1) Cystic fibrosis registries gather detailed patient data that can yield significant findings about effective treatments through analysis. 2) Registries are collaborating with researchers and pharmaceutical companies to enhance disease knowledge, evaluate new medicines, and assess patient responses to treatments over time. 3) A new software platform upgrade allows electronic, encounter-based reporting accessible to consultants and includes a module for a common European cystic fibrosis registry of over 40,000 patients.

1. USING REGISTRIES TO
INNOVATE AND DIRECTLY
IMPACT PATIENT CARE
Abi Jackson, PhD
Research Fellow
Cystic Fibrosis Registry of
Ireland
4th October 2016

2. BACKGROUND
Cystic fibrosis (CF) is an inherited condition that affects many body functions such as
breathing, digestion, and reproduction. It is a lifelong condition for which there is no cure.
The symptoms and severity of CF vary from person to person. Better treatment strategies
have helped to improve the length and quality of life of people with CF.
CF registries gather information on all aspects of a patient’s condition. They act as
information storehouses for infection and treatment statistics. Detailed analysis of this
information can yield significant findings about the most effective treatments for CF. It is
through these analyses that better management of CF may be achieved.

3. USING REGISTRIES TO DRIVE
INNOVATION
People with CF are using
registries to drive health
innovation and impact care
Innovative collaborations
Innovative health
technologies

4. INNOVATIVE COLLABORATIONS (1)
With international academic researchers
 Enhanced disease knowledge potentially influencing care practices e.g.
1. CF-related liver disease study:- knowing when & how to screen for liver disease
2. CF newborn CF screening study:- evaluate benefits of early detection and intervention
3. CF genetic study:- understanding the function of the CF gene product, correlating
clinical symptoms with mutations
4. CF survival estimation:- better prognostic information, international comparisons,
raises new issues e.g. independent living

5. INNOVATIVE COLLABORATIONS (2)
 With the pharmaceutical industry*
 Evaluate new medicines in real-world settings (known as pharmacovigilance)
Clinical trials conducted on relatively small number of patients with a specific profile for a
limited length of time
 Allows identification of adverse events
 Tracks how patients respond to particular medicines over time
 Complies with EU regulation for new medicines
 Can assist regulators
 assess whether approvals can be expanded to other patient groups
 assess whether expectations underpinning decision by healthcare payer to reimburse are
being met
* Patient-identifiable data is never shared with pharma. Data analysis

6. INNOVATIVE TECHNOLOGIES
Software platform upgrade 2015
Secure, electronic, web-based
Care encounter-based reporting
Accessible by CF consultant(s)
Reporting module
Common platform with European
CF Registry (>40,000 patients)

7. PRELIMINARY STUDY: CF
PATIENT/PARENT SURVEY
Perspectives on patient electronic access to registry health records (HR): an Irish-
Slovene online survey
Ireland*
Sep 2015
Slovenia
*
Nov 2015
N 147 (12 %
1)
N 41 (44 %
1)
1 % of IE/SI patients in European CF
Registry
Home internet access
Interested in on-line HR
access
100
 18
yrs
Currently keeping HR on
paper
Currently keeping HR in
own electronic database
0
%
97
99
86
88
58
56
7
13
≥ 18
yrs
Slide c/o Dr Uros Krivec presented at the European CF
Ireland
Sloveni
a
*With thanks to CF Ireland & CF Association of

8. EVALUATING OUTCOMES
IN EUROPEAN CYSTIC FIBROSIS
PATIENTS
WITH ACCESS TO THEIR
REGISTRY RECORDSA randomised control trial of a Registry patient
portal
Lead PI: Dr Abi Jackson, CFRI
Mr Godfrey Fletcher, CFRI
Dr Ed McKone, UCD & SVUH
Dr Gerardine Doyle, UCD
Prof Cecily Kelleher, UCD
IE, DE & SL site investigators
www.cfview.eu
CF REGISTRY PATIENT
PORTAL

9. www.cfview.eu
Your CF data on your
phone
See your data in a
list…
Or on a chartA simple interface
REGISTRY PATIENT
PORTAL

10. www.cfview.eu

11. PATIENT PARTICIPATION Study
started
?
No. of participants
(% of people
needed)
Dublin, IE √ 100 (100%)
Dublin, IE √ 25 (63%)
Dublin, IE √ 11 (92%)
Dublin, IE √ 14 (88%)
Dublin, IE √ 25 (89%)
Galway, IE √ 11 (92%)
Limerick, IE √ 28 (100%)
Cork, IE √ 56 (100%)
TOTAL 270 (96%)
No. of patients needed in Ireland : 280
Aarhus,
Denmark
October -
Ljubljana,
Slovenia
December -

12. WILL CFVIEW HAVE AN IMPACT ON
PATIENT CARE?
Areas under investigation
1. Patterns of usage by participants
2. Clinical outcomes
3. Usage of health services
4. Health literacy
5. Patient-reported quality of life
6. Cost of care
Comparing outcomes in participants with access to CF View and
educational videos
Participants
randomly
allocated
CF View
Videos
CF View &
videos
Usual standard
of care
A randomised control trial

13. CONCLUSIONS
Patients can drive health innovation
 Participate in your registry
 Participate in research
People with CF are having a direct impact on their own care!
 Sharing information that can be used for future-proofing CF services
 Development of new tools which may aid self-management of their condition
 Monitoring of the safety and effectiveness of CF medications
 Contributing to improved clinical decision making

14. ACKNOWLEDGEMENTS
Congratulations IPPOSI
on your 10th birthday
Special thanks to:
CF Registry participants & their parents