Patient registries in the field of rare diseases
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17 Anonymised data are useful in allowing information sharing for research purposes with minimum risk. Anonymisation techniques should be standardized to ensure their robustness.
Patient organisations (or patients, when no organisation exists) should be involved in the elaboration of the consent form and the consent form process in order to ensure the best understanding of the information provided.
As a principle, research use of identifiable patient information is subject to legal requirements. The identifiability can be direct or indirect. Especially in the field of RD, a patient with a specific diagnosis, located in a defined place may be easily identified as being the only one. It is recommended to consult with the appropriate national authorities in charge of data protection to ensure compliance with requested standards.
Transparency, oversight and ownership
Transparency contributes to public and professional confidence in the scientific integrity and validity of registry, and promotes inquiries from other interested parties. Registry developers achieve transparency by making the PR objectives, governance, eligibility criteria, sampling and recruitment strategies, general operating protocol, sources of data and of funding, available to anyone. Creating a website describing all these elements is one way to achieve transparency.
Good security is essential for legal, ethical and operational considerations. It should not be underestimated. Security is easier to maintain on a dedicated server, but that also requires a backup strategy and an intrusion detection system on the server itself to avoid hackers.
The independence of a PR depends more on its governance and the funding conditions than on the origin of the funding. Possible governing structures can vary widely depending on the project. The PR developer can be the sole decision-maker, but usually there is a governing board including all stakeholders: the data providers, the patient organisation(s), the funding agency, the professionals running the PR (clinical researchers, statisticians, information technology specialists). It is also desirable to appoint an independent advisory board to provide oversight of registry operations, particularly regarding the scientific independence. RDTF Report on patient registries in the field of rare diseases (June 2011)
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The governing structure has to control that provision is specified to ensure continued care of the database under any circumstances.
The concept of ownership does not fit health information comfortably as it fails to acknowledge individual patient privacy interests in health information. Alternatively, the legal concept of custody is useful. Custodians have legal rights and responsibilities, among which to preserve privacy and dignity of individual patients. Custody is also transferable from one custodian to another, which is particularly relevant for PR which are long-term projects.
Agreements about ownership of data and access to data should be determined by multi- party contracts and not regulated by legislation. Practices should be based on the following principles:
information directly derived from it. Once the information has been processed, it becomes research data (i.e. data) unless there is agreed private ownership. The processor and/or principle investigator of data should be considered as the guardian of the data. As such, it is up to this person to take all the appropriate steps to protect the data, its storage, use and access. It follows that the researcher holds ultimate intellectual property with due consideration for benefit sharing data.
the donation of data by the subject to the researcher eliminates the subjects expectation of an individual compensation, but no the possibility of commercialisation by the researcher through traditional intellectual property rules.
Use of collections by third parties should be allowed providing that there is no transfer of ownership and that the use is in agreement with the present guidelines.
PR can also receive Copyright protection as they satisfy the statutory definition of a compilation.
Finally, careful attention to the ethical considerations related to the design and operation of a PR, as well as the applicable legal requirements, contributes to the success of a PR and ensures the realisation of its social and scientific benefits. A quality procedure should be implemented to ensure the compliance to the present guidelines.
RDTF Report on patient registries in the field of rare diseases (June 2011)
19 Bibliography used for establishing this document
1. Gliklich RE, Dreyer NA, eds. Registries for Evaluating Patient Outcomes: a user’s guide. AHRQ Publication N°. 07-EHC001-1. Rockville, MD: Agency for Healthcare Research and Quality. April 2007. 2.
biomedical research: informed consent, confidentiality, quality issues, ownership, return of benefits. A professional perspective. Eur J Hum Genet. 2003 Dec;11 Suppl 2:S88-122 3.
