European Health Technology Assessment (HTA) and Registry ... · EUnetHTA project EUnetHTA...

European Health Technology Assessment (HTA) and Registry Requirements

Reinhard Busse, Prof. Dr. med. MPH FFPHFG Management im Gesundheitswesen, Technische Universität Berlin

(WHO Collaborating Centre for Health Systems Research and Management)&

European Observatory on Health Systems and Policies

Questions to be asked regarding a (new) technology• Is it safe to use (in the short term)?• Does it function in healthy?• Does it function in ill persons?• Does it work compared to doing nothing?• Does it work better compared with an

alternative (study conditions)?• Does it work better compared with an

alternative under real life conditions?• Is it cost-effective (vs. alternative)?• Is it safe to use (in the long run)?

Phase I

Phase II

uncontrolledone-armed

studies

controlledstudies

vigilance monitoring/ product registries

Phase III

pharma-ceuticallicensing

HTA(in theory)

Methodologies and requirements

HTA(in practice)

medicaldevice

certification“clinical investigation”

Measure of effect under

“real life” conditions

and vs. alternatives

EffectivenessEfficacy

Measure of effect under

ideal conditions

Cost-effectiveness

Relationships between

costs and benefits

Safety

Measure of adverse effects

Licencing/ certification/market access

Health TechnologyAssessment (HTA):

covered in benefit basket?reimbursement price?

In short … and highlighting terminological confusion

Positioning of “performance”? “function”

• incontinence pads• wheel chair• pregnancy test

• knee (endo-)prosthesis• Implantable Cardio-Defibrillator• coronary stents

• endoscope (for e.g. gastroscopy)• operating room equipment• imaging devices: X-ray, CT, MRI

Example technologies

products which are prescribed, paid and given to an individual patient

Category I“medical aids”

Category III“assistance forprofessionals”

technical equipment supporting professionals in diagnostics and/or treatment with two-stage financing:

Category II“artificial body parts”

medical devices which stay at or in the patient (e.g. knee endoprostheses, stents): only one component of an often DRG-financed “service package” to implant or adapt the “(hardware) product” to the individual patient

• IIIa: investment• IIIb: refinancing via use(diagnostics/treatment)

Classification of Medical Devices is usually based on risk only (the first question that interests us) … but one on coverage, reimburse-ment and usage in health care system would look very different

• incontinence pads• wheel chair• pregnancy test

• knee (endo-)prosthesis• Implantable Cardio-Defibrillator• coronary stents

• endoscope (for e.g. gastroscopy)• operating room equipment• imaging devices: X-ray, CT, MRI

Example technologies

products which are prescribed, paid and given to an individual patient

Category I“medical aids”

Category III“assistance forprofessionals”

technical equipment supporting professionals in diagnostics and/or treatment with two-stage financing:

Category II“artificial body parts”

• IIIa: investment• IIIb: refinancing via use(diagnostics/treatment)

Device constitutestechnology for

coverage decision

Device can beused for different

procedures,e.g. brain CT scanfor headache …

… and would be useful also for HTA separating categories by purpose and HTA focus

medical devices which stay at or in the patient (e.g. knee endoprostheses, stents): only one component of an often DRG-financed “service package” to implant or adapt the “(hardware) product” to the individual patient

Device is(important)component

of a procedure(hip implant

hip implantation)

The process in France (after certification)

The Medical Devices‘ and the HTA worldare often still strangers I – some “arguments”

• Short product cycles (step-wise innovations) evaluation takes too long and impedes access (but do we want access to devices of unproven benefit?)

• Small patient groups randomisation/ control group not possible (untrue, think of orphan drugs!)

• Placebo often not possible good use of data under routine conditions necessary, through(i) Coverage for Evidence Development (new technologies) and (ii) registers (disease-, not product-oriented for control group)

• No evaluations done to be used in HTA !? !? !?

