On February 27 – the World Day of Rare Diseases – Minister of Health Luiz Henrique Mandetta announced in the Chamber of Deputies that Brazil seeks to adopt a new purchase model for high-cost medicines for rare diseases. This would see the government only finance medicines for rare diseases where there is evidence of their effectiveness in treating patients; that is, not pay for them no matter what, as has been the case up to now. The pilot test of the new model will begin with the muscular medicine Spinraza, bidding for which will happen in the next few days.

Under the proposed new model, the government will only pay for those drugs that result in effective treatments. To this end, once the purchase is accepted, the government will monitor the success or failure of the treatment and where the drug is deemed ineffective, the laboratory involved will have to reimburse the cost of the drug. In Mandetta’s words: “we will be sharing for the first time the risks of that medication” with the pharmaceutical industry, so that in this way “not only is access to medicines promoted” but also guarantees that they do not have “unnecessary use”.

According to the minister, the application of this new modality for the purchase of high-cost medicines will be agreed and evaluated on a case-by-case basis. For now, however, drugs currently purchased by the ministry will continue to be done so in line with the existing model. According to Mandetta, the first medicine to be tested under the new scheme is Spinraza, used for the treatment of spinal muscular atrophy.

Spinraza is expected to be available in Brazil by the end of March. If the new purchase model is considered a success, the government says it will expand the list of drugs covered under this new scheme.