EUnetHTA Training course for Stakeholders - Patient involvement (Sophie WERKO)

How can patients and providers best
contribute to the HTA process?

Sophie Werko, SBU
werko@sbu.se
HTAi Interest Sub-Group for Patient/Citizen Involvement in HTA
Co-Chair Working Group on Methods and Impact
EUnetHTA Stakeholder Training: 16 January 2014

Patients & Providers contributing to HTA
• Providers

• Patients

SBU:s process for systematic reviews
Specify questions
Determine inclusion and exclusion criteria

Search the literature

Select articles

Review quality of studies

Tabulate the studies

Summarise the results and grade the quality
of evidence

GRADE

SBU strives to minimize the "bias" in the
assessment and synthesis of knowledge
•
•
•
•
•

Systematic process
Broad area of expertise in the project groups
Reproducible and transparent process
Review by independent experts
Meticulous and clear declaration of conflicts of
interests

Project process
SBU Board
Selection and prioritization
of projects

Review pair / project group
Writing

External reviewer
Reviewing

Project group
Formulation of questions and
Inclusion/exclusion criteria

Synthesis of results and
weighing of evidence

SBU Board
SBU Scientific Advisory Board
Feedback

SBU
Printing

Review pair
Literature search

Assessment of quality and
relevance of the studies

SBU / Project group
Dissemination

Project group
Formulation of questions
and
Population
Intervention
Control
Outcome
Example:
Population: Diabetics type 1 and 2
Intervention: food interventions
Control: diabetes food according to present convention
Outcome: mortality, diabetic complications, quality of
life, side effects…

Review pair
Literature search
("diabetes mellitus"[MeSH Terms] OR diabetes[Title/abstract] OR diabetic[Title/abstract] OR
NIDDM[title/abstract] OR IDDM[title/abstract] OR "prediabetic state"[MeSH Terms] OR "hyperglycemia"[MeSH
Terms] OR "prediabetes"[Title/Abstract] OR prediabetic[title/abstract] OR "hyperglycemia"[Title/Abstract] OR
"hyperglycemic"[Title/Abstract] OR "glucose intolerance"[title/abstract] OR "impaired fasting
glucose"[title/abstract] OR "high fasting glucose"[title/abstract] OR "impaired fasting plasma
glucose"[title/abstract] OR "high fasting plasma glucose"[title/abstract] OR "impaired fasting blood
glucose"[title/abstract] OR "high fasting blood glucose"[title/abstract] OR "impaired glucose
tolerance"[title/abstract] OR "igt"[title/abstract] OR "ifg"[title/abstract]) AND ("diet, carbohydrate
restricted"[MeSH Terms] OR "carbohydrate restricted"[Title/Abstract] OR "low carbohydrate"[Title/Abstract] OR
"low carb"[Title/Abstract] OR "south beach"[Title/Abstract] OR "atkins diet"[Title/Abstract] OR "high fat
diet"[Title/Abstract] OR "high protein diet"[Title/Abstract] OR "lchf"[Title/Abstract] OR "lchp"[Title/Abstract] OR
"lckd"[title/abstract] OR "ketogenic diet"[Title/Abstract] OR "dietary carbohydrates"[MeSH Terms] OR (("dietary
fats"[MeSH Terms] OR "dietary proteins"[MeSH Terms]) AND (carbohydrate[title/abstract] OR
carbohydrates[title/abstract] OR carb[title] OR carbs[title] OR carbohydrates[MeSH Terms]))) AND ("randomized
controlled trial"[Publication Type] OR randomized[title/abstract] OR random[title/abstract] OR
randomly[title/abstract] OR multicenter[title/abstract] OR controlled[title] OR "controlled clinical
trial"[publication type] OR "multicenter study"[publication type] OR (review[publication type] AND
"systematic"[title/abstract]) OR "meta analysis"[publication type] OR "cohort studies"[MeSH Terms] OR
"cohort"[Title] OR "prospective"[Title] OR "long term"[Title] OR "observational"[title] OR "secondary
prevention"[Title/Abstract] OR "case control"[Title/Abstract] OR "case control studies"[MeSH Terms] OR
"risk"[title] OR "incidence"[title] OR "incident"[title] OR "risk factors"[MeSH Terms]) NOT ("animals"[MeSH
Terms] NOT "humans"[MeSH Terms])

Review of the literature – flow chart
Literature search
Reviewer 1

Title and abstract
inclusion and exclusion

A

A

B

Reviewer 2

Individual review of abstracts
Include or exclude?

A

B

B

If A and B say include = order fulltext
If A or B says include = order fulltext
If A and B say exclude = exclusion

Ordering of articles in fulltext
Fulltext
inclusion and exclusion

A

General individual review of articles
Include or exclude?