• Possible wider economic effects (due to necessary organizational changes etc.) need to be taken into account

• Some medical devices can be diagnostic (true separate methods needed)

• Greater effect of operator learning curve (true assessment time important)

device

drug

The Medical Devices‘ and the HTA worldare often still strangers II – some more “arguments”

Source: Henschke C et al., 2015

Developing a Medical Devices taxonomy relevant for HTA

Testing the taxonomy by looking at >1200 HTA reports

63%, n=879

4%, n=51

33%, n=466

Dg. 29% Th. 71%

B: 23% A: 6% C: 71%

Source: Fuchs S et al., 2019

0

20

40

60

80

100

120

140

2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015

red cells yellow cells green cells total

Reports (n=1,237) by 33 European HTA institutions, total and per colour tier used in the taxonomic model (green n= 879, yellow n= 467, red n=50), 2004-2015

Trend over time


LoE 1a: 83 (9%)

LoE 1b: 264

(29%)

LoE 2a: 60 (7%)

LoE 2b: 41 (5%)

LoE 3: 25 (3%)

LoE 4:425 (47%)

Base: 898 studies in 93 HTA reports

Which evidence base can HTA use to assess high-risk Medical Devices? (1) a question of study design

Source: Olberg B et al., 2017 almost in every other case, the study design is insufficient

14 (17%)29 (11%)

52 (63%)

162 (61%)

17 (20%)

73 (28%)

0

50

100

150

200

250

300

1a 1b

Num

ber o

f stu

dies

Level of Evidence

Good quality Moderate/poor quality No information about quality

38%

only 43 of 898 studies (<5%) with high Level of Evidence and good qualitySource: Olberg B et al., 2017

Which evidence base can HTA use to assess high-risk Medical Devices? (2) a question of study quality


Transparency

Coordination

Challenges for HTA of Medical Devices – the view of European HTA institutions (n=16; e.g. NICE, IQWiG, KCE) I

Evidence base

Weak licensing requirements(no effectiveness data)


Challenges for HTA of Medical Devices – the view of European HTA institutions (n=16; e.g. NICE, IQWiG, KCE) II

Broader perspective (e.g. treatment and diagnostic companion)

PharmaceuticalsPharmaceuticals

1%

88%

2%

62%

Cons

titut

es im

prov

emen

tDoes what it should do

Consequently, often no evidence for therapeutic/ diagnostic benefit …

… here results for France in

2018… and in

comparison to pharmaceuticals

What can/ should be done?1. Strengthening legal and methodological requirements (helping national HTA)2. Foster European cooperation, from voluntary to mandatory

e.g. Canadian EXCITE program

Fuchs S et al., 2016


Recommendations for improving (national) HTA of Medical Devices –the view of European HTA institutions (n=16; e.g. NICE, IQWiG, KCE)

A proposal for study/ data requirements for licensing and coverage/ reimbursement

Expand HTA’s PICO scheme (Population –Intervention –Comparator –Outcomes) to include modifying factors if looking at therapeutic Medical Devices

Source: Schnell-Inderst P et al., 2018

1994 – 2002 EUR-ASSESS HTA-Europe ECHTA / ECAHI

Harmonization of methods for HTA Information sharing / lessons

learned Emerging technologies and priority

setting Identifying topics for joint work Linking HTA to policy and practice

change

More European cooperation:A short walk through >25 years of European HTA

RCTs other types of evidence

global local


2006 – 2009 EUnetHTA project EUnetHTA Collaboration

2004: Need for a sustainable network for HTA in Europe

Build on learnings from previous European projects

Reduce duplication of effort Tools to facilitate collaboration

EUnetHTA HTA Core Model®Domains1. Health problem and current use of technology2. Description and technical characteristics3. Safety4. Clinical effectiveness5. Costs and economic evaluation6. Ethical analysis7. Organisational aspects8. Patient and social aspects9. Legal aspects



2010 – 2012 EUnetHTA

Joint Action (JA) 1

JA with “official” members Develop methodological

guidance and practical online tools

“Putting into practice” -testing of tools in joint work




Joint Action (JA) 1


JA2

“Strengthening practical application”

