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SCIENTIFIC AND SOCIAL VALUE JUDGMENTS FOR ORPHAN DRUGS IN HEALTH TECHNOLOGY ASSESSMENT

Published online by Cambridge University Press:  14 September 2016

Elena Nicod  and
Panos Kanavos
Elena Nicod
Affiliation:
London School of Economics and Political Scienceelena@nicod.com
Panos Kanavos
Affiliation:
London School of Economics and Political Science
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Abstract

Objectives: We explore how broader aspects of a treatment's value and the impact of the condition on patients not captured by routine health technology assessment (HTA) methods using clinical and economic evidence, defined as “other considerations,” may influence HTA processes in different settings.

Methods: Countries included were England, Scotland, Sweden, and France. Data sources were the publicly available reports on HTA recommendations. Ten drugs with European Medicines Agency orphan designation and appraised in England were selected. Qualitative thematic analysis was used to systematically identify and code all “other considerations” based on a previously developed methodological framework, which also coded whether it was provided by stakeholders, and how it influenced the decision.

Results: A classification framework of scientific and social value judgments was developed and used throughout the study. A total of 125 “other considerations” were identified and grouped into ten subcategories based on the information provided. Eighteen to 100 percent of these, depending on the agency, were put forward as one of the main reasons for the final decision potentially contributing to accepting a higher incremental cost-effectiveness ratio or uncertain evidence. Some of these were nonquantified or nonelicited and pertained to the assessor's judgment. A taxonomy of these value judgments was created to be used in future cases. Results also contributed to better defining the determinants of social value and improving accountability for reasonableness.

Conclusions: The systematic identification of the scientific and social value judgments enables to better understanding the dimensions of value, which can be used to improve their transparency and consistent use across decisions and settings.

Keywords

Orphan drugScientific value judgmentsSocial value judgmentsHTAThematic analysis
Type
Assessments
Information
International Journal of Technology Assessment in Health Care , Volume 32 , Issue 4 , 2016 , pp. 218 - 232
Copyright
Copyright © Cambridge University Press 2016 

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References

REFERENCES

1. Brouwer, WBF, Culyer, AJ, van Exel, NJA, et al. Welfarism vs. extra-welfarism. J Health Econ. 2008;27:325–338.Google Scholar
2. Luce, B, Drummond, M, Jonsson, B, et al. EBM, HTA, and CER: Clearing the confusion. Milbank Q. 2010;88:256–276.CrossRefGoogle ScholarPubMed
3. O'Donnell, JC, Pham, SV, Pashos, CL, et al. Health technology assessment: Lessons learned from around the world-an overview. Value Health. 2009;12:S1-S5.Google Scholar
4. Rawlins, M. De Testimonio: On the evidence for decisions about the use of therapeutic interventions. Clin Med. 2008 8:579–588.Google Scholar
5. Black, N. Why we need observational studies to evaluate the effectiveness of health care. BMJ. 1996;312:1215–1218.Google Scholar
6. Rawlins, MD, Culyer, AJ. National Institute for Clinical Excellence and its value judgments. BMJ. 2004;329:224–227.Google Scholar
7. Rawlins, MD. Evidence, values, and decision making. Int J Technol Assess Health Care. 2014;30:233–238.Google Scholar
8. Garau, M, Shah, K, Towse, A, et al. Assessment and appraisal of oncology medicines: NICE's approach and international HTA experience. Report for the Pharmaceutical Oncology Initiative Group (POI), 2009. https://www.ohe.org/publications/assessment-and-appraisal-oncology-medicines-nices-approach-and-international-hta (accessed July 2016).Google Scholar
9. Daniels, N, Sabin, JE. Accountability for reasonableness - An update. BMJ. 2008;337:a1850.Google Scholar
10. Earnshaw, J, Lewis, G. NICE guide to the methods of technology appraisal - Pharmaceutical industry perspective. Pharmacoeconomics. 2008;26:725–727.Google Scholar
11. Drummond, MF, Wilson, DA, Kanavos, P, et al. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007;23:36–42.Google Scholar
12. Drummond, M, Towse, A. Orphan drug policies: A suitable case for treatment. Eur J Health Econ. 2014;15:335–340.CrossRefGoogle ScholarPubMed
13. NICE. Ultra orphan drugs. London: NICE Citizens Council Report, 2004.Google Scholar
14. Desser, AS, Gyrd-Hansen, D, Olsen, JA, et al. Societal views on orphan drugs: Cross sectional survey of Norwegians aged 40 to 67. BMJ. 2010;341:c4715.Google Scholar
15. McCabe, C, Stafinski, T, Menon, D. Is it time to revisit orphan drug policies? BMJ. 2010; 341:c4777.Google Scholar
16. Simoens, S, Dooms, M. How much is the life of a cancer patient worth? A pharmaco-economic perspective. J Clin Pharm Ther. 2011;36:249–256.Google Scholar
17. Nicod, E, Kanavos, P. Developing an evidence-based methodological framework to systematically compare HTA coverage decisions across countries: A mixed methods study. Health Policy. 2016;120:35–45.CrossRefGoogle ScholarPubMed
18. Onwuegbuzie, A, Combs, J. Emergent data analysis techniques in mixed methods research: A synthesis. In: Tashakkori, A, Teddlie, C, eds. Sage handbook of mixed methods in social & behavioral research, 2nd ed. Los Angeles: SAGE Publications; 2010.Google Scholar
19. QSR International Pty Ltd. NVivo qualitative data software (Version 10). Doncaster, Victoria, Australia: QSR International Pty Ltd; 2012.Google Scholar
20. Eurordis. Rare diseases: Understanding this public health priority. Paris: Eurordis; 2005.Google Scholar
21. European Union. Regulation EC No 141/2000 of the European Parliament and of the Council of December 16, 1999 on orphan medicinal products. Official Journal of the European Union; 1999. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_141_cons-2009-07/reg_2000_141_cons-2009-07_en.pdf (accessed July 2016)Google Scholar
22. The Dental and Pharmaceutical Benefits Agency. Guide for companies when applying for subsidies and pricing for pharmaceutical products, Version 2.0 2012.Google Scholar
23. Commission of the European Communities. Communication from the Commission to the European Parliament, the Council, the European Economic and Social Committee adn the Committee of the Regions on Rare Disease's: Europe's challenges. 2008.Google Scholar
24. Nicod, E. Why do HTA coverage recommendations for orphan drugs differ? Applying a mixed methods framework in four European countries. Eur J Health Econ. 2016; accepted for publication. doi:10.1007/s10198-016-0823-0 Google Scholar
25. Schwappach, DL. Resource allocation, social values and the QALY: A review of the debate and empirical evidence. Health Expect. 2002;5:210–222.Google Scholar
26. Baker, R, Bateman, I, Donaldson, C, et al. Weighting and valuing quality-adjusted life-years using stated preference methods: Preliminary results from the Social Value of a QALY Project. Health Technol Assess. 2010;14:1–162.CrossRefGoogle ScholarPubMed
27. EUnetHTA Joint Action 2. Work Package 8. The HTA Core Model®. 2015. http://www.corehta.info/BrowseModel.aspx (accessed December 2015).Google Scholar
28. Dolan, P, Shaw, R. A note on a discussion group study of public preferences regarding priorities in the allocation of donor kidneys. Health Policy. 2004;68:31–36.Google Scholar
29. Daniels, N. Accountability for reasonableness. BMJ. 2000; 321:1300–1301.Google Scholar
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