On October 1, 2025, the Federal Commission for the Protection against Sanitary Risks (COFEPRIS) published the Guide for structuring the file in accordance with the Common Technical Document (CTD) for submitting applications for recognition and modification of orphan drugs.

This document seeks to standardize and make transparent the regulatory processes related to these drugs, following international best practices.

Orphan drugs are those intended to prevent, diagnose, or treat rare diseases, defined as those that affect no more than five people per 10,000 inhabitants. The low prevalence of these pathologies means that the availability of treatments for those who suffer from them is limited, which necessitates clear regulations that facilitate access.

WHOM DOES IT IMPACT?

The direct impact is on pharmaceutical companies, health registration holders, patients with rare diseases, healthcare professionals, and medical institutions.

CONCLUSION:

The COFEPRIS initiative represents a significant advance in the modernization of Mexican healthcare regulation. By aligning procedures with international standards, it strengthens trust in the regulatory system and promotes equitable access to innovative medicines for patients with rare diseases.