Alpha-1
Advocacy – A Concerted Effort:
Public Policy Advancement
Charles Waller,
Director RPP Healthcare
[email protected]
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Outline
1. Overview of EU competence in public health
2. Advocacy as a change driver
3. Alpha-1 European Expert Group Recommendations 2017
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Competences in public health
EU
 ensuring that all EU policies and actions
provide a high level of health protection
 assisting Member States to coordinate
their action and collaborate on health
 joint action with member states on
'threats to health' particularly where
there is a cross border dimension
 standard setting and regulation in
specific areas such as pharmaceuticals,
medical devices, blood products, organs
for transplantation
National level
 Financing, organising,
managing and running
health services
 Reimbursement
decisions
 Regulatory Compliance
Post-2020: the EU might set up of a
legal framework for EU cooperation on
HTA
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EU Public Health toolbox
Soft tools
•
•
•
•
Health Strategy
Legislation
Agencies (EMA, ECDC, EFSA)
Public Health Programmes
• Working groups
• Think tanks
• Public consultations
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Advocacy as a change driver:
RPP Contribution to EU Rare Disease Space
Legislative Credentials:
1. Worked with EC on inserting Rare Disease definition in Cross Border Healthcare
Directive
2. Worked to insert the ‘ultra-rare’ definition in the Clinical Trials Regulation
3. Developed the term ‘treatable rare diseases’
Stakeholder Credentials
1. Represented Patient Groups covering 400+ Rare Disorders
– Rare Bleeding Disorders, PID’s, AATD, Rare Cancers, Childhood Dementia
etc.
Impact on Policy
1. Rare Disease Plan Implementation work (Sweden, Denmark, Belgium,
Germany)
2. National campaigns pushing for implementation for Newborn screening for SCID
3. Patient Training (Ireland, UK, etc.)
4. Reimbursement in Lithuania
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Advocacy as a change driver: example of IPOPI & NBS
Europe
Italy: ADA-SCIDs is effectively carried out in Tuscany and Umbria
Spain: as of 23 March, the Spanish Ministry of Health will evaluate
SCID newborn screening to be a common national service.
Catalonia: SCID incorporated in the neonatal screening
programme (as of January 2017)
Pilot projects at regional levels are currently underway in: Italy,
France, Slovenia, & UK
Worldwide
US: 47/50 states have SCID newborn screening or soon will
implement it
Canada: Province of Ontario has a SCID newborn screening
programme
New Zealand: SCID screening to start as from mid-2017
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Advocacy as a change driver:
e.g. Childhood Dementia in Lithuania
•
2014 - 9 patients
•
List price of therapy between €500,000 and €1 million per patient per year
Today
• 8 are treated
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Alpha-1 Expert Group Recommendations 2011
Recommendation 2011
Status of achievement 2017
Diminishing health inequalities
affecting Alpha-1 and other RD
patients
Partially achieved (through CrossBorder Health Directive)
Increased access to treatment through Partially achieved
implementation of Cross-Border
More opportunities for treatment
Health Directive
abroad but not many patients are
using them
Better standardisation of treatments
and devices supporting breathing
Partially achieved
Revised Medical Devices Regulation
(as from 2019)
Alpha-1 groups (research, patients)
are supported by the EU and Member
States
Partially achieved
€449.4m (2014-2020) though 3rd
Health Programme, with a particular
focus on rare diseases
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The Process
2011
Alpha-1 in the EU
Expert Group
Recommendations
Dissemination of the
Recommendations
Inserting Rare
disease definition
in CBHD
2016
National Rare
disease plans
development
Re-evaluation of
the Alpha-1
community needs
Re-engagement
with Alpha-1
Expert Group
2017
Alpha-1 European
Expert Group
Recommendations
22 March
Alpha-1
Awareness
Day in Europe
November
National level:
application of
Recommendations
Alpha-1 Expert Group Recommendations 2017
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Alpha-1 Expert Group Recommendations 2017
The use for policy
makers
 To improve policy for
patients
 To make healthcare
systems more
sustainable
 To consider Alpha-1
patients in wider policy
topics
The use of healthcare
professionals
The use for individuals
and carers
 To increase awareness  To increase knowledge
when to test for Alpha-1
about Alpha-1 condition
 How to connect with
 To encourage all COPD
centres of excellence
patients to be tested for
 To recommend good
Alpha-1
networking practices
 Know what to say to
your doctor and what
more you can do
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2017 Recommendations: Policy level changes:
 Member states to develop an Alpha-1 diagnosis programme as part of
rare disease plan
 Augmentation therapy has been proven to be cost-effective, it should be
made available for all patients who need it
 An EU project generating an Alpha-1 centre of excellence accreditation
 Inclusion of Alpha-1 in all policies (e.g. environmental policy) & other
respiratory/liver conditions
 Proof of concept at the national level: pilot project in Scandinavian countries
discussed.
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2017 Recommendations: Changes in diagnosis & care
 Increased knowledge and awareness on Alpha-1 among healthcare
professionals
 Increased rates of diagnosis
 Improved treatment and care
 Enhanced networking among healthcare professionals to share expertise
in Alpha-1
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Where are we?
 Alpha-1 European Expert Group Recommendations’ website
 Political and expert momentum established at the Europe level
Options
 Consider how best to incorporate this new tool into the work at national
level
 Look for milestones, e.g. Alpha-1 Awareness day
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2017 Recommendations: What do we want to achieve?
Empowered Alpha-1 community
Increased awareness
on Alpha-1 esp. during
Alpha-1 Awareness
Month, November
Increased funding
opportunities for
Alpha-1 research
Reduced inequalities in
Alpha-1 diagnosis,
treatment and care
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