1. Transferability of HTA between countries
Zoltán Kaló
Professor of Health Economics
Eötvös Loránd University (ELTE)
Syreon Research Institute
Transferability of HTA between countries

2. Pragmatic approach to evidence based health policy
Too complicated and time-consuming to rank all available health care technologies according to their cost-effectiveness  cost-effectiveness criteria are assessed mainly for new and expensive therapies
For innovative pharmaceuticals, the mandatory economic evaluation represents the fourth hurdle to market access, as registration already includes assessment of the efficacy, safety and quality.
In addition to considering the health gain, the risk-benefit ratio and cost- effectiveness, public payers take into account several other factors in their decisions, including unmet medical need, budget impact, equity, incidence and prevalence of the disease.
All these factors are incorporated into a formal health technology assessment process in several countries, prior to the reimbursement and formulary listing of new pharmaceutical therapies

3. Importance of NICE
National Institute for Health and Clinical Excellence (NICE) in England and Wales is one of the most prominent public institutions to incorporate economic evaluation and health technology assessment into its recommendations
As NICE publishes health technology assessment reports that are considered to be unbiased references, public decision- makers in many other countries implicitly take into account the NICE recommendations in their own decisions (O’Donnel et al., 2009)

4. Central-Eastern European situation
Presentation Information
Central-Eastern European situation
HTA capacity is limited in CEE countries
Public payers and policymakers have a greater need to rely on NICE recommendations in their own decisions, often without understanding the limitations of the transferability of health technology assessment reports.
Reliance on NICE recommendations is especially common for new technologies with significant budget impact.
Malignancies
the public health burden of malignancies in CEE is huge
innovative pharmaceutical therapies are very expensive
NICE recommendations in oncology may receive even greater attention in CEE than in any other therapeutic areas.
CONFIDENTIAL 4

6. Welte’s knock-out criteria for HTA transferability
“General knock-out” criteria preclude transferability of cost- effectiveness results when either the investigated technology or the comparator are irrelevant, or the methodological quality of the cost-effectiveness study does not meet local standards, meaning that the starting points of the study are irrelevant to local decision-makers.
“Specific knock-out criteria” apply when cost-effectiveness results are only transferable after adjustment for differences in treatment patterns, in unit costs, or other aspects for which adjustment may be required.
Ref: Welte et al. Pharmacoeconomics, , 857–876.

7. Policy vs data driven HTA determinants
Policy-driven determinants:
If the local policy is similar to the NICE policy, there is no need for local adjustment of that particular determinant
If the local policy is different from the NICE policy, the transferability of recommendations becomes more limited.
Data-driven determinants:
require local adjustment, when the data is different.

8. Determinants of health technology assessment
HTA section
Determinant
Policy driven
Measure
burden of disease
number of patients no
prevalence; incidence
health status of the target population; disease progression
life expectancy; healthy life expectancy; comorbidities; standardized mortality
subgroup of patients
age; proportion of patients in different disease stages; pharmacogenomic status
unmet medical need
added innovation
availability of effective therapeutic alternatives; first in class; mechanism of action
public health need
priority setting
yes
local public health program
special regulation
orphan drug status; end of life therapy
comparator
positioning of new therapy in local therapeutic guidelines
first line, second line, etc.
relevance of the comparator
reimbursement status; local practice for standard therapy

9. Determinants of health technology assessment
HTA section
Determinant
Policy driven
Measure
health gain
baseline risk no
mortality; risk of clinical endpoints
efficacy or relative effectiveness
absolute risk reduction, relative risk reduction
real world benefit
adherence / compliance
health state valuation
partly
utility estimates
cost- effectiveness
unit cost
production function of health care services; relative prices of medical technologies
resource utilization
local treatment practices and patient routes
methodology of economic evaluation
time horizon
yes
projection of health gain and cost (in years)
discount factor %
perspective
health care or societal perspective; inclusion of indirect costs
CE threshold
explicit or implicit threshold
opportunity cost
budget impact
expected costs in local currency
equity
accessibility
access and utilization according to geographical and socioeconomic status

10. Incidence of malignant neoplasms per 100,000 persons (2008)
Incidence & mortality
Incidence of malignant neoplasms per 100,000 persons (2008)
Standardized death rate of malignant neoplasms per 100,000 persons (2009 or nearest year)
Czech Republic
288.5
254
Estonia
230.1
275
Hungary
286.6
316
Poland
225.1
272
Slovak Republic
260.6
263
Slovenia
264.8
266
United Kingdom
269.4
199
Source: OECD (2011), Health at a Glance 2011

11. Mortality burden of malignancies 2005
Source: WHO Health for All, 2010

12. 5-year survival probability,
Disease progression Colorectal cancer 5-year survival and standardized mortality
5-year survival probability,
( or nearest period)
Colorectal cancer age standardized mortality / 100,000, (2005 or nearest year)
male
female
Czech Republic
45.6%
48.5%
31.0
Hungary -
31.9
Poland
34.7%
39.3%
20.8
Slovak Republic
29.8
United Kingdom
50.4%
53.1%
17.6
Source: OECD, Health at a Glance, 2009

