Orphan Drugs & Rare Diseases
Keen to learn more about orphan drugs & rare diseases in Panama? Read on! Prepared in association with AFRA, a leading global law firm, this is an extract from The Pharma Legal Handbook: Panama, available to purchase here for USD 99.
Orphan Drugs in Panama are regulated by Law 1 January 10, 2001, Executive Decree 40 of May 14, 2019 and Executive Decree 1217 of December 7, 2015; Rare Diseases are regulated by Law 28 of October 28, 2014 and Executive Decree 1217 of December 7, 2015.
1. What is the definition of Rare Diseases in your country?
Rare, less common and orphan diseases are defined by Law as those that are chronically debilitating or severe diseases that are life-threatening and endangering to the human life that may be genetically originated or for unknown causes, that require specialized and permanent therapy, with a dominance lower than 1 per every 2,000 individuals.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Yes, our legislation defines Orphan Drug as the drug or biologic product used for the treatment of rare conditions or diseases that affects only a small proportion of the population. It refers to potential active principles wherein there is no interest from the manufacturing laboratories for the commercial development of the drug, since it does not represent an economic incentive, even though it may satisfy health needs.
The following products are included in this definition:
- Drugs indicated for the diagnostic, treatment or prevention of rare conditions or diseases.
- Antidotes indicated for unusual intoxications.
- Active principles excluded from the market due to adverse effects but useful for other diseases.
- Active principles that do not have commercialization sponsors or that are no longer commercialized despite their therapeutically efficiency and being products that are withdrawn from the market due to low selling price, inefficient consumption or because they are not patentable since are natural products.
Therefore, in Panama the definition of Rare Diseases does not correspond to the definition of Orphan Drug, but they are related.
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?)
- Law No. 1 of January 10, 2001 which regulates Drugs and other Human Health related products.
- Executive Decree No. 40 of February 15, 2019 whereby the Law No. 1 of January 10, 2001 is regulated.
The regulatory framework is based on Rare Diseases status.
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
No. The clinical trial and scientific evidences for Orphan drugs are the same as for other drugs.
5. Is there an expedited pathway for Orphan Drugs?
The only regulated expedited pathway is if the Orphan Drug is under the process of authorization and if for humanitarian reasons is required, the Ministry of Health will approve the exception of the sanitary registration for importation purposes.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Foreign marketing authorizations are not recognized for authorization purposes.
However, part of the requirements for the obtention of the authorization certificate is to file a Notarial Declaration wherein the manufacturer of the Orphan Drugs must include the countries where the Orphan Drug is registered and the year of registration or, in absence of this information, a certification issued by Foreign Sanitary Authorities from the origin or manufacturing country.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan drugs?
To the best of our knowledge to date there is no specific regulation for the reimbursement of Orphan Drugs.
8. How are the prices of Orphan Drugs regulated?
Prices of Orphan Drugs are not specifically regulated. However, it is established that due to the nature of the Orphan Drug, the commercialization can not generate high dividends for the company.
9. In case of reference price based on a basket of countries, what countries are included?
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
No to the best of our knowledge.
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?
No to the best of our knowledge.