasty Register (SHAR) (http://www.shpr.se/en) is presented as an illustrative example of registry effectiveness. The registry started in 1979, a web-based reporting system has been in place since 1999, and since 2002 it has measured patient reported variables.[note 23] In 2005 the registry also started collecting data about partial arthroplasty. The registry has excellent coverage (patient coverage 98% and hospital coverage 100% in year 2009) [24]. The registry is governmentally funded, and no device-manufacturing industry funding is present (although the registry sells data to industry, without identifiers). The Swedish legal context enables undisturbed data collection. The data are collected after surgery and reported to the SHAR through the internet. In accordance with the Swedish Data Act, all patients are informed about the registry and are free to give up their participation in the registration at any point. Analyses of registry data focus primarily on re-operations, short-term complications revisions (surgeries to replace devices) and patient-reported outcomes. Revision rates for hip implants in Sweden declined substantially over the years, which is largely due to the registry’s success in detecting devices for hip replacement surgery which have longer survival rates. Judging by the registry's success, for instance in comparison with other countries such as the U.S., the survival of hip replacement implants among Medicare patients in the United States (1997-2005) and patients aged 65 and older in Sweden, the failure rate is about three times higher in the U.S. [25].
From its original focus on the devices themselves, the registry has moved on to analyse the whole process surrounding hip implant surgery to find predictors of good and poor outcomes [26]. Also, beyond the registry’s quality improvement purpose, the data in SHAR have been used for research, including several doctoral dissertations and a stream of publications on outcomes associated with different prostheses and surgical techniques; age, ethnic, and socioeconomic predictors of outcomes of hip replacement surgery; the occurrence of rare adverse events; and patient-reported outcomes. The creation of the Nordic Arthroplasty Register Association that pools data from the Swedish, Norwegian, Danish, and Finnish registries is creating additional research opportunities both because of larger numbers and because the countries have different user profiles [27].

2.2.3 Health Services Registries

Another type of patient exposure that can be used to define registries is exposure to a health care service. The focus of health service registries is on providing information used in the management of health services. They are based on service generated data derived from health facilities and patient–provider interactions. Health care services that may be used to define inclusion in a registry include individual clinical encounters, such as office visits or hospitalizations, procedures, or full episodes of care.

Health care service registries are sometimes used to measure and improve the quality of care, defined as “the degree to which health services for individuals and populations increase the likelihood of desired health outcomes and are consistent with current professional knowledge” [28].

Hospital discharge data are a specific type of health service registry data. They are widely available and very useful for monitoring the quality of health services. This source almost always includes individual records that capture different dimensions of the interactions between the health service and the individual (measuring or paying costs, basic statistics of procedures and diagnosis etc.). Hospital discharge data have been used in quality-of-care research and, recently, as an input for effective coverage assessment. As a result, it is advisable to regularly assess the quality of health service data and to help ensure some basic standardization, to the extent possible, to better serve national and regional interests. Regular monitoring also helps to better understand the aggregate capacity of a health system to provide care (productivity of care) [29].

Quality improvement registries (QI registries) seek to use systematic data collection and other tools to monitor and improve quality of care at the local, regional, or national level, as well as to broadcast clinical research. QI registries generally fall into two categories: registries of patients exposed to particular health services (e.g., procedure registry, hospitalization registry) around a relatively short period of time (i.e., an event); and those with a disease/condition tracked over time through multiple health services [7].

QI may be used for various purposes, such as: to monitor trends in the use of certain procedures and to evaluate trends in healthcare usage; to examine provider adherence to safety protocols and best practice guidelines; to monitor the impact of prevention efforts and public health awareness campaigns; to survey the quality of care patients receive.

