Table 2 Checklist for assessing gene therapies

Clinical Effectiveness

Surrogate endpoint used

□

□

Validation given?

Rare disease

□

□

Prevalence _____

Serious condition

□

□

Single-armed trial

□

□

Matched historical cohort used?

Pediatric population

□

□

Age range _____

Reporting of adverse consequences and risks

□

□

Size of clinical trial

_____ number of patients

Length of clinical trial

_____ duration in months

Extrapolation to long-term outcomes

_____ duration in months

Elements of Value

Quantification

Severe disease

□

□

Value to caregivers

□

□

Insurance value

□

□

Scientific spillovers

□

□

Lack of alternatives

□

□

Substantial improvement in life expectancy

□

□

Other Considerations

Notes

Discounting

Different discount rates explored

□

□

Uncertainty

Alternative payment models explored

□

□