Orphan Drugs
Trench Rossi e Watanabe / Brazil
Join industry executives in staying informed on the market access and HTA process in Brazil.
1. What is the definition of an orphan drug in your country?
There is no specific definition for orphan drug in Brazilian current rules. However, we understand that it corresponds with the definition of rare diseases.
As per ANVISA Resolution RDC 205/2017, rare diseases are defined as diseases with incidence of up to 65 cases for every 100,000 inhabitants.
2. Describe the pricing process of orphan drugs in your country.
The pricing of orphan drugs follows the rules foreseen in CMED Resolution 02/2004, as do other types of drugs, as explained in Chapter 4, Question 1.1.
Companies must submit to CMED the request for price approval, after receiving marketing authorization from ANVISA and submit economic data on the product and propose a suggested price. Then CMED we will define the maximum price of the drug, using ERP and/or IRP.
3. Describe the reimbursement process of orphan drugs in your country.
There is no specific reimbursement process for orphan drugs. They follow the procedure applicable for other drugs, as explained in Chapter 3, Question 3. Recently, Conitec issued a guideline for setting cost-effectiveness thresholds in health decisions. Scenarios were also discussed in which the threshold could have its values relaxed (including rare diseases, neglected endemic diseases, diseases in children, diseases affecting individuals at the end of life expectancy and high effectiveness in terms of QALY).
4. Other than the clinical studies required, do the data and studies required for the pricing and reimbursement of orphan drugs differ from that of other drugs?
No, there is no difference.
