The Future of HTA in Europe

1
The Future of HTA in Europe

• Panos Kanavos, Ulf Persson
• and Michael Drummond
• Prague, 18 February 2009

2
Agenda

• Background
• The challenge
• Impact of HTAs
• Data and Methods
• Report structure/issues analysed
• Results
• Key messages

3
HTA - Overview

• While HTA systems increasingly play a role in
  supporting decision-making, they are not without
  controversy
• Questions abound surrounding the following
  issues
• Role of HTA in decision-making and
  priority-setting
• Methods employed during the assessment process
• Impact on innovation and access
• Role of stakeholders

4
The Challenge

• Health systems wish to maintain high quality,
  innovative, and sustainable health care while
  managing health care budgets, safeguarding
  equity, access and choice
• What is the role of HTA in that context?
• Where are we now and where do we need to go?

5
Impact of Health Technology Assessments

• Can lead to a refusal to reimburse or cover the
  technology concerned
• More often it leads to restrictions to access to
  technologies (eg 2nd or 3rd line use, only for
  some patient groups, etc.)
• Some restrictions are harder to enforce than
  others, and the implementation of recommendations
  is not automatic
• Sometimes the mere intention to conduct an HTA
  can impact on use of the product

6
NICE Appraisal Consultation Document. Prevention
and treatment of osteoporosis in postmenopausal
women

• Bisphosphonates are recommended as treatment
  options for postmenopausal women younger than 65
  years of age with a fragility fracture if they
  have either of the following
• T-score below 3.2SD established by a DEXA scan
• T-score below 2.5SD and either a history of
  maternal hip fracture or long-term use of
  systematic corticosteroids
• Bisphosphonates are not recommended for the
  treatment of osteoporosis in postmenopausal women
  of any age who do not have a fragility fracture

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Media View on Osteoporosis Treatments
8
Study Methods

• To meet these objectives, the study employed both
  primary and secondary methods
• Current literature related to HTA was
  systematically reviewed
• Evidentiary base included both peer-reviewed
  journals and grey literature sources
• Where needed, reports and other information
  sources were translated to English
• To supplement the secondary data collection and
  address any information gaps, HTA experts were
  consulted
• Focus on nine countries
• UK, Germany, France, Italy, Spain, The
  Netherlands, Sweden, Denmark, Finland

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Towards the future of HTA

• A wide body of evidence exists on good and bad
  practice
• Identify a set of principles that can help assess
  existing or establishing new HTA activities
• Themes identified around HTA use in
  decision-making
• I. Structure of HTA activities
• II. Methods
• III. Processes for the conduct of HTA
• IV. HTA use in health care decision-making

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I. Structure of HTA activities
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1. Goal and scope of HTA must be explicit and
relevant to its use

• Encompasses a wide spectrum of activities,
  agencies, bodies
• Use in defining reimbursement, but also in
• Delivering information to providers via practice
  guidelines
• Supporting decisions re investment acquisition
  of health tech
• Important considerations
• Define scope of appraisal
• Define questions to be addressed by HTA
• Initiate a broad multi-disciplinary perspective
  involving all stakeholders
• Operate at arms length of payers (e.g. NICE,
  SMC, TLV, IQWiG)
• Operate at national, regional or local level
  (e.g. HTA by TLV vs mini-HTA)
• May take one or more of several forms
• HTA at national, regional or local level
• Formal regulatory role vs. (informal) advisory
  role
• Used explicitly in decision-making vs. implicitly
• Arms length vs integrated approach
• Use of economic evidence vs. only use of clinical
  evidence
• Encompassing role vs. technology-specific
• Variability can lead to confusion about
  objectives, roles, and division of labour
• A stronger coordinating action could produce
  benefits

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Mini-HTA

• Increasingly pursued at regional/local or micro
  (provider) level
• Rationale remains the attempt to understand the
  implications of introducing new technologies or
  processes
• May lead to more appropriate prioritization, but
  could be subject to bias and influenced by
  self-interest
• Evidence from DK, S, but also ESP

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2. HTA as an open, unbiased, rigorous and
transparent exercise

• Transparency
• Process and methods, accountability, stakeholder
  involvement, appeal, conflict of interest
• Independent, arms length agencies in principle
  fare better
• Explicit value judgements
• An explicit/fixed threshold, vs. a flexible one
• Other criteria beyond TE need to feature in
  decision-making (usefulness, equal value of all
  human beings, severity of illness, need and
  solidarity, marginal utility)

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Explicit Value Judgements IIEquity /need
adjusted reimbursement decisions compared with a
constant cost-effectiveness threshold
Cost/QALY
Adjusted threshold
Netherlands, Sweden
Threshold
UK
1.0
0.9
0.5
0.1
0.2
0.3
Degree of severity/need
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3. All inclusiveness in HTA

