RIO DE JANEIRO (CN) — After a workplace accident left Diogo Brollo with a traumatic spinal cord injury, his sister contacted a lawyer in hopes of getting access to an experimental treatment.

Rômulo Luiz de Aquino Colly, the lawyer for the glazier from Friburgo in Rio de Janeiro state, obtained a court order for Brollo for polylaminin — an experimental treatment still in early-stage clinical trials and unavailable to the public.

Polylaminin acts as a biological matrix to enable regeneration of nerve fibers disrupted by trauma. The protocol calls for a single application to the spinal cord, preferably within 72 hours of injury, considered critical for its potential effect.

The use of the substance outside the clinical trial has been justified in lawsuits like Brollo’s primarily under Brazilian health regulator Anvisa’s “compassionate use” framework, which allows exceptional access to unique experimental drugs, subject to agency review within 45 days.

In the case of polylaminin, that regulatory timeline clashes with the recommended three-day application window — leading to patients’ justification for their emergency requests in court, saying they risk losing therapeutic benefits if they have to wait.

Colly said, “The administrative deadlines simply do not keep pace with the clinical urgency of a patient with acute spinal cord injury.”

Discovered by researchers at the Federal University of Rio de Janeiro and developed in partnership with pharmaceutical company Cristália, polylaminin is a protein derived from human laminin found in the placenta.

Even before Anvisa authorized the start of phase 1 clinical trials on Jan. 5, patients began obtaining court orders allowing them to receive the substance. As of Jan. 14, Brazilian courts had authorized six spinal cord injury patients access to polylaminin, according to the laboratory developing the drug.

This stage of the trial, focused on evaluating safety, is to include five patients between 18 and 72 years old with complete acute thoracic spinal cord injuries who have surgery within three days of trauma.

An independent committee created specifically to track data in real time is monitoring the study, expected to last six months.

Split decisions

According to Colly, his client’s suit was based primarily on the constitutional right to health and life, which imposes on the state the duty to guarantee access to treatment when no effective alternatives are available. “We also emphasized the principle of human dignity and the patient’s right to make an informed and conscious decision about his own treatment,” he said.

In addition to medical reports and imaging exams, the lawsuit included scientific studies, academic references, news reports about the substance’s potential and the patient’s formal consent, “who assumed the risks because it is an experimental therapy,” Colly added.

For the lawyer, the judiciary has shown sensitivity and responsibility in analyzing cases involving ongoing research, especially when fundamental rights and irreversible situations are at stake. “Health regulations are essential, but many of them lag behind today’s scientific advances,” he said.

But there is no clear consensus about the courts’ role in decisions on safety and efficacy.

Judicial opinions on access to polylaminin have not been uniform. While some courts have authorized its use based on clinical urgency and lack of therapeutic alternatives, others have denied similar requests based on more restrictive technical and regulatory criteria.

In a December ruling, the Federal Regional Court of the Sixth Circuit, which covers the southeastern state of Minas Gerais, authorized polylaminin for a patient with acute spinal cord injury from a car accident.

The court found delays in Anvisa’s administrative review would compromise the substance’s effectiveness, given the stated therapeutic window. It also noted the drug would have no cost to the public treasury, since Cristália offered to donate it.

In the same ruling, the court distinguished its decision from Supreme Court precedent, holding the state is not obligated to provide experimental drugs.

According to the Sixth Circuit in that decision, supply under compassionate use if compliant with applicable regulations is an exception to that prohibition, especially when supported by technical documents and opinions indicating an acceptable safety level for the start of clinical trials.

In a separate ruling in January, the Federal Regional Court of the Fourth Circuit, which covers the southern states of Rio Grande do Sul, Santa Catarina and Paraná, denied a similar request.

In that case, the court denied access for a patient with complete paraplegia who didn’t meet the study’s anatomical criteria and whose request was reviewed about 30 days after the trauma — far beyond the recommended 72-hour window.

‘Interfering’

Henderson Fürst, a health law attorney and director of the Brazilian Society of Bioethics, said decisions like these reveal a structural lack of understanding of how clinical research regulation and drug development work in Brazil.

