IPPOSI CEO Dr Derick Mitchell outlines what 'access' means to patients at the Pharmaceutical Managers of Ireland Masterclass.

1. What
does
ACCESS
actually
mean
to
patients?
PATIENTS – SCIENCE – INDUSTRY
PMI Masterclass: October 19th, 2016

2. Overview
• IPPOSI? - the patient-led perspective
• Patient Involvement in Access to Innovations
• Broader Questions

4. Vision
Early,
equitable
access
to
Health
Innovation for
improved
patient
outcomes
Mission We
are
the
collective
voice
of
Patients,
Science
and
Industry
in
enabling
meaningful
involvement
in,
and
improved
access
to,
Health
Innovation
4
IPPOSI Vision + Mission

5. IPPOSI Members (examples)
100+ Patient Organisations
100+ Scientific/Regulatory/Agencies
(200+ individuals)
16 Industry members

6. Partnership Approach
Other Stakeholders
Patient
Experts
• Encourage open, constructive
dialogue and interaction
• Highlight value that patients can bring
• Develop new opportunities for patients
to influence decision-making

7. Public-Private Partnership
• Grant from Department of Health
(via Health Research Board)
• Industry membership fee

8. Patient-led activities
Health Hacks
Workshops
Conferences
Consultations
Round-Tables
Working Groups
Training Days
Twitter Chats
Webinars
eHealth
Clinical Research
Health Information
Health Economics
Patient Registries
Rare Diseases
Biobanking
Patient Data

9. European Patients Academy on
Therapeutic Innovation (EUPATI)
www.eupati.eu

10. Patients have a key role in all aspects of
health-related research
Public
Research
Ethics
Committees
Competent
authorities
Policy makers
/
Research
Policy
HTA
agencies
&
committees
Research
subject
Info
provider
Advisor
Reviewer
Co-­‐researcher
Driving force
Clinical
Research
Research
design
Informed
consent
Ethical
review
Value
assessment
Health
policy

11. Why is patient involvement important in
decisions around access to innovations?
..’brings a balance by clarifying what matters to patients..’

12. HTA impacts patient access
Figure from Julia Voloschuk, Patient Access Director, Novartis Pharma AG, Heart Failure Patient Academy, May 2016

13. HTA bodies + regulators who engage patients
Body
(Country)
Patient
organisation
partnerships
by
the
HTA
body
Patient
Reps
interviews
the
HTA
conduct
Patient
insights
submitted
by
applicant in
HTA
submission
Patient
organisation
submissions
accepted
Public
meetings
conducted
by
HTA
body
FDA
(USA) X X
EMA
(EU) X
G-­‐BA
(Germany) X
NICE
(UK) X X X X
SMC
(Scotland) X X X
NCPE
+
HIQA
(Ireland)
X X
CADTH
(Canada) X X X
PBAC
(Australia) X X X
Japan X

14. 1. Unmet need for HTA
What patient data is relevant?
• Information that is relevant for HTA bodies and that may not be available in the
published literature or quality of life measure (i.e. that only patients, carers can bring)
• What is it like to live with condition x? Not really
• How the new innovation can improve unmet medical and social needs? Yes
• (e.g. symptom improvement, social functioning)
• Improvements in Health-related Quality-of-Life? Yes
• Impact on general health, wellbeing, daily life
• Impact on Social Life
• Financial Impact + Ability to work
• Psychological impact
• Impact on Family life and relationships
• Current treatment burden incl. side-effects

15. My advice to patient organisations?
• Objectivity is key. Not just advocating for….
• Move from individual testimonials to clear evidence
• e.g. people with improvement ‘x’ are 25% more likely to be employed
• Pay close attention to ongoing trials, standards of care
• Work closely with medical / scientific professionals to obtain unbiased scientific opinion
• If possible obtain feedback from individuals on relevant trials – can be very revealing
• Having the tools is important but having an understanding of the environment is
crucial!

16. 2. Patient involvement in healthcare decision-making?
• Shared decisions with prescribers
• Clinical Care Programmes
• Health Technology Assessments
• Regulatory - EMA Committees
Ø Adherence
ØNew medicines (e.g. biosimilars)
Ø Steering Committees
Ø Adjusting clinical / review criteria
Ø Patient preferences in QALY
Ø Between NCPE recomm.+ decision
Ø Benefit-Risk Assessments,
Ø Scientific Advice

17. 3. Outcomes demonstration (RWE)
A number of Tools and Platforms available
• SurveyMonkey
• Focus Groups
• Patient Registries
• Online patient information platforms

18. Patient Registries
• Evaluating new treatments in real-world settings
(Pharmaco-vigilance)
• Rare Disease Reference Centres of Excellence
Courtesy
of
Abigeal Jackson,
CFRI,
IPPOSI
10-­‐year
anniversary
event,
Oct
2016

19. Rareconnect
Aggregates
documents
and
people
Provides
aggregate
data
in
the
form
of
bar
charts
and
rating
Crohnology

20. Umotif, PatientslikeMe, Carenity
Graphically
display
charts,
timelines
and
various
representations
of
different
measures

21. Patient Involvement today?
• Agreed Values?
• Harmonized processes?
• Quality Standards?
• Metrics to demonstrate impact?

22. Values and Quality Standards for PPI
• HIQA
• Guidelines for Stakeholder
Engagement in HTA
• EUnetHTA
• Stakeholder Forum
• HTAi
• Vision: that patient and citizen
perspectives improve HTA
• EUPATI
• Guidance for HTA agencies
http://www.htai.org/interest-­‐groups/patient-­‐and-­‐citizen-­‐involvement/pcig-­‐resources/hta-­‐resources.html

23. What is IPPOSI doing in this space?
Active Partnerships with HTA Agencies
• HTA scoping (HIQA)
• Live HTA Feed from NCPE on IPPOSI website
• Events + Consultations
• HTA Training for patient organisations
• Future: Irish patient education programme
• Based on EUPATI course (modular)

24. Annual HTA & Decision-Making Training
JULY 2016:
“incorporating the
patient perspective”

25. Broader Questions….
• Regulators + HTA Agencies to consider using measures of
value that recognize outcomes important to patients
• Patient-Reported Outcomes and Measures of…
• Increased transparency on the true cost of
interventions
• Rawles Rule of Rescue….

26. Orphan Drugs
• National Plan for Rare Diseases (2014)
• Oversight Committee for Plan Implementation
• Orphan Drugs Technology Review Committee
• Learning from international examples

27. Accelerated Access Review (UK)
‘I Statements’
• Priorities for research should be set in partnership with people
• Our voices and expertise should be valued at every stage of research and
innovation
• Decisions about access to treatment should be made in partnership with
people
• Patients should be empowered to make decisions about using treatments
Slide adapted from Hilary Newiss, Chair, National Voices, eyeforpharma Patient Summit Europe, Oct 2016

28. Take Home Messages
• Develop clear policies for patient involvement
• Early & Ongoing Involvement
• Need Education/Training for all

29. “Every patient is an expert in their chosen field –
namely themselves and their own life”
- Emma Hill
Editor, The Lancet

30. Thank you!
@IPPOSI www.ipposi.ie
@DerickOMisteal dmitchell@ipposi.ie