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European network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.eu

The EUnetHTA submissiontemplate to support production ofcore HTA information and rapidassessments

Zoe Garrett, National Institute Health and Care Excellence

WP 7 Methodology development and evidence generation: Guidelines andpilots production - SG4

Third WP2 Face to Face Training Course for EUnetHTA stakeholders,Brussels, 23rd of April 2015

European network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.eu2

Background

• Before a health technology is reimbursed, countries maycomplete 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 haveevidence requirements e.g. checklists, templates, applications

• There is duplication in the assessment of evidence as agenciesin different countries often look at the same products and usesimilar evidence

• Joint working across countries and harmonisation of methodsand processes, may enable a more efficient assessment process

• The EUnetHTA submission template is one of the tools beingdeveloped to help facilitate joint working

April 23rd 2015


Activity 1: Quiz

• We contacted 33 European countries about theirevidence 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 manyrequested 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


Development of submission template

• Aim: to develop a submission template that can be used tosupport reimbursement processes in European countries, andwhere 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 fortheir HTA and reimbursement decisions

Addresses the questions that individual national agencies ask

• Submission template will be flexible, agencies would be able tochoose 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

Evidencerequirementsreceived forpharmaceuticals

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

Evidencerequirementsreceived formedical devices

6

April 23rd 2015


Methodology

• Framework was developed based on the HTA CORE model,domains, topics and assessment elements

• Framework was piloted and adjusted to ensure information inevidence requirements could be categorised

• EUnetHTA partners involved were asked to data extract theevidence requirements into the framework

• Either completed independently by two people before beingreconciled, or completed by one person and quality assured byanother with differences reconciled

• Data extractions analysed for similarities and differencesbetween countries and used to develop draft template

April 23rd 2015


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 thedevelopment and updating of their own submission templates tosupport their national and regional processes

• Targeted consultation on draft with EUnetHTA partners, othernational reimbursement agencies, WP7 stakeholder advisorygroup, deadline April 30th.

• Currently exploring ways in which to present the template so thatagencies can use it flexibly e.g. to choose modules andquestions relevant to their decision making criteria

April 23rd 2015


Activity 2: Develop your own submissiontemplate

• You are a health insurance fund deciding which productsyou 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 apharmaceutical 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 thequestions they chose and we will compare

40 minutes

April 23rd 2015


Activity 2 reminder: description of the first 4domains in the HTA CORE model

• Health problem and current use of the technology: describesthe disease, target groups, management, epidemiology and theavailability and patterns of current use of the technology.

• Description and technical characteristics of technology:describes the technology and its characteristics, i.e. mode ofaction, when was it developed and for what purpose(s). Alsoincludes regulatory and reimbursement information.

• Clinical effectiveness: the relative benefits of a technologyversus comparator under experimental conditions or routineconditions

• Safety: the harmful effects of a technology. The harms areidentified, quantified in terms of frequency, incidence, severityand seriousness, and compared to those of comparators.

April 23rd 2015


Most frequently requested pieces ofinformation 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 healthtechnology in the pathway of care

• The alternatives currently used in the pathway of care for whichthe 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


Most frequently requested pieces ofinformation domain 1

% of evidence requirements requesting informationEvidence 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

Diseasedescription

Prevalenceand

incidence

Clinicalmanagement

Proposeduse

Alternatives Size targetpopulation

Effect ofintroduction

Experienceof use of

technology


April 23rd 2015


Contents of the submission template

Theme Information requested

Describing thedisease

Definition/description of disease, prevalence andincidence, natural course of the disease, symptomsand burden to patients, aspects of burden targeted bytechnology, consequences to society, causes or riskfactors

Current clinicalmanagement

Current clinical pathway (management anddiagnosis), variation in management, uncertainties

Target population Target population / proposed position andjustification, alternative treatments and justification,size of target population, effect of introducing thetechnology on care pathway

Current use oftechnology andalternatives

Experience of using the technology, amount ofcurrent 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


Summary domain 1

• There is a common set of frequently requested information

• Information requests are similar for pharmaceuticals and medicaldevices, but for medical devices, evidence requirements morefrequently request information on proposed use, effect ofintroduction and existing experience of using the medical device

• Information in this domain covers a small number of themes withfew of the questions being asked only very rarely


April 23rd 2015


Most frequently requested pieces ofinformation 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) andrecommended 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 devicesApril 23rd 2015


Most frequently requested pieces ofinformation domain 2 (pharmaceuticals)

% of evidence requirements requesting informationEvidence requirements: Used for pharmaceuticals (N=29)


0

10

20

30

40

50

60

70

80

90

100

Description Regulation Requirementsand changes

April 23rd 2015


Most frequently requested pieces ofinformation domain 2 (medical devices)

% of evidence requirements requesting informationEvidence requirements: Used for devices (N=20)


0

10

20

30

40

50

60

70

80

90

100

Description Regulation Requirementsand changes

April 23rd 2015


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 ofpackaging, 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 andcatalogues, used materials, accessories andancillary substances, development history,different models or versions

