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3 min read
In this three-part series, we explore how Mexico, Brazil, and Colombia have built their own regulatory frameworks for orphan drugs, each with particular features that, together, provide a representative overview of some Latin American countries.
The first entry of the Freyr 3D Series on orphan drugs in LATAM examines Mexico’s regulatory framework. Upcoming articles will address Brazil and Colombia, providing an integrated perspective of key countries in the region.
In Mexico, orphan drug regulation is inspired by international standards, particularly those of the FDA and the EMA, and is consolidated through COFEPRIS rare diseases as the health authority. A drug is classified as an orphan when it affects fewer than 5 individuals per 10,000 inhabitants, according to the criteria established for sanitary registration in Mexico
About Rare Diseases and Orphan Drugs
Rare diseases, although infrequent, represent a major challenge for healthcare systems and a unique opportunity for the pharmaceutical industry. Orphan drugs in Mexico —those intended to treat conditions affecting very small populations— are a bridge between innovation, social impact, and sustainability.
Regulatory Framework
- Formal recognition of orphan drugs in Mexico through the General Health Law (Articles 224 Bis and 224 Bis 1, reformed in 2012), which incorporated this figure into Mexican legislation for the first time.
- COFEPRIS’s rare diseases guidelines, which operationalize these articles through technical criteria: prevalence definition (fewer than 5 cases per 10,000 inhabitants), dossier requirements, and Mexico’s pharmacovigilance provisions.
- References to international standards, such as the FDA and EMA, allow for harmonization and FDA EMA reliance on external certifications.
The Mexican regulatory framework is relatively young and less developed than that of other countries in the region. However, it is clearly aligned with international benchmarks such as the FDA and the EMA. This alignment grants legitimacy and enables Mexico to join the global movement toward differentiated access. Still, the country lacks solid financial mechanisms to ensure sustainable long-term access.
Moreover, formal recognition does not always translate into effective availability for patients, as decisions regarding pricing, reimbursement, and coverage remain limited and subject to negotiation. Consequently, while the legal structure opens the door to innovation, actual access depends on economic, political, and funding factors that remain unresolved challenges.
Incentives and Benefits
- Differentiated evaluation processes that allow reduced documentation requirements in certain cases.
- pathways for critical needs that help shorten COFEPRIS response times for priority therapies.
- on foreign certifications (CPP, GMP), which facilitates acceptance of international evidence to streamline dossiers.
Recently, COFEPRIS reported on its website the recognition of nearly one hundred orphan drugs, reflecting the growing interest of the industry.
Technical Requirements
- Submission of a dossier in CTD format, with information on quality, safety, and efficacy.
- Inclusion of sufficient clinical and preclinical evidence, adapted to the target population.
- Establishment of a pharmacovigilance unit in Mexico, essential for post-marketing monitoring.
Although there are clear incentives, the technical requirements of COFEPRIS for rare diseases are rigorous. The real challenge for companies lies in translating international evidence into the local context, which requires investment in certified translations, formal adaptations, and harmonized documentation processes for sanitary registration in Mexico.
Challenges
- High costs for small populations put pressure on the economic viability of therapies.
- Limited clinical evidence makes it difficult to build strong regulatory dossiers.
- Accessibility and reimbursement challenges, with significant pressures due to high prices that impact both patients and healthcare systems.
Role of a Strategic Partner
In Mexico, where regulation prioritizes speed of evaluation and international reliance, the challenge for companies is not only to prepare a solid dossier but also to adapt global evidence to the Mexican regulatory framework and meet the requirements of COFEPRIS rare diseases.
Freyr LATAM supports organizations in turning that speed into tangible results: harmonizing dossiers, ensuring the quality of certified translations, and anticipating local requirements so that access becomes faster, safer, and more sustainable.
Explore how your company can transform regulatory complexity into a clear path toward access in Mexico. Let’s discuss how to anticipate your strategy together.
