Orphan Drugs & Rare Diseases
An insight into orphan drugs & rare diseases in Russia. Prepared in association with Lidings, a leading global law firm, this is an extract from The Pharma Legal Handbook: Russia, available to purchase here for USD 99.
1. What is the definition of Rare Diseases in your country?
The definition of rare diseases is stipulated by art. 44 of the Federal law of 21.11.2011 No. 323-FZ “On fundamental healthcare principles in the Russian Federation” (“Law on fundamental healthcare principles”). Rare (orphan) diseases are diseases that have a prevalence of no more than 10 cases per 100 thousand people. A list of rare (orphan) diseases is maintained by the Russian Ministry of Healthcare on the basis of statistical data and is placed on its official website.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Orphan drugs are regarded as drugs intended solely for diagnosis or pathogenic treatment (treatment aimed at the mechanism of development of the disease) of rare (orphan) diseases (clause 6.1 of the Federal Law of 12.04.2010 No. 61-FZ “On drugs circulation” (“Law on drug circulation”)).
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 authorization of orphan drugs includes Law on fundamental healthcare principles, Law on drug circulation, which provisions are supplemented with the regulations adopted by the Government of the Russian Federation and Russian Ministry of Healthcare (including, inter alia, “Administrative regulations of the Russian Ministry of Healthcare on the provision of state services for the state registration of medical drugs” approved by the Order of the Russian Ministry of Healthcare of 21.09.2016 No. 725n).
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Yes. Pursuant to clause 3.1 art. 26 of the Law on drug circulation with respect to orphan drugs it is possible to provide the results of clinical studies of orphan drugs performed outside of the Russian Federation but in accordance with standards similar to Russian good laboratory practice and good clinical practice (whereas for drugs in general it is specifically required to perform clinical studies in Russia).
5. Is there an expedited pathway for Orphan Drugs?
The accelerated procedure of examination (examination of documents in the registration dossiers, quality control and expertise of benefit-risk balance) for the purpose of state registration of orphan drug is carried out by the decision of the Russian Ministry of Healthcare pursuant to the applicant’s application within a period not exceeding 80 business days (whereas in general examination takes 110 business days).
In addition, it is possible to provide the results of clinical studies of orphan drugs performed outside of the Russian Federation but in accordance with standards similar to Russian good laboratory practice and good clinical practice.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
No, foreign marketing authorizations are not recognized in Russia. For orphan drugs there are no exceptions.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
There are two possible options for orphan drugs reimbursement:
- Some orphan drugs are included in the List of vital essential and necessary drugs (“EDL”). Reimbursement of the cost of EDL orphan drugs is carried out at the expense of the federal budget;
- Other orphan drugs are included in the List of high-cost nosologic diseases and thus are reimbursed at the expense of the relevant regional budget pursuant to the Law on fundamental healthcare principles.
In order to effectively provide citizens suffering from rare (orphan) diseases with drugs and specialized medical food products, the federal register of persons suffering from rare (orphan) diseases is maintained.
8. How are the prices of Orphan Drugs regulated?
State registration of drug prices is executed for those orphan drugs that are included in the EDL. For EDL drugs maximum sale prices must be submitted by the manufacturers or their authorized representatives and must be subject to state registration with the Russian Ministry of Healthcare.
Purchasing prices in the public procurement sector must not exceed the price calculated on the basis of such a registered price considering the relevant maximum wholesale mark-up.
In addition, the Russian Federal Antimonopoly Service (“FAS”) supervises the Russian market in general regarding monopolistically high or low prices establishment by manufacturers (including drug manufacturers) which is a violation of Russian antimonopoly laws. In its official Statement of 10.02.2016 (“Statement”) FAS clarified definitions of monopolistically high and low prices:
- Monopolistically low price of goods is the price, which is (1) below the amount necessary for the production and sale of such goods, expenses and profits; and (2) lower the price formed in competitive conditions at the comparable commodity market (if such market exists in Russia or abroad).
- Monopolistically drug high price of goods is the price, which is (1) above the amount necessary for the production and sale of such goods, expenses and profits; and (2) above the price formed in competitive conditions at the commodity market comparable in composition of buyers or sellers of goods, conditions of goods circulation, conditions of access to the commodity market, state regulation, including taxation and customs and tariff regulation (if such market exists in Russia or abroad).
In order to prevent establishing monopolistically high or low prices FAS developed certain mechanisms so that to identify timely the fact of monopolistically high or low-price establishment and to take relevant prevention measures. Due to recent FAS practice, this approach is applied not only to EDL drugs but to all drugs in general.
Thus, for drug manufacturers it is recommended to establish an economically justified price that considers all pricing factors (i.e. purchase volume, forms and terms of payment, the presence of delays and arrears in payment, packing forms, etc.).
9. In case of reference price based on a basket of countries, what countries are included?
Proceeding from the FAS Statement comparable country is to be established based on the following criteria: this country is to be comparable in terms of the composition of drug buyers or sellers, the conditions of drugs circulation, the conditions of access to the market, state regulation, including taxation and customs tariff regulation. At the same time, