Budget Impact of Inotersen for the Treatment of Stage I or II Polyneuropathy in Adult Patients With Hereditary Transthyretin Amyloidosis (hATTR) in Public Health Mexico

OBJECTIVES: To perform a budget impact from the perspective of the Mexican public health sector of the use of inotersen for the treatment of polyneuropathic AhTTR in Coutinho stages I and II.

METHODS: Comparators were selected based on recommendations from international clinical practice guidelines as well as options available for the same indication in México. Estimation of annual costs was performed through a Markov model, where the evidence of efficacy was obtained through a systematic review of the literature. Direct medical costs related to the interventions evaluated were considered and a time horizon of 5 years was used. Results are presented in dollars. Total expenditure was calculated for two scenarios; expenditure in current scenario is that incurred by institutions using only standard care (defined by a Delphi panel and includes symptom monitoring). The new scenario is the one that contemplates the use of inotersen based on its market access rate, which was considered to be 3% in the first year, with an annual increase of 3%, reaching 15% in the fifth year of the analysis. Budget impact was expressed as thedifference between the two scenarios, expressed as a percentage of the institutions' budgets.

RESULTS: A cohort of 111 prevalent cases was estimated for year one with four incident cases for subsequent years. The introduction of inotersen resulted in an average annual impact of 2.3 million, which corresponds to 0.036% of the health budget, which could be considered an affordable impact.

CONCLUSIONS: Inotersen is a valuable option for the treatment of polyneuropathic hATTR in Coutinho stages I and II, since it would be the only and first pharmacological option available in Mexico for this condition.