Workshop 3 - Registries "Presentation of the concept, definitions and terminology" Ulrike Pypops, CF Belgium

1. CONCEPT, DEFINITION AND
TERMINOLOGY OF REGISTRIES
Ulrike Pypops
 Lawyer at the Belgian CF Association
 Patient Representative of CF Europe in the ECFS
Patient Registry Executive Committee
 Legal adviser of two hospital ethics committees
(protocol review & ethics)
 Member of the Board of RaDiOrg.be

2. 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, …

3. 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

4. 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

5. 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

6. SETTING UP A REGISTRY
1. Designing and planning
2. Data elements
3. Data sources
4. Data collection
5. Ethics, data ownership and privacy
6. Analysis & interpretation of data
7. Reporting

7. 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

8. PATIENT REGISTRY = SCIENTIFIC PROJECT
Use (3)
 Use of data
 Update
 Evaluation

9. ETHICS, PRIVACY AND DATA PROTECTION
Ethics
 Respect for persons
 Beneficience
 Justice
→ can lead to different conclusions
But … has lead to practice (and legal requirement)
of informed consent

10. INFORMED CONSENT: INFORMATION (1)
 Information on:
 Which info is gathered
 Why it’s gathered
 How it’s protected
 How to withdraw
 Who has access

11. INFORMED CONSENT: CONSENT (2)
 Consent to:
 Registry creation by
compilation of patient
information
 Research purpose(s)
 Use of registry data
 Etc.

12. 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 explicit
consent to process sensitive data
→ Directive 95/46/EC (the data protection
Directive)
→ Compliance with national/local protection
legislation

13. PRIVACY
 Protecting your identity?
= Setting “defence” mechanisms
1. Anonymous data
2. 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?

14. 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?

15. PITFALLS
 Incomplete cohort
 Poor representativity
 Bad precision
 Improperly observations
 Pollution and errors
 Inaccurate measure
 Misunderstanding
 Loss of the work
 Unethical work
 Incomplete data

16. 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!

17. ROLE OF PATIENT ORGANISATIONS
Design Creation
 Idea  Participation in
 Purposes
management
 Recruitment
 Communicate
Use Evaluation
 Family help & info
 Improve diagnosis
 Social policy

18. WANT TO KNOW MORE?
 Eurordis Fact Sheet – RD patient registries
 Registries for evaluating patient outcomes : a
user's 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