2. Policy Debate I
Valuing Innovation: Patient
perspectives from across Europe
Prof Rosanna Tarricone (Chair)
Executive Director EHTI
Professor, Università Bocconi

3. Policy Debate I
Valuing Innovation: Patient
perspectives from across Europe
Klara Zalatnai
Associate Prof Dr Zoltan Kalo
Ros Meek
Prof Bruce Campbell

5. Policy Debate I
Valuing Innovation: Patient
perspectives from across Europe
Klara Zalatnai
President
Hungarian Osteoporosis Patient Association (HOPA)

6. Medical devices
patients’ perspective from Hungary
Klara Zalatnai
Hungarian Osteoporosis Patient Association Hungary (HOPA)
MedTech Forum
Policy Debate I Valuing innovation
Brussels 2010

7. Challenges and opportunities
• Public Health System • Emerging needs
• Medical device industry (e.g. developing a shared
understanding of future
healthcare goals)
• Increasing expectation
• (e.g. overcoming health
inequalities)
• Societal changes
(e.g. ageing society)
• New medical technologies
(e.g. e-health technologies)

8. Public Health Challenges
• Demographic pressures
– (e.g. ageing of population, low birth rate, etc)
• Increasing prevalence of age and life-style related
chronicle disease
• Previously fatal diseases . . . into chronic conditions . . .
• Patient in the public health - patients’ empowerment
– Increasing compliance
– Decision making
– Self making
– Self care
– Self management

9. Public Health Policies expectations
• Well formulated patient needs
• Patient-centered approaches in healthcare systems
• Clarity and visibility with regard to public health policies
and strategies
• Harmonization on best practices and guidelines between
Member States

10. Public Health Policies in Hungary
from patients’ perspective
• Patient societies are isolated
– patients’ needs are formulated not sufficiently
• Lack of the resources (human and financial)
• No coalition of the patient societies
– Poor advocacy
• Poor patient-centered approaches in healthcare
systems
• Poor clarity and visibility with regard to public
health policies and strategies

11. Involvement, education and training
of different actors expectations
• The role of all actors needs to be acknowledged
• Development new skills and professional
curricula training and skills for healthcare
professionals and also patients
• Education and interaction platforms needed
between technology, patients and healthcare
professionals
• Necessary for all stakeholder to develop a
shared understanding of future education
needs

12. Education and training of patients
in Hungary
• „Patients’ university”
program under
preparation
• Exampels for thematic
sites on the facebook
– „Ne ess, ne törj” Don’t fall,
don’t break!,
– „Osteoporosis and
dance”
• should be extended –
almost unlimited sites and
groups

13. Patient Safety & Quality of Care
expectations
• Critical component of quality of healthcare
• Patient can be injured or be exposed to adverse events
• The exchange of information on best practice and on
technologies improving patient safety

14. Patient Safety & Quality of Care
in Hungary
• Adverse / side effect reporting - works
– at the medicines
– at medical devices ??
• The exchange of information on best
practice and on technologies improving
patient safety

15. Value of and access to medical
devices expectations
• Measure appropriately the value of medical devices,
• Enhance better access of patients to medical devices
– looking at the different factors including
• pricing and reimbursement policies.
• waiting lists
• well defined patients routs
• Huge inequity
• Patient involvement in
– measurement the value of medical devices - clinical trials
– decision making process
– Quality of the patients’ contribution

16. Quality health information for
empowered patient expectations
• Identifying the fundamental need of patients for
health information
• Tools that aid assessment and use of relevant health
information
• Obstacles to accessing relevant health information
• Positive action in Europe and across the globe
– Health authorities
– Medical products agencies
– Healthcare assessment agencies
– Healthcare providers
– Healthcare professionals
– Consumer organizations
– Patients’ associations

17. Patient voice - Partnership –
Communication expectations
• Well articulated, formulated, powerful
– quality and quantity
• Partnership - strengths
– health professionals – medical doctors
– medical devices manufacturers, suppliers
– other patient organizations
• Media
– written
– electronic
• Social Web sites - information exchanges

18. Patient Involvement in Health
Technology Assessment Process
expectations
• HTA Evaluation of health care technology aiming at
providing objective information to support health care
decision
– Technology: drug, procedure, medical devices
– Providing decision makers with appropriate
information
– Need to develop patients’ expertise to enable the
patients to put the case forward in a satisfactory and
meaningful way

19. Recommendation
• Investing in Health Literacy and training
• Patients’ having access to information in a concise and
timely way
• Develop patients’ expertise to enable the patients to put
the case forward in a satisfactory and meaningful way
• European wide platforms to support patients
organisations in participating in HTA
• Patient’s organisation themselves should be able to
improve the way they interact with each other and with
decision-makers
Source: EPF