• Justified on the basis that potential
  inefficiencies exist in all forms of healthcare
• Consequently all technologies should be potential
  candidates for HTA
• Failure to do so may lead to distortion in the
  allocation and use of resources
• Specific areas that require attention public
  health interventions, e-Health
• Good practice NICE, SBU, DACEHTA

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HTA and Public Health

• There is significant opportunity to reduce
  mortality from avoidable causes as well as reduce
  avoidable hospitalisations
• There are agencies that are working on public
  health/health promotion agenda NICE, DACEHTA,
  SBU
• Evidence base is starting to shape up in several
  areas (e.g. screening for cancer, etc), but still
  remains fragmented
• Expand current remit of agencies to include PH
  interventions
• Address methodological issues associated with
  public health interventions
• This would mean increasing level of resources and
  strengthening implementation strategies
• Countries with no sufficient capacity need to
  resort to adaptation of results

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HTA and eHealth

• Development of health portals in a number of
  countries
• Use of ICT and telephony
• Telemedicine initiatives and imaging services
  enabling faster access to services
• Re-engineering of (primary) health services
• Potential positive implications for efficiency
  and cost
• Need for evaluation
• Very little in terms of national strategies on
  eHealth
• Several initiatives, several pilots, need for HTA

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Re-engineering health services with the use of
ICT - Finland
Total call volume
G U I D A N C E
Health advice services
10
Large part of the services provided by phone only
1/3 self care
Appointment services assessment of the need
of care
65
1/3 Nurse appointment
Only those who have a definite need for care
after proper assessment will be guided into the
system
1/3 Doctors appointment
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Calls outside office hours
THE KEY IS THE PROFESSIONAL ASSESSMENT AND
GUIDANCE FOR CARE
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4. A clear system for setting priorities

• Need to have a priority-setting system
• Where not all technologies are assessed there may
  be scope for inefficiency and distortion in
  decision-making about investment and use of
  scarce resources
• In most cases selection criteria are ill defined
  or not existent sometimes ad hoc
• Priority-setting criteria
• Burden of disease
• Resource impact
• Assessment of incremental value of new
  technologies compared with existing ones
• Clinical and policy importance
• Presence of inappropriate variation in practice
• Timeliness of guidance
• Likelihood of guidance having an impact
• Examples of good practice criteria
  implementation vary

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II. Methods for the conduct of HTA
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Key issues

• 1. Incorporating appropriate methods for
  assessing costs and benefits
• Sweden allow all costs to be included
• UK, The Netherlands allow only/mainly direct
  costs
• France pricing negotiation with CEPS usually
  led from narrow, short-term budget perspective
• 2. Should consider a wide range of evidence and
  outcomes
• (Over-) Reliance on RCTs
• Considering additional sources of evidence
• 3. Encourage a full societal perspective when
  undertaking HTAs
• Some countries do take into account a societal
  perspective (e.g. TLV, DACEHTA, CFZ)
• Often relates to appropriately defining the HTA
  question
• Doubts remain about whether a societal
  perspective is used when deciding about funding
• 4. Addressing uncertainty
• Taking into account the long-term effects of
  individual technologies
• 5. Address generalisability and transferability
• Transferable components systematic reviews of
  efficacy data
• Country-specific components clinical
  interpretation against local practice
  demographics and unmet need health policy
  agenda local comparators costs used in economic
  models CE thresholds ethical considerations
  local resource use treatment patterns

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Spectrum of Value (Payers) Broad Sources and
Perspective of HTAs
Complexity and breadth of data required to
demonstrate value
Source of Value
Societal Value
Therapeutic Value
Health System Value
Relative Budget
Other Budgets (e.g. pensions, social security)
Pharmaceutical Budget
Health System Budget
Sweden
Representative Country
France
UK
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III. Appropriate processes for the conduct of HTAs
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1. Involvement of and input by all key
stakeholder groups

• Role of stakeholders
• Examples of good practice TLV, NICE
• Interaction and contact relationship with HTA
  agency prior to assessment
• Generate a forum for consultation and providing
  scientific evidence
• HTA agencies in general poorly equipped to fulfil
  this role
• Appeal
• No conflict of interest, e.g. separating
  assessment from appraisal

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2. Seek all available data Ex-ante vs ex-post
assessment

• Ex-ante
• Enables appraisal at time of launch
• Data sources are RCTs
• Use of modelling
• Ex-post
• Delayed appraisal
• Real life data through observational studies
• Acceptance of these data?
• Ability to study disease management implications,
  particularly of chronic patients
• Who requests the studies, who owns the data?
• HTA Systems should allow the opportunity of
  inclusion of fresh evidence from real life
  settings

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3.Foresight in HTA

• Horizon scanning
• Early warning systems on new emerging
  technologies NL, S, UK
• Should enable optimal scheduling of technology
  reviews anticipate important innovations
• Work together with innovators to develop CTs
• Avoid bias against new technologies
• Encouraging early contact with stakeholders
• Understanding HTA requirements
• Potentially treating certain technologies
  differently (e.g. orphans)

