Legal & Regulatory > Brazil > Orphan Drugs & Rare Diseases: Brazil

The key facts about orphan drugs & rare diseases in Brazil. Prepared in association with Trench, Rossi e Watanabe one of Brazil’s most prestigious law firms, this is an extract from The Pharma Legal Handbook: Brazil, which can be purchased for GBP 75, here.

1. What is the definition of Rare Diseases in your country?

According to Resolution RDC No. 205/2017 issued by the National Health of Surveillance Agency (“ANVISA”), “rare diseases” are defined as diseases with incidence of up to 65 cases for every 100,000 inhabitants.

2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)

No, there is no specific definition for “Orphan Drug” in Brazilian current rules. However, we understand that it corresponds with the definition of Rare Diseases.

3. What is the regulatory framework for the authorization of an Orphan Drug? (Is this regulatory framework based on Rare Disease status or can it alternatively be based on Orphan Drug foreign status?)

The main applicable rule 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.

4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?

Yes. Aimed at encouraging companies to request ANVISA’s approval for the conduction of clinical trials in Brazil with experimental drugs for rare diseases, and at making the registration of drugs for rare disease faster, Resolution RDC No. 205/2017 provides for different procedures and criteria when compared to other conventional procedures.

As an example, it allows the company to propose the designation of a drug to treat a rare disease, with ANVISA responsible for the validation stage. In addition, the interested company and ANVISA will meet prior to the submission of the registration application, in order to align with ANVISA about the product and related documents. Also, the rule provides alternatives for cases in which the company applying for registration has not yet completed the entire process of developing a new drug for rare disease.

Also, ANVISA created this year (2020) a new category of products called “advanced therapy medicinal products”, a special category of new drugs comprising the advanced cell therapy product, the tissue engineering product and the gene therapy product, through Resolution RDC No. 338/2020. According to the Resolution, the advanced therapy medicinal products intended for rare diseases will have its registration request analysed with priority.

5. Is there an expedited pathway for Orphan Drugs?

According to information disclosed by ANVISA, the registration pathway related to drugs intended for rare diseases is three times faster than the regular procedure, due to Resolution RDC No. 205/2017.

According to Resolution RDC No. 338/2020 the pathway for advanced therapy medicinal products intended for rare diseases is also expedite.

6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?

No. It is mandatory to obtain a registration for Brazil, to be granted by ANVISA.

7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan drugs?

Yes, the reimbursement is possible but there is no specific procedure. Note, however, that according to court precedents on Federal Law No. 8,080/1990, known as the Brazilian Organic Health Law, the payment or reimbursement of national or imported drugs that are not registered before ANVISA is prohibited.

8. How are the prices of Orphan Drugs regulated?

The prices of Orphan Drugs are regulated by Brazilian Drug Market Regulation Chamber (“CMED”). The authority sets limits on drug prices, adopts rules that stimulate competition in the industry, monitors marketing, and enforces penalties when its rules are trespassed.

The company must submit to CMED the request for price approval, after gaining marketing authorization from ANVISA. In order to determine the maximum product price, companies must submit economic data on the product and propose a suggested price. External reference pricing is used to define the price of new drugs.

9. In case of reference price based on a basket of countries, what countries are included?

The prices in Brazil cannot exceed the lowest price charged between nine different countries, which are the United States, New Zealand, Australia, Greece, Portugal, Italy, Spain, France, and Canada.

10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?

On May 2019, Brazilian Federal Supreme Court rendered a decision of general repercussion nature for defining that (i) the State cannot be obliged to provide experimental drugs; (ii) the absence of registration with ANVISA prevents, as a general rule, the supply of drug by judicial decision; (iii) it is possible, exceptionally, the judicial grant of an unregistered drug, in case of unreasonable delay of ANVISA in considering the request (deadline longer than provided for in Law No. 13.411 / 2016), when three requirements are met: (a) the existence of application for registration of the drug in Brazil (except in the case of orphan drugs intended for rare and ultra-rare diseases), (b) the registration of the drug with renowned regulatory agencies abroad, and (c) the lack of a therapeutic substitute registered in Brazil.

11. Are there proposals for reform or significant change to the regulation of Orphan Drugs? If yes, when are they likely to come into force?

As the Resolution RDC No. 205/2017 was recently issued by ANVISA, we do not expect any changes to it in the near future. Note, however, that certain Bills related to orphan drugs are ongoing, such as:

• Bill No. 2233/19: grants tax benefits for drugs intended to rare diseases. In addition, the Bill provides for a simplified registration process for orphan drugs;
• Bill No. 2036/19 creates the temporary special registration of drugs aimed at treating diseases without effective alternatives in the country.
• Bill No. 2.657/2015: provides for the registration and importation, by individuals, of orphan drugs, provides for different criteria for the evaluation and incorporation of orphan drugs and also specifies process for price comparison.
• Bill No. 1606/2011: aims at setting a new national policy on rare diseases.

As the processes are ongoing, we cannot estimate if and when such Bills will come into force.