Adrian TowseDirector of the Office of Health EconomicsVisiting Professor London School of Economics

HTAi Tokyo May 2016

Availability, accessibility and applicability of evidence: Transferability - Data versus pragmatism

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

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

Ways in which the results from studies conducted in other jurisdictions are used in middle income countries?

(N=number of organizations)

0

1

2

3

4

5

6

7

8

9

8

4

2 2

1

3

5

1

0

2 2

6

Often Sometimes Never

Source: Drummond et al., 2015; presented at HTAi Washington 2014

Categories of foreign data used when conducting local studies (N=number of responses)

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

0

1

2

3

4

5

6

7

8

9

10

3

9

0 0

2

6

0

3

0

6

0 0

6

9

1

Often Sometimes Never

Source: Drummond et al., 2015; presented at HTAi Washington 2014

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;

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

HEALTH ECONOMIC EVALUATION OF BIBLIOMETRIC TRENDS

IN SUB-SAHARAN AFRICADr 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 2016Tokyo, Japan 2016

www.pricelesssa.ac.za

HTAi Tokyo May 2016

Collaborations between first author and co-authors

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).

HTAi Tokyo May 2016

Results (1)

-10,000 -5,000 0 5,000 10,000

Finland IcelandWideTrial SwedenDenmark Norway

Figure 1. Distribution of INMB

HTAi Tokyo May 2016

Results (2)

Source: Zamora, Marsden, Towse (forthcoming)

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

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.

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?

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.

HTAi Tokyo May 2016

Differences in self-reporting

Source: Feng et al (2016)

HTAi Tokyo May 2016

Gerlinger et al., 2012

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

HTAi Tokyo May 2016Knott, Black, Hollingsworth, Lorgelly, 2016

Anchoring Vignettes

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.

Adrian TowseThe Office of Health Economics

Registered address Southside, 7th Floor, 105 Victoria Street, London SW1E 6QT

Website: www.ohe.org  Blog: http://news.ohe.orgEmail: atowse@ohe.org

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