Judicialization of delandistrogene moxeparvovec in Brazil: a cross- sectional study on clinical eligibility, litigation patterns, and public expenditure

In the modern world of medicine, a new frontier has opened for treating rare and devastating genetic diseases. For decades, patients with conditions like Duchenne muscular dystrophy, a progressive muscle-wasting disorder that primarily affects young boys, relied on treatments that could only slow the disease's march. Recently, a different approach emerged: gene therapy. These are one-time treatments designed to fix the underlying genetic error by delivering a working copy of a missing gene directly into the body. While this offers a glimmer of hope where there was none before, it brings a complex set of challenges. These therapies are incredibly expensive, often costing millions of dollars per patient, and the long-term proof that they work as well as hoped is sometimes still being gathered. In countries with public health systems, where the government pays for care, this creates a difficult balancing act. How do officials decide whether to spend vast sums on a treatment that is promising but not yet fully proven, especially when there is no guarantee it will work for everyone?

This tension came to a head in Brazil regarding a specific gene therapy called delandistrogene moxeparvovec. The country's health regulators had given the drug special permission to be sold because of the severity of the disease it treats, but a separate government committee, which evaluates whether new technologies should be added to the public health system, had not yet recommended it for widespread use. In the gap between these two decisions, a wave of legal action began. Families and lawyers started filing lawsuits against the government, demanding that the state pay for the treatment for their children. A new study set out to understand exactly what was happening in these court cases. Researchers gathered data on every single lawsuit filed against the Brazilian Ministry of Health between January 2024 and February 2026 that sought this specific drug. They looked at who was asking for the medicine, whether those patients actually met the strict medical rules for using it, how the courts handled the requests, and how much money the government was spending to comply with court orders.

The researchers found a picture that was far more complicated than a simple story of patients fighting for their rights. They identified 223 individual lawsuits, all involving boys with Duchenne muscular dystrophy. However, when they checked the medical details against the official rules for the drug, a startling pattern emerged. The drug is approved for a very specific group: boys between the ages of four and seven who can still walk. Yet, only about one in six of the lawsuits involved patients who fit both of these requirements. Nearly half of the cases involved boys who were either too young or too old, or who had already lost the ability to walk. In a significant number of other cases, the court files simply did not contain enough information to know if the patient could walk or not, even though the patient was within the correct age range. Despite these missing details or clear mismatches with the rules, the courts had already ordered the government to provide the drug in 15 cases.

The financial cost of these decisions was immense. For the 15 cases where the government fully complied with the court orders, it spent nearly 194 million Brazilian reais. When the researchers broke this down, they found that only three of those patients actually met the official age and walking criteria. The other twelve patients either did not meet the criteria or lacked the necessary medical records to prove they did. This suggests that the legal system was sometimes granting access to the drug for patients who fell outside the safety and effectiveness boundaries established by regulators. The study also highlighted a stark inequality in who was able to use the courts. Almost all the lawsuits were filed by private lawyers hired by families, while very few were handled by public defenders who represent those who cannot afford legal fees. This indicates that the ability to secure this expensive treatment through the courts may depend heavily on a family's resources and access to private legal help.

Looking at the bigger picture, the researchers calculated what would happen if the government were forced to pay for every single one of the 223 lawsuits. The cost would range from nearly 2.4 billion to over 4 billion Brazilian reais, depending on the price of the drug in each case. This massive potential expense raises serious questions about how a public health system can remain sustainable when individual court decisions can redirect such huge amounts of money toward treatments that may not be suitable for all patients. The study suggests that the current system is struggling to manage the arrival of high-cost gene therapies. It points out that while the courts are trying to protect the right to health, they are often making decisions without complete medical information, and in doing so, they may be delaying treatment for the few patients who clearly qualify while spending public funds on those who may not benefit. The author argues that to fix this, the country needs better ways to check medical records before a judge makes a decision, and new models for paying for these therapies that balance the urgent needs of individual patients with the long-term health of the entire system.