OBJECTIVES: To estimate the cost-effectiveness of inotersen + standard care (BSC) vs. BSC for the treatment of stage I or II polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR) in Colombia.

METHODS: A Markov model was used to simulate the progression of patients through the stages of Coutinho classification. Adult patients with stage I or II polyneuropathy were included, using information from the NEURO-TTR clinical trial. The model considered the loss of utility associated with disease progression (base case). Additionally, caregiver disutility (scenario 1) and family grief following the premature death of the patient (scenario 2) were considered.

RESULTS: Inotersen + BSC showed favorable outcomes in terms of disease progression compared to BSC. It presented a higher number of life years (LYs) (29.50 vs 25.29), more time in stages 1 and 2 (18.03 vs 3.54), shorter duration in stage 3 (11.46 vs 21.75), and higher quality-adjusted life years (QALYs) (base case: 6.33 vs 2.52, scenario 1: 5.55 vs 1.14, scenario 2: 5.42 vs 0.96). Inotersen + BSC was associated with increased costs, and the cost per additional QALY exceeded the local cost-effectiveness threshold.

CONCLUSIONS: Inotersen + BSC offers significant health gains, with more total life years, more time in stages 1 and 2, and less time in stage 3 and QALYs. The results are consistent with those obtained by health agencies such as NICE, ICER, and CADTH. The intervention did not prove to be cost-effective, a common phenomenon in treatments for rare diseases.