TAFAMIDIS FOR TTR-FAP
Tafamidis for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP)
Background
Transthyretin familial amyloid polyneuropathy (TTR-FAP) is one of three types of familial amyloid polyneuropathy (FAP). It is due to inheritance of a mutation in a protein called transthyretin (TTR), which is mainly made in the liver. This results in deposits of amyloid in nerves and the heart, leading to nerve damage and heart failure. Symptoms include sensory motor neuropathy such as limb weakness and loss of sensation; autonomic dysfunction affecting the bladder, bowel and sexual function; the heart is frequently affected and other organs including the kidneys can also be involved. Currently treatment involves supportive therapy and liver transplantation. Liver transplantation aims to stabilise the disease by replacing the main source of mutant TTR with a liver that makes normal transthyretin.
Tafamidis is a new drug treatment that is claimed to work by binding to TTR protein in the blood and reducing its propensity to aggregate abnormally and form amyloid.
The aim of this project is to assess the clinical effectiveness and cost-effectiveness of tafamidis for the treatment of TTR-FAP compared to current treatment options of supportive therapy, including liver transplantation. The assessment will include an appraisal of the manufacturer's submission to AGNSS. A systematic review will be undertaken to ensure that all relevant data on tafamidis and the comparator interventions are included in the assessment. A critical appraisal will be undertaken of any previous published cost-effectiveness studies as well as the economic model submitted by the manufacturer. If necessary the manufacturer's model will be extended or amended to explore key areas of uncertainty.
This project has now been completed and a report will be issued.
Conducted by: Catriona McDaid1
, Mark Corbett1, Lisa Stirk1, Rita Faria2, Simon Walker2, Stephen Palmer2