2009 http://www.epposi.org/upl/1/en/doc/Registries%202009%20final.pdf
The correct form when quoting this document is: S. Aymé, A. Kole, C. Rodwell “RDTF Report on Patient registries the field of rare diseases: Overview of the issues surrounding the establishment, governance and financing of academic registries”, June 2011. http://www.eucerd.eu/EUCERD/upload/file/RDTFReportRegistriesJuly2011.pdf
Annex 1: Participants at the workshop 13 March 2008
Fabrizia Bignami –Eurordis, France
Centre (LUMC), Netherlands
Genzyme Europe B.V., Netherlands
Studio e la Cura dei Tumori, Italy
Europe B.V.
RDTF Report on patient registries in the field of rare diseases (June 2011)
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Hematology and Bone Marrow Transplantation Unit, Italy
Department, University of Padua, Italy
Maggiore Hospital Mangiagalli and Regina Elena Foundation, University of Milan, Italy
Carlos III, Spain
Hungary
Research Centre for Rare Diseases, Italy
Debré, France
Other contributors to the report in addition to workshop participants Helen Dolk - EUROCAT Tsveta Schyns - European Network for Research on Alternating Hemiplegia Liesbeth Siderius - Stichting Shwachman Syndrome Support Netherlands
RDTF Report on patient registries in the field of rare diseases (June 2011)
21 Annex 2: List of rare disease registries in Europe
Orphanet Report Series on Disease Registries in Europe (January 2011) http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf
Disease Registries in Europe O rphanet Report Series Rare Diseases collection January 2011 www.orpha.net HEALTH-F2-2008-201230
2 http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Orphanet Report Series - Disease Registries in Europe - January 2011 Methodology 3 Summary 4 1- Distribution of registries by country 4 2- Distribution of registries by coverage 5 3- Distribution of registries by institution 5 4- Network registries 5 Distribution of registries by country 6 European registries 28 International registries 30 Network registries 31 Table of contents 3 http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Orphanet Report Series - Disease Registries in Europe - January 2011 Patient registries and databases constitute key instruments to develop clinical research in the field of rare diseases (RD), to improve patient care and healthcare planning. They are the only way to pool data in order to achieve a sufficient sample size for epidemiological and/or clinical research. They are vital to assess the feasibility of clinical trials, to facilitate the planning of appropriate clinical trials and to support the enrolment of patients. Registries of patients treated with orphan drugs are particularly relevant as they allow the gathering of evidence on the effectiveness of the treatment and on its possible side effects, keeping in mind that mar- keting authorisation is usually granted at a time when evidence is still limited although already somewhat convincing. This report gather the information collected by Orphanet so far, regarding systematic collections of data for a specific disease or a group of diseases. The concept of coverage (regional, national, european or international) that is associated with patient registries reflects the area of collection of a single physical database, to which several clinical entities are contributing. The notion of networks corresponds to the relationship existing between several databases (or patient registries), whether a coordinating database exists or not. Cancer registries are listed only if they belong to the network RARECARE or focus on a rare form of cancer. American registries are listed only if they cover the European area and focus on a rare disease uncollected in Europe. For any questions or comments, please contact us: contact.orphanet@inserm.fr Methodology 4 http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Orphanet Report Series - Disease Registries in Europe - January 2011 Summary 1- Distribution of registries by country CouNTRy REgIoNal NaTIoNal EuRopEaN global NoT DEfINED ToTal AT - Austria 1 12
