Bolsonaro government authorizes purchase of the most expensive medicine in the world for up to R$ 6.5 million

The Jair Bolsonaro government (PL) approved raising the maximum selling price in Brazil for zolgensma from R$2.9 million to R$6.5 million, a treatment considered the most expensive in the world.

Used in children with SMA (spinal muscular atrophy), the drug has a price tag since the end of 2020, but it can cost public coffers more than R$10 million per patient.

The government has not yet released the new value. The information is contained in the minutes of the ministers’ meeting that approved the change, obtained by the leaf.

As the product was not launched in Brazil and is outside the SUS (Unified Health System), the government pays for some treatments by order of Justice.​

In a note, the Ministry of Health said that it had already paid for 75 deliveries of zolgensma, disbursing R$ 715.7 million.

With the change in the maximum price, members of Saúde hope to gain greater bargaining power with the manufacturer Novartis and reduce the price of judicialization.

The definition of the ceiling value is also a necessary step to negotiate the eventual incorporation of the drug into the list of medicines offered by the SUS.

The pharmaceutical, however, asked for R$ 9.7 million as a ceiling value, a figure that is equivalent to the lowest price in the international market.

Novartis said it has not yet been notified of the new price.

“We reaffirm our commitment to the patient community and all actors in the healthcare system and we remain open to dialogue to advance the challenges and opportunities to bring innovation to everyone’s health,” the company said in a statement.

The decision on the new value of zolgensma was taken on January 25 during a meeting of the ministers of the CMED (Medicine Market Regulation Chamber), the last instance of the body that sets the price of drugs in Brazil.

Minister Anderson Torres (Justice and Public Security) suggested, at the meeting, that the amount be set at R$ 5.1 million. Paulo Guedes (Economy) defended the manufacturer’s recommended price, while Marcelo Queiroga (Health) proposed R$ 6.5 million.

This price is used for commercialization by laboratories and distributors and does not consider the ICMS charge, which can reach 20% in Rio de Janeiro.

Government sources recognize that the increase in the maximum price does not in itself guarantee the viability of an agreement with the manufacturer for the acquisition of the drug, but the issue remains under monitoring by the folders.

Anvisa (National Health Surveillance Agency) approved the registration of zolgensma in August 2020. In December of the same year, CMED set the cost at around BRL 2.9 million.

The figure was calculated from the value of two years of SMA treatment with spinraza, a competing drug. To reach approximately R$ 6.5 million, Saúde considered the comparison of 5.2 years of use of spinraza.

Novartis has questioned the price set in 2020 on the grounds that the drug is an innovative gene therapy that has lasting results, while the competitor offers a continuous treatment.

“The use of single-dose gene therapy lessens the impact related to multiple intrathecal applications, need for regular visits to physicians and healthcare services, which determines a better quality of life for patients and their caregivers,” Novartis said in the resource. .

The government is still wading through the debate on incorporating high-cost drugs into the SUS. In 2019, Health tested a “risk sharing” model with Biogen for spinraza delivery, but the plan was terminated.

Patients still complain that there are many restrictions on access to drugs that are in the public network, which keeps the door to judicialization open.

Lawyer and coordinator of the Health program at Idec (Brazilian Institute for Consumer Protection), Ana Carolina Navarrete stated that there are weaknesses in the criteria used to define prices in Brazil.

Navarrete argues that the center of the debate on access to high-cost treatments should not be pitting individual interests against the collective. “Before asking when society is willing to pay for a life, there is another question: why does medicine cost so much?”

“The way forward may be to give more transparency to pricing criteria and to think of policies that are not limited to setting a value, such as mobilizing the health industrial complex [para produção dos fármacos no Brasil]said Navarrete.

In August 2020, Marina Moraes de Souza Roda was the first child to receive a single dose of zolgensma in the country. The procedure was performed at Hospital Israelita Albert Einstein, in São Paulo.

SMA is a rare disease caused by a change in the gene responsible for encoding the protein necessary for the proper development of muscles. The disease therefore causes weakness, hypotonia, atrophy and progressive muscle paralysis and can lead to death.

The government monitors with concern the actions in the courts to provide treatment. In July 2021, the president of the Federal Supreme Court (STF), Luiz Fux, ordered the Union to supply zolgensma to a child.

In 2017, Gianlucca Trevellin, now 9 years old, was the first patient to receive in court the right to treatment against SMA with spinraza funded by the Ministry of Health.

The drug only arrived the following year. After four years of infusions, the patient’s father, Renato Trevellin, stated that there is a clear improvement in health.

“Today he is able to stay off the ventilator for periods, which would be impossible for a 9-year-old patient. He can also play on the computer with hand movements and can stand up. Before, he was in a lot of pain,” he said.

Trevellin presides over an institute named after his son and supports other AME patients. “With this high amount, practically everyone has to go to court. It’s usually a year of fighting.”

“A person who looks healthy doesn’t know if he has the rare disease and may need a drug of that cost tomorrow. He has to show solidarity. Are you going to condemn the child to death because of the price?”, said Trevellin, who also charges that industry to reduce drug prices.