The Brazilian Supreme Court’s innovative approach towards patients’ lawsuits claiming access to gene therapies

Anderson Ribeiro

Souto Correa, São Paulo

anderson.ribeiro@soutocorrea.com.br

Lucas Calabria

Souto Correa, São Paulo

lucas.calabria@soutocorrea.com.br

The ordinary pathway for drug reimbursement/health litigation

The regulatory path for a medicine to become commercially available in Brazil begins with the submission for marketing authorisation (MA) to Anvisa, which will analyse the quality, safety and efficacy of the product.

Once approved by Anvisa, the next step is the price request before the Drug Market Regulation Chamber (CMED), which will decide the maximum sale price of the product to consumers and the government. After that, the medicine can be duly marketed and promoted in Brazil.

However, for the Brazilian public health system (Sistema Único de Saúde or SUS) to provide it free of charge, there is an additional step: the health technology assessment (HT) of the therapy, which is performed by a commission linked to the Ministry of Health (MoH) called the National Commission for the Incorporation of Technologies (CONITEC).

As a rule, the steps above are necessary to access medicines in Brazil, with MA as the initial milestone. However, since health is a right of every Brazilian and an obligation of the government guaranteed by the Federal Constitution, many patients seek access to treatments through court orders, including medicines without MA. This phenomenon is famous in Brazil and is called judicialização.

In the specific case of medicines without MA, the Brazilian Supreme Court (STF) ruled between 2019 and 2020 that requirements must be verified by judges before granting patients access to therapies as follows:

1. The government cannot and is not obliged to provide experimental drugs.
2. The government is not obliged to provide unregistered drugs, except when there is a delay by Anvisa in deciding an MA request and:
   1. there is a request for MA (this requirement does not apply in the case of orphan drugs for rare or ultrarare diseases);
   2. the medicine has MA by reputable foreign agencies; and
   3. there is no therapeutic substitute in SUS.

Based on this STF ruling, several patients filed lawsuits and obtained injunctions to access medicines in Brazil, with Elevidys being a recent important case.

The Elevidys case

Elevidys is gene therapy indicated for Duchenne muscular dystrophy (DMD) which, at the time of writing this article, has a MA request pending decision by Anvisa.

Given its high cost (close to BRL 18m or US$3m) and the existence of more than a hundred lawsuits, the Attorney General’s Office (AGU), fearing that the supply of the product could undermine the stability of the SUS (according to AGU, all lawsuits would correspond to a cost of BRL 1.8 bn or US$300m), requested STF to stay all lawsuits and injunctions (Complaint RCL #68709).

In August 2024, the AGU’s request was accepted, and a few injunctions were upheld for patients who were close to the age limit of the treatment window. In parallel, STF determined that all entities involved in the supply of the product – including the pharmaceutical company seeking the MA, ANVISA, MoH, CONITEC, and CMED –should be part of conciliation meetings to discuss sustainable ways to supply the drug.

The case is under seal, but STF informed that the following topics were discussed in the meetings: the possibility of compassionate use, discounts, risk-sharing agreements, patient monitoring and centralised purchasing.

In November 2024, after four meetings, STF accepted a joint request from the pharmaceutical company and MoH, ruling on new parameters for the case, which are summarised as follows:

1. Suspension of conciliation meetings until the product is approved by Anvisa.
2. Maintenance of the injunctions’ stay, except for patients close to leaving the age range for which the medicine is indicated.
3. Monitoring of patients by the SUS, with follow-up by the MA holder.
4. If the MA is granted, the MA holder will request the price from CMED within five days.
5. The MoH will request ANVISA, CMED and CONITEC fast-track procedures for assessing the pending submissions related to the product.

Future implications

Aware that several other medicines are as expensive as Elvidys but are also the best or, in many cases, the only options for serious diseases, the above case may become an important example for companies interested in bringing gene therapies to Brazil.

For the first time under STF’s management, a process is underway in which various stakeholders are involved in ensuring access to medicines and are assessing the availability of a drug prior to its approval by ANVISA. In other words, high-cost products that might face significant hurdles in gaining reimbursement approvals from SUS could benefit from a coordinated approach among all relevant authorities.

Therefore, the future outcome of the case could become a precedent for gene therapy companies to rethink the usual way of marketing medicines in Brazil, prioritising prior alignment with all the entities involved.