21. Policy Debate I
Valuing Innovation: Patient
perspectives from across Europe
Associate Prof Dr Zoltan Kalo
Director of Health Economics Research Centre
at Eötvös Loránd University
Past President Hungarian Health Economics
Association (META)

22. Role of HTA and transparent
decision-making criteria to
improve the access of patients
to medical devices:
the case of Hungary
Zoltán Kaló
Health Economics Research Centre
Eötvös Loránd University
Budapest, Hungary

23. Affordability and reimbursement of
medical devices
 Lack of EU guidance to member states to improve
transparency of their pricing and reimbursement systems for
medical devices (potential key elements: individual decisions;
verifiable and objective criteria; predictability of timing; availability of
remedies for negative decisions)
 Difficult to assess the value of medical devices  countries pay
attention to credible HTA sources, e.g. NICE appraisals
– Caveat: effectiveness and cost-effectiveness of medical devices is
more country / health care system specific than pharmaceuticals
 Price of medical devices is usually adjusted to local settings
and purchasing power  increased likelihood of reimbursement of
innovative medical devices in lower income countries (as opposed
to pharmaceuticals)
 In many countries budget impact / affordability is the main
criteria for reimbursement  innovative medical devices are
reimbursed with volume control mechanisms

24. Reimbursement algorithm of
medical devices in Hungary
Before 2010
 no objective and verifiable criteria: decisions without
explanation
 no explanation  there is no basis for remedy
 no guidelines for budget impact analysis
 cost-effectiveness is not defined – no official threshold
 no criteria on when, why and how to revise previous
decisions
 timelines are not predictable
 inconsistencies due to individual deals and trade-offs

25. Access of patients to medical devices:
reimbursement in Hungary
Before 2010
 non-transparent criteria for reimbursement and
formulary listing
 no involvement of patients into decisions, lack of civil
control
 Unpredictability of decisions: lucky winners and
unfortunate losers
 Even winners were controlled by local budget caps
 Huge inequity: non-transparent waiting lists, gratuity
 Lack of managed patient routes  poor health
outcomes in complex diseases e.g. oncology, diabetes

26. New regulation in 2010
 Reimbursement and formulary
decisions only once a year (1st July)
 Transparent point system including
HTA elements
 No experience yet

27. The Hungarian Point System
I Health Care Priority 20 point
I.1. Public Health Programme 6 point
I.2. Health Policy priorities 7 point
I.3. Aggregated health gain of population 7 point
II. Severity of disease 15 point
II.1. Life-threatening acute disease 13-15 point
II.2. Life-threatening chronic disease 10-12 point
II.3. Non life-threatening acute disease 8-9 point
II.4. Non life-threatening chronic disease 6-7 point
III. Equity 15 point
III.1. Size of patient population (i.e. rare diseases) 8 point
III.2. Accessability 7 point
IV. Cost-effectiveness, QoL benefit 30 point
IV.1. ICER 15 point
IV.2. QALY gain per patient 15 point
V. Aggregated budget impact 10 point
VI. Local and National professional opinion 10 point
VI.1. Opinion of Professional College 3 point
VI.2. International guidelines 3 point
VI.3. Level of scientific evidence 4 point
Total 100 point

28. Health Policy Priorities
 Services to improve the efficiency of health
care ( technical efficiency)
 Services to reduce inpatient care
 Telemedicine
 Minimal invasive of non-invasive care
 Services to improve rehabilitability of patients
 Services to replace symptomatic care with
targeted curative care
 Preventive services

29. Market access of medical devices:
Assessment of cost-effectiveness
 Methodological problems
– multiple indications (e.g. diagnostics)
– frequent product modifications
– low level of clinical evidence base
 head-to-head RCTs
- no registration criteria
- problems with randomization & blinding
 observational studies
- subject to selection bias
- only after reimbursement decisions

30. Market access of medical devices:
Assessment of cost-effectiveness (2)
 Methodological problems
– ICER is not constant over time
 learning curves
 role of medical personnel (e.g. laparoscopic surgery)
– local production function
 economies of scale (e.g. technologies with high fixed costs)
 need for organisational changes, training etc.
– calculation of costs
 list prices or internal transfer prices (see dialysis providers)
 costs (average or marginal)
 charges
 reimbursement

31. Other problems
 Capacity of assessors (including HTA Office)
 Training
 Budget for single HTA
 Revision of decisions
 Frequency of reimbursement:
– Pharmaceuticals: continuously
– Medical devices: once a year
 Progressivity:
– where to place innovative medical devices?
– How to facilitate equitable access?

