How to use the EUnetHTA submission template to support production of core Health Technology Assessments?

European network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.eu
The EUnetHTA submission
template to support production of
core HTA information and rapid
assessments
Zoe Garrett, National Institute Health and Care Excellence
WP 7 Methodology development and evidence generation: Guidelines and
pilots production - SG4
Third WP2 Face to Face Training Course for EUnetHTA stakeholders,
Brussels, 23rd of April 2015

2
Background
• Before a health technology is reimbursed, countries may
complete an assessment of evidence to consider its value
• Companies are asked to provide evidence to support the process
• To help companies provide relevant evidence agencies have
evidence requirements e.g. checklists, templates, applications
• There is duplication in the assessment of evidence as agencies
in different countries often look at the same products and use
similar evidence
• Joint working across countries and harmonisation of methods
and processes, may enable a more efficient assessment process
• The EUnetHTA submission template is one of the tools being
developed to help facilitate joint working
April 23rd 2015

3
Activity 1: Quiz
• We contacted 33 European countries about their
evidence requirements for reimbursement
– how many provided a national standardised evidence
requirement used for pharmaceuticals?
– …..and for medical devices?
• Of the total templates or checklists received, how many
requested that the company provide:
– The size of the population eligible for the product
– The locations of manufacture
– Reimbursement information in other countries
– A list of relevant clinical studies
– An assessment of the quality of the evidence base
April 23rd 2015
10 minutes

4
Development of submission template
• Aim: to develop a submission template that can be used to
support reimbursement processes in European countries, and
where appropriate, joint assessments.
• Remit:
– A submission template for ‘relative effectiveness assessment’,
– Based on national agencies existing evidence requirements
• Ideal situation: any agency can use the submission template for
their HTA and reimbursement decisions
 Addresses the questions that individual national agencies ask
• Submission template will be flexible, agencies would be able to
choose the questions relevant to their decision making
April 23rd 2015

European network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.euEuropean network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.eu
31 of 33 countries confirmed
their requirements
Received 29 evidence
requirements from 29
countries
Of these, 23 used only
for pharmaceuticals and
6 were used for any
health technology
Evidence
requirements
received for
pharmaceuticals
5
April 23rd 2015

European network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.euEuropean network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.eu
25 of 33 countries confirmed
their requirements
Received 20 evidence
requirements from 17
countries
Of these, 14 only used
for medical devices
and 6 were used for any
health technology
Evidence
requirements
received for
medical devices
6
April 23rd 2015

7
Methodology
• Framework was developed based on the HTA CORE model,
domains, topics and assessment elements
• Framework was piloted and adjusted to ensure information in
evidence requirements could be categorised
• EUnetHTA partners involved were asked to data extract the
evidence requirements into the framework
• Either completed independently by two people before being
reconciled, or completed by one person and quality assured by
another with differences reconciled
• Data extractions analysed for similarities and differences
between countries and used to develop draft template
April 23rd 2015

8
Validation and dissemination
• Submission template is currently in draft format
• Draft is being piloted by WP5 in context of joint assessment
• Draft is also being used by 2 agencies as part of the
development and updating of their own submission templates to
support their national and regional processes
• Targeted consultation on draft with EUnetHTA partners, other
national reimbursement agencies, WP7 stakeholder advisory
group, deadline April 30th.
• Currently exploring ways in which to present the template so that
agencies can use it flexibly e.g. to choose modules and
questions relevant to their decision making criteria
April 23rd 2015

9
Activity 2: Develop your own submission
template
• You are a health insurance fund deciding which products
you will make available for reimbursement
– List the information that you would ask a company to provide to
support the reimbursement of their product (areas on next slide)
– Why is this information important
– Is the information relevant to both reimbursement of a
pharmaceutical and a medical device, or just one or another?
• Work in small groups
• You have 20 minutes to write down your response
• After each group will be asked to go through the
questions they chose and we will compare
40 minutes
April 23rd 2015

10
Activity 2 reminder: description of the first 4
domains in the HTA CORE model
• Health problem and current use of the technology: describes
the disease, target groups, management, epidemiology and the
availability and patterns of current use of the technology.
• Description and technical characteristics of technology:
describes the technology and its characteristics, i.e. mode of
action, when was it developed and for what purpose(s). Also
includes regulatory and reimbursement information.
• Clinical effectiveness: the relative benefits of a technology
versus comparator under experimental conditions or routine
conditions
• Safety: the harmful effects of a technology. The harms are
identified, quantified in terms of frequency, incidence, severity
and seriousness, and compared to those of comparators.
April 23rd 2015