These registries may identify disparities in the availability of care, identify and investigate sub-optimum practice and processes, as well as demonstrate potential improvement opportunities. The steadily increasing costs of health care (for OECD countries annual health expenditure averaging almost 5% growth rate over the period 2000-2009) [30] imply the need to justify health care interventions and plans with accurate cost/benefit measures and by showing the impact of interventions on relevant outcomes. Without a valid system for monitoring outcomes within institutions there is little space for management to be aware of how their services truly compare with services elsewhere or with pre-determined quality standards. Since a registry can continuously record data, it has the potential to identify unnecessary or inappropriate variations of healthcare quality and incite its improvement by creating a feedback loop which can pinpoint areas of poor quality [30]. Longitudinal data also provides the needed understanding in order to act as an early warning system if quality declines.

Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization [7].

Registries can drive quality in a variety of ways, be it indirectly – through stimulating competition, or directly – through evaluating adherence with best practices and through affecting healthcare policy (pricing and regulation). In order to improve measurement of quality health care indicators, one should fully exploit the potential of (national) registries, particularly through the implementation of unique patient identifiers, secondary diagnostic coding and present-on-admission flags.[note 24]

As creating and maintaining a registry of this type may require (considerable) resources, narrower focus of quality registries should thus be concentrated on conditions and procedures where outcomes are thought to vary and where improvements in quality of care have the greatest capacity to improve quality of life, and reduce costs [31] (i.e. monitoring renal transplantation outcomes, as poor outcome from this procedure forces patients to revert to haemodialysis, with subsequent consequences of lower quality of life means much higher costs to society). Finally, the ultimate purpose of data from quality of care registries should be to inform clinical practice, policy development and resource allocation.

Although these registries share common objectives in improving quality and can prove a powerful tool in improving health care value, their usefulness on carrying out the objectives varies depending on the registry’s stakeholders (research or health policy oriented), scope, quality of data and finally utilization of registry information by policy makers.

The EC is supporting such initiatives, and one such is example is HoNCAB (http://honcab.eu) – a pilot network of hospitals related to payment for care for cross-border patients, with the main objectives to determine patients' rights in terms of access to cross-border health assistance and entitlement to reimbursement of such treatment, to ensure access and provision of safe, high-quality, efficient and quantitatively adequate healthcare abroad, to support collaboration between MSs regarding healthcare, and finally to obtain a better understanding of the financial and organisational requirements that may arise as a result of a patient receiving healthcare outside the MS affiliation. The network of hospitals features a functioning organisational structure and established means of communication, supported by a web-based database to collect and exchange information, all with the aim to share between MS practical experiences, problems and solutions related to cross-border care.

The benefits of quality of care registries are apparent. For example, the registry set up by the Danish Lung Cancer Group through feedback of indicators of high-quality care derived from registry data to those delivering care has been largely responsible for improvement in 30-day, 1-year and 2-year survival rates for people with lung cancer of 1.6%, 8% and 10%, respectively [32].

There is an international momentum gathering to develop new clinical registries as quality-improvement measures.

At EU MS level the biggest effort in developing quality of care registries is in Sweden, where a system of national quality registers (http://www.kvalitetsregister.se) has been established (since the 1970s). The system is recently on the rise, going hand in hand with a number of initiatives at both the national and local Swedish government levels and suggesting that governance of health care services is guided by an emerging performance paradigm. Although the traditional objectives of distributive justice and cost control are still valid, they have been complemented by objectives concerning efficiency and value for money spent on health care services [33].

Today, Sweden boasts 89 certified national quality registries of various types: interventions or procedures (e.g. hip fracture repair and cardiac surgery); diagnoses and episodes of care (e.g. myocardial infarction and stroke); and chronic disease (e.g. diabetes and leukaemia). National quality registers cover more than 25% of total national health expenditures, about one third of the registries collect patient data on more than 90% of all Swedish patients diagnosed with a given condition or undergoing a particular procedure, and many have been in place long enough to provide unique longitudinal information on patient cohorts.

Thus, in addition to being a comprehensive primary data source for comparative studies, the high percentage of coverage of health services enables Swedish registry data to play an important role in the monitoring and evaluation of health care quality, as well as help in developing nationwide health care policy[note 25], while constantly being a resource for research, one of the registries’ common feats.

