Orphan Drugs & Rare Diseases
An intro to the legal situation for orphan drugs and rare diseases in Portugal. Prepared in association with Cuatrecasas, a leading Iberian law firm, this is an extract from The Pharma Legal Handbook: Portugal, available to purchase here for GBP 99.
1. What is the definition of Rare Diseases in your country?
There is no legal definition for Rare Disease within the national legislation. However, within the European Union, diseases that affect less than five in ten thousand persons (5 in 10 000) are defined as Rare Diseases, sometimes called an orphan disease.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Yes. Decree-Law 176/2006 refers to Regulation (EC) no 141/2000 of the European Parliament and of the Council of 16 December 1999, whereby a medicinal product shall be designated as an orphan medicinal product if its sponsor can establish: (i) that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in ten thousand persons (5 in 10 000) in the Community when the application is made, or (ii) that it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the Community and that without incentives it is unlikely that the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment. (iii) Alternatively the sponsor can also establish that there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Community or, if such method exists, that the medicinal product will be of significant benefit to those affected by that condition.
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?)
Orphan Drugs are governed by the regulatory framework specified on Regulation (EC) no 141/2000 of the European Parliament and of the Council of 16 December 1999. Although this regulation is from the European Union it is directly applicable in Portugal.
4. Does your country have pro- visions for relaxed clinical trial/ scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Please refer to the previous answer.
5. Is there an expedited pathway for Orphan Drugs?
Under Regulation (EC) no 141/2000 Orphan Drugs Orphan Drugs are autho-rized by a centralized procedure with regards to their marketing authorizations evaluations.
The sponsor of an orphan medicinal product may also request protocol assistance to the European Agency for the Evaluation of Medicinal Products, such as advice from the Agency on the conduct of the various tests and trials necessary to demonstrate the quality, safety and efficacy of the medicinal product. There is also support with all the fees payable under the applicable legislation.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Yes. With the implementation of Regulation EC No. 726/2004, all applications for marketing authorizations for drugs with orphan designation are mandatorily assessed through a centralized process. This represents access to a single EU market, since the medicinal product will be recognized as an orphan drug within all member states of the EU.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
Yes, Prices of drugs are regulated by the System of Assessment of Health Technologies (SiNATS). SiNATS was created by Decree-Law 97/2015, 1 June 2015, which established the provisions applicable to pricing, reimbursement and prior evaluation procedures. There is no specific reimbursement procedure for orphan drugs.
8. How are the prices of Orphan Drugs regulated?
Please refer to the previous answer.
9. In case of reference price based on a basket of countries, what countries are included?
For a reference-based price the basket countries of reference consist of Spain, France, Italy and Slovenia (Ministerial Order 297/2020).
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
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?