This document summarizes a presentation on the transferability of data and evidence between countries for healthcare decision making. It discusses guidelines around transferring treatment effects, baseline risks, costs and utilities. It also describes approaches to improve transferability, such as identifying networks for collaboration, assessing efficacy transferability through value of information analysis, and addressing differences in patient-reported outcome reporting across cultures. The presentation evaluates challenges to transferring evidence internationally and innovative methods that could help overcome issues with accessibility, applicability and availability of data between jurisdictions.

1. Adrian Towse
Director of the Office of Health Economics
Visiting Professor London School of Economics
HTAi Tokyo May 2016
Availability, accessibility and
applicability of evidence: Transferability
- Data versus pragmatism

2. HTAi Tokyo May 2016
Agenda: Transferability - Data
versus pragmatism
• Barbieri et al. 2010, Drummond et al.
2015
• OHE HTAi Asia Policy Forum 2014
• Innovative approaches:
• Identifying / Monitoring Networks
• Efficacy Transferability
• PRO Transferability

3. HTAi Tokyo May 2016
Evidence from pharmacoeconomic
guidelines
• A review of HTA pharmaceoeconomic guidelines
(Barbieri et al 2010) found:
• relative treatment effect (after adjusting for
differences in comparators) was broadly
considered to be highly transferable
• baseline risk and unit costs were consistently
considered to be of low transferability
• resource use and utility values were
considered to be of low transferability in the
majority of cases

4. Ways in which the results from studies conducted in other
jurisdictions are used in middle income countries?
(N=number of organizations)
8
4
2 2
1
3
5
1
0
2 2
6
0
1
2
3
4
5
6
7
8
9
General background To check the validity of the data or
assumptions in the local dosser from
the manufacturer
To compare the conclusions in the
local dossier with the conclusions in
other jurisdictions
As a basis for making a local decision,
based on the foreign study's
recommendations
Often Sometimes Never
Source: Drummond et al., 2015; presented at HTAi Washington 2014

5. Categories of foreign data used when conducting local studies
(N=number of responses)
3
9
0 0
2
6
0
3
0
6
0 0
6
9
1
0
1
2
3
4
5
6
7
8
9
10
Data on epidemiology of
disease or baseline risk
Data of relative treatment
effect
Data on resource use Unit costs/prices Health state preference
values/utilities
Often Sometimes Never
Source: Drummond et al., 2015; presented at HTAi Washington 2014

6. HTAi Tokyo May 2016
2014 HTAi AHPF on Transferability
• Transfer of HTA may represent an efficient approach to building
the local HTA evidence base
• Concerns around the transfer of HTA included:
• The applicability of transferred HTA in terms of data inputs, clinical
practice patterns, and social value judgements;
• The accessibility of external HTA reports due to language barriers,
poor reporting, and different timelines;
• The different focus of HTA for middle and low income counties
• Increased collaboration, and development of various Asia specific
resources, could help with transferability of HTA in this region:
• An Asia specific core model which could guide methodology and
development of HTA, and standardise reporting;
• An Asia specific transferability tool to highlight important factors to
be considered when considering the use of external HTA reports;

7. HTAi Tokyo May 2016
Agenda: Transferability - Data
versus pragmatism
• Barbieri et al. 2010, Drummond et al.
2015
• OHE HTAi Asia Policy Forum 2014
• Innovative approaches:
• Identifying / Monitoring
Networks
• Efficacy Transferability
• PRO Transferability

8. HEALTH ECONOMIC EVALUATION OF
BIBLIOMETRIC TRENDS
IN SUB-SAHARAN AFRICA
Dr Karla Hernandez-Villafuertea, Dr Ryan Lib, Professor Karen J Hofmanc
aOffice of Health Economics, bNICE International, c PRICELESS SA, Wits/MRC Burden of
Disease Unit, University of Witwatersrand, School of Public Health
HTAi 2016
Tokyo, Japan 2016
www.pricelesssa.ac.za

9. HTAi Tokyo May 2016
Collaborations between first
author and co-authors

10. HTAi Tokyo May 2016
Transferability of Efficacy in HTA
• Existing literature on transferability advises that multinational
trials should report country-specific cost-effectiveness
results, yet typically these country specific results are only
used to assess the between-location variability for improving
the mean estimate and variance.
• We use value of information (VOI) analysis to show that
country specific results can be used to calculate the value of
transferability (transfer from the wide trial to the country of
interest) in terms of the Expected Value of Sample
Information (EVSI).
• To test this method on the results of the Scandinavian
Simvastatin Survival Study published by Cook et al. (2003).

11. HTAi Tokyo May 2016
Results (1)
-10,000 -5,000 0 5,000 10,000
Finland Iceland
WideTrial Sweden
Denmark Norway
Figure 1. Distribution of INMB

12. HTAi Tokyo May 2016
Results (2)
Source: Zamora, Marsden, Towse (forthcoming)

13. HTAi Tokyo May 2016
Agenda: Transferability - Data
versus pragmatism
• Barbieri et al. 2010, Drummond et al.
2015
• OHE HTAi Asia Policy Forum 2014
• Innovative approaches:
• Identifying / Monitoring Networks
• Efficacy Transferability
• PRO Transferability

14. HTAi Tokyo May 2016
Transferability of PRO data
• PROs data have been shown to be good proxies for clinical
outcomes
• But can patient reported outcomes data collected in one
country (or across multiple countries) be assumed to be a
good proxy for the outcomes likely in another country?
• PROs provide a systematic way of capturing patients’
subjective experience of their own health.
• Transferability of these data depends on:
(a) The underlying concepts being equally relevant cross-
culturally
(b) The labels for underlying concepts translating across
languages to mean the same thing
(c) People responding to self-report questions in a similar way
(d) The relevance of PRO summary scores/values across
different countries.