32. Conclusion for Hungary
(and for other CEE countries)
 compared to Western Europe
– worse health status
– even more limited health care resources
– strategic pricing of new health care technologies is
adjusted to large Western European countries
 minimal prospective health economic data collection
 few trained health economists
 low public budget for health technology assessment
 no excuse: must improve the appropriateness of
reimbursement decisions

33. General conclusions
 Improvement is necessary
– EU registration criteria: more information on patient benefits,
failure rates, risks
– health technology assessment: more information on value for
money
– reduce differences between market access processes for
pharmaceuticals and medical devices
 EU guidance to member states to improve transparency of
their pricing and reimbursement systems for medical
devices
 Sustainability of innovation is essential
 Better involvement of patients into decisions is necessary
 Ramsey pricing supports innovation and access to medical
devices  benefits from no international price referencing
 Performance based agreements may increase evidence
base for medical devices without delaying patient access

35. Policy Debate I
Valuing Innovation: Patient
perspectives from across Europe
Ros Meek
Director, ARMA

36. Reflections on ARMA engagement
with NICE
Ros Meek
Director
Arthritis and Musculoskeletal Alliance UK

37. Challenges facing the NHS
 Ever higher expectations
 Demand driven by demographics
 Health in age of information and connectivity
 Changing nature of disease
 Advances in treatment
 Changing workplaces
 Cost containment, Quality, Care closer to home

38. What can ARMA offer?
 Holistic view of the needs of people living with
arthritis and musculoskeletal conditions, their
clinicians, AHPs and the research community
 34 member organisations
 local networks which can reflect our membership
and work as a powerful advocate locally

39. What do patients/users want?
(Quality)
 Availability and Accessibility
 Technical Competence
 Communication Skills
 Interpersonal Attributes of Care
 Continuity of care
 Range of On-Site Services

40. PIN Patients in NICE
 3rd sector organisations are a crucial part of NICE’s
stakeholder community raising concerns and
promoting partnership between patients, carers,
communities and the public
 PIN group has a large number of members that
meet independently of NICE
 NICE also had a Partners Council

41. NICE Engagement with 3rd Sector
 Guidance and work programmes “stakeholder”
undertaking responses to consultations and
submission of evidence which would otherwise
not be accessible
 Consultations on support manual production and
individual projects such as the recent one on
innovation
 Opportunity to support dissemination and
implementation
 Equality Forum

42. Patients in NICE (PIN) Remit
 share information, including via an e-mail group
 provide a support network for group members
including a mentoring/buddying scheme
 raise areas of mutual concern with NICE with a view
to finding collaborative solutions where possible; but
without compromising the position of organisations
that wish to challenge decisions made by NICE
 raise areas of mutual concern, connected with NICE,
and with other bodies such as the Department of
Health

43. PIN remit cont:
 raise the profile of the role that patient groups play in
NICE technology appraisals and other NICE guidance
 identify any training needs for organisations engaging
with NICE that are not being met
 replying as a group to NICE wide consultations,
including the end of life consultation and value of
innovation project.
 PIN’s independent “critical friend “ role is crucial to
NICE
 But concern about the value placed on their HTA
appraisal contributions and impact on decision
making

44. Patient and Public involvement
training
 Advice support and training for 3rd sector
organisations to participate and engage with NICE
Work programmes
 Appropriate submissions of evidence
 Training on NICE Methodologies – health
economics, critical appraisal, systematic reviewing
etc

45. Strengthening relationships cont;
 encouraging more organisations to become
members of PIN to reflect the diversity of the third
sector stakeholders with whom NICE engages.
include mental health in the NHS, and groups with
an interest in our public health guidance. Support in
marketing PIN to the wider stakeholder community
 formalising relationships between the PIN group and
members of NICE’s Senior Management Team

46. Strengthening relationships cont;
 exploring other formal mechanisms for mutual
feedback between PIN and NICE’s SMT could be
established (such as regular meetings between
NICE’s Chief Executive/Deputy Chief Executive and
the Chair of PIN)
 encouraging PIN to engage with wider aspects of
NICE’s work, as its involvement has historically been
largely with the technology appraisals programme

47. Into the future?
 provide a sounding board for developments within
NICE’s activities, including new developments, work
streams etc
 provide a forum to allow testing of social and ethical
issues
 Allow feedback on ongoing, developing and new
NICE activities
 provide a dissemination route out to stakeholders on
the work of NICE and provide NICE with feedback
on stakeholders’ engagement experiences
 promote a greater understanding of both the
extent and limitations of the work of NICE

49. Policy Debate I
Valuing Innovation: Patient
perspectives from across Europe
Prof Bruce Campbell
Chair Committees on Procedures and Technologies
NICE (UK)

50. Patient involvement in
producing NICE guidance
Bruce Campbell
Chair, Interventional Procedures and
Medical Technologies Advisory Committees
NICE, UK.
Brussels 13th Oct 2010

51. NICE Guidance
• Technology Appraisals - Clinical and cost
effectiveness
• Interventional Procedures - Safety and efficacy
Not cost
• Clinical Guidelines - Specific conditions:
Management/care pathway
• Public Health
• Medical Technologies devices/diagnostic adoption
• Diagnostics - new