A

Fulltext
reviewing and exclusion

A

B

B
If A and B say include = review
If A or B says include = review
If A and B say exclude = exclusion, to list of excluded studies

Reivew articles with quality assessment tool

A
A

B

B
B

Discussion about how the studies have been validated, consensus.
If A and B say exclude = exclusion, to list of excluded studies

Tabellation of results for furthur discussion in the project group

Assessment of relevance and quality of the studies


Evidence table

Wound infections associated with surgical treatment of jaw fractures,
antibiotic prophylaxis compared to placebo
Studies
Patients

Design

Quality

3
461

RCT
⊕⊕⊕⊕

-1 *

Consistency

0

GR
AD
E

Directness

Imprecise
results

Publication
bias

Effect
size

Dose
response

Confounding

0**

-1***

0

0

0

0

*Lack of blinding
**Different antibiotics
***Few outcomes, together with ** -1

15

Project process
SBU Board
Selection and prioritization
of projects

Writing

External reviewer
Reviewing

Project group
Formulation of questions and


SBU Board
SBU Scientific Advisory Board
Feedback

SBU
Printing

Review pair
Literature search

Assessment of quality and
relevance of the studies

SBU / Project group
Dissemination

Patients?
• Patient
• Service User
• Consumer
•Carer/Care-giver

Patients’ and carers’ experiences
• Living with an illness
– ‘No one knows better what it is like to live with an
illness day in, day out, than those who are doing this –
the patients and their family and friends who care for
them.’

• The technology
– Their needs and preferences, and benefits and
unwanted effects

Understanding HTA. Health Equality Europe. 2008
(Available in several languages). http://www.htai.org/index.php?id=744

HTA and decision making
• HTA can be considered as a bridge between
scientific evidence and decision making
• Patient perspectives’ can
illuminate the bridge by
identifying appropriate research
questions, helping translate
efficacy into effectiveness,
clarifying the determination of
value, helping us write clear
recommendations.

How can patients get involved in
HTA?
• A patient’s view is an individual’s subjective
experience
- Is it representative?
- Is it biased by industry influence?

• How can patients’ and carers’/care-givers’
perspectives be combined with evidence from
controlled clinical trials or complicated
economic models of cost and benefit?

‘Patients’ perspectives in HTA: a route to
robust evidence and fair deliberation’
(Int. J. Tech Assess Health Care, 2010, 334-340)
Karen Facey,
Scotland
Javier Gracia,
Spain
Helle Ploug Hansen,
Denmark
Alessandra Lo Scalzo, Italy
Jean Mossman,
Health Equality Europe

Antoine Boivin,
Canada
Ann Single,
Australia

Effective patient participation in HTA
• Patients’ views and preferences contributing to
HTA:
o In the form of robust evidence
o Through engagement in the process

Patient Evidence

Describing burden of illness
• Nature of illness (chronic, common, rare, life
threatening, etc)
• Impact of illness daily life (home, work,
social activities,…)
• Psychological and social issues (stigma,
exclusion, mental wellbeing,…)
• The most difficult aspects of the illness


Patient Evidence

Impacts of Technology
• How current treatments are taken and
issues arising – side effects, alteration
of dosing,…
• How easily technology fits into daily life
• Outcomes from a treatment that would
be most valued by patients, e.g.
– relief of symptoms to allow return to work,
ability to dress;
– fewer visits to hospital

Patient evidence
•
•
•
•

Concise and balanced
Presents a range of patients perspectives
Variations in clinical practice
Personal experience with existing and new
technologies
• Expectations from new technology
• Views on who should be treated and for how
long
• Evidence and facts, not emotion

1. PARTICIPATION in the HTA process
At every stage:
•
•
•
•
•
•
•
•
•
•
•
•

Study design to produce evidence
HTA topic selection
Scoping
Submission of evidence
Presentation of patient experience to expert committee
Sitting on an HTA decision-making committee
Providing consultation comments on draft HTA report
Patient friendly summaries
Dissemination/communication
Designing & reviewing patient engagement processes
Use HTA to inform charity investments
Contributing to governmental review of HTA

Scoping
• Population
– What patients will benefit?
• Intervention
• Comparators
– What treatments are currently given?
• Outcome
– What matters to patients?

