Table 2.

Categories and definitions for required data inputs

Category	Subcategory	Specific components	Description/examples
Clinical outcomes (probabilities)	Screening phase	Screening outcomes	Data on sensitivity, specificity, and duration of time for following up test results
	Medical evaluation phase and clinical outcomes	Natural history	Epidemiologic data for short- and long-term outcomes for the condition identified via clinical identification or with newborn screening; includes incidence of condition, mortality rates
		Treatment characteristics	Estimates of the effectiveness of treatment for short- and long-term health outcomes; adherence rates; adverse events
Costs	Screening phase	Test costs	Cost of the initial screen
	Medical evaluation phase	Costs of following up out-of-range test result	All costs associated with medical evaluation following an out-of-range test result
	Treatment	Direct medical costs	Hospitalizations, outpatient visits, drug treatments, procedures, diagnostic tests, medical equipment, other costs
		Direct nonmedical costs	Transportation costs, special education, home modifications, other costs
		Opportunity costs	Patient time for testing and treatment, informal caregiver time
Valuation of health outcomes		Screening results	Public or parent values for false-positive results as valued using QALYs
		Health outcomes	Public or parent/patient values for short- and long-term health outcomes included in the natural history model as valued using QALYs
		Treatments	Loss in health-related quality of life associated with treatment regimens measured using QALYs. Loss in health-related quality of life could include difficulty of adhering to dietary treatments or losses in relation to painful and difficult transplant procedures.
		Treatment-related adverse events	Loss in health-related quality of life for adverse events associated with recommended treatment regimens measured using QALYs

QALYs, quality-adjusted life years.