Introduction:

The regulation of specific registrations for the so-called "orphan drugs" has been a strategy of the largest drug agencies in the world to promote access and monitoring of treatment for diseases and conditions of low prevalence or market interest.

Aims:

This study aimed to identify the profile of drugs that fall into this category internationally and explore possible gaps in registration generated by the absence of a specific health standard in Brazil.

Methods:

Orphan drug registration databases from countries of the European Union and the United States of America were analyzed and the results were compared with the database of the Brazilian Agency.

Results:

A total of 369 drugs registered as orphans in European and US agencies were identified, totaling 801 clinical indications. Most of the drugs registered internationally were antineoplastic agents and immunomodulators (N=135; 36.59 %) and drugs that acted on the digestive system and metabolism (N=48; 13.01 %). Of the orphan drugs registered and marketed internationally, almost half, 177 (47.97 %), did not have active registrations in Brazil and meet 327 clinical indications (40.82 %).

Conclusion:

Brazil must analyze in depth the impacts of the absence of an orphan drug registration flow, which can directly affect access to treatment for certain rare and neglected diseases.