Table 2.

Checklist for assessing gene therapies

Item	Yes	No	Notes
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	□	□

Source: Drummond et al., 2019⁴