What does ACCESS actually mean to patients?
PATIENTS – SCIENCE – INDUSTRY
PMI Masterclass: October 19th, 2016
Overview
• IPPOSI? - the patient-led perspective
• Patient Involvement in Access to Innovations
• Broader Questions
VisionEarly, 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
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IPPOSI Vision + Mission
IPPOSI Members (examples)
100+ Patient Organisations
100+ Scientific/Regulatory/Agencies
(200+ individuals)
16 Industry members
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
Public-Private Partnership
• Grant from Department of Health (via Health Research Board)
• Industry membership fee
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
European Patients Academy on Therapeutic Innovation (EUPATI)
www.eupati.eu
Patients have a key role in all aspects ofhealth-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 designInformed consentEthical review
Value assessment Health policy
Why is patient involvement important in decisions around access to innovations?
..’brings a balance by clarifying what matters to patients..’
HTA impacts patient access
Figure from Julia Voloschuk, Patient Access Director, Novartis Pharma AG, Heart Failure Patient Academy, May 2016
HTA bodies + regulators who engage patients
Body(Country)
Patient organisationpartnerships by the HTA body
Patient Reps interviews the HTA conduct
Patient insights submitted by applicant in HTA submission
Patientorganisationsubmissions accepted
Public meetings conducted by HTA body
FDA (USA) X XEMA (EU) XG-‐BA (Germany) XNICE (UK) X X X XSMC (Scotland) X X XNCPE + HIQA (Ireland)
X X
CADTH (Canada) X X X
PBAC (Australia) X X XJapan X
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
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!
2. Patient involvement in healthcare decision-making?
• Shared decisions with prescribers
• Clinical Care Programmes
• Health Technology Assessments
• Regulatory - EMA Committees
Ø AdherenceØNew medicines (e.g. biosimilars)
Ø Steering CommitteesØ Adjusting clinical / review criteria
Ø Patient preferences in QALYØ Between NCPE recomm.+ decision
Ø Benefit-Risk Assessments, Ø Scientific Advice
3. Outcomes demonstration (RWE) A number of Tools and Platforms available
• SurveyMonkey
• Focus Groups
• Patient Registries
•Online patient information platforms
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
Rareconnect
Aggregates documents and people Provides aggregate data in the form of bar charts and rating
Crohnology
Umotif, PatientslikeMe, Carenity
Graphically display charts, timelines and various representations of different measures
Patient Involvement today?•Agreed Values?
• Harmonized processes?
•Quality Standards?
•Metrics to demonstrate impact?
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
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)
Annual HTA & Decision-Making Training
JULY 2016:
“incorporating the patient perspective”
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….
Orphan Drugs
• National Plan for Rare Diseases (2014)
• Oversight Committee for Plan Implementation
• Orphan Drugs Technology Review Committee
• Learning from international examples
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
Take Home Messages
•Develop clear policies for patient involvement
• Early & Ongoing Involvement
•Need Education/Training for all
“Every patient is an expert in their chosen field –namely themselves and their own life”
- Emma HillEditor, The Lancet