Orphan Drugs & Rare Diseases
Keen to learn more about orphan drugs & rare diseases in Argentina? Read on! Prepared in association with Marval, O’Farrell & Mairal, a leading global law firm, this is an extract from The Pharma Legal Handbook: Argentina, available to purchase here for GBP 119.
1. What is the definition of Rare Diseases in your country?
According to Law No. 26,689 on Rare Diseases, rare diseases are considered to be those whose prevalence in the population is equal to or less than one in two thousand (1:2000) people, in accordance to the national epidemiological situation. On February 12, 2021, the Ministry of Health issued Regulation No. 641/2021 which approved a list of rare diseases, according to the prevalence of such diseases in the country.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Yes. ANMAT Regulation No. 4,622/2012 states that Orphan Drugs are those intended for the prevention, diagnosis and/or treatment of rare diseases, for which there are no available, effective and safe treatments or that they are inadequate.
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 regulatory framework for the authorization of an Orphan Drug in Argentina is provided in ANMAT Regulation No. 4,622/2012. It establishes that medicinal specialties intended for the prevention, diagnosis and/or treatment of rare diseases may be registered and granted “under special conditions”. This regulatory framework is based on Rare Disease status, as defined by Law 26,689 and Annex I of ANMAT Regulation No. 4,622/2012.
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Pharmaceutical companies can request the registration of orphan drugs by submitting complete information from the early phases of investigation (preclinical phase, and phases I and/or II of pharmacological clinical trials), as stated in ANMAT Regulation No. 4,622/2012 and clinical trials regulations.
5. Is there an expedited pathway for Orphan Drugs?
There is an expedited pathway for registering orphan drugs that are already registered in one of the countries included in Annex I of Executive Decree No. 150/1992 (countries with high sanitary surveillance, such us: Denmark, Japan, the U.S.A., Israel, Germany, the U.K., Spain, Italy, and France) and that have completed the pharmacological clinical trials phase III. This procedure consists on registering the orphan drug by submitting the foreign marketing authorization, bioavailability data and a project of label, leaflet and prospect. The orphan drug will be registered under the same conditions of registration and commercialization authorized in the Annex I country.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Yes, but only for product registration and for compassionate use purposes. For registration purposes, the countries recognized are listed in Annexes I and II of Executive Decree No. 150/1992. For compassionate use, the only countries recognized are the ones listed in Annex I of Executive Decree No. 150/1992. Please see Question 5 above for more detail.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan drugs?
Orphan Drugs can be reimbursed by public and/or private healthcare insurance providers if such drugs are included in the Mandatory Medical Plan (PMO, after its acronym in Spanish). The Decree No 794/2015 (which regulates Law No. 26,689 on Rare Diseases) states that patients with rare diseases will have PMO treatment coverage. However, it does not determine the extent of the additional treatment coverage of medicines/treatment beyond the scope of the PMO. According to Law No. 26,689, there will be a special coverage for Orphan Drugs though it has not been implemented yet.
8. How are the prices of Orphan Drugs regulated?
There is no Orphan Drugs pricing regulation in Argentina so far. Since 2014, ANMAT has issued regulations aimed at compelling the holders of marketing authorization certificates to report the suggested price of sale of their products, in order to publish such reference prices on its website in the National Vademecum of Medicines.
Notwithstanding the lack of a more specific regulatory framework regarding pricing, be noted that there have been precedents where the MoH fixed prices for certain high-cost orphan drugs.
9. In case of reference price based on a basket of countries, what countries are included?
No reference prices are in place in Argentine regulations for Orphan Drugs.
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
There are vast legal/judicial developments in relation to orphan drugs in Argentina. Leading cases involve health claims filed by patients requesting treatment coverage against their public or private healthcare insurance providers to cover high cost drugs, including orphan drugs. The sanction of Law No. 26,689 on Rare Diseases and ANMAT Regulation No. 4,622/2012 has led to a more regulated scenario for orphan drugs in Argentina since 2011. In addition, there has been vast case law involving orphan drugs treatment coverage.
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?
- Creation of the Argentine Health Technology Assessment Agency (“AGNET”, after its acronym in Spanish). The Bill was filed in March 2022, so it should be filed again to be voted. The AGNET is a very sensitive issue for the local pharmaceutical industry, as it will ultimately be the body that determines through certain studies which medicines, medical devices and procedures, among other products, will be incorporated into the healthcare system’s benefits for their financing and/or coverage. The Bill states that the AGNET will be the consulting body in judicial proceedings where health issues are being discussed. Currently, reports are under the scope of the CONETEC.