impact of european rare disease policies on access to therapies
TRANSCRIPT
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19th International Plasma Protein Congress (IPPC)
Ruediger Gatermann
CSL Behring, Public Affairs Europe
Vice Chair, PPTA Health Policy Steering Committee
Dublin
March 05, 2013
Impact of European Rare Disease Policies on Access to Therapies
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Agenda for today
European challenges in health policy for rare diseases
Current healthcare challenges
European influence in health policy
EU legislation initiatives
Key EU legislation affects rare (plasma) diseases
EU Sectoral Study on Blood and Plasma (Blood Directive)
Healthcare infrastructure – patient access to therapies
National Plans for Rare Diseases
Health Technology Assessment – market access for therapies
EUNetHTA core model and assessment pilots
Rare Diseases business models and pricing challenges
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Healthcare challenges increase in times of financial crisis
Overall healthcare costs are rising in response to key demographic and economic factors
Consequently, governments and payers across major markets are implementing numerous cost-containment policies with direct impact on drug margins
Health Technology Assessment is focusing on most expensive therapy areas, leading to new challenges for rare disorders including plasma therapies
Success of the plasma industry is determined, to a great extent, by the external environment in which we operate
Political interest for specific areas (innovation, biotech, rare diseases…) may lead to favourable sector policies despite general cost cutting efforts
Patient empowerment adds new options for alliances if mutually agreed and established in an open and transparent manner
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On European level, EU institutions become more influential in health policy
EU Commission and Parliament increasingly take the lead
in health policy agenda setting
Rare diseases are widely recognized as health priority
Plasma disorders are unique, treatable but still often neglected
Plasma industry’s public affairs efforts are focused on positioning plasma disorders as a key area in the context of rare diseases
Recent EU policy initiatives: European Conferences on Rare Diseases
European Commission Task Force on Rare Diseases, now EUCERD
Rare Diseases in EU Community Research Framework Programme
EC Communication and Council Recommendation on rare diseases
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On national level, for all pharmaceuticals price control policies are used extensively across major markets…
Price cuts, freezes and ceilings
Discounts & rebates
Profit controls
Reference pricing
…and reimbursement controls become even more popular
Pharmaco-economics, Health Technology Assessment (HTA)
Risk sharing
Volume limitations
Formulary positive / negative lists
Key concern: Patient access to therapies
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EU policy agenda: Key legislation affects Rare Diseases (RD)
Cross Border Health Care Directive (new)
European Directive came into force in 2011
Clarifies patients rights on access to cross-border healthcare services
Articles 13, 15 (Rare Diseases, HTA cooperation)
Transparency Directive (under revision)
Initial Directive from 1989 - EU Commission proposal of March 2012
Commission proposal more binding for MSs (Time-limits, sanctions)
EU Parliament amendments: Time-limits extended and removal of sanctions (Tough
negotiation with the EU Council, strong opposition from MSs)
Caveat: Vehicle to push “transparency” on industrial pricing / cost structures
Clinical Trials Directive (under revision)
Change from Directive to Regulation - New EU Commission proposal of July 2012
Emphasis on EU collaboration - Strong alignment EU Commission and EU
Parliament
Major changes: Increase transparency of clinical trials data; Facilitate clinical trials in
medicines used off-label to treat particularly rare diseases
Feb-13
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EU Commission study on blood and plasma sector
Background:
« Blood Directive » is 10 yrs old / Average life of EU legislation
EC needs to prevent potential « crisis »
Study Objectives: (European Commission / EC DG SANCO*)
Improve EC understanding of the supply chain of blood and blood components in
Europe
Identify discrepancies in the functioning of the market of blood and blood
components
Desired outcomes (PPTA):
Clear EC understanding and recognition of current EU situation (Clinical/patient
needs)
Avoid conclusions which could undermine patient access to plasma therapies
Clear distinction of blood (transfusion) and plasma (fractionation) in EU legal text
*DG SANCO = Directorate General “Health and Consumers”
Feb-13
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EC Sector Study: Work Plan/ Milestones 2013/2014
DG SANCO to issue report at the end of the EC sectoral study
DG SANCO to address Member States regarding:
• Implementation of the current Blood Directive
• Issues for potential Blood Directive reform
• Country reports
DG SANCO to consult with stakeholders on impact analysis
DG SANCO to make recommendations for way forward to other Directorate Generals, EU Council & Parliament (?)
2/28/2013
June 2014
New EU
Parliament
End 2014
New EU
Commission
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National Plans for Rare Diseases – Healthcare infrastructure
EUROPLAN project (co-funded by the EC Program for Public Health 2008-2013)
EU Council Recommendation supports adoption of national plans and strategies for
responding to rare diseases (non binding EU legislation)
Policy goals
Elaborate and adopt a plan or strategy by the end of 2013 at the latest
Structure actions in rare diseases within their health and social systems
Integrate initiatives at local, regional and national levels into plans or strategies
Define a number of priority actions with objectives and follow-up mechanisms
Key areas
Definition, codification and inventory
Research
Centers of expertise and European reference networks
Gathering the expertise on rare diseases at European level
Empowerment of patient organizations
Feb-13
Member States:
Implementation?
