PATIENTS – SCIENCE – INDUSTRY

UCD Rare Disease Module, March 28th, 2017

Derick Mitchell, PhD

Overview• Context

• Environment IPPOSI exists in / was created to address• IPPOSI – aims/objectives• Rare diseases in Ireland & Europe

• Centres of Expertise – European Reference Networks

• Orphan drugs & technologies

• Your feedback

May you live in changing times….

Clara’s Story• At birth, Clara was diagnosed with

Achondroplasia - a rare bone disease that no one knew about in the local hospital.

• With no experts in the local area, Inés, Clara’s mum, became the expert, directing the medical teams

• Clara had to go through months of countless exams and medical appointments

• She had three surgeries, one of which was life threatening and possibly avoidable

• Meanwhile, Inés had to cope with the worry, take numerous days off work and leave behind her 3 year-old son with his grandmother

Slide courtesy of A. Daly, EURORDIS

Patient‘s organisations have unique insights in “real life“ and “real needs“ of patients:

Clinical Research DesignPriority SettingResearch Policy

Building a new environment for health care and research

5

In a changing system - the two loops theory

Trailblazers

Protectors

Firefighters

Illuminators

Platform

• https://vimeo.com/194851031

IPPOSI? Who?IPPOSI

A patient-led organisation

that works with patients, government, industry,

& science to put patients at the heart of health

innovation

“Turning Patients into

Partners”

IPPOSI Members (examples)

100+ Patient Organisations

100+ Scientific/Regulatory/Agencies

(200+ individuals)

20 Industry members

Partnership ApproachSc

ience

Industry

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

Patient-led activities

Health HacksWorkshopsConferencesConsultationsRound-tablesWorking GroupsTraining Days

Rare DiseaseseHealthClinical ResearchHealth InformationHealth EconomicsPatient RegistriesBiobanking

Rare Diseases in Europe

• Rare diseases in EU = 5 in 10,000

• 6-7,000 rare diseases found (Orphanet)

• Up to 6% - 8% of EU population affected at one point in life = 30 million people in the EU are affected or will be affected by a Rare Disease

• Republic of Ireland = 157,000 have a Rare Disease • 200,000 in the population yet to be diagnosed

Rare Diseases in European policy

Early 2000s• Rare Diseases not a recognised as a public health priority and there

were no public health policies for RD

2017• EU regulations on medicines: orphan, paediatric, advanced therapies,

(Regulation EC NO 141/2000)• EU Health Strategy (2008-2013): Rare Diseases = a strategic objective• EU Research FrameWork Programme (2007- 2013): RD = a priority• National Plans on Rare Diseases

EU Council Recommendation on Action in the Field of Rare Diseases (June 2009)

• This recommendation is important because it calls for concerted action at EU and national level in order to:

• Ensure that rare diseases are adequately coded and classified

• Enhance research in the field of rare diseases

• Identify Centres of Expertise by the end of 2013 and foster their participation into European Reference Networks

• Support the pooling of expertise at European level

• Share assessments on the clinical added value of orphan drugs

• Foster patient empowerment by involving patients and their representatives at all stages of the decision making process

• Ensure the sustainability of infrastructures developed for rare diseases

EU Committee of Experts on Rare Diseases ‘EUCERD’

• Multi-stakeholder• Provided an opportunity to enhance the partnership

between all rare disease stakeholders - patient representatives, academic experts, industry, national and European decision makers

• Comprehensive• Provided the opportunity to discuss, promote and guide EU

policy in all main fields of action relevant for rare diseases

Rare Diseases in Ireland

• National Plan – Ireland 2014-2018 (published May 2015)

• Patient Involvement – key to success • Member of the steering committee to develop the plan

• IPPOSI Head of subgroup on Orphan Drugs• Member of the plan Implementation Oversight Committee

• June 2015• Launch of the National Rare Disease Office – Mater Hospital

www.rarediseases.ie

Centres of ExpertiseEUROPEAN REFERENCE NETWORKS

ADDRESSING THE NEED

Integrated action and collaboration

The traditional short duration of contracts hampers the sustainability of research

• Better coordination and better optimisation of research activities, including standards and tools, and research funding

• Reinforced public funding both at the EU and national levels• In this framework, the potential of ERNs to embed and boost research activities should be

exploited

The lack of coordination in initiatives, tools and standards causes a waste of limited resources

To better coordinate both policy and funding within a strategic framework

To ensure the long-term continuity of research and limit the loss of precious funds

Slide courtesy of A. Daly, EURORDIS

HOW PATIENTS CAN DELIVER

Full involvement of expert & empowered patients as actors of research

• co-funding, leveraging & private-public partnerships

• identification of unmet research needs

• gap analysis

• definition of the research strategy

• clinical trial design

• identification of participants for clinical trials

• source of data

Slide courtesy of A. Daly, EURORDIS

WHERE THERE ARE CHALLENGES THERE ARE OPPORTUNITIES

• Science has taken enormous steps over the last 10-15 years scientific and technological advances, mostly but not only limited to genetics

Opportunity to address societal and ethical questions that these advances raise, not least of all how to address the deepening of inequalities across countries & regions in access to results of scientific research, i.e. improved care & therapies

• Cooperation in the field of research can bring about significant results as shown by voluntary initiatives such as E-Rare (funding), and IRDiRC

We need nhanced and structured cooperation in research

• Full integration of ERNs into national healthcare systems. Creation of the ERNs that will pool of experts, patients, researchers:

Will enable the creation of Clinical Research Networks for Rare Diseases strategically aligned with the ERNs

• Increasingly complex system in the field of rare disease research

Clustering training and capacity building activities aimed to patients but also to young scientists, clinicians etc.

Slide courtesy of A. Daly, EURORDIS

ERNs & Research Infrastructure

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Always between a patient and a doctor in a hospital Data sharing is the key to unlock the potential of ERNsOptimising the use of existing knowledgeGenerating new knowledgeAiming for clinical excellence and improved patients outcomes

Research Infrastructure

Clinical Research Networks

European Reference Networks

Slide courtesy of A. Daly, EURORDIS

Orphan Drugs

What we know….

• Irish health system is unique + requires an Irish solution

• 2014: 9 ODs assessed, majority eventually approved

• High budget impact = decisions take longer

• No funding process in place

What we don’t know…..

• What the final decisions are based on?

• How many ODs are currently available in Ireland?

• What reimbursement of ODs actually costs?

• How ODs are being prescribed/monitored?

IPPOSI views on Orphan Drugs (ODs)

• Separate assessment system with different criteria for assessing OD in Ireland

• Criteria should consider • where patient perspective can be incorporated• the cost to the state of not treating someone

• Transparency and Communication

Health Technology Assessment

Active Partnership with HIQA + NCPE• HIQA have patient involvement on the advisory

board of individual HTAs

• Annual HTA Training for patients through IPPOSI

• Acknowledgement that data provided by patient organisations is invaluable to NCPE assessments

• Irish patient groups exploring measuring patient outcomes with the NCPE

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

Roles change as a result of EUPATI course- creating patient leaders Role Before 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.

Irish Patient Education Programme

Patient Narrative Project

Person-Centered, Coordinated Care

• A set of generic descriptors for what person-centred, coordinated care looks and feels like from the Irish service user perspective

• A definition for ‘person-centred coordinated care’ that is shared, understood and used by service users and staff at all levels of the health service in Ireland