The key facts about orphan drugs and rare diseases in Ecuadorian pharma. Prepared in association with Corral Rosales, a leading global law firm, this is an extract from The Pharma Legal Handbook: Ecuador, available to purchase here for GBP 75.

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

Our law states a specific definition for rare diseases which is the following:

They are the ones that are mainly considered life threatening or that debilitate in the long term, with low prevalence and high complexity that constitute a wide set of disease. That are characterized by being chronic and disabling. Their therapeutic resources are limited and expensive, some are in the experimental stage.

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

Orphan Drugs. – Those listed in the CNMB (NATIONAL TABLE OF BASIC MEDICINES) are manufactured by biotechnology or by complex pharmaceutical technology processes, are sold at high prices, generally have only one manufacturer worldwide, are not sold as generics, and its supply is difficult.

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?)

National Agency for Regulation, Control and Sanitary Surveillance (ARCSA) is the regulating agency that establishes the requirements to authorize orphan drugs. The regulations that establish and regulate orphan drugs are:

• Regulation of Sanitary Registration for Medicines in General
• Instructions for the Treatment of Rare and Catastrophic Diseases
• Technical Standard Import Medications, Specialized Treatments
• Substitute technical regulations to authorize import by exception and import by donation drugs, biological products, medical devices and biochemical and diagnostic reagents
• Replacement regulation to authorize the acquisition of medicines that are not included in the National Basic Medicines Chart – current CNMB.

There is a proceeding to authorize the import and commercialization of orphan drugs and recognize the authorizations in other countries; however, it is necessary to take into account so that in order for the regulatory authority to issue the corresponding permit it is necessary to evidence that the drug is necessary.

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

There are no provisions that establish requirements for clinical trials or scientific evidence for Orphan Drugs.

5. Is there an expedited pathway for Orphan Drugs?

The proceeding to import orphan drugs is quicker than that to import other drugs. The importation of the goods that have the authorization by exception (orphan drugs), must be made during the period of six (6) months following the issuance of the authorization from ARCSA (regulating entity).

This authorization will also be valid for partial shipments that due to their volume or nature require several shipments to complete the authorized import quantity, they will be covered by or referred to the same shipping document and in the same way must be imported within a period of six (6) months from the issuance of the authorization.

Once the authorization is granted, the applicant must notify ARCSA within a two (2) months term, the date and place of arrival or departure of the product, indicating its expiration date; batch or serial number (as applicable), invoice or other documents that the authority deems necessary, if not submitted with the initial application.

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

In the case of drugs for rare diseases or orphan drugs, our legislation requires that they have the respective health notification in force from the country from where originates from or from the country where it is sold.

In order for the drug to be recognized, it must be authorized and sold in at least one of the countries from the Pan-American Health Organization (PAHO) or the World Health Organization (WHO), since they are the Regional Authorities; or, they must have a health notification issued by the Authorities in: the United States, Canada, Australia, Japan, by the European Medicines Agency (EMA) and the Ministry of Food and Drug Safety of the Republic of South Korea. However, the recognition from one of these foreign authorizations is only one of the requirements to obtain the authorization to import the goods.

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

There are not procedures regarding reimbursement of orphan drugs.

8. How are the prices of Orphan Drugs regulated?

The price regulation is the same as that of a general medicine, once the health registration of the medicine has been obtained, the “regime confirmation” must proceed, which will be carried out in the Technical Secretariat for the Approval of Prices for Drugs, who will determine whether the price is regulated (established by the authority) or free (determined by the owner of the goods).

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

There is no reference based on other countries. The price parameter is fixed based on the analysis of the competent Authority.

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

No developments exist regarding these types of orphan drugs.

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?

There are many proposals regarding regulations of these types of drugs, especially for establishing timeframes to import them. However, there is no priority interest with respect to the authorities.