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Condition-specific preference-based measures: benefit or burden?

Matthijs M Versteegh1, Annemieke Leunis, Carin A Uyl-de Groot

  • 1iMTA/iBMG, Institute of Health Policy and Management/Institute for Medical Technology Assessment, Erasmus University of Rotterdam, Rotterdam, The Netherlands. versteegh@bmg.eur.nl

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
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PubMed
Summary
This summary is machine-generated.

Condition-specific preference-based measures (PBMs) offer improved sensitivity for disease impacts but may reduce comparability and miss side effects. Their use in cost-utility analysis requires careful consideration.

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Area of Science:

  • Health Economics
  • Patient-Reported Outcomes
  • Quality of Life Measurement

Background:

  • Generic preference-based measures (PBMs) may lack sensitivity to disease-specific improvements.
  • Condition-specific PBMs (CS-PBMs) are emerging to address this limitation.
  • Comparative data between generic and CS-PBMs are limited.

Purpose of the Study:

  • To develop and compare CS-PBMs derived from condition-specific questionnaires with a generic measure.
  • To evaluate the sensitivity of CS-PBMs to disease-specific effects, comorbidities, and side effects.

Main Methods:

  • Developed CS-PBMs from the Health Assessment Questionnaire (arthritis), Quality of Life Questionnaire for Cancer 30 (cancer), and Multiple Sclerosis Impact Scale 29 (multiple sclerosis).
  • Conducted a time trade-off study (N=402) to derive utility weights.
  • Compared utility values from CS-PBMs against the EuroQol five-dimensional (EQ-5D) questionnaire across four datasets.

Main Results:

  • CS-PBMs generated higher utility values than the EQ-5D.
  • The arthritis CS-PBM was insensitive to comorbidities.
  • CS-PBMs for cancer and multiple sclerosis showed equal or better discrimination of comorbidities and side effects than EQ-5D, and superior sensitivity for mild impairments.

Conclusions:

  • CS-PBMs enhance sensitivity to disease-specific effects but may compromise comparability of utility values.
  • Potential trade-offs include insensitivity to side effects and comorbidity in some cases.
  • The application of CS-PBMs in cost-utility analysis is permissible only under specific, stringent conditions.