The vision for quality registries and competence centres is to constitute an overall knowledge system actively used at all levels (health provider, hospital, regional, state) for continuous learning, and evaluation, development, quality improvement and management of all health care services [34].

A national quality registry contains individualized data concerning patient problems, medical interventions and outcomes after treatment; within all healthcare production. It is annually monitored (quality control) and approved for financial support by an executive committee. Funding comes from central state level and is allocated to a few competence centres, where several registries share the costs of staff and systems which it would not be possible for a single registry to fund. The successful development of the Swedish National Quality Registries is explained largely by their decentralized nature. Caregivers that have the greatest use for data also have the main responsibility for developing the system and its contents, and databases are spread out among different clinical departments throughout Sweden. Another potential reason for success could be relatively liberal legal provisions concerning personal data in Sweden, where special permission can be obtained that allows national personal data to be recorded and processed[note 26] (even in universities) [35].

Also, data quality of registries in the national quality list is quite high and as a result sufficient for use in clinical research [36].

Outside the EU, Australia is also trying to establish a national base of clinical quality registries with goals similar to those of Sweden, and with certain advantages (national level policy) and disadvantages (existing registries lack nationwide coverage). Clinical quality registries in Australia are envisioned as indicated in Figure 2.1 [37].

Figure 2.1: Dependencies of clinical quality registries in Australia

To summarize, when considering a clinical quality registry, collection and feedback of data must be based on an effective central governance structure, with strong clinical leadership, and a regulatory framework that provides incentives for quality improvement and dedicated approaches for managing poor performance. Local clinical leaders should ensure that registry outcomes drive quality improvement.

2.3 Diversity in Use of Patient Registries

As illustrated in the previous chapter, a patient registry can be a powerful tool for a number of potential needs: to understand variations in treatment and outcomes, to examine factors that influence prognosis and quality of life, to describe care patterns, to assess effectiveness, to monitor safety and harm, and to measure quality of care. Through functionalities such as feedback of information, registries are also being used to study quality improvement [38].

Registries today vary by organization, condition and type, and have different strengths and limitations accordingly. Different stakeholders perceive and may benefit from the value of registries in different ways. For a clinician, registries can collect data about disease presentation and outcomes on large numbers of patients rapidly, thereby producing a real-world picture of disease, current treatment practices, and outcomes. For an organization of physicians, a registry might provide data that can be used to assess the degree to which clinicians are managing a disease in accordance with evidence-based guidelines, focus attention on specific aspects of a particular disease that might otherwise be overlooked, or provide data for clinicians to compare themselves with their peers [39]. From a private payer’s perspective, registries can provide detailed information from large numbers of patients on how procedures, devices, or pharmaceuticals are actually used including data for evaluating their effectiveness in different populations. This information may be useful for determining coverage policies [40]. Furthermore, for a drug or device manufacturer, a registry-based study might demonstrate the performance of a product in the real world, develop hypotheses, or identify patient populations that will be useful for product development, clinical trials design, and to identify individuals eligible to participate in research. The use of patient registries varies by priority condition, with cancer and cardiovascular disease having a large number of registries and areas such as developmental delays or dementia, far fewer. Overall, the use of patient registries appears to be active and growing [7].

2.4 Overview of European Registries

The current European registry landscape is often viewed as a collection of divergent registries. Design, development, and maintenance of patient registries revolve around registry platforms (software tools for managing registries’ data). This approach leads to creation of segregated silos, resulting in expensive and inflexible IT systems. Often, registries are built for a single purpose, with their own data stores and for limited user profiles. Furthermore, registries have different legislative and governance rules and obligations and are spread across different European countries and types of organizations. As a result, patient registries implement only a subset of the registry functions, using and producing only a fraction of the registry data, and often not applying existing interoperability approaches (standards, best practices). Thus these registries manifest themselves as islands of data and governance rules.

However, some efforts are being made to improve the situation. Through performing a literature review numerous such projects have been identified and recognized as best practices (briefly presented in chapter 2.2).

