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

4

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

Thank you!

@IPPOSI www.ipposi.ie@DerickOMisteal dmitchell@ipposi.ie