Medical devices from patients' perspectives

Policy Debate IValuing Innovation: Patient

perspectives from across Europe

Prof Rosanna Tarricone (Chair)Executive Director EHTI

Professor, Università Bocconi

Klara Zalatnai

Associate Prof Dr Zoltan Kalo

Ros Meek

Prof Bruce Campbell



Klara ZalatnaiPresident

Hungarian Osteoporosis Patient Association (HOPA)



Medical devices patients’ perspective from Hungary

Klara ZalatnaiHungarian Osteoporosis Patient Association Hungary (HOPA)

MedTech Forum

Policy Debate I Valuing innovationBrussels 2010

Challenges and opportunities

• Public Health System

• Medical device industry

• Emerging needs

(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)

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

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

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

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 neededbetween technology, patients and healthcare professionals

• Necessary for all stakeholder to develop ashared understanding of future education needs

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

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

Patient Safety & Quality of Carein 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

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

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

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

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

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

Associate Prof Dr Zoltan KaloDirector of Health Economics Research Centre

at Eötvös Loránd University

Past President Hungarian Health Economics

Association (META)



Role of HTA and transparentdecision-making criteria to

improve the access of patients to medical devices:the case of Hungary

Zoltán KalóHealth Economics Research Centre

Eötvös Loránd UniversityBudapest, Hungary

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 settingsand 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

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

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

New regulation in 2010

Reimbursement and formulary decisions only once a year (1st July)

Transparent point system including HTA elements

No experience yet

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

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

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

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

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?

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

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

Ros MeekDirector, ARMA



Reflections on ARMA engagement

with NICE

Ros Meek

Director

Arthritis and Musculoskeletal Alliance UK

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

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

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

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

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

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

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

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

Strengthening relationships

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

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

Strengthening relationships cont;

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

Prof Bruce CampbellChair Committees on Procedures and Technologies

NICE (UK)



Patient involvement in

producing NICE guidance

Bruce Campbell

Chair, Interventional Procedures and

Medical Technologies Advisory Committees

NICE, UK.

Brussels 13th Oct 2010

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

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

….. 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

Interventional Procedures

guidance (whole UK)

• First guidance published 2002

• Now >350 procedures

• Many being reviewed

……. Patient involvement mandated 2006


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

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

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)

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

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

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

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?

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)

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)

Examples of findingsQuality 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”

New NICE Evaluation Pathway for

Medical TechnologiesAim: 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

Medical Technologies Advisory Committee (MTAC)

Routing of “selected” products

• To other NICE Programmes

– Technology Appraisals, Diagnostics,


• MTAC guidance recommendations

– Advice on adoption (+ clinical and cost data)

– Research (may foster research collaborations:

recommendations on data collection)

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

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.

Health & Medicine

Medical devices from patients' perspectives