The criteria for recognizing best practices are in accordance with overall PARENT aims, and include projects, organizations, initiatives, registries etc. working on national, regional or international levels in the fields of:

  • Recognizing and converging similar sources of data (based on disease, device and/or service) in order to improve surveillance, quality, outcomes, safety and/or effectiveness.
  • Tackling different levels of data exchange (individual or aggregated level, metadata) between similar (group of registries) or different sources of data (registries – EHR – insurance databases).
  • Addressing healthcare data exchange issues such as standards, interoperability, metadata, platform, common datasets etc.
  • Defining needs for efficient health information exchange on different levels (patients, health care providers, researchers, payers, decision makers etc.) and ways to address those.
  • Promoting collaboration, reducing redundancies, and improving transparency among patient registry holders.
  • Aligning patient registries classification, definitions, taxonomy, purpose, development and governance.
  • Adding value through evaluating information produced by secondary use of health data.

The next subchapter presents a list of patient registries and a short descriptive analysis of some of their features.

2.4.1 Member State level registries overview

To date, PARENT WP4 team in collaboration with project partners has compiled a list of registries identified as suitable for being taken into consideration as regional/national/county and/or local level patient registries. The list[note 27] currently contains 1028 registries and is continuously growing as additional information arrives (newly discovered data sources, literature, and information from project partners). It should be noted that the results presented in this chapter below are based on responses from project partners.

Most of the registries from European countries are located in Spain, mainly due to the specific organisational principle of Spanish healthcare registries. The vast majority of patient registries in Spain are county-based, which means that each contains equivalent registries (e.g. Basque Country Cancer Registry, Murcia cancer registry, etc.), while many other countries may collect the same type of data at a national level (e.g. Polish national cancer registry). Other highly represented countries are also characterized by a comparatively high-level of organization of healthcare registries at a national level. These often provide meta-registries or registry lists (UK DocDat, IR HIQA, PT DIS, SE National Quality Registers, etc.) which provide information on a large number of patient registries that are, or have been operating within a certain country. Less prominently featured countries often have a smaller number of active patient registries in total, but may also be underrepresented due to a lower level of international visibility. This may be due to organizational issues, lack of connectedness between registries at a national level and/or lack of other specialized focal organizations at an international level. Although these international organizations often contain comprehensive lists of patient registries, they are often characterized by a specific focus (like Orphanet, which contains the most comprehensive list of data on 641 patient registries, but consists only of rare diseases registries), which is why it is likely that there would be immense benefits from an establishment of a general cross-border meta-registry organized around collecting data on all active patient registries. There are also already several multi-country registries in our list which collect data from several countries at once. These may be either international registries of specific conditions such as coronary events, or specialized international studies collecting patient data.

Table 2.2 Distribution of identified registries across European countries
Country N Country N
Spain 191 Latvia 17
UK 139 Estonia 16
France 82 Slovenia 15
Portugal 66 Netherlands 14
Ireland 65 Multi-country 13
Germany 41 Czech Republic 11
Hungary 40 Switzerland 10
Austria 38 Malta 9
Italy 38 Cyprus 8
Finland 32 Greece 7
Sweden 29 Romania 6
Croatia 28 Lithuania 4
Poland 24 Serbia 2
Norway 23 Albania 1
Belgium 19 Bulgaria 1
Denmark 19 Georgia 1
Slovakia 18 Turkey 1
Total 1028

Based on our general classification (primary purpose) we recognized that the majority (64%) of patient registries were disease/condition based, followed by service (26%) and product based patient registries (10%).

Figure 2.2: Breakdown of all registries based on primary purpose (N=1028)

Further categorizing them into disease/condition based patient registries according to entry criteria definition (particular disease or condition), we recognized several sub-categories based on organ system (cardiovascular, neuromuscular etc.) or clinical field (cancer, rare, congenital, occupational) irrespective of body part focus. The largest number of disease/condition based registries in our list falls under the coronary/vascular subcategory (27%)[note 28], followed by cancer/tumour/haematological (20%), infectious (9%), rheumatic (8%) and pulmonary (7%). Although rare diseases contribute only to 6% of registries in our list, the extended list[note 29] contains 641 rare disease registries in total (to be integrated as a joint activity of PARENT and Orphanet). All other subcategories account for 23% of total disease/condition registries in the list.

