Toward Leadership: How Brazil can Unlock Clinical Trials with Regulation, Transparency and Technology

Brazil has unique characteristics that position it as a natural candidate to become a strategic hub for the conduct of clinical research. In addition to being the world’s ninth-largest pharmaceutical market and having the largest urban and economically active population in Latin America, Brazil’s average cost of conducting clinical studies is approximately 44% of the cost in the United States.

Nevertheless, a complex regulatory landscape and the lack of appropriate incentives mean that Brazil accounts for only about 2% of the clinical studies initiated annually. The country ranks twentieth globally, behind countries such as Egypt, Turkey, and Taiwan.

The Legal Framework for Clinical Research (Statute No. 14,874/2024), together with recent initiatives by the Brazilian FDA (Anvisa) (Rules No. 338/2024 and No. 9445/2024), represents an important step toward consolidating a modern and internationally harmonized regulatory environment. The proposed update to the Clinical Research Action Plan demonstrates the Government’s intention to create incentives to consolidate Brazil’s position in the global clinical research landscape.

The path ahead remains long. The lack of implementing regulations for the new Legal Framework for clinical research—even one year after its enactment—and the bifurcated pathway with still-lengthy timelines for study approvals must be overcome.

For Brazil to reach the level of countries such as China and the United States, it must not only advance regulatory frameworks—as it has been doing—but also implement broader strategies grounded in the integration of new technologies, such as the use of Artificial Intelligence in Real-World Evidence. This article analyzes Brazil’s progress, presents current limitations, and indicates future pathways.

Regulatory Overview: Advances and What Still Needs to Be Done

The regulatory environment has historically been one of the principal disincentives to conducting clinical studies in Brazil. Regulatory approval—both sanitary and technical—and ethical approval are rendered by distinct actors in parallel procedures. While sanitary/technical approval falls to the Brazilian Health Regulatory Agency (Anvisa), ethical approval is rendered by a Research Ethics Committee (CEP) or by the National Research Etichs Comission(CONEP), depending on its sensitivity.

The Legal Framework for Clinical Research (Statute No. 14,874/2024), enacted in May 2024, was conceived in response to the need to confer greater legal certainty on the system governing clinical research involving human subjects in Brazil. Previously, the subject was governed by infra-legal norms, and the ethical review process was regarded as inefficient and slow.

On the one hand, the Legal Framework introduced a series of material advances. Under the new statute, the ethical approval process for conducting a study is subject to a defined timeline that ranges from 30 to 140 business days. Regulatory approval by Anvisa has also been assigned new deadlines. With the adoption of Rule No. 945/2024, the prior 90-business-day timeline that applied only to the analysis of the Clinical Development Dossier for Medicinal Products (DDCM) now applies to the analysis of both the DDCM and the Clinical Trial-Specific Dossier (DEEC). If Anvisa does not issue a decision within the prescribed timeframe, the clinical study is deemed tacitly approved and may be initiated (provided that ethical approval has been granted).

Another notable advance at Anvisa is the incorporation of a reliance instrument for approving clinical studies, which now allows use of decisions issued by Foreign Trusted Regulatory Authorities (AREE) to streamline the review and approval of clinical studies.

Moreover, although the new regulatory framework has been welcomed by industry, the statutehas also drawn criticism. A major point of debate—criticized by CONEP itself (the National Research Ethics Commission)—was the rule allowing sponsors to cease supplying the investigational product to a volunteer five years after its commercial availability. CONEP’s position is that supply should continue for the duration of the volunteer’s treatment, without a fixed time limit. The provision was initially vetoed by President Lula on the grounds that it contravened the public interest and the principles of dignity, beneficence, and justice. Congress, however, overrode the veto and reinstated the five-year period on the grounds that an indefinite supply obligation would undermine sponsors’ economic predictability and could discourage investment in clinical research in Brazil.

Next Steps for Brazil: Implementing Regulations and Measures Already Underway

Despite notable advances, the sector still awaits implementing regulations for approximately 30 provisions of the New Legal Framework. Delays in regulation risk squandering momentum while generating legal and operational uncertainty.

Among the provisions that still require regulation are:

(i) Establishment of the National Research Ethics Body (INAEP): a technical collegiate body that will establish guidelines for the field;

(ii) Classification of Study Risk: this determination will dictate whether the protocol must be reviewed by an accredited Research Ethics Committee (CEP) or may be reviewed by a credentialed (non-accredited) CEP only. The processing pathway varies with complexity, making this definition important for procedural agility;

(iii) Accreditation of CEPs: it is necessary to rapidly expand and adapt the number of accredited committees in light of the New Legal Framework to ensure greater agility in processing.

In addition to implementing regulations, the sector awaits the update to the Clinical Research Action Plan. In August and September 2025, the Ministry of Health conducted Public Consultation No. 69/2025 to collect input for the Plan’s update. The focus is on identifying bottlenecks (bureaucracy, regional disparities, scarcity of financing) and opportunities (digitalization, regulatory harmonization), thereby strengthening the legitimacy of policies and their alignment with the sector’s actual needs.

Next Steps for Brazil: Intellectual Property Protection, Transparency and AI.

A central dilemma in clinical research that requires greater attention in Brazil is balancing transparency regarding studies with the protection of intellectual property (IP), which is essential to preserving innovation incentives.

In the United States, the Final Rule (42 C.F.R. Part 11) mandates registration/disclosure on ClinicalTrials.gov, with fines for non-compliance. In the European Union and the United Kingdom, similar policies require publication of protocol synopses and final reports while safeguarding sensitive data.

In Brazil, notwithstanding the advances under the Legal Framework and Anvisa’s rules, robust mechanisms ensuring transparency regarding clinical studies remain lacking. At present, Anvisa’s search tools and the Plataforma Brasil contain errors and make it difficult, for example, to readily identify the molecules under study. It is therefore advisable to improve such tools, facilitating public access to essential elements (molecule under study, objective, design, eligibility criteria, general endpoints) while preserving under confidentiality strategic IP-sensitive information.

To reach the level of leading countries, Brazil must go beyond regulation and support strategies grounded in the integration of new technologies, such as the use of Artificial Intelligence (AI) in Real-World Evidence (RWE).

The parallel capabilities of AI and RWE can constitute an imperative for modernizing Brazil’s clinical research ecosystem. Among the principal benefits, the use of AI and RWE advances the development of medicines for under-represented and underserved populations, such as women of childbearing potential and individuals who are vulnerable or have rare diseases. For rare diseases, where the small number of patients precludes traditional randomized trials, RWE-augmented clinical development is particularly relevant.

Machine Learning (ML) has the potential to enable predictive modeling of treatment effects from Real-World Data (RWD). That is, combining AI with RWD can not only assist in clinical study planning but also assess - or even predict - the effect a drug may have on a given patient group. For example, in the case of medicinal effects in women who are pregnant, it takes approximately 10 to 15 years after marketing approval for reliable information for this population to be included in product information documents. As disclosed at DIA Europe 2025Conference, this period can be reduced to five years.

Conclusion

The private sector already exhibits palpable optimism. The growth in multinational investment, new strategic partnerships (such as Rede D’Or and NEXT Oncology) ), the arrival of Contract Research Organizations (CROs),, and a significant increase in job openings in the sector all indicate that the international market is focusing on Brazil.

Although full implementation of the regulatory framework—especially defining study risk classification and achieving timelines comparable to international models—remains an urgent imperative, the current trajectory is one of progress. Brazil is positioning itself to maximize its advantage in cost-effectiveness and in population and epidemiological diversity, supported by a new legal framework and government initiatives.

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