Dr Jean Saunders, University of Limerick, and IPPOSI Board Member (Science) presents at the 5th World Congress on Advanced Clinical Trials and Clinical Research on public and patient involvement in clinical trials.

1.
PPPI – The involvement of
patients/people in the Design and
Development of Clinical Trials
within Ireland and Europe
May 14May 14thth
20182018
5th World Congress on
Advanced Clinical Trials and
Clinical Research
Dr. Jean Saunders, Executive Director,Dr. Jean Saunders, Executive Director,
Statistical Consulting Unit/CSTAR @ UL,Statistical Consulting Unit/CSTAR @ UL,
University of Limerick, Limerick, IrelandUniversity of Limerick, Limerick, Ireland

2. AcknowledgmentAcknowledgment
• IPPOSI slides kindly provided byIPPOSI slides kindly provided by
Derick Mitchell, Chief Executive,Derick Mitchell, Chief Executive,
IPPOSI (IPPOSI (Irish Platform for
Patients’ Organisations with
Science & Industry)

3. My BackgroundMy Background
• Consultant Statistician been involved in ClinicalConsultant Statistician been involved in Clinical
Trials and Medical Research for over 20 yearsTrials and Medical Research for over 20 years
• C. Stat & C.Sci (RSS + Consultancy)C. Stat & C.Sci (RSS + Consultancy)
• Been involved in medical research ethicsBeen involved in medical research ethics
committees for over 20 yearscommittees for over 20 years
• Currently a member of 3 research ethicsCurrently a member of 3 research ethics
committees, Vice Chair of one and Chair of anothercommittees, Vice Chair of one and Chair of another
• Have been interested in patient involvement inHave been interested in patient involvement in
clinical trials and medical research for many yearsclinical trials and medical research for many years
as a statistician involved in planning the studiesas a statistician involved in planning the studies
and as a member of ethics committees.and as a member of ethics committees.
• Became a non-executive Board Member of IPPOSIBecame a non-executive Board Member of IPPOSI
about 18 months agoabout 18 months ago

4. BackgroundBackground
• Clinical Trials were originally designed and developed with
input from various experts, statisticians, clinicians, nurses
etc.
• Gradually there was a recognition that maybe the patients
(or participants) in the actual trial could contribute although
at first this was mainly limited to their involvement in the
choice of outcome measures.
• The difficulty of involving patients in clinical trial design was
mainly attributed to patients not having sufficient
understanding of the trials to be able to make more than
simple design changes.
• However Patients’ Organisations have developed within
Ireland and Europe (and worldwide!) and have become more
and more influential at all levels of treatment and health
provision.
• Up until recently the patients/organisations still had very
little influence over clinical trial design leading to the
patients and patients’ organisations criticising the trials
when they were taking place or completed.

5. Background continuedBackground continued
• This has led recently to the idea of the ‘expert’ patient.
• This is the concept that patients often know quite a lot
about their disease so the only thing stopping them from
providing useful input into a clinical trial is their
comparative lack of knowledge of interpreting the
medical literature and/or the design of efficient and valid
clinical trials.
• To address this various programmes of education for
patients have been devised in Ireland and within Europe
training them how to ‘read’ medical papers and
understand clinical trials.
• This talk provides the history behind these initiatives and
the results obtained.
• It is hoped that in the future very few trials will be
planned in Ireland and the EU without some input from
patients or patient groups.

6. IPPOSI? Who?IPPOSI? Who?
IPPOSIIPPOSI
A patient-led organisation
within Ireland
that works with patients,
government, industry,
& science to put patients at
the heart of health
innovation
“Turning Patients into Partners”
“Turning Patients into Partners”

7. IPPOSI Priorities + MembersIPPOSI Priorities + Members
20
Actively advocate
for improved +
equitable patient
access to Health
Innovation
Actively advocate
for improved +
equitable patient
access to Health
Innovation
Promote meaningful
patient involvement
in Health Research
and Policy
Promote meaningful
patient involvement
in Health Research
and Policy

8. Practical “Roadmap” on patient involvement inPractical “Roadmap” on patient involvement in
medicines R&Dmedicines R&D
Research design
and Planning
Design of Protocol
Informed Consent Study reporting
Post-study
communication
Patient Info
Leaflet
Trial steering committee
Investigators Meeting
Level of expertise in the disease area required:
mediumhigh
Data Monitoring CommitteePractical
considerations
Health Technology
Assessment
Protocol
Synopsis
Research
priorities
Setting
research
priorities:
Information to trial
participants
Research conduct and
operations
Regulatory affairs
Dissemination,
communication,
post-approval
Source: Geissler, Ryll, Leto, Uhlenhopp (2017) Therapeutic Innovation & Regulatory Science. (doi: 10.1177/2168479017706405)
Fundraising
for research
Ethics Review