52. Technology assessment and guidelines
• Patient organisations contribute to scope
• Individual patients/carers attend committee
• Guidelines may use workshops, interviews
• Public consultations on scope and
guidance open to all

53. ….. in addition
• All NICE committees have lay members
• Meetings are open to the public as observers
(except certain parts)
….and
• Anyone can notify interventional procedures

54. Interventional Procedures
guidance (whole UK)
• First guidance published 2002
• Now >350 procedures
• Many being reviewed
……. Patient involvement mandated 2006

55. Interventional Procedures
Guidance on safety and efficacy
• Procedures used for treatment or
diagnosis that involve:
– incision, puncture or entry into body cavity
– ionising, electromagnetic, acoustic energy
• First use in the NHS (or private sector)
• New concerns about efficacy or safety
Procedures not specific devices

56. Evidence used by the Interventional
Procedures Advisory Committee (IPAC)
• Published studies (abstracts for safety only)
• Specialist Medical Advisers (questionnaires)
• Committee Members’ knowledge (lay members)
• Public consultation (public, patients)
• 2006 >>> Patient Commentators

57. Patient input to IP guidance
What kind of input? For example:
• Patients with condition on benefits ~ risks?
• Personal experience of procedure?
Some concerns about personal experience:
• Influenced by expectations – what patients
have been told (asked about this)
• No experience of alternative management
• “Lifesaving” procedures – only survivors
(likely to be satisfied)

58. Patient input to IP guidance
- practical difficulties
• Identifying patients who have had procedure
• Procedures done as part of another
• Procedures used in palliative care
• Data protection and ethics legislation
• Numbers of procedures: many each meeting

59. Semi structured questionnaire via
hospitals
• Less time and resources
• Compliance with data protection and ethics
• Avoids direct NICE contact with patients
• Patients may feel less inhibition
BUT
• No chance for interactive exploration
• Some do not reach patients
• No opportunity for NICE to chase

60. Sending and retrieving patient
questionnaires (“packs”)
• NICE identifies hospitals doing procedure
• Hospitals identify patients and send packs
• Patients return packs to NICE (anonymous)
• If < 5 responses committee sees all
• If >5 summary and individual quotes
• Lay committee members always see all

61. Questions 1- 10:
“Epidemiological” / general information
***Questions 11 – 22 include***:
• How well procedure worked
• Effect on different specified areas of life
• Pain before/during/after procedure
• Side effects
• Concerns about safety (before and after)
• Would you have again if in same situation?
• Would you recommend to a friend?

62. Retrospective study (early 2009)
73 questionnaires on 9 procedures
• Transcatheter aortic valve implantation
• Laparoscopic gastrectomy for cancer
• Laparoscopic cystectomy
• Thoracoscopic epicardial RFA for AF
• Deep dermal gel injection for HIV facial lipoatrophy
• Single-incision suburethral tape insertion
• Brachytherapy alone after breast cancer excision
• Allogenic pancreatic islet cell transplantation for
Type 1 diabetes; and after pancreatectomy (x2)

63. Committee Members asked to score
every response for 1-2 procedures
• Usefulness (5 point score)
• Nature (thematic categories)
– No new insights
– Insights help understanding of other evidence
– New and original “evidence”
– Insights on sociocultural or equalities issues
– Suggests lack of understanding
– Not relevant (e.g. about local matters)

64. Examples of findings
Quality and Safety in Healthcare 2010 (in press) Campbell B
• Most “useful”: How well it worked; Effects on
different areas of life*; Would have again?
• Least useful: About pain; About information
and consent (these questions now abandoned)
• Most frequent category: “Supports other
evidence but no new insights”
• Only 0-3% (7%*):“New and original evidence”

65. New NICE Evaluation Pathway for
Medical Technologies
Aim: identify >>> evaluation >>> adoption
Devices and diagnostics
Notified by manufacturers
Selected by MTAC
for measurable improvements in:
• patient outcome or experience
• use of resources – facilities, staff, other tests
• cost, sustainability

66. Medical Technologies Advisory Committee (MTAC)
Routing of “selected” products
• To other NICE Programmes
– Technology Appraisals, Diagnostics,
Interventional Procedures
• MTAC guidance recommendations
– Advice on adoption (+ clinical and cost data)
– Research (may foster research collaborations:
recommendations on data collection)

67. Patient input to Evaluation Pathway
and MTAC
Through relevant patient organisations
• Questionnaire at selection stage
• Committee identifies key areas for patient
information (e.g. insights into living with
the condition)
• More specific questionnaire + statement
• Public consultation – organisations and
interested individuals notified

68. Patient input to Evaluation Pathway
and MTAC
• Limited experience so far
• Most patient organisations have not
offered responses
• A very early stage: all this will evolve.