Health Technology Assessment:

Opportunities and Limitations

Rosanna Tarricone, PhD

Director CERGAS

Scientific Director EHTI

EHF Gastein, October 2nd 2013

Agenda

1.  HTA in Europe: where do we stand? 2.  HTA: is it and accomplished task? 3.  HTA: the way forward

HTA in Europe: where do we stand? (1)

•  HTA is a multi-disciplinary process studying the medical, economic, social and ethical implications of diffusion and use of health technology aimed at informing policy-decisions to efficiently allocate scarce resources

•  Over the past decades, the quality of clinical evidence has become the primary criterion for accepting and funding technologies (i.e. capacity to benefit from the technology)

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HTA in Europe: where do we stand? (2)

•  Increasing concerns around healthcare costs escalation has brought the attention of policy-makers to combine clinical effectiveness to economic efficiency (i.e. capacity to benefit from the technology per Euro spent)

•  Economic Efficiency and Clinical Effectiveness dominate HTA activities and economic evaluation studies, namely Cost-Effectiveness Analysis, normally make the most of HTAs

HTA in Europe: where do we stand? (3)

•  A large number of organisations undertake or commission HTAs across the world and many of them increasingly link HTA to a particular decision about reimbursement, coverage, access and use of technologies

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Explicit use of HTA: Countries

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Australia

Belgium

Denmark

Brazil

Finland

Canada

Germany

Hungary

Korea

Ireland

ItalyMexicoNetherlands

Norway

New  Zealand

Portugal  Slovakia  

Sweden  

Taiwan  UK  USA  

HTA: is it an accomplished task?

•  Although largely diffused, several challenges remain unsolved: –  Source and quality of clinical evidence –  Timing of assessment –  Costs –  Organisational aspects –  ICER & decision rule –  Types of technologies (e.g. medical devices vs.

drugs) –  Harmonisation vs. duplication

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Source and quality of clinical evidence (1)

Meta

analysis

RCT

Cohort studies

Case series

Animal research

In-vitro research

Incr

easi

ng

valid

ity

•  If experimental studies are (most often) reliable, are they also relevant for policy-making? –  Protocol-driven studies vs real-world data (e.g. observational

studies, registries) •  Provided that experimental studies hold the highest

degree of evidence, which ones do we consider? –  Comparator(s) –  Head to head vs. placebo –  Direct vs. indirect comparisons

•  See Spinner et al. “Do different clinical evidence bases lead to discordant health-technology assessment decisions? An in-depth case series across three jurisdictions” ClinicoEconomics and Outcomes Research 2013;5:69-85.

Source and quality of clinical evidence (2)

•  Are experimental studies always the golden standard for HTA? –  Unethical (when evidence is clear) –  Not possible (e.g. surgical procedures) –  Difficult (e.g. patients refuse randomisation) –  What do we compare?

•  Clinical effectiveness of new program vs. current practice? Or inexperience with the new program vs. experience of the current practice?

•  Learning curve poses the question of timing of assessment

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Source and quality of clinical evidence (3)

Timing of assessment

•  Buxton’s Law*: “It is always too early [for rigorous evaluation] until, unfortunately, it’s

suddenly too late”

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*Buxton MJ. Problems in the economic appraisal of new health technology: the evaluation of heart transplants in the UK. In Drummond MF, Economic appraisal of health technology in the European community. Oxford: Oxford Medical Publications, 1987: 103-118.

Unit of measurement for effectiveness (1)

•  HTA asks for measuring the impact of technologies onto final outcomes: – Number of Life Years Saved – Quality Adjusted Life Years

Did she invent QALYs?

“It’s not the years in your life, but the life in your years, that counts.”