13. Unmet medical need Cervical cancer screening,
percentage of women screened aged 20-69
Standardized death rate cervix carcinoma (<70 year/100,000 women)
Czech Republic
47.7
4.0
Estonia
52.0
5.4
Hungary
23.7
5.2
Poland -
6.5
Slovak Republic
22.9
5.9
Slovenia
72.3
3.3
United Kingdom
78.7
2.1
Source: OECD (2011), Health at a Glance 2011

14. Unmet medical need
Until 2010 NICE reviewed 5 innovative drug therapies to treat renal cell carcinoma (bevacizumab, sorafenib, sunitinib, temsirolimus and everolimus).
NICE recommended only one first-line therapy (sunitinib) within a special patient access scheme (2009) and refused all second-line sequential treatments (2009).
Despite sorafenib and temsirolimus are orphan drugs in this indication, NICE guidance quotes that survival data is immature and questions the robustness of life extension and sub-group analyses. It is not surprising that overall survival data is immature and non-robust for orphan drugs.
The moral question is why an accelerated registration process should be granted for oncology drugs if later patient access to those medicines is denied by the lack of reimbursement.

15. Unit cost Annual remuneration of employed health professionals (latest available years between ; US$ exchange rate)
2007
General practitioners
Specialist physicians
Hospital nurses
Czech Republic
(35 167*)
30 859
15 987
Estonia
24 488
31 191
14 749
Hungary
23 410
26 086
12 603
Slovak Republic
25 984 -
12 095
Slovenia
65 447
75 487
28 207
United Kingdom
111 580 (212 144*)
51 673
Source: OECD Health Data 2010, June

16. Differential pharmaceutical pricing in the European Union: mission impossible?
Parallel trade
International price referencing
Transparent pricing strategy
Price convergence
Launch sequence

17. Impact of European Union: narrow pricing corridor for new medicines

18. Resource utilisation
The ratio of practising nurses to physicians is 3.6 to 1 in the United Kingdom and less than 2.4 to 1 in Central-Eastern European countries (OECD, 2011).
The system of health service providers is different in the NHS, e.g. gatekeeping function of general practitioners.
Mammography (OECD, 2011) and cervical cancer screening rates are higher in the United Kingdom.
There are more acute care beds in Central-Eastern European countries and the number of hospital discharges for cancer cases is 2-3 times higher than in the United Kingdom (OECD, 2011).
Greater inequity in the accessibility of health care services in CEE than in the UK (OECD, 2011).

19. Survey of HTA agencies in LatAm / CEE / Asia: In what ways are results from studies conducted in other jurisdictions used?
Ref: Drummond M, Augustovski F, Kaló Z, Yang BM, Pichon-Riviere A, Bae EY, Kamal-Bahl S. Challenges faced in transferring economic evaluations to middle income countries. Manuscript in submission, 2014

20. Survey of HTA agencies in LatAm / CEE / Asia: Which categories of data from foreign studies are most often used in submissions you receive?
Ref: Drummond M, Augustovski F, Kaló Z, Yang BM, Pichon-Riviere A, Bae EY, Kamal-Bahl S. Challenges faced in transferring economic evaluations to middle income countries. Manuscript in submission, 2014

21. Survey of HTA agencies in LatAm / CEE / Asia: Obstacles to transferring economic evaluationss from other jurisdictions
Ref: Drummond M, Augustovski F, Kaló Z, Yang BM, Pichon-Riviere A, Bae EY, Kamal-Bahl S. Challenges faced in transferring economic evaluations to middle income countries. Manuscript in submission, 2014

22. Number of times mentioned
Survey of HTA agencies in LatAm / CEE / Asia: Which categories of foreign data do you consider to be transferable?
OBSTACLE
Number of times mentioned
Other practice patterns, or the availability of facilities, are often different in my jurisdiction 10
The current standard of care/ relevant comparator is often different in my jurisdiction 9
Studies are often conducted in countries with a higher GDP, so results do not apply in my jurisdiction 8
Studies are often badly reported, or not enough details are given
It is often difficult or impossible to obtain an electronic copy of the model 7
The patient population is often different in my jurisdiction 6
Often, it is not possible to find local data to re-populate the model
Studies often have methodological deficiencies 5
Decision-makers in my jurisdiction much prefer a locally designed study
Studies often use methods that are too advanced for decision-makers in my jurisdiction 4
Other obstacles (please list and rank) 3
Lack of local technical capability 1
Decision-makers in my jurisdiction much prefer non-data driven arguments
Different resources & costs used in other jurisdictions
Ref: Drummond M, Augustovski F, Kaló Z, Yang BM, Pichon-Riviere A, Bae EY, Kamal-Bahl S. Challenges faced in transferring economic evaluations to middle income countries. Manuscript in submission, 2014

23. Conclusion
Transferability of good quality HTA reports could be beneficial to prevent duplication of efforts and save resources for local HTAs.
Certain elements of HTA reports are transferable, but adjustment to local data is absolutely necessary.
Copying recommendations based on international HTA without local adjustment may do more harm than good.