PowellPACarltonJRowenD, et al. Measuring what matters: little evidence supporting the content validity of EQ-5D in people with Duchenne muscular dystrophy and their caregivers. Med Decis Making. 2021.
2.
CrossnohereNLFischerRLloydAProsserLABridgesJFP. Assessing the appropriateness of the EQ-5D for Duchenne muscular dystrophy: a patient-centered study. Med Decis Making. 2021;41(2):209–21.
3.
PerfettoEMOehrleinEMBoutinMReidSGaschoE. Value to whom? The patient voice in the value discussion. Value Health. 2017;20(2):286–91.
4.
MokkinkLBTerweeCBPatrickDL, et al. The COSMIN checklist for assessing the methodological quality of studies on measurement properties of health status measurement instruments: an international Delphi study. Qual Life Res. 2010;19(4):539–49.
5.
CrossnohereNLBrundageMCalvertMJ, et al. International guidance on the selection of patient-reported outcome measures in clinical trials: a review. Qual Life Res. 2021;30(1):21–40.
KhadkaJKwonJPetrouSLancsarERatcliffeJ. Mind the (inter-rater) gap: an investigation of self-reported versus proxy-reported assessments in the derivation of childhood utility values for economic evaluation: a systematic review. Soc Sci Med. 2019;240:112543.
8.
ProsserLAHammittJKKerenR. Measuring health preferences for use in cost-utility and cost-benefit analyses of interventions in children. Pharmacoeconomics. 2007;25(9):713–26.
9.
CrossnohereNLFischerRCrossleyEVroomEBridgesJF. The evolution of patient-focused drug development and Duchenne muscular dystrophy. Exp Rev Pharmacoecon Outcomes Res. 2020;20(1):57–68.
10.
TsaiJ-HCrossnohereNLStrongTBridgesJF. Measuring meaningful benefit-risk tradeoffs to promote patient-focused drug development in Prader-Willi syndrome: a discrete-choice experiment. MDM Policy Pract. 2021;6(2):23814683211039457.
11.
LavelleTACrossnohereNLBridgesJF. Quantifying the burden of hyperphagia in Prader-Willi syndrome using quality-adjusted life-years. Clin Ther. 2021;43(7):1164–78.
12.
PowellPACarltonJRowenDChandlerFGuglieriMBrazierJE. Development of a new quality of life measure for Duchenne muscular dystrophy using mixed methods: the DMD-QoL. Neurology. 2021;96(19):e2438–50.
