Proposed structure of economic evaluation (decision-analytic) Table 4: Summary of extended PICO to define research question that assessment will investigate Patients Intervention Comparator Reference Outcome claims Prior tests standard Genetic testing for No genetic testing Not available Safety: 1. Patients without Tissue biopsy TTR mutations and standard Any adverse family history of and use of care* events arising TTR-FAP, who tafamidis in from the genetic have neurological patients with *Standard care test or cardiac confirmed TTR includes symptom symptoms of management and Analytic and orthoptic liver performance of amyloid transplant with the test lifelong - Specificity - Sensitivity (for - NPV eligible patients) - PPV 2. Patients with N/A Genetic testing for family history of TTR mutations Effectiveness: TTR-FAP, who and use of - Neuropathy have neurological tafamidis in Impairment or cardiac patients with Score symptoms of confirmed TTR - Norfolk QoL mutation (Diabetic neuropathy) - Modified BMI - Composite endpoints measuring small and large nerve fibre function PASC also requests details on: - # patients tested per case detected - # patients tested per case treated - cost of testing per case detected - cost of testing per case treated
Proposed structure of economic evaluation (decision-analytic) Table 4: Summary of extended PICO to define research question that assessment will investigate Patients Prior tests Intervention Comparator Reference standard Outcome claims 1. Patients without family history of TTR-FAP, who have neurological or cardiac symptoms of and amyloid Tissue biopsy Genetic testing for TTR mutations and use of tafamidis in patients with confirmed TTR No genetic testing and standard care* *Standard care includes symptom management and orthoptic liver transplant with lifelong (for eligible patients) Not available Safety: Any adverse events arising from the genetic test Analytic performance of the test - Specificity - Sensitivity - NPV - PPV Effectiveness: - Neuropathy Impairment Score - Norfolk QoL (Diabetic neuropathy) - Modified BMI - Composite endpoints measuring small and large nerve fibre function PASC also requests details on: - # patients tested per case detected - # patients tested per case treated - cost of testing per case detected - cost of testing per case treated 2. Patients with family history of TTR-FAP, who have neurological or cardiac symptoms of N/A Genetic testing for TTR mutations and use of tafamidis in patients with confirmed TTR mutation
1222-TTR-FAP-FinalDAP-Accessible.docx