Latin America Roundup: ANVISA to allow rolling submission of clinical trial data

The Brazilian Health Regulatory Agency (ANVISA) agency said it has revamped its rules governing clinical research, including enabling the rolling submission of clinical trial data. The changes “focus on reducing bureaucratic obstacles and facilitating the clinical development of medicines, without reducing the technical parameters necessary to prove their safety and effectiveness,” ANVISA said in a statement 28 November.

Among the key changes, the agency said, is the introduction of a mechanism that will allow researchers to make ongoing or continuous submissions of safety and efficacy data before submitting a formal application for marketing approval. This will allow ANVISA to assess incoming data in real time, potentially speeding approvals.

Continuous submissions, which were first used by ANVISA during the COVID-19 pandemic to assess vaccines, “will allow ANVISA to begin evaluating applications earlier, while other data and information is generated,” the agency said. ANVISA did not stipulate what categories of products the mechanism would be available for.

The new rules, which have yet to be published, will also more precisely define risk categories, study phases, and technical requirements, the agency said, as well as delineate how decisions by what Brazil calls equivalent foreign regulatory agencies (AREEs), can be used to speed analyses and reduce redundancies. The changes followed a public consultation phase in which the agency received and evaluated some 240 comments.

Also on 28 November, ANVISA published its updated regulatory agenda for 2024-2025.

Statement (Portuguese)

Novel therapies approved in Brazil and Mexico

A gene therapy costing $3 million per treatment in the United States has been approved by ANVISA for use in children with Duchenne muscular dystrophy (DMD), a genetic disease that causes progressive muscle degeneration in young boys.

ANVISA’s decision on Roche’s Elevidys (delandistrogen moxeparvoveque), announced on 2 December, would make the treatment available in Brazil, which sees an estimated 300 new DMD cases per year. The approval, granted under a 2021 rule facilitating approvals for rare-disease treatments, covers children aged 4-7 years with the condition. A post-marketing observational study will be carried out by the manufacturer as a condition of the approval.

How Elevidys will be priced in Brazil and whether Brazil’s public health system will pay for it remain to be determined. Elevidys is the third gene therapy to be approved by ANVISA for a pediatric genetic disease. With Zolgensma (onasemnogene abeparvovec-xioi) a gene therapy for spinal muscular atrophy approved by ANVISA in 2022, families initially had to go to court to get treatment covered, but coverage is now automatic for the most severe cases.

In Mexico, a treatment for Alzheimer’s disease that costs more than $25,000 per year in the United States was approved by the Federal Commission for the Protection Against Health Risks (COFEPRIS), which announced the move on 2 December. The approval of Eisai’s Leqembi (lecanemab) comes more than a year after the drug’s approval by the US Food and Drug Administration and just weeks after the European Medicines Agency’s recommendation for the drug.

“By guaranteeing access to innovative medicines, COFEPRIS reaffirms its commitment” to Alzheimer’s patients in Mexico, the agency said in its statement.

Both the Brazil and Mexico approvals come amid fierce debates in Latin America over the merits of prioritizing regulatory review of high-cost, innovative therapies. Industry and patient-advocacy groups frequently report that approvals are too slow, leaving patients without access. Others, including some heads of national regulatory agencies, have countered that middle and low-income countries are not ideal markets for such treatments and that assuring steady supplies of affordable generic medications – an issue that Mexico, in particular, has struggled with – should be the priority.

ANVISA (Portuguese)

COFEPRIS (Spanish)

Argentina institutes frequent updates to medicines register

Regulators in Argentina have updated the country’s national register of medicines and eliminated from it about 60 products that, it says, are no longer licensed or commercially available.

The National Administration of Drugs, Food and Medical Devices (ANMAT) said in a statement 21 November that it will repeat these revisions every three months, to “guarantee the system can count on having up-to-date information.”

The register, known in Argentina as the Vademécum Nacional de Medicamientos or VNM, was created in 2014 as an official, searchable reference of all medicines authorized for commercial sale in Argentina. The new, more frequent schedule of updates was instituted to avoid confusion as to which products are actually available, ANMAT said.

It is unclear why the VNM had fallen out of date. However, the news outlet Infobae reported on 27 November that some Argentinian manufacturers had been using unavailable or “ghost” drugs to gain an advantage during large tenders by public institutions. By appearing to deeply discount drugs that were listed but in fact unavailable, these businesses gained favor during the tender process.

The regular updates are part of a broader government plan to improve the efficiency and transparency of pharmaceutical purchases for the public health system, according to the Infobae report.

Statement (Spanish)

Panama hosts international regulators’ meeting

Panama’s health ministry hosted regulators from 14 Latin American countries as well as Spain, Portugal and Andorra, a tiny principality in the Pyrenees between Spain and France.

At the Ibero-American Network of Medicines Authorities (EAMI) meeting, which took place from 26-28 November in Panama City, Spain’s top regulator, María Jesús Lamas, said she aimed to share her country’s best practices and technical and regulatory experiences regarding medical devices.

The broader theme of the meeting was “regulatory convergence to improve universal access to medicines,” according to a 26 November statement by the Panamanian health ministry.

Panama health secretary Fernando Boyd Galindo said in the same statement that the role of regulatory authorities “is not only to evaluate and approve medications, but also constant market surveillance, education and training of health professionals, as well as collaboration with the pharmaceutical industry and other key actors.”

Statement (Spanish)

Latin America Roundup: ANVISA to allow rolling submission of clinical trial data

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