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Table 5 Utility values of patients treated with cerliponase alfa with mapping to the EQ-5D-3L

From: Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Disease stage Cerliponase alfa
Mean value Standard error Median value Minimum value Maximum value Difference between EQ-5D-5L and mapped EQ-5D-3L utility values
6 0.985 0.015 1.000 0.879 1.000 0.005
5 0.762 0.011 0.747 0.747 0.836 0.088
4 0.629 0.019 0.636 0.541 0.710 0.116
3 0.464 0.036 0.510 0.325 0.555 0.038
2 0.424 0.056 0.468 0.101 0.555 0.050
1 0.218 0.078 0.267  − 0.166 0.531 0.120
0  − 0.163 0.033  − 0.159  − 0.352  − 0.071 0.292
0 + VL  − 0.198 0.060  − 0.147  − 0.594  − 0.071 0.317
0 + VL + PC  − 0.211 0.058  − 0.154  − 0.594  − 0.095 0.315
  1. Disease progression increases with decreasing disease stage. Utility values are given on a scale where 1 is equivalent to perfect health, and 0 equivalent to death. Summary values were produced from the returned EQ-5D-5L questionnaires and results were mapped to equivalent EQ-5D-3L utility values using the Van Hout algorithm [23]. UK value set
  2. PC, palliative care; VL, vision loss