Adapted for use withmedical devices

For pharmaceuticals only

For medical devices only




Contents of the submission template (cont)Theme Information requested

Regulatory status Authorisation status, wording of indication,date of approval, authorisation abroad,product launch, contraindications, conditions oflicence, approval type, ongoing procedures

Reimbursement status Status in European countries, level ofreimbursement, indications covered, restrictions,date of decision

Requirements to usetechnology

Requirements to ensure correct use, suppliesneeded, equipment needed, personnel required,context and level of care, concomitanttreatments, infrastructure and premises required

Investments, disinvestmentsand changes in serviceorganisation

Changes to services, disinvestments,additional skills and training, additional humanresources, further tests and monitoring, additionalequipment and infrastructure

Adapted for use withmedical devices




Contents of the submission template (cont)


Manufacture, distribution andfollow up

Location of manufacture, mechanism ofdistribution, availability of spares andreplacements, 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 andpeople involved, technical platform, software,anaesthesia, differences betweenprocedures that may be used with device







Summary domain 2

• For medical devices there is a common set of informationfrequently requested, there is a different set of informationfrequently requested for pharmaceuticals

• For medical devices there is more variation in informationrequested, and there are also a large number of itemsinfrequently requested

• Information about changes to services and possibledisinvestments is more frequently requested for medical devicesthan for pharmaceuticals


April 23rd 2015


Most frequently requested pieces ofinformation in domains 3 and 4

• List of the relevant studies (copies of reports and publicationsoften also requested)

• Description of the studies to include: design, population,interventions, follow up, primary outcomes, secondary outcomesand number of participants

• Individual study results: method of presentation not generallyspecified

– where specified to include values for treatment groups, relative andabsolute differences, confidence intervals and p values

• Summary and interpretation of the evidence base: outcomes toconsider when interpreting the evidence base generally notspecified in the evidence requirements

– where specified most commonly mentioned: mortality, morbidity, (health-related) quality of life

Clinical effectiveness and safety

April 23rd 2015


Most frequently requested pieces ofinformation domains 3 and 4

% of evidence requirements requesting informationEvidence 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




Identification andselection of clinicaleffectiveness studies

Databases and registries, search dates, searchstrategies, inclusion and exclusion criteria, flowchart, research question, methods for identifyingongoing and unpublished studies, citation hits

List of relevant studies Study reference, study dates, registrationname/number, conflicts of interest, study location,source of identification, references to linkedpublications, status

Study description Design, population, intervention, comparator,follow up, primary and secondary outcome,study objective, randomisation methods, methodsof analysis, methods blinding, methods allocationconcealment






Individual studyresults: clinicaloutcomes

Study results, patient withdrawal, baselinecomparison, sample size determination

Individual studyresults: safetyoutcomes

Study results, exposure, discontinuation andwithdrawal of treatment, susceptible patient groups

Risk of bias studylevel (RCT)

Randomisation sequence, allocation concealment,blinding, complete outcome reporting, other aspects ofbias

Risk of bias studylevel (observationalstudies)

Determination of treatment group, baselinecomparability, minimisation of bias, complete outcomereporting, ITT implementation, other aspects of bias

Risk of bias outcomelevel

Blinding of outcome assessor, ITT implementation,complete outcome reporting, other aspects of bias






Methods of evidencesynthesis

Type of synthesis, outcomes in synthesis,justifications, methods used for synthesis,heterogeneity, consistency, publication bias, sensitivityanalyses

Conclusions: clinicaleffectiveness

Interpretation of results, relative effects on mortality,morbidity, quality of life, satisfaction

Conclusions: patientsafety

Interpretation of results, harms (absolute and vscomparator), dose relationship, onset, changes overtime, susceptible groups

Subgroups Identification, description, justification, plausibility,methods of analysis, results

Strengths andlimitations

Internal validity, relevance of evidence base to scope,factors influencing external validity


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




Manufacturer vigilance data List of incidents, corrective measures, recalls,modifications, methods of optimising orlimiting service to minimise risk

Safety Risk management Methods of optimising or limiting service tominimise risk, changes to marketingauthorisation as a result of safety, otherharms appearing after granting of marketingauthorisation


For pharmaceuticals only




Summary domains 3 and 4

• There is a common set of frequently requested pieces ofinformation

• Agencies don’t tend to include a lot of very specific informationrequests for clinical effectiveness and safety – requests tend tofocus on obtaining evidence

• With the exception of vigilance information, the informationrequests for medical devices and pharmaceuticals are similar


April 23rd 2015


Conclusions

• There is a common set of information frequently requested byreimbursement agencies which can be used to create a commonsubmission template

• However the responses to the same question may not betransferable between countries because of differences in context,agency methods and processes

• Further work is needed to assess the extent of the potentialdifferences in response and reasons for these

• There are other questions less frequently asked which if includedin a single submission template may make the templateoverwhelmingly large for some agencies

• Final submission template will be published October 2015

April 23rd 2015

European network for Health Technology Assessment | JA2 2012-2015 | www.eunethta.eu

Thank you!

This presentation arises from the EUnetHTA

Joint Action 2 which has received fundingfrom the European Union, in the frameworkof the Health Programme

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