Table 4 References to the various elements in HTA reports for onasemnogene abeparvovec (Zolgensma)

Severe disease

Yes

Yes

Yes

Yes

Yes

Yes

N/A

Value to caregivers

Yes

Yes

Yes

Yes

N/A

Yes

N/A

Insurance value

N/A

N/A

N/A

N/A

N/A

N/A

N/A

Scientific spillovers

Yes

N/A

N/A

N/A

N/A

N/A

N/A

Lack of alternatives

No

No

No

No

No

No

N/A

Substantial improvement in life expectancy

Yes

Yes

Yes

Yes

Yes

Yes

N/A

Discounting

Yes

Yes

N/A

Yes

Yes

Yes

N/A

Different discount rates employed

Yes

Yes

N/A

Yes

Yes

Yes

N/A

Uncertainty

Yes

Yes

Yes

Yes

Yes

Yes

Yes

Alternative payment models explored

No

N/A

No

No

Yes

N/A

N/A