15. HTAi Tokyo May 2016
(a) Conceptual relevance
•HRQoL not a precisely defined construct (what is
‘health related’, and what is not?)
•Different cultural groups may have different views
about what aspects of health most effect HRQoL
•Or about what HRQoL is, e.g. Going broader than
health related, so measure Quality of Life or
Wellbeing, also suffers from this.
• For example growing research on capabilities; Sen
defined as capability to lead the life that that individual
values, so the construct is very subjective
•Key question: What are we are measuring and is
it transferable across different countries?

16. HTAi Tokyo May 2016
Translating PRO instruments such as EQ-5D
• In China, approximately 20 % of participants in a rating scale exercise assigned the
translated version of ‘severe’ a higher (worse) score than ‘extreme’. To avoid possible
confusion, the Chinese version of EQ-5D-5L therefore uses ‘severe’ and ‘very severe’
in the PD and AD dimensions, compared to ‘severe’ and ‘extremely severe’ in English
• In Brazil, using a preference based approach (e.g. which do you prefer ‘severe pain’ or
‘extreme pain’), it was found that a high proportion of respondents (approx 62%)
preferred ‘severe pain’ over ‘extreme pain’, i.e. preference inversion, because the
Portuguese word for ‘severe’ (‘graves’) can suggest a serious underlying health
problem which is not the case with ‘extreme’.
• When valuing the ICECAP-A capability instrument for Australia, ‘quite a lot’ was
deemed to be more than ‘a lot’ for a non-trivial number of respondents, whereas in
Britain where the instrument was developed it means less.
• Self-report of health status might vary between countries, even for those in an
objectively similar health state e.g. ‘bathing’ in Japan can be a more demanding
activity than in western countries because of repeated entry and exit from the bath-
tub – therefore ‘slight’ problems with bathing might mean something different
• Traditional New Zealand maori views of health put as much emphasis on spiritual and
‘whanau’/family as on individual physical and mental health (Perkins, Devlin, Hansen
2004) Whanau is now included in PHARMAC assessment of the effect of interventions.

17. HTAi Tokyo May 2016
Differences in self-reporting
Source: Feng et al (2016)

18. HTAi Tokyo May 2016
Gerlinger et al., 2012

19. HTAi Tokyo May 2016
Differences in self-reporting
• Differential item functioning (DIF) (differential reporting
behaviour / response-scale heterogeneity / reporting
heterogeneity) is when respondents use response scales
differently
• Two groups may have the same underlying latent health, but
interpret the scales of a PRO measure differently, such that inter-
group comparisons may suggest differences that aren’t actually
difference in their true health
• Anchoring Vignettes other one approach to identifying and adjusting
for DIF. Shown to offer promise with the EQ-5D (Knott, Black,
Hollingsworth, Lorgelly, 2016)
• The novelty of the anchoring vignette approach is that it does not just
identify DIF, but also allows for an adjustment of DIF.
• e.g. adjusted EQ-5D values can be used in analyses
• Work in progress (see Harris, Knott, Lorgelly & Rice, 2015). Still to
understand what effect this has on CEA

20. HTAi Tokyo May 2016
Knott, Black, Hollingsworth, Lorgelly, 2016
Anchoring Vignettes

21. HTAi Tokyo May 2016
References (i)
Barbieri, M., Drummond, M., Rutten, F., et al. What do international pharmacoeconomic guidelines say
about economic data transferability? Value in Health 2010;13(8):1028-37.
Cook, J. R., Drummond, M., Glick, H. and Heyse, J. F. (2003), Assessing the appropriateness of combining
economic data from multinational clinical trials. Statist. Med., 22: 1955–1976. doi:10.1002/sim.1389
Drummond, Augustovski, Kaló, Yang, Pichon-Riviere, Bae and Kamal-Bahl (2015). Challenges Faced In
Transferring Economic Evaluations To Middle Income Countries. International Journal of Technology
Assessment in Health Care, 31, pp 442-448. doi:10.1017/S0266462315000604.
Feng Y. et al. (2016). An Exploration of the Differences Between Japan and Two European Countries in the
Self-Reporting and Valuation of Pain and Discomfort on EQ-5D. Poster at ISPOR Washington DC May 2016.
Gerlinger et al. Treatment of endometriosis in different ethnic populations: a meta-analysis of two clinical
trials. BMC Women's Health 2012, 12:9 http://www.biomedcentral.com/1472-6874/12/9
Harris, Knott, Lorgelly & Rice (2015) Survey self-assessments, reporting behaviour and the use of
externally collected vignettes. Bankwest Curtin Economics Centre Working Paper, no. 15/8, Curtin
University.
Hernandez-Villafuerte K, Li R, Hofman K (submitted). Health Economic Evaluation Of Bibliometric Trends
In Sub-Saharan Africa
Knott, Black, Hollingsworth, Lorgelly (2016). Response-Scale Heterogeneity in the EQ-5D. Health
Economics
Zamora, Marsden, Towse (2016). Value of Transferability and Efficiency in HTA. Poster at ISPOR
Washington DC Meeting May 2016.

22. Adrian Towse
The Office of Health Economics
Registered address Southside, 7th Floor, 105 Victoria Street,
London SW1E 6QT
Website: www.ohe.org Blog: http://news.ohe.org
Email: atowse@ohe.org
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