Figure 2.3: Breakdown of Condition and Disease based registries (N=655)

While the number of product-based patient registries represented a minority of all registries in our list, two subcategories can be further identified: device registries (most prominently featuring devices such as pacemakers or arthroprosthetics) and pharma registries (registries collecting data on pharmacological products). Less than 20% of product registries belong to the latter, while the much larger proportion of product registries were identified as medical device-based registries.

Figure 2.4: Breakdown of Product based registries (N=103)

Apart from condition/disease and product registries, our review of patient registries yielded a third category of registries which we categorize as service-based patient registries. This group is the most heterogeneous of all and consists of registries whose primary definition and focus is ostensibly based upon healthcare services. The largest identified subcategory contains registries evaluating preventative services, quality of care, and health monitoring. It accounts for exactly a third of all service-based registries and includes population, permanent sample and vulnerable groups’ registries and registries used for evaluating preventative screening programs or monitoring population health. The second biggest subgroup contains various specific medical procedures registries (24%) which monitor specialized surgical procedures, therapeutic or diagnostic services or emergency interventions. All other observed service based registries accounted for less than a half of this group and were subcategorized as registries of transplant procedures and/or donors (blood, bone marrow, organ etc.), various obstetric and gynaecological services registries (births, abortions, medically assisted fertilization), immunization, causes of deaths registries, hospital discharges registries and registries for health/social insurance purposes.

Figure 2.5: Breakdown of Service based registries (N=270)

2.5 Key issues arising within registries

Within this subchapter, we will briefly look into the most important and emerging issues arising from registries. It should be noted that issues presented here are based on the questionnaire survey of registry holders (n=131; registry list available in Appendix A) performed by the PARENT project team. Therefore, the results do not reflect a regulatory perspective, from which other issues within registries may be recognized.

Possible solutions and proposals are listed in subsequent chapters. Emerging issues at national and regional level differ from issues at EU level, in line with differences in setting-up and running a registry. At the same time, it is necessary to point out that the majority of EU-level registries are based on secondary data sources.

Besides, there are different views to key issues regarding the role of the reader: major concerns of registry holders might differ from major concerns of data users. The issues mentioned below are trying to address both sides, but again different views cause different perspective of the same issue. Therefore, one can recommend to browse the list below and refer to later chapters of the guidelines where these challenges are further elaborated.

Not all of the issues listed below are relevant for national or EU registries, but when setting-up the registry, all of them should be considered.

  1. The most important issues among EU registries are unstable funding and therefore limited sustainability. At this point, the differences among national (or regional) registries and EU-level registries are important and influence the sustainability: Funding of national registries by national authorities might not be stable; as mentioned in ‘PARENT - Deliverable 5: Registry analysis and Report’, only half of the registries are currently funded by national government authority, about 16 % have “no specific funding”. EU registries are funded either by an umbrella organisation or by a certain project, which again introduces instability and limited sustainability.
  2. Table 2.3 Funding source
    Funding source (question 7) Initial registry funding - set-up Current registry funding
    N % N %
    National government authority 58 36% 76 52%
    No specific funding 27 17% 24 16%
    Regional Authority 18 11% 18 12%
    University/Research Institute 14 9% 1 1%
    Foundation 12 8% 8 5%
    EU commission agency 10 6% 3 2%
    Hospital 10 6% 5 3%
    Industry 9 6% 5 3%
    Patient Association 2 1% 6 4%
    Total 160* 100% 146** 100%