Increased focus on production of joint work / joint activities


2006 – 2009 EUnetHTA project EUnetHTA

Collaboration


Joint Action (JA) 1


JA2


JA3

“Turning pilots into standard practice”

Focus on production and uptake of joint work – but limited*

Early Dialogues and post-launch evidence generation

Sustainability * 8 pharmaceuticals; 23 other technologies in 2016-2019 (2, resp. 6/ year)

Is a continuation of these 25 years enough? Definitely not … more evidence is required, and to make that work, reliable but mandatory requirements must exist

In early 2018, the European Commission proposed a HTA regulation that would …• create “Joint Clinical Assessments” for

pharmaceuticals (licensed by EMA) and high-risk medical devices, which have to be taken into account in national decision-making

• offer Joint Scientific Consultations to technology developers

• prepare annual reports on emerging health technologies

What would that mean for Medical Devices?

• Is it safe to use (in the short term)?• Does it function in healthy?• Does it function in ill persons?• Does it work compared to doing nothing?• Does it work better compared with an

alternative (study conditions)?• Does it work better compared with an

alternative under real life conditions?• Is it cost-effective (vs. alternative)?• Is it safe to use (in the long run)?

Phase I

Phase II

uncontrolledone-armed

studies

controlledstudies

vigilance monitoring/ product registers

Phase III

pharma-ceuticallicensing

HTA(in theory)

HTA(in practice)

medicaldevice

certification“clinical investigation”

Filling the evidence gap

We need to develop a common understanding of study designs and data collection tools for generating data with high external validity

uncontrolled one-armed studies controlled studies

Different types of “registries” need to be distinguished

Population sample(independent of already existent

disease)

COHO

RT S

TUDY Persons with

a certain disease(often “all”)

DISEASEREGISTRY

Enables formation of arms for(randomised) controlled studies

Example: rare disease registries in Europe

Persons having receiveda particular device

DEVICEREGISTRY

Not useful for controlled studies(long-term safety only)

In conclusion,1. Medical Devices have a too limited evidence base (both in terms

of study design and quality) for making HTA-based decisions on their patient benefit.

2. An expansion of this evidence base towards controlled studies producing real life evidence requires a mind-change for policy-makers, MD manufacturers and HTA agencies.

3. HTA should become mandatory and routine for MDs with common requirements across the EU (as in the HTA proposal).

4. HTA methodology has to acknowledge MD specifics – with a broadening of methodologies and data sources (e.g. controlled studies generated within disease registries).

Cited references• FUCHS S, OLBERG B, PERLETH M, BUSSE R, PANTELI D (2019): Testing a new taxonomic model

for medical devices: is it plausible and applicable? Insights from HTA reports and interviews with HTA institutions in Europe. Health Policy 123(2): 173-181

• SCHNELL-INDERST P, HUNGERA T, CONRADS-FRANKA A, ARVANDIA M, SIEBERT U (2018): Ten recommendations for assessing the comparative effectiveness of therapeutic medical devices: a targeted review and adaptation. J Clin Epidemiol 94: 97-113

• FUCHS S, OLBERG B, PANTELI D, PERLETH M, BUSSE R (2017): HTA of medical devices: Challenges and ideas for the future from a European perspective. Health Policy 121(3): 215-229

• OLBERG B, FUCHS S, PANTELI D, PERLETH M, BUSSE R (2017): Scientific Evidence in Health Technology Assessment Reports: An In-Depth Analysis of European Assessments on High-Risk Medical Devices. Value in Health 20(10): 1420-1426

• FUCHS S, OLBERG B, PANTELI D, BUSSE R (2016): Health Technology Assessment of medical devices in Europe – processes, practices and methods. Int J Technol Assess Health Care 31(5): 324-330

• HENSCHKE C, PANTELI D, PERLETH M, BUSSE R (2015): A taxonomy of medical devices in the logic of HTA. Int J Technol Assess Health Care 31(5): 324-30

European Health Technology Assessment (HTA) and Registry ... · EUnetHTA project EUnetHTA...

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