Horizon scanning (HS) is an important tool for guiding health policy formulation and the decision-making process in Brazil. In 2016, the Ministry of Health started to draft Brazilian clinical practice guidelines for transthyretin familial amyloid polyneuropathy (TTR-FAP), which is a rare disease caused by a mutation of the transthyretin gene. An initial HS report was conducted that provided information about new and emerging technologies for TTR-FAP. The HS identified five drugs that were based on two mechanisms of action: transthyretin stabilization (diflunisal, tafamidis, and tolcapone) and gene silencing (ALN-TTR02 and ISIS-TTR-Rx). At that time in Brazil there were no drugs registered for the treatment of TTR-FAP. However, a few months later tafamidis was licensed in Brazil. In early 2017 the manufacturer submitted an application to the National Committee for Health Technology Incorporation (CONITEC), with the aim of incorporating tafamidis into the Brazilian health system. As a result the HS report was updated to support the assessment by CONITEC. This study aims to show how HS is being used to support CONITEC in this issue.

As per the EuroScan toolkit, we performed a reassessment of the technologies included in the initial HS report. We searched clinical trial registers, the websites of pharmaceutical companies, conference proceedings, scientific journals, HS databases, and regulatory websites for further information. The data were synthesized and a reformulated landscape of the technological environment for TTR-FAP therapy was presented to the CONITEC Plenary.

The main difference between the initial and final HS output was that tafamidis was approved for use in Brazil, making it the only registered drug for TTR-FAP. Another difference was related to the start of a new clinical trial with diflunisal for TTR-FAP, indicating that this drug could be a potential competitor for tafamidis. It was also possible to add published positive results from a clinical trial with ISIS-TTR-Rx, which were unavailable when the first report was written. Beyond that, it appears that there are two promising gene silencers on the horizon that could represent potential competitors for tafamidis.

The analysis of tafamidis for incorporation into the Brazilian health system is ongoing, but HS was able to deliver strategic information that could affect the final recommendation of CONITEC.