Important Outcome (NICE example)
• Skin condition
• Clinical data focussed on overall body
• Patients’ priority was to reduce coverage
on hands and face, and then overall body

Delloite, Eli Lilly – Enhancing Consumer Involvement in Medicines HTA - 2009

Patient evidence
• Patient evidence can identify limitations in
published research
– In particular the failure to capture the true
concerns of individual patients related to
quality of life

• Committee is looking for a concise and
balanced overview that reflects the range
of patients perspectives
Briefing paper for the update to Methods Guide - 2012

Draft Patient Submission Template
• Consultation until 28 February 2014 on form for
patients to submit information when medicines
being assessed
http://www.htai.org/index.php?id=776

• Please give us your comments!
• Amended form piloted by HTA organisations in
May 2014
• Consultation in Spring 2014 on form for patients
to submit information when health technologies,
other than medicines, are being assessed

Commitment in the draft template
• All members of HTA Committee will have access
to all patient submissions.
• All submissions from patients will be summarised
in HTA advice with indication of how the
information was considered in the development of
conclusions or recommendations.
• Will provide feedback about usefulness of your
submission in our deliberation
• Will share some submissions on website as
examples (with permission)

Evidence on patient’s perspectives
• Literature searching
•
•
•
•
•
•

Surveys (QOL, resource use, experience)
Interviews, focus groups – qualitative research
Review of members’ helpline enquiries
Patient stories
Discussion fora
Social networking

Evaluation and synthesis of studies using
qualitative methods of analysis (SBU, 2012)
When the aim of a study is to achieve a
deeper understanding of a person’s
subjective perception of – for example –
quality of life, a person’s individual
perceptions, experiences, impressions and
actions, then qualitative research methods
may be more relevant. Such methods offer
an understanding of associations from the
individual’s perspective.
http://www.sbu.se/en/Assessment-and-Evidence/SBUs-Handbook/
(C) Karen Facey 2009

NICE
Briefing paper for update to Methods Guide 2012

Views from expert patients can inform the debate
about:
•Variations in clinical practice
•Personal perspectives about benefits and
difficulties with the technology
•Views on rules for starting and stopping treatment

Public partner on HTA Committee
• Equal member of committee
– Participation in discussion
– Presentation of information from patient
organisations
– Voting
– Conflicts of Interest

• Requirements
– Clear description of role and timely reimbursement
of expenses
– Understandable, accessible information
– Support to contribute (training, one-to-ones with
researchers, understanding of Chairman)

Tips for writing your consultation response
(NICE-IPP)
• (Evaluation of efficacy and safety - not costs)
• Doesn’t need to be lengthy or comment on
everything
• Short, focussed response
• Be specific about the procedure
• Balanced - positive and negative
• No local issues

Patient Decision Aids (O’Connor, 2005)
• Interventions designed to help patients discuss
treatment options with their clinicians
and make specific, informed choices
(as an adjunct to ‘counselling’)
• Examples for HTAs in breast cancer treatment
(Spain) and breast cancer screening (Finland)

Patient Decision Aids (PtDAs)
Essential elements
• Facts on condition, options, outcomes
• Risk communication (probability of outcomes
and uncertainty)
• Values clarification (to help patient determine
which benefits, harms and uncertainties
matter most to them)
• Structured guidance in deliberating and
communicating with clinician
• Balanced display of positive and negative
features of options

Improving HTA patient engagement
processes
• Strategic group of public
partners/patient representatives
• Materials and training to explain the
process for patient engagement
that are developed by patients
• Publish a policy for patient
engagement
• Publish an annual report on patient
engagement, highlight successes
and priority areas for development
(C) Karen Facey 2009

DRAFT Values for
patient involvement in HTA

Unique knowledge
Patients provide perspectives and experiences that can
inform HTA value judgements and decision-making.
Mutual respect
Contributions from all participants in HTA are respected and
valued.
Equity
Patient involvement in HTA addresses issues of equity by
ensuring that fair processes are used to understand the
needs of those with one particular medical condition in the
context of a health system that must provide care for all.
Legitimacy
Patient involvement is important for an HTA organisation’s
accountability for decision-making; ensuring openness and
transparency.

DRAFT Standards
Overarching issues
•Those working in HTA have a policy for patient
involvement in HTA that includes a commitment to
patient involvement across key organisational
functions.
•HTA organisations assign a budget and resources
for patient involvement.
•HTA organisations identify a named contact whose
role is to ensure that patients can contribute
effectively to HTA.
•HTA contributors (patients, HTA committees,
researchers, staff etc) receive appropriate education
and training about HTA and patient involvement.

DRAFT Standards
HTA process
•Communications between parties involved in HTA use
plain language (simple, accessible, no jargon).
•The HTA process provides advance notice of patient
involvement deadlines so that input from a range of
st
patients can be obtained.
•Patients are given access to appropriate information to
enable them to fully contribute to HTA.
•Processes for patient involvement, and how patients’
perspectives have affected HTA outputs, are clearly
reported.
•Feedback is given to individual contributors about the
value of their input to an HTA.
•HTA outputs are presented in a form that can be
communicated to patients and understood by them.