Financing?
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Health Technology Assessment (HTA): Cross functional strategies are needed
Medical evaluation (“assessment“)
Data collections, dossier
Evidence, (Added-)benefit
Recommendations, guidelines
Economical evaluation(“appraisal“)
„Value“
Influence of financial situation
Pricing and Reimbursement
Political framework
Inclusion of partners (“stakeholders“): Industry, patients…
Differentiation of HTA in rare diseases
Differentiation of HTA for plasma proteins
Feb-13
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Feb-13
Timing of Health Technology Assessment - An assessment continuum rather than an “on/off switch”
Pre-clinical phase
Clinical trials
Marketing authorization
process
Scientific evaluation
of HTA
Economic evaluation
of HTA
Pricing and reimburse
ment
Product launch
HTA reassessment
Marketing authorization
„It is always too early to assess a
technology, until suddenly it is too late.“
Martin Buxton 1987
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Feb-13
Where and how value can be demonstrated
RCTs
Clinical efficacy &
safety
Added
therapeutic value
Health system
value
Societal
value
Comparative
data
Real world data
& modeling
Various
Co
mp
lex
ity o
f d
ata
to
dem
on
str
ate
va
lue
EMA/FDA
Budget N/A
France
Drug budget
UK
Healthcare budget
Sweden
Other budgets (e.g.
social security)
Source: Mel Walker, GSK (HTA World Europe, 2010)
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HTA: A new take on an old story… perceptions matter!
Price Innovation
Value
Evidence
Cost
Cutting
Budget
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Model Rapid REA
Health problem &use of
technology
Technical characteristics
of the technology
Safety
Effectiveness
Cost and economic
considerations
Ethical analysis
Organizational analysis
Social aspects
Legal aspects
HTA Core Model
Health problem & use of
technology
Technical characteristics
of the technology
Safety
Effectiveness
Cost and economic
considerations
Ethical analysis
Organizational analysis
Social aspects
Legal aspects
Model Rapid REA
Health problem & use of
technology
Technical characteristics
of the technology
Safety
Effectiveness
Cost and economic
considerations
Ethical analysis
Organizational analysis
Social aspects
Legal aspects
1.
M
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PILOTS
C
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C
K
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HTA “á la carte”?: Domains, Models and Pilots…
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Pricing of rare disease therapies is broadly discussed by stakeholders and is now a key public policy issue
WSJ; 2013-01-30: “Drug Makers See Profit Potential in Rare Diseases”
BBC; 2013-01-16: “Orphan drugs: Remarkable drugs at remarkable prices”
Reuters; 2013-01-03: “Entering the age of the $1 million medicine”
To be continued…
Source: www.bbc.co.uk
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Feb-13
Numerous European policy initiatives for rare diseases,
yet national market access remains challenging
Increasing recognition for differentiation of
rare disease therapies / OMPs
EunetHTA Joint Action (I) 2010 –
2012; (II) 2012-2015
(Core HTA Model, Rapid REA pilots)
Transparency Directive
(Minimizing times for
P&R processes)
National Plans on Rare Diseases
(Improving healthcare infrastructure, e.g. registries, CoE…)
Cross Border HC Directive
(Patient rights, Art. 15 on HTA Network)
DG SANCO / CAVOD
(Minimizing market
access delays – EMA /
EUNetHTA interaction)
EUCERD
(Future EU Policies and
initiatives on rare
diseases)
DG Enterprise /
MOCA
(Coordinated access to OMPs
– still under development)
New Clinical Trials
Directive / Regulation
(Greater EU collaboration)
New Pharmacovigilance
Directive
(Enhanced risk management)
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The RD policy challenge: Dialogue for Differentiation
Better awareness and (early) diagnosis
Best practices on rare diseases care
Equal patient access to therapies, including orphan drugs
Paradigm shift: Costs = investment in health
Level playing field for market access
Differentiation
Rarity versus severity / Rare Diseases versus common diseases?
Plasma protein therapies?
Rare Disease business models and industry reputation
HTA Domains for assessment and appraisal
Pricing challenges
Dialogue
Stakeholder inclusion and alignment
Patients
Physicians
Experts
Industry
Policy makers
Regulators
Payers
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THANK YOU FOR YOUR ATTENTION !
Contact:
Ruediger Gatermann
Director, Public Affairs Europe
CSL Behring Biotherapies for Life ™
P.O. Box 1230 | 35002 Marburg | Germany
+49 (0) 6421 39 4264 phone | +49 (0) 6421 39 5550 fax
+49 (0) 171 3035465 mobile