0 0 15 BE - Belgium 2 16 1 1 0 20 BG - Bulgaria 0 4
0 0 4 CH - Switzerland 1 5 1 2 0 9 CY - Cyprus 0 1
0 0 1 CZ - Czech Republic 0 4 0 0 0 4 DE - Germany 4 57
5 0 86 DK - Denmark 1 3 0 0 0 4 EE - Estonia 0 2
0 0 2 ES - Spain 4 25 3 0 0 32 FI - Finland 0 5
0 0 5 FR - France 17 92 11 1 1 122 GR - Greece 0 2
0 0 2 HR - Croatia 0 1 0 0 0 1 HU - Hungary 0 3
0 0 3 IE - Ireland 4 6 0 0 0 10 IL - Israel 0 2
0 0 2 IS - Iceland 0 2 0 0 0 2 IT - Italy 9 35
5 0 51 LT - Lithuania 0 1 0 0 0 1 LU - Luxembourg 0 1
0 0 1 LV - Latvia 0 1 0 0 0 1 MK - Republic of Macedonia 0 1
0 0 1 MT - Malta 0 1 0 0 0 1 1 6
5 0 15 NO - Norway 0 3 1 0 0 4 PL - Poland 3 6
0 0 9 PT - Portugal 1 8 0 0 0 9 RO - Romania 0 2
0 0 2 RS - Serbia 0 4 0 0 0 4 SE - Sweden 0 15
3 0 18 SI - Slovenia 0 2 0 0 0 2 SK - Slovakia 0 2
0 0 2 TR - Turkey 0 4 0 0 0 4 UA - Ukraine 0 1
0 0 1 UK - United Kingdom 13 38 6 1 0 58 US - United States of America 0 0
6 0 6 TOTAL 61 373 50 29 1 514 5 http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Orphanet Report Series - Disease Registries in Europe - January 2011 2- Distribution of registries by coverage 3- Distribution of registries by institution INSTITuTIoN NuMbER of REgISTRIES ACADEMIC
490 PATIENT ORGANISATION 8 PRIVATE COMPANY 16 TOTAL
514 NETWoRKS NuMbER of NETWoRKS TOTAL
9 4- Network registries CoVERagE NuMbER of REgISTRIES REGIONAL
61 NATIONAL
373 EUROPEAN
50 GLOBAL
29 NOT DEFINED 1 TOTAL
514 6 http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Orphanet Report Series - Disease Registries in Europe - January 2011 Distribution of registries by country aT - auSTRIa (15 registries) ENglISh labEl of ThE aCTIVITy CoVERagE INSTITuTIoN Austrian acromegaly registry National Academia
Austrian alpha-1 antitrypsin deficiency registry - contributes to the Alpha One International Registry (AIR) National Academia
Austrian brain tumor registry National
Academia Austrian cancer registry - contributes to the RARECARE project National
Academia Austrian chronic myeloid leukemia registry National Academia
Austrian cystic fibrosis patient registry - contributes to the EUROCARE CF registry National Academia
Austrian Haemophilia Registry National
Academia Austrian Huntington disease registry National Academia
Austrian myeloma registry National
Academia Austrian registry for inborn errors of metabolism National Academia
Austrian severe chronic neutropenia patient registry - contributes to the SCN international registry (SCNIR) National Academia
EMSA-SG: central patient registry of the European multiple system atrophy network European Academia
ENRAH: European alternating hemiplegia and rare epilepsies registry in childhood European Academia
MDS: Austrian myelodysplastic syndromes patient registry National
Academia Styrian registry of congenital anomalies - contributes to the EUROCAT network Regional
Academia bE - bElgIuM (20 registries) ENglISh labEl of ThE aCTIVITy CoVERagE INSTITuTIoN ACROBEL: the Belgian registry on acromegaly, epidemiology and quality of care National
Academia Antwerpen registry of congenital anomalies - contributes to the EUROCAT network Regional
Academia Belgian alpha-1 antitrypsin deficiency registry - contributes to the Alpha One International Registry (AIR) National
Academia Belgian cystic fibrosis patient registry (BMR-RBM) - contributes to the EUROCARE CF and the ECFS registries National
Academia Belgian familial adenomatous polyposis registry National Academia
Belgian Neuromuscular Disease Registry National
Academia Belgian patient database for Wilson disease - contributes to the EuroWilson registry National
Academia Belgian patient registry for Duchenne and Becker muscular dystrophy - part of the TREAT-NMD network National
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