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 USD 99.

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

Our law states a specific definition for rare diseases or orphan drugs 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 are the ones to treat rare diseases, orphan or catastrophic.

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

There is a proceeding to authorize the import and commercialization of orphan drugs and recognize the authorizations in other countries; however, it is neces- sary 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 120 days following the issuance of the authorization from ARCSA (regulating entity)

Once the authorization is granted, the applicant must notify ARCSA of the date when the goods would arrive at least 5 days in advance.

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 on the basis of 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.