11
Most frequently requested pieces of
information in domain 1
• Definition and description of the disease
• The prevalence and/or incidence of the disease
• The pathway of care for the disease and relevant guidelines
• The target population and the proposed position of the health
technology in the pathway of care
• The alternatives currently used in the pathway of care for which
the technology is an alternative or will be added to
• The size of the target population
• How the pathway of care will change with the new technology
• How the technology is currently being used
Health problem and use of the technology
April 23rd 2015

12
information domain 1
% of evidence requirements requesting information
Evidence requirements: Used for devices (N=20); Used for pharmaceuticals (N=29); Total (N=43)
0
10
20
30
40
50
60
70
80
90
100
Disease
description
Prevalence
and
incidence
Clinical
management
Proposed
use
Alternatives Size target
population
Effect of
introduction
Experience
of use of
technology
April 23rd 2015

13
Contents of the submission template
Theme Information requested
Describing the
disease
Definition/description of disease, prevalence and
incidence, natural course of the disease, symptoms
and burden to patients, aspects of burden targeted by
technology, consequences to society, causes or risk
factors
Current clinical
management
Current clinical pathway (management and
diagnosis), variation in management, uncertainties
Target population Target population / proposed position and
justification, alternative treatments and justification,
size of target population, effect of introducing the
technology on care pathway
Current use of
technology and
alternatives
Experience of using the technology, amount of
current use of technology, amount of use of alternatives,
variations in use of alternatives
Bold type = requested in 40% or more of evidence requirements; Normal black type = requested in 20-40%;
Normal gray type = requested in less than 20% April 23rd 2015

14
Summary domain 1
• There is a common set of frequently requested information
• Information requests are similar for pharmaceuticals and medical
devices, but for medical devices, evidence requirements more
frequently request information on proposed use, effect of
introduction and existing experience of using the medical device
• Information in this domain covers a small number of themes with
few of the questions being asked only very rarely
April 23rd 2015

15
information in domain 2
• The names, class, active substance (p) and product codes
• Description of the claimed benefits
• A detailed description of the medical device (md)
• Purpose of the medical device (md)
• Description of how the device is used (md)
• The package contents, administration (p), dosing (p) and
recommended course of treatment (p)
• The authorisation status and indication being assessed
• The regulatory and reimbursement status in other countries
• The requirements to ensure correct use and supplies required
• Changes in the organisation of care following introduction
• Any disinvestment opportunities
Description and technical characteristics of the technology
Information specific to (p) pharmaceuticals specific to (md) medical devices April 23rd 2015

16
information domain 2 (pharmaceuticals)
Evidence requirements: Used for pharmaceuticals (N=29)
0
10
20
30
40
50
60
70
80
90
100
Description Regulation Requirements
and changes
April 23rd 2015

17
information domain 2 (medical devices)
Evidence requirements: Used for devices (N=20)
0
10
20
30
40
50
60
70
80
90
100
Description Regulation Requirements
and changes
April 23rd 2015

18
Contents of the submission template
Overview of features Proprietary and non proprietary name,
form, product and classification codes,
claimed benefits, mechanism of action,
active substance, names in other countries
Administration and dosing Administration mode, description of
packaging, total volume in packaging,
recommended course of treatment,
dosing, posology, defined daily dose
Detailed characteristics Detailed description, how device is used,
package contents, package inserts and
catalogues, used materials, accessories and
ancillary substances, development history,
different models or versions
Adapted for use with
medical devices
For pharmaceuticals only
For medical devices only

19
Contents of the submission template (cont)
Regulatory status Authorisation status, wording of indication,
date of approval, authorisation abroad,
product launch, contraindications, conditions of
licence, approval type, ongoing procedures
Reimbursement status Status in European countries, level of
reimbursement, indications covered, restrictions,
date of decision
Requirements to use
technology
Requirements to ensure correct use, supplies
needed, equipment needed, personnel required,
context and level of care, concomitant
treatments, infrastructure and premises required
Investments, disinvestments
and changes in service
organisation
Changes to services, disinvestments,
additional skills and training, additional human
resources, further tests and monitoring, additional
equipment and infrastructure
Adapted for use with
medical devices

20
Manufacture, distribution and
follow up
Location of manufacture, mechanism of
distribution, availability of spares and
replacements, maintenance requirements,
quality control, medical surveillance,
statistics of repairs
Duration of life, guarantees,
warranties
Lifetime of device and component parts,
details of guarantees and warranties
Procedures used with the device Type of approach, steps within approach and
people involved, technical platform, software,
anaesthesia, differences between
procedures that may be used with device