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HS Council for Health Research (NL)

• Advises government on issues related to health
  research
• Primary scanning activities focus on
• Preventive and curative health care
• Nutrition and food quality
• Environment and health
• Work and health
• Topic selection related to governments agenda
• Supports research on HTA in new technologies
• Output based on systematic lit review and
  consultation with expert groups

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4. Monitoring the implementation of HTA findings

• Process safeguarding implementation of decisions
• Evaluation of the impact of technology in terms
  of
• Budgetary allocations
• Health gain
• Additional remit from payers may be needed for
  arms length agencies

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IV. Use of HTA in Decision-Making
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Use of HTA in Decision-Making

• 1. Timeliness of HTA and independence from
  regulatory (licensing) process
• 2. Appropriate communication to different
  decision-makers
• 3. Affordability in the context of HTA
• Rigid thresholds?
• Other parameters included? (unmet need, ethics,
  clinical judgement, prevalence
• 4. Use of HTA in Value-based pricing static vs.
  dynamic efficiency
• 5. HTA and dis-investment
• 6. Appropriate resourcing

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Pricing issues - Ex ante VBP

• Ex ante assessment of pricing plus periodic ex
  post adjustment
• Trade-off innovation incentive and market
  incentive
• IF ex ante assessment based on product value and
  coupled with ex post adjustment based on product
  value
• Innovative incentives reduced
• Ex ante pricing based on efficacy does not
  necessarily reward innovation
• Could apply risk-sharing more widely

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Pricing issues - Ex post VBP

• If ex post pricing is adopted, the protection of
  innovation is weakened and the risk of innovation
  is returned to the firm
• Firms may choose less risky innovations
  (potentially high value products)
• Firms may not choose high risk innovation
  strategies (as payoff to discovery tends to zero
  relative to the ex post reimbursement inducement
  to minimise risk)
• Variation in innovation decreased
• Variance of risk minimised

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HTA and dis-investment

• Not a leading priority although some HTA agencies
  have subscribed to it on paper
• Genericisation likely to root out older
  technologies
• For newer technologies, relative effectiveness
  may, in principle, decide on investment -
  disinvestment
• Implementation?

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Increasing international collaboration in HTA (1)

• International standards for HTA
• What standards? What can be legitimately
  standardised?
• Costs occurring in future years should be
  discounted to present values international
  standard
• Increasing international collaboration?
• Standardise (some of) the methods of HTA
• Bilateral or trilateral arrangements HAS, NICE,
  IQWiG)
• EUNetHTA and output, e.g. core HTA

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Increasing international collaboration in HTA (2)

• Common methods
• International standards in methods can increase
  the comparability between HTAs and reduce burden
  of submissions
• Feasibility methods, a fruitful form of
  collaboration
• Common assessments
• core HTA approach common template avoid
  duplication
• Feasibility great potential in the systematic
  reviews of clinical data than in the economic
  evaluation component of HTAs (cost variation,
  clinical practice variation
• Common decisions?
• Unlikely to occur in the medium-term in an
  environment of subsidiarity

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Key messages - I

• Use of HTA has increased significantly in the
  past decade in the study countries
• HTA and economic evaluation is now a formal
  criterion of assessment of new medicines in most
  countries
• In some cases, HTA has assumed a pivotal/leading
  role in assessment of value of new medicines
  claiming price premia in relation to other
  criteria (e.g. UK, Netherlands, Finland, Sweden,
  Italy)
• HTA is not always related to economic evaluation
  only, but can be related to clinical assessment
  of value as well (France)
• There are both arms length agencies (e.g. UK,
  Sweden) and integrated approaches to HTA
  (Netherlands, Denmark, Finland)

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Key messages - II

• The independence of HTA agencies and, in
  consequence, independent reviews should be
  favoured over a process, which is guided by
  political expediency, recognizing that such a
  process offers both advantages and disadvantages
• Stakeholders (patient groups, medical sector,
  industry) should have the opportunity to actively
  participate in person in HTAs, submit evidence,
  as well as comment on draft reports and be able
  to access the rationale for the final decision
• Independent HTA reviews lend greater transparency
  and bring broader perspectives to the assessment
  process
• An appeals process should be created and be
  available to resolve disputes over reports that
  are not resolved through consultation between HTA
  agencies and stakeholders
• The HTA process should not linked to the
  affordability question the latter falls into the
  realm of political decision-making

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Key messages - III

• Early warning systems can generate scope for
  collaboration between HTA agencies and
  stakeholders
• When using HTA in decision-making, other criteria
  beyond TE need to feature
• For the purposes of HTA
• a wide perspective would be desirable to the
  extent possible
• Real life data should be generated
• HTA on its own, whether ex-ante or ex-post does
  not necessarily foster innovation as such
  additional tools may be needed
• Significant need to strengthen evidence base on
  public health and disease prevention
  interventions
• Opportunities to evaluate eHealth programmes and
  the benefit they bring to users and providers