Table 2 Assumptions related to health-related quality of life (HRQoL) and % panel agreement

Apart from the impact of comorbidities, short stature (final height < 140 cm) likely has an independent negative impact in individuals with achondroplasia on:

 Physical health-related quality of life

73

8

 Mental health-related quality of life

64

8

In individuals with achondroplasia, vosoritide likely increases HRQoL through lifetime if long-term treatment is started before puberty (Tanner stage > 1)

82

8

A positive impact of vosoritide on HRQoL is more likely in individuals with achondroplasia starting long-term treatment at an earlier age than in those starting treatment later

100

0

Increased frequency of surgeries relative to the general population has a substantial negative short-term impact on HRQoL in individuals with achondroplasia

100

0

A positive impact of vosoritide on the incidence of surgeries is more likely in individuals with achondroplasia starting long-term treatment at an earlier age than in those starting later

90

17

A positive impact of vosoritide on chronic pain through lifetime is more likely in individuals with achondroplasia starting long-term treatment at an earlier age than in those starting later

88

33

A positive impact of vosoritide on work participation through lifetime is more likely in individuals with achondroplasia starting long-term treatment at an earlier age than in those starting later

78

25

A positive impact of vosoritide on activities of daily living through lifetime is more likely in individuals with achondroplasia starting long-term treatment at an earlier age than in those starting later

73

8