Delphi 1 stage consultation
process runs until
26 January 2014

Available in English, Spanish, Italian, Polish, Mandarin, Greek
www.htai.org/index.php?id=545

HTAi consumer and patient glossary
A beginner’s guide to words used in
health technology assessment
Compiled by Ann Single and Biotext Pty Ltd
with contributions from Eleanor Ahern,
Tony Culyer, Helena Dahlgren, Karen Facey,
Karen MacPherson, Margaret Reid,
Karen Ritchie, Tania Stafinski,
Durhane Wong-Rieger
Version: 1
October 2009
www.htai.org/fileadmin/HTAi_Files/ISG/PatientInvolvement/Glossary/
HTAiPatientAndConsumerGlossaryOctober2009_01.pdf

• HTAi promotes development and use of HTA, with over
1,000 members in 50 countries – from all stakeholders
• Interest Group on Patient/Citizen Involvement in HTA
• Working Groups:
 Involvement and Education
 Methods and Impact
 Citizen and Community perspectives
• Travel grants to HTAi Annual Meeting
• Reduced subscription

Interest-Sub Group on
Patient/Citizen Involvement in HTA
• Quarterly ebulletin and email list
• Contributions to annual HTAi conference
• Website of resources
www.htai.org/index.php?id=545

HEE Guide to HTA in 5 languages
Glossary for HTA and clinical research
Free Access themed edition of HTA journal
Organisational reports
Information on HTA Agency approaches to
patient/public involvement
 Links to training courses
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



Patient influenced HTAs

Value
Judgements

Value
Judgements
Recommendations
Recommendations

Making HTA more patient-centered
• Engage with those doing research to ensure clinical
studies include outcomes that are important to patients
• Work with academics to do qualitative research
• Work with HTA Agencies to develop their processes for
engaging patients so that patients can help
demonstrate the value of a new technology
• Engage with politicians to ensure that robust HTA
systems lead to rational and consistent decisions by
healthcare payers
• Encourage collaboration among stakeholders to
ensure rapid access to technologies that demonstrate
value

HTA and You!
• HTA is used to help health systems make investment
decisions
• Patients and carers have important perspectives that
can contribute to HTA
• Patient evidence should include clear facts about
experience and views
• Patients and carers may be included in the HTA
process at all stages
• Find out what happens in your local HTA process.
Ask how you can be involved!
http://www.htai.org/index.php?id=545

SBU's conference on collaboration with healthcare users

SBU – Kunskapscentrum för hälso- och sjukvården I Swedish Council on Health Technology Assessment

www.sbu.se

Multiple Sclerosis Research Trust
• Questionnaire issued by MSRT
• To people with multiple sclerosis (MS) and
their carers with whom they had contact in
previous 6 years who volunteered to provide
personal details
• Characteristics of form of MS (type of
disease, number and frequency of relapses,
disability score, cognition), treatment,
quality-of-life using the EQ-5D instrument

MSRT input to NICE appraisal
• 1555 respondents covering forms of disease
• 152 had relapsing remitting or secondary
progressive MS and were receiving
treatment with one of the products
considered by NICE appraisal at the time of
the questionnaire
• Utilities from these 152 were used in the
revised NICE model to replace UK values
provided by leading Swedish economist

Prevention of relapse in alcohol
dependence (NHS QIS HTA3)
• 4 effective psychosocial therapies and 2 medicines found
to be cost saving to the NHS over 20 years
• Even in controlled conditions, more than half the patients
relapsed at the end of the (short) study period
• 45 patients interviewed in 3 hospitals – felt they were
failures, didn’t know about alternatives, put to the bottom
of the list for future treatment
• HTA recommended that a choice of treatments should be
available, for initial choice and after failure

Communicating to a committee
• Understand your audience and how you are
allowed to participate (responding to questions
and posing questions)
• Identify what other experts will be involved and
seek to provide unique knowledge
• Be prepared with written evidence to refer to
• Know how long you have to speak - top 3
messages
• Facts, not emotion

Communicating benefit that changes a
committee decision (Scotland)
• Combination product
• More expensive, limited clinical evidence
of benefit
• Patient organisations explained that a key
symptom of disease is dry mouth, making
swallowing pills difficult

Delloite, Eli Lilly – Enhancing Consumer Involvement in Medicines HTA - 2009

EUnetHTA Training course for Stakeholders - Patient involvement (Sophie WERKO)

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