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)

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

Disease stage	Cerliponase alfa
Disease stage	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

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
PC, palliative care; VL, vision loss

ISSN: 1750-1172