118
Views
1
CrossRef citations to date
0
Altmetric
Original Research

A Discrete Choice Experiment to Derive Health Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency in France

, ORCID Icon, ORCID Icon, , , & show all
Pages 21-30 | Published online: 25 Jan 2022

References

  • Kojima K, Nakajima T, Taga N, et al. Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency. Brain. 2019;142(2):322–333. doi:10.1093/brain/awy331
  • Wassenberg T, Molero-Luis M, Jeltsch K, et al. Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency. Orphanet J Rare Dis. 2017;12(1):12.
  • Dolan P. Modeling valuations for EurQol health states. Med Care. 1997;35:1095–1108. doi:10.1097/00005650-199711000-00002
  • Haute Autorité de Santé (HAS). Choices in methods for economic evaluation; 2020.
  • Landfeldt E, Edström J, Sejersen T, Tulinius M, Lochmüller H, Kirschner J. Quality of life of patients with spinal muscular atrophy: a systematic review. Eur J Paediatr Neurol. 2019;23(3):347–356. doi:10.1016/j.ejpn.2019.03.004
  • Landfeldt E, Lindgren P, Bell CF, et al. Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study. Dev Med Child Neurol. 2016;58(5):508–515. doi:10.1111/dmcn.12938
  • Lloyd AJ, Thompson R, Gallop K, Teynor M. Estimation of the quality of life benefits associated with treatment for spinal muscular atrophy. Clinicoecon Outcomes Res. 2019;11:615–622. doi:10.2147/CEOR.S214084
  • Pickard AS, Knight SJ. Proxy evaluation of health-related quality of life: a conceptual framework for understanding multiple proxy perspectives. Med Care. 2005;43(5):493–499. doi:10.1097/01.mlr.0000160419.27642.a8
  • Torrance GW. Measurement of health state utilities for economic appraisal. J Health Econ. 1986;5:1–30. doi:10.1016/0167-6296(86)90020-2
  • Smith AB, Retzler J, Taylor MJ. Standard gamble to derive utility health states for limbal stem cell deficiency. Clinicoecon Outcomes Res. 2020;535:546.
  • Smith AB, Hanbury A, Buesch K. Eliciting health state utilities for aromatic L-amino acid decarboxylase (AADC) deficiency: a UK vignette study. J Patient Rep Outcomes. 2021;5(1). doi:10.1186/s41687-021-00403-0
  • Ryan M, Farrar S. Eliciting preference for healthcare using conjoint analysis. BMJ. 2000;320:1530–1533. doi:10.1136/bmj.320.7248.1530
  • Toumi M, Millier A, Cristeau O, Thokagevistk-Desroziers K, Dorey J, Aballéa S. Social preferences for orphan drugs: a discrete choice experiment among the French general population. Front Med. 2020;7:323. doi:10.3389/fmed.2020.00323
  • López-Bastida J, Ramos-Goñi JM, Aranda-Reneo I, Taruscio D, Magrelli A, Kanavos P. Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy. Orphanet J Rare Dis. 2019;14(1):154. doi:10.1186/s13023-019-1126-1
  • Gu NY, Botteman MF, Gerber RA, et al. Eliciting health state utilities for Dupuytren’s contracture using a discrete choice experiment. Acta Orthop. 2013;84(6):571–578. doi:10.3109/17453674.2013.865097
  • Hanbury A, Smith AB, Buesch K. Deriving vignettes for the rare disease AADC using parent, caregiver and clinician interviews to evaluate the impact on health-related quality of life. Patient Relat Outcome Meas. 2021;12:1–12.
  • Smith AB, Hanbury A, Ortiz de Zarate IB, Hammes F, de Pourvourville G, Buesch K. Eliciting health state utilities for aromatic L-amino acid decarboxylase (AADC) deficiency: a vignette study in France. Patient Relat Outcome Meas. 2021;12:237–246. doi:10.2147/PROM.S306228
  • Smith AB, Hanbury A, Whitty JA, Buesch K. A discrete choice experiment to derive health utilities for aromatic L-amino acid decarboxylase (AADC) deficiency. Patient Relat Outcome Meas. 2021;12:97–106. doi:10.2147/PROM.S294628
  • Marshall D, Bridges JFP, Hauber B, et al. Conjoint analysis applications in health - how are studies being designed and reported? Patient. 2010;3(4):249–256. doi:10.2165/11539650-000000000-00000
  • Orme BK. Getting Started with Conjoint Analysis: Strategies for Product Design and Pricing Research. Madison Wisconsin: Research Publishers LLC; 2006.
  • Rose JM, Collins AT, Bliemer MCJ, Hensher DA. Ngene [Computer Program]. Version 1.1.1 ed. ChoiceMetrics Pty Ltd; 2012.
  • Feeny D, Furlong W, Boyle M, Torrance GW. Multi-attribute health status classification systems: health utilities index. Pharmacoeconom. 1995;7(6):490–502. doi:10.2165/00019053-199507060-00004
  • Feeny DH, Furlong WJ, Torrance GW, et al. Multi-attribute and single-attribute utility functions for the health utilities index mark 3 system. Med Care. 2002;40:113–128. doi:10.1097/00005650-200202000-00006
  • Costet N, Le Galès C, Buron C, et al. French cross-cultural adaptation of the health utilities indexes mark 2 (HUI2) and 3 (HUI3) classification systems. Clinical and Economic Working Groups. Qual Life Res. 1998;7:245–256. doi:10.1023/A:1008830115246
  • Le Galès C, Buron C, Costet N, Rosman S, Slama G. Development of a preference-weighted health status classification system in France: the health utilities index 3. Health Care Manag Sci. 2002;5:41–51.
  • Econometric Software Inc. NLOGIT [Computer program]. Version 5 ed. Plainview, NY: Econometric Software, Inc.; 2012.
  • Matza LS, Stewart KD, Lloyd AJ, Rowen D, Brazier JE. Vignette-based utilities: usefulness, limitations, and methodological recommendations. Value Health. 2021;24:812–821. doi:10.1016/j.jval.2020.12.017