    * Multiple choice question

    ** The difference in numbers of initial and current registry funding registries is due to missing answers.

  3. There are many legal issues concerning registry set-up, data protection and re-use. Legal backgrounds in Member States differ greatly. At this stage, the preparation of the new regulation on data protection should be pointed out as it might influence the future of the majority of patient registries in EU. Much more on these issues is described in chapter 5.
  4. Within the phase of development (or setting-up) the registry and also later on, the roles of different stakeholders are very important and, in many cases, not very clear. There are different possible roles: data owners, data holders, data users, etc. Much more on these issues is described in chapter 6
  5. Modes of data collection: almost half of the EU registries are still based on paper-and-pen mode (paper based questionnaires, paper based health records and laboratory results). The situation is burdensome for data providers and causes lower data quality. One should point out that paper-and-pen data collection mode is nowadays not desirable since it is costly, time consuming and does not allow any control of the data filled in. To read more on this topic, see chapter and
  6. Table 2.4 Sources of data for a registry
    Sources of data (question 17) N %
    Paper based questionnaires 67 22%
    Electronic health care records 56 18%
    Online questionnaires 53 17%
    Paper based health records 44 14%
    Paper based laboratory results 34 11%
    Electronic laboratory results 26 8%
    Directly from clinical examinations 17 5%
    Interviews 14 5%
    Total 311* 100%

  7. Lack of awareness of existing standards and standard processes when building or maintaining a patient registry. These standards are actually wanted by registry holders. More information on standards is available in chapter and 10.11.
  8. Balance between accuracy and timeliness is usually skewed in favour of accuracy, resulting in low timeliness. Comparability over time and/or space (as another quality component) is often limited due to set-up procedures, specific funding, etc.
  9. Data quality (including completeness) is often compromised. There is low awareness of existing quality standards and there is also a lack of knowledge on quality assessment. On the other hand, only 20 % of registry holders would like to have a common quality control tool (see ‘PARENT - Deliverable 5: Registry analysis and Report’). More on registry data quality is described in chapter 4.
  10. Registry transparency and openness with the emphasis on data access for research purposes: a majority of registries are closed to researchers from other institutions than the data holder. There should be protocols enabling users/researchers to access the data under certain conditions (see project Data Without Boundaries.
  11. Insufficient data dissemination: a minority of registries actually disseminate their aggregated data on the websites allowing users to get easy access to the first results. The commendable exceptions are cancer registries with wide dissemination (see http://eu-cancer.iarc.fr/EUCAN/Default.aspx, http://eu-cancer.iarc.fr/EUREG/Default.aspx, http://eu-cancer.iarc.fr/EUREG/Default.aspx). These registries have established standards that should be followed by other registries. On the other hand, it should be pointed out that dissemination standards differs from country to country; in general, the data in all forms are much easier accessible in, for example, Nordic countries, UK, France,… than in some other countries. To read more on data dissemination, see chapter 8.1.6.


