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In this second edition of the Freyr 3D Series | Orphan Drugs in LATAM, we analyze the regulatory framework of Brazil. In the first entry, we explored the case of Mexico, and in the next one, we will address Colombia, offering an objective and complementary view of these countries in the region.
Regulatory Framework
In Brazil, the recognition of orphan drugs is primarily based on:
- Law No. 12.401/2011 which regulates the incorporation of health technologies into the Unified Health System (SUS).
- ANVISA Resolution RDC No. 205/2017, which establishes criteria for the priority review of drugs intended for rare and serious diseases.
- The role of CONITEC (National Committee for the Incorporation of Technologies into SUS), which evaluates and recommends the inclusion of new technologies in the public system.
This legal framework reflects an effort to integrate rare diseases into the SUS, ensuring that treatments are evaluated not only from a regulatory perspective but also in terms of coverage and public health financing.
The Brazilian regulatory framework is more structured than Mexico’s in terms of formal processes, but less agile. While it ensures the incorporation of new therapies into the public system, decisions are often constrained by budget limitations and lengthy evaluation procedures. This means that, although there is clarity in regulatory steps, access speed may be compromised. Unlike Colombia, which has a more robust and earlier legal framework, Brazil relies on the logic of the SUS, which reinforces public coverage but adds significant sustainability pressures.
Incentives and Benefits
- Priority review: designed to accelerate the assessment of medicines for high-impact rare diseases.
- Fee waivers or reductions: partially reduce financial burden in certain cases.
- International cooperation (IPRP, PANDRH): promotes regulatory convergence and allows leveraging global experiences.
- Early dialogue with ANVISA: offers companies an opportunity to reduce regulatory uncertainty before formal submission.
Despite these incentives, ANVISA’s requirements for orphan drugs are highly rigorous. The main challenge for companies is not only to present a complete technical dossier, but also to navigate lengthy review timelines, demonstrate economic viability, and align technical requirements with the SUS coverage logic. These factors make the process more complex and can delay the availability of treatments, even when robust international clinical evidence already exists.
Technical Requirements
- Submission of a complete dossier in CTD/NeeS format with quality, safety, and efficacy data.
- Inclusion of clinical and preclinical evidence, even from studies with small populations.
- Compliance with Brazilian pharmacovigilance standards, including a Risk Management Plan adapted to the local context.
- Adjustment of labeling and information materials according to ANVISA requirements.
Challenges
- High costs for small patient populations put pressure on the economic viability of therapies.
- Limited clinical evidence, which undermines the robustness of regulatory dossiers.
- Accessibility and reimbursement challenges, with significant pressures due to high prices, affecting both patients and health systems.
Role of a Strategic Partner
Success in obtaining regulatory approval in Brazil and ensuring timely access of orphan drugs to the market depends on anticipatory regulatory preparation: harmonizing dossiers, conducting gap analyses, and developing robust pharmacovigilance plans in Brazil. On this path, Freyr LATAM brings together global expertise and local presence to support companies closely and effectively.
In summary, Brazil combines regulatory clarity and a focus on public health, but faces strong financial restrictions. For the industry, the main challenge is not only achieving regulatory approval but ensuring that incorporation into the SUS is viable and sustainable over time.
Let’s discuss how to anticipate your next steps in the largest pharmaceutical market in the region. Discover with Freyr new ways to make access to orphan drugs in Brazil viable and sustainable.