Mae West

Unit of measurement for effectiveness (2)

•  HTA asks for measuring the impact of technologies onto final outcomes: – Number of Life Years Saved – Quality Adjusted Life Years

•  Although not all jurisdictions use QALYs, there is a tendency to incorporate QoL into outcomes measurement

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Unit of measurement for effectiveness (3)

•  QALY. Is equivalent to 1 year of life in full health. Product of the length of time spent in a health state and the quality of life assigned to that health state: Utility Weights, i.e. Values for State of Health

•  What utility measures? –  Direct Measurement (RS, TTO, SG) –  Health Status Classification System (QWB,

EuroQoL 5D, UI) •  Different utility measures bring to different results

and different results bring to different policy recommendations!

Costs: where do we stop?

•  Healthcare direct costs (e.g. hospital admissions, diagnostic tests, lab exams, drugs,..)

•  Non healthcare costs (e.g. informal care) •  Productivity losses •  Societal perspective asks for all cost components to be

considered but the majority of HTA Agencies limit the assessment to healthcare costs falling under the NHS budget: –  Silos budgeting does not consider relevant implications of new

technologies onto other stakeholders

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Organisational implications

•  Many technologies are likely to have relevant organisational implications once introduced in clinical practices (e.g. new skills to be developed, new way of delivering same services, modifications of inter-professional relations, logistics,..)

•  In a recent literature review, only one article out of 210 addressed the organisational aspect of HTA*

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*Nielsen et al. Health technology assessment: research trends and future priorities in Europe. Journal of Health Services Research and Policy 2011;16(2):6-15.

HTA in EU: the case of TAVI Austria UK Belgium France NL IT-P Croatia IT-L

Source of evidence:

RCTs Yes Yes Yes Yes Yes Yes Yes Yes

No-RCTs Yes, but not the same

Other HTA reports No Yes No No Yes Yes Yes No

Clinical guidelines No Yes Yes Yes Yes No Yes Yes

Other CEA Na Na Na Na Na Na Yes Yes

Comparator(s) No No Yes, AVR No No No No No

Costs No Yes but not specified

Valve Intervention & hospital stay

No DRG Valve & consumables

No

Organisational aspects

No No No Medical staff & No of procedures (200)

Medical staff & No of procedures (50)

No Medical staff

Medical staff

ICER Inoperable 37,400€ /QALY High risk 750,000€ /QALY

Final recommendations

Positive but restricted to some centres & codtl reimbursement

No recommendation

Positive for inoperable Negative for high risk patients

Positive for inoperable patients

Positive for inoperable patients

No recommendation

Positive for inoperable patients

No recommendation

HTA: the way forward (1)

•  Harmonisation: If desirable in principle, it seems not entirely feasible in practice today

•  JA2 endeavoured to develop a toolkit to adapt HTA reports to other jurisdictions: –  An attempt in Italy was not successful: too much time to adapt

•  Unless clear convergence exists around key issues, it would be difficult to reduce duplications in EU: –  Clinical evidence –  Utility measures –  Type of costs –  Type of technologies (e.g. medical devices vs. drugs) –  Organisational and ethical aspects –  Stakeholders involvement (patients, industry,..) 19

HTA: the way forward (2)

•  EU recently funded research project to deep dive into those issues: –  MEDTECHTA (CERGAS, Italy) aims at developing methods to

assess medical devices by studying the characteristics that make MDs different from drugs

–  ADVANCE-HTA (LSE, UK) aims at analysing concepts of value assessment and value for money, assessment of rare diseases, elicitation of preferences and capacity building of HTA

–  ADHOPHTA (Fundació Privada Clínic per a la Recerca Biomèdica, Spain) aims at strengthening the use and impact of excellent quality HTA results in hospital settings

–  INTEGRATE-HTA (Bremen, Germany) aims to develop concepts and methods for HTA to enable a patient-centered, integrated assessment of the effectiveness, and the economic, social, cultural and ethical issues of complex technologies (i.e. palliative care)

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HTA: the way forward (3)

•  Evidence from these studies would bring new insights and guidelines to improve harmonisation across jurisdictions in EU

•  Although socio-economic contexts and institutional arrangements of healthcare systems in EU will always influence final recommendations, harmonisation in methods is likely to increase efficiency in the HTA process

•  However, equity of access across EU patients still remains unclear and opens further area of research

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Thank You

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