SCIENTIFIC and SOCIAL VALUE JUDGMENTS for ORPHAN DRUGS in HEALTH TECHNOLOGY ASSESSMENT

International Journal of Technology Assessment in Health Care

Volumn 32, Issue 4, 2016, Pages 218-232

  Nicod, Elena ^a   Kanavos, Panos ^a  

^a LONDON SCHOOL OF ECONOMICS   (United Kingdom)

Author keywords

HTA; Orphan drug; Scientific value judgments; Social value judgments; Thematic analysis

Indexed keywords

COST EFFECTIVENESS;

EUROPEAN MEDICINES AGENCIES; HEALTH TECHNOLOGY ASSESSMENTS; METHODOLOGICAL FRAMEWORKS; ORPHAN DRUG; SCIENTIFIC VALUES; SOCIAL VALUES; SYSTEMATIC IDENTIFICATION; THEMATIC ANALYSIS;

PATIENT TREATMENT;

ORPHAN DRUG;

ARTICLE; COST EFFECTIVENESS ANALYSIS; DECISION MAKING; DISEASE SEVERITY; ENGLAND; FRANCE; HEALTH TECHNOLOGY ASSESSMENT; HUMAN; QUALITATIVE ANALYSIS; SCOTLAND; SOCIAL PSYCHOLOGY; SWEDEN; TECHNOLOGY; THEMATIC ANALYSIS; BIOMEDICAL TECHNOLOGY ASSESSMENT; COST BENEFIT ANALYSIS; DRUG MANUFACTURE; ECONOMICS; EUROPE; HEALTH CARE PLANNING; LIFE EXPECTANCY; PROCEDURES; QUALITATIVE RESEARCH; QUALITY OF LIFE; SEVERITY OF ILLNESS INDEX;

COST-BENEFIT ANALYSIS; EUROPE; HEALTH PRIORITIES; HUMANS; JUDGMENT; LIFE EXPECTANCY; ORPHAN DRUG PRODUCTION; QUALITATIVE RESEARCH; QUALITY OF LIFE; SEVERITY OF ILLNESS INDEX; SOCIAL VALUES; TECHNOLOGY ASSESSMENT, BIOMEDICAL;

EID: 84987618854     PISSN: 02664623     EISSN: 14716348     Source Type: Journal    
DOI: 10.1017/S0266462316000416     Document Type: Article

References (29)

1. Brouwer WBF, Culyer AJ, van Exel NJA, et al. Welfarism vs. extra-welfarism. J Health Econ. 2008;27:325-338.
2. Luce B, Drummond M, Jonsson B, et al. EBM, HTA, and CER: Clearing the confusion. Milbank Q. 2010;88:256-276.
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.
4. Rawlins M. De Testimonio: On the evidence for decisions about the use of therapeutic interventions. Clin Med. 2008 8:579-588.
5. Black N. Why we need observational studies to evaluate the effectiveness of health care. BMJ. 1996;312:1215-1218.
6. Rawlins MD, Culyer AJ. National Institute for Clinical Excellence and its value judgments. BMJ. 2004;329:224-227.
7. Rawlins MD. Evidence, values, and decision making. Int J Technol Assess Health Care. 2014;30:233-238.
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).
9. Daniels N, Sabin JE. Accountability for reasonableness-An update. BMJ. 2008;337:a1850.
10. Earnshaw J, Lewis G. NICE guide to the methods of technology appraisal-Pharmaceutical industry perspective. Pharmacoeconomics. 2008;26:725-727.
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.
12. Drummond M, Towse A. Orphan drug policies: A suitable case for treatment. Eur J Health Econ. 2014;15:335-340.
13. NICE. Ultra orphan drugs. London: NICE Citizens Council Report, 2004.
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.
15. McCabe C, Stafinski T, Menon D. Is it time to revisit orphan drug policies? BMJ. 2010; 341:c4777.
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.
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.
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.
19. QSR International Pty Ltd. NVivo qualitative data software (Version 10). Doncaster, Victoria, Australia: QSR International Pty Ltd; 2012.
20. Eurordis. Rare diseases: Understanding this public health priority. Paris: Eurordis; 2005.
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)
22. The Dental and Pharmaceutical Benefits Agency. Guide for companies when applying for subsidies and pricing for pharmaceutical products, Version 2.0 2012.
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.
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
25. Schwappach DL. Resource allocation, social values and the QALY: A review of the debate and empirical evidence. Health Expect. 2002;5:210-222.
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.
27. ®. 2015. http://www.corehta.info/BrowseModel.aspx (accessed December 2015).
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.
29. Daniels N. Accountability for reasonableness. BMJ. 2000; 321:1300-1301.