The National Committee for Incorporation of Technologies (CONITEC, Comissão Nacional de Incorporação de Tecnologias) has rejected the inclusion of nusinersen (Spinraza) into the SUS (Brazil’s universal health scheme).
Spinraza belongs to a class of medicines known as advanced therapy medicinal products (ATMPs), and is currently the only medicine available worldwide to treat spinal muscular atrophy (SMA). SMA is classified as a rare disease as it affects approximately 7 to 10 babies per 100,000 live births.
The review by CONITEC was unusual as in this instance they used cost-effectiveness analysis to support the decision outcome, something that CONITEC has rarely done previously when assessing orphan drugs.
There is a consultation open on the decision until 17 September that will allow relevant stakeholders to give their opinion. Details of the consultation can be found here.