Snapshot

The Orphan Drug Route pathway has been developed by the Agência Nacional de Vigilância Sanitária (ANVISA) of Brazil.

This pathway can be used where The main applicable rule for orphan drugs is ANVISA Resolution RDC No. 205/2017, which provides for a special procedure for approval of clinical trials, good manufacturing practices certification and registration of new drugs for rare diseases. The conditions for the application of this regulation are: (i) the drug is intended for the treatment, diagnosis or prevention of a rare disease; (ii) the drug is used in a serious and debilitating condition; and (iii) it proposes to change, in a clinically substantial way, the development of the disease or allows a disease’s remission. Definitions: 1. Rare disease: one that affects up to sixty-five people in every hundred thousand individuals, as defined by the National Policy for Integral Attention to People with Rare Diseases, based on official national data or, when non-existent, on data published in technical-scientific documentation. 2. New drug: a drug with an active pharmaceutical ingredient (API) unprecedented in the country for the specific rare disease. .

This pathway accelerates the regulatory review process.

It is an abridged review (a reliance pathway).

To submit the drug development dossier for rare diseases, the sponsor must request a pre-submission meeting to present the dossier to ANVISA, which will be held within 60 days of the request. After the meeting, the sponsor must then submit for evaluation the regular dossier, along with the specific documentation required per the resolution. The analysis will be made by ANVISA, and they will issue a demand or approval letter within 30 days upon submission. The analysis of the request for registration of a new drug will be made within 60 days after submission, with issuance of a demand or approval letter. In addition, it also encourages the marketing of registered medicines through the criteria of this resolution, establishing a period of up to 365 days to be marketed after approval of the registration.