9. IPPOSI &IPPOSI & The European Patients AcademyThe European Patients Academy
• EUPATIEUPATI (European Patients Academy on(European Patients Academy on
Therapeutic Innovation)Therapeutic Innovation) produces Expertproduces Expert
Patients on Medicines Research &Patients on Medicines Research &
DevelopmentDevelopment
• Provides Training & EducationProvides Training & Education
• Disseminates through national platformsDisseminates through national platforms
The project received support from the Innovative Medicines
Initiative Joint Undertaking under grant agreement n°
115334, resources of which are composed of financial
contribution from the European Union's Seventh Framework
Programme (FP7/2007-2013) and EFPIA companies.
www.eupati.eu

10. The Impact of PatientThe Impact of Patient
EducationEducation
Role Befor
e
EUPATI After
Member of patient organisation, not actively
involved
17% 2%
Active role in a patient organisation 62% 71%
Leadership role in a patient organisation 62% 71%
Employee of a patient organisation 25% 23%
Volunteer role in a patient organisation 60% 67%
Presenting at conferences, workshops etc. 63% 83%
Advising a pharmaceutical company 13% 44%
Advising a regulatory agency 21% 42%
Advising a reimbursement agency 4% 8%
EUPATI Fellows are increasingly taking leadership roles and are engaging with pharma,
regulators and HTA bodies. Role changes also imply identity shifts.

11. 2017-20182017-2018
Pilot Irish Patient EducationPilot Irish Patient Education
ProgrammeProgramme
7 months of ‘blended learning in7 months of ‘blended learning in
totaltotal
3 x 6-week Modules3 x 6-week Modules
•Understanding Clinical TrialsUnderstanding Clinical Trials
•Regulatory, Medicines Safety, PVRegulatory, Medicines Safety, PV
•HTA Principles + PracticesHTA Principles + Practices
Irish Education PartnersIrish Education Partners
2 Universities, 1 Regulator, 1 HTA Agency,2 Universities, 1 Regulator, 1 HTA Agency,
1 LMS website:1 LMS website: www.patientsinvolved.iewww.patientsinvolved.ie
21 Students from 9 counties21 Students from 9 counties
(36 applications)(36 applications)
6-member application review panel6-member application review panel
Finished March 12
th , 2018
Finished March 12
th , 2018

12. Patient OrganisationsPatient Organisations
applyingapplying
• Alpha -1 Action GroupAlpha -1 Action Group
• Arthritis IrelandArthritis Ireland
• Irish Children's Arthritis NetworkIrish Children's Arthritis Network
• Irish Cancer SocietyIrish Cancer Society
• National Council for the Blind, IrelandNational Council for the Blind, Ireland
• Fighting Blindness (4)Fighting Blindness (4)
• Vasculitis Ireland Awareness (3)Vasculitis Ireland Awareness (3)
• Migraine Association of IrelandMigraine Association of Ireland
• Spinal Injuries IrelandSpinal Injuries Ireland
• Irish Neonatal Health Alliance
• FibroIreland
• Tick Talk Ireland
• Cancer Trials Ireland PAAG
• ISGO PPI (Gynae Oncology)
• Patients for Patient Safety Ireland
• EASO Patient Council (Obesity)
• Sound Advice (Hearing)
• Order of Malta
• Chronic Pain Ireland

13. LMS (Moodle site) –LMS (Moodle site) –
www.patientsinvolved.iewww.patientsinvolved.ie

14. Promotional Video +Promotional Video +
Grad PhotosGrad Photos
VIDEOVIDEO
https://vimeo.com/2https://vimeo.com/2
61284514/7f547ebb61284514/7f547ebb
7878
PHOTOS
https://www.flickr.com/photos/
136386396@N03/albums/7215
7666910530478

15. Evaluation + ImpactEvaluation + Impact

16. EUPATI Irish NationalEUPATI Irish National
PlatformsPlatforms
• Network of 20 NationalNetwork of 20 National
Platforms coordinated byPlatforms coordinated by
IPPOSIIPPOSI
• Irish National PlatformIrish National Platform
– Tom O’Leary - industryTom O’Leary - industry
– Deirdre Hyland - scienceDeirdre Hyland - science
– IPPOSI CEO - patientIPPOSI CEO - patient
• ENP NetworkENP Network
– AGM, May 8AGM, May 8thth
, 2018, 2018

17. Not the end of the story?Not the end of the story?
• Just the beginningJust the beginning
• Patient involvement at allPatient involvement at all
stages of CTs not universalstages of CTs not universal
• Some trials still have ‘token’Some trials still have ‘token’
patient participationpatient participation
• Patient involvement in HTAPatient involvement in HTA
process at Government levelprocess at Government level
now being promoted by IPPOSInow being promoted by IPPOSI
• Access to medicines initiativeAccess to medicines initiative

18. Questions?Questions?

19. Dr Jean Saunders C.Stat, C.SciDr Jean Saunders C.Stat, C.Sci
Executive Director,Executive Director,
Statistical Consulting Unit/CSTAR@UL,Statistical Consulting Unit/CSTAR@UL,
Dept. of Mathematics and StatisticsDept. of Mathematics and Statistics
UNIVERSITYUNIVERSITY ofof LIMERICKLIMERICK
OLLSCOIL LUIMNIGHOLLSCOIL LUIMNIGH
Tel: 00353 61 213471Tel: 00353 61 213471
Email: jean.saunders@ul.ieEmail: jean.saunders@ul.ie