21
Summary domain 2
• For medical devices there is a common set of information
frequently requested, there is a different set of information
frequently requested for pharmaceuticals
• For medical devices there is more variation in information
requested, and there are also a large number of items
infrequently requested
• Information about changes to services and possible
disinvestments is more frequently requested for medical devices
than for pharmaceuticals
April 23rd 2015

22
information in domains 3 and 4
• List of the relevant studies (copies of reports and publications
often also requested)
• Description of the studies to include: design, population,
interventions, follow up, primary outcomes, secondary outcomes
and number of participants
• Individual study results: method of presentation not generally
specified
– where specified to include values for treatment groups, relative and
absolute differences, confidence intervals and p values
• Summary and interpretation of the evidence base: outcomes to
consider when interpreting the evidence base generally not
specified in the evidence requirements
– where specified most commonly mentioned: mortality, morbidity, (health-
related) quality of life
Clinical effectiveness and safety
April 23rd 2015

23
information domains 3 and 4
Evidence requirements: Used for devices (N=20); Used for pharmaceuticals (N=29); Total (N=43)
0
10
20
30
40
50
60
70
80
90
100
Common elements of study description
April 23rd 2015

24
Identification and
selection of clinical
effectiveness studies
Databases and registries, search dates, search
strategies, inclusion and exclusion criteria, flow
chart, research question, methods for identifying
ongoing and unpublished studies, citation hits
List of relevant studies Study reference, study dates, registration
name/number, conflicts of interest, study location,
source of identification, references to linked
publications, status
Study description Design, population, intervention, comparator,
follow up, primary and secondary outcome,
study objective, randomisation methods, methods
of analysis, methods blinding, methods allocation
concealment

25
Individual study
results: clinical
outcomes
Study results, patient withdrawal, baseline
comparison, sample size determination
Individual study
results: safety
outcomes
Study results, exposure, discontinuation and
withdrawal of treatment, susceptible patient groups
Risk of bias study
level (RCT)
Randomisation sequence, allocation concealment,
blinding, complete outcome reporting, other aspects of
bias
Risk of bias study
level (observational
studies)
Determination of treatment group, baseline
comparability, minimisation of bias, complete outcome
reporting, ITT implementation, other aspects of bias
Risk of bias outcome
level
Blinding of outcome assessor, ITT implementation,
complete outcome reporting, other aspects of bias

26
Methods of evidence
synthesis
Type of synthesis, outcomes in synthesis,
justifications, methods used for synthesis,
heterogeneity, consistency, publication bias, sensitivity
analyses
Conclusions: clinical
effectiveness
Interpretation of results, relative effects on mortality,
morbidity, quality of life, satisfaction
Conclusions: patient
safety
Interpretation of results, harms (absolute and vs
comparator), dose relationship, onset, changes over
time, susceptible groups
Subgroups Identification, description, justification, plausibility,
methods of analysis, results
Strengths and
limitations
Internal validity, relevance of evidence base to scope,
factors influencing external validity
Normal gray type = requested in less than 20%
April 23rd 2015

27
Manufacturer vigilance data List of incidents, corrective measures, recalls,
modifications, methods of optimising or
limiting service to minimise risk
Safety Risk management Methods of optimising or limiting service to
minimise risk, changes to marketing
authorisation as a result of safety, other
harms appearing after granting of marketing
authorisation
For pharmaceuticals only

28
Summary domains 3 and 4
• There is a common set of frequently requested pieces of
information
• Agencies don’t tend to include a lot of very specific information
requests for clinical effectiveness and safety – requests tend to
focus on obtaining evidence
• With the exception of vigilance information, the information
requests for medical devices and pharmaceuticals are similar
April 23rd 2015

29
Conclusions
• There is a common set of information frequently requested by
reimbursement agencies which can be used to create a common
submission template
• However the responses to the same question may not be
transferable between countries because of differences in context,
agency methods and processes
• Further work is needed to assess the extent of the potential
differences in response and reasons for these
• There are other questions less frequently asked which if included
in a single submission template may make the template
overwhelmingly large for some agencies
• Final submission template will be published October 2015
April 23rd 2015

Thank you!
This presentation arises from the EUnetHTA
Joint Action 2 which has received funding
from the European Union, in the framework
of the Health Programme

How to use the EUnetHTA submission template to support production of core Health Technology Assessments?

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