“When a lawyer asks the judiciary for access to a scientific research product, he is mistaken about how the right to access research functions,” Fürst said. “When the judiciary grants it, it ends up ratifying and interfering in a methodological process and in the resources allocated to that development.”

Fürst compares Brazil’s situation to other countries. In the United States, he explained, there is a so-called “right to try,” which allows patients to access experimental treatments even without proof of safety or effectiveness, provided certain conditions are met.

In Brazil, Fürst said, there is no equivalent right formalized in law. Still, judges have been allowing access to experimental therapies, but unlike the U.S. model, costs are then transferred to the public health system or private insurers.

Guillermo Glassman, a partner in the life sciences practice at São Paulo-based L.O. Baptista, said the polylaminin case “is neither the first nor the last” in which Brazil’s judiciary has intervened to allow access to substances still under research.

He cited the case of synthetic phosphoethanolamine, known as the “cancer pill,” distributed by court orders between 2015 and 2016 despite the absence of scientific proof of its safety and effectiveness. At the time, hundreds of lawsuits ordered cancer patients be supplied with the drug before clinical trials concluded and without prior Anvisa approval.

The episode prompted Congress to enact a law authorizing its use, which Brazil’s Supreme Court struck down in 2020, reaffirming the regulator’s technical authority over drug approvals.

Leo Rosenbaum, a partner at São Paulo-based Rosenbaum Advogados, which specializes in health law, said the polylaminin case reflects “a more advanced and sophisticated phase of health care litigation in Brazil,” as citizens turn to courts to secure access to treatment.

According to Rosenbaum, unlike past cases involving drugs already approved by Anvisa but unevenly distributed by the public health system, this case represents “anticipatory” litigation, seeking supply of a substance still in the experimental phase without conclusive evaluation of safety and effectiveness. This strains the system by shifting to judges’ decisions that primarily belong to technical agencies like Anvisa and scientific bodies of the state.

“Decisions involving treatments supported by early scientific evidence place judges in a central dilemma,” he said. “On one hand, there is the constitutional duty to protect the fundamental right to health and life, especially in severe conditions without proven alternatives. On the other, there is the risk of disregarding essential technical criteria such as safety, cost-effectiveness and equal access.”

Lenir Santos, a public health specialist and founder of the Institute for Applied Health Law, said although judges and lawyers are likely treating these requests as cases of compassionate use, she considers the situation “absurd.”

“It is a risk, because it overrides legislation, and legislation exists to protect people’s health,” she said.

She added, “Health care litigation in Brazil has gained autonomy and now lives for itself.”

According to Santos, the country has a structure of forums, committees and commissions designed to turn health litigation into “an institution,” a situation she considers unlikely to be reversed.

Fürst and Rosenbaum, however, highlighted the role of Brazil’s Judicial Technical Support Center, a system that provides trial courts and appellate panels with technical opinions grounded in scientific evidence to assist in analyzing requests involving medical procedures and drug supply.

“There is no way to control lawyers, as they have the right to file whatever requests they want, but it is up to the judiciary to be technical in knowing what should and should not be provided,” Fürst said. “In a situation like this, involving a substance still in early-stage clinical development, the system would flag that.”

Rogério Almeida, Cristália’s vice president for research and development, said the laboratory “is not here to discuss or judge court orders, but simply to execute them.”

“It is not the ideal tool, but putting yourself in the place of patients and families, it is a tool that exists,” he said. “If you consider how debilitating and psychologically complex this disease is, and you know there is even a minimal possibility of improvement, you will try to access that treatment in every possible way.”

According to Almeida, the company has received more than 2,000 requests for access through its patient service channels. Its standard response: The product is not registered and cannot be sold commercially yet.

Almeida said if all goes well, the company expects to apply for final approval between late 2027 and 2028.

He said the company considers the substance to have an acceptable safety profile, based on available data. However, the clinical study authorized by Anvisa is still evaluating the product’s safety.

Anvisa said the authorization is limited to the use of the product exclusively within the clinical trial protocol and added that all court decisions must be complied with regardless of regulatory review.

Courthouse News reporter Marília Marasciulo is based in Brazil.

Publicado em COURTHOUSE NEWS