  1. The terms “register” and “registry” are often used interchangeably. However some authors differentiate between these two terms taking the position that the ‘’registry’’ is the organisation and process that supports one or a number of individual ‘’registers’’ [41]. In this paper we are using the term “registry”, the only exceptions are registries’ official names.
  2. This classification is by no means definite or indisputable but subject to change and modification.
  3. A “population registry” is a registry that intends to cover all residents in a given geographic area within a given time period. The coverage of the specific registry may, however, be incomplete, but it is nevertheless a population registry if the aim is to include all the individuals in the target population. A population is defined by geographical boundaries, but usually only residents (or citizens) within a given time period are included in the definition [42].
  4. The term “population-based registry” should be used when all persons with a given trait, exposure or event, are intended to be included in the registry. If the registry includes everyone in the population (even the oldest), it becomes a population registry. Intention rather than performance defines the terms. A population-based disease registry aims at including everyone with the disease in the population, be it self-reported, clinically diagnosed or detected at screening. Population and population-based registries may be further classified as of good or bad quality depending on coverage or other characteristics [43].
  5. EUROBIROD Deliverable D5.1: Common dataset. Available at: http://www.eubirod.eu/documents/downloads/D5_1_Common_Dataset.pdf
  6. EURORDIS (http://www.eurordis.org), as a non-governmental patient-driven alliance of patient organisations, is also bridging the gap between patients, addresses their needs and is active in promoting health policies and services and research policies and actions related to RD.
  7. List of core recommendations is available at: http://www.eucerd.eu/wp-content/uploads/2013/06/EUCERD_Recommendations_RDRegistryDataCollection_adopted.pdf
  8. EPIRARE Deliverable D5: Delivering a European Platform for Rare Disease Registries. Available at: http://www.epirare.eu/_down/del/D5_DevelopingaEuropeanPlatformforRareDiseaseRegistries%20FINAL.pdf
  9. EPIRARE Deliverable D4: Guidelines for data sources and quality for RD Registries in Europe. Available at: http://www.epirare.eu/_down/del/D4_GuidelinesfordatasourcesandqualityforRDRegistriesinEurope.pdf
  10. EPIRARE Deliverable D9.3: Common Data Set and disease-, treatment and other specific modules. Available at: http://www.epirare.eu/_down/del/D9.3_ProposalforCDE_FINAL.pdf
  11. Guide is available at: http://www.treat-nmd.eu/downloads/file/registries_toolkit/UK_SMA_registry_protocol.pdf
  12. Other sources provide more information about defining and reporting of device-related AEs and product problems, and about post marketing studies (including those involving registries), such as: Baim DS MR, Kereiakes DJ, et al. Postmarket surveillance for drug-eluting coronary stents: a comprehensive approach. Circulation 2006; (113):891–7.
  13. (AHRQ) Registries for Evaluating Patient Outcomes: A User’s Guide, 3Ed, Volume 2. In: Guide, editor, 2012.
  14. The term 'medical device' covers all products, except medicines, used in healthcare for the diagnosis, prevention, monitoring or treatment of illness or disability
  15. Available at: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2004:136:0001:0033:en:PDF
  16. Available at: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2001:311:0067:0128:en:PDF
  17. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000345.jsp
  18. http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=CONSLEG:1993L0042:20071011:en:PDF
  19. e.g. ISO 14971 – Risk management for medical devices
  20. Private, for-profit third party bodies that are devices certified for marketing approval
  21. MEDDEV 2.12-1 Rev8 “Guidelines on a Medical Devices Vigilance System” and MEDDEV 2.12-2 Rev2 “Post-Market Clinical Follow-up (PMCF) Studies”. Available at: http://ec.europa.eu/health/medical-devices/documents/guidelines/index_en.htm
  22. Evaluation of EUDAMED from 2012 is available at: http://ec.europa.eu/health/medical-devices/files/pdfdocs/eudamed_evaluation_en.pdf
  23. The patients complete a question form about satisfaction and health-related quality of life, serving as a baseline for comparisons one, six, and ten years after surgery.
  24. OECD Health Ministerial Meeting. Improving Value in Health Care: Measuring Quality. Forum on Quality of Care, Paris, 7-8 October 2010. Available at: http://www.oecd.org/health/ministerial/46098506.pdf
  25. A recent assessment of quality in Swedish health care, including the country's register system, made by the Boston Consulting group, found that the registries are improving quality and efficiency in health care. The report from BCG recognized the potential of registries to increase value in health expenditures, and they estimated that investing in registries in the Swedish context would generate a significant cumulative return over the next years because of improvements in quality. Available at: http://www.bcg.com/documents/file64538.pdf
  26. Possible importance of privacy legislation for success of a registry – see subchapter 2.2.2] regarding the Swedish Hip Arthroplasty Register.
  27. Available at: http://www.parent-ror.eu/#!state/list_all
  28. Mainly due to integration of European Society of Cardiology (ESC) meta registry data – dynamic portal (http://www.esc-crt.org/workstream/Pages/dynamic-portal.aspx Accessed: 9th June 2014)
  29. Orphanet list of rare diseases registries, January 2014 (http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf)


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