Workshop 3 - "Presentation of the concept, definitions and terminology"
CONCEPT, DEFINITION ANDTERMINOLOGY OF REGISTRIESUlrike Pypops Lawyer at the Belgian CF Association Patient Representative of CF Europe in the ECFSPatient Registry Executive Committee Legal adviser of two hospital ethics committees(protocol review & ethics) Member of the Board of RaDiOrg.be
CONCEPT & DEFINITION A patient registry is an On-going, exhausive system of data collection Of patients with the same disease From a geographically defined population Over an extended period of time≠ other databases: patient information management systems, ad hoc (observational) surveys, …
PURPOSE Increasing knowlegde on RD Determine clinical effectiveness of health care Supporting health care service planning Support research by pooling data in order to achieve a sufficient sample size for: Epidomiological research Clinical research Surveillance of drugs used off-label Post-marketing orphan drug surveillance
AND THE RD PATIENT? RD patient registries are only existing source of information on the natural history of the disease e.g. sypmtoms, different patient profiles plus evolutions epidemiology optimal clinical management social management QOL outcomes
AND THE RD PATIENT?Patient registries on RD can improve the quality of care (for ex. guidelines/ standards of care) improve life expectancy better prepare for drug clinical trials if potential treatment arise assess “real” effectiveness of treatment outside controlled context of CT
SETTING UP A REGISTRY1. Designing and planning2. Data elements3. Data sources4. Data collection5. Ethics, data ownership and privacy6. Analysis & interpretation of data7. Reporting
PATIENT REGISTRY = SCIENTIFIC PROJECT Design (1) Creation (2) Objectives Protocol Knowledge Data collection Available stuff Recording data Stakeholders Controlling Timeschedual Quality points Money Development→ Pilot study
PATIENT REGISTRY = SCIENTIFIC PROJECT Use (3) Use of data Update Evaluation
ETHICS, PRIVACY AND DATA PROTECTIONEthics Respect for persons Beneficience Justice→ can lead to different conclusionsBut … has lead to practice (and legal requirement)of informed consent
INFORMED CONSENT: INFORMATION (1) Information on: Which info is gathered Why it’s gathered How it’s protected How to withdraw Who has access
INFORMED CONSENT: CONSENT (2) Consent to: Registry creation by compilation of patient information Research purpose(s) Use of registry data Etc.
PRIVACY Personal data: any information concerning an identified or identifiable person Sensitive data (for ex. concerning health):In principle, such data cannot be processed.Derogation is tolerated under very specific circumstances.These circumstances include the data subject’s explicitconsent to process sensitive data→ Directive 95/46/EC (the data protection Directive)→ Compliance with national/local protection legislation
PRIVACY Protecting your identity?= Setting “defence” mechanisms1. Anonymous data2. Coded data (codes which do not reflect your initials and/ or day of birth) What with ultra RD’s? Or with ultra rare mutations for a in RD’s?
DATA PROTECTION Securing the access at the entrance point Securing the access at the storage center Securing cross referencing of data Securing the access of “demanding” parties Who gets access to which information? Securing/ limiting the 3rd party use of data → is it beneficient to the patient?
PITFALLS Incomplete cohort Poor representativity Bad precision Improperly observations Pollution and errors Inaccurate measure Misunderstanding Loss of the work Unethical work Incomplete data
PATIENT’S CONRIBUTION Patients and their families bring knowlegde and experience about daily life and their illness to the physician/ researcher The physician/ researcher adds epidemiological and clinical data If you collect all this you have a firm base for a patient registry Without patients … no patient registry!
ROLE OF PATIENT ORGANISATIONS Design Creation Idea Participation in Purposes management Recruitment Communicate Use Evaluation Family help & info Improve diagnosis Social policy
WANT TO KNOW MORE? Eurordis Fact Sheet – RD patient registries Registries for evaluating patient outcomes : a users guide, Agency of HealthCare Research and Quality, September 2010, 367 pages http://www.effectivehealthcare.ahrq.gov/ Directive 95/46/EC of the European Parliamentand of the Council of 24 October 1995 on the protection of individuals with regard to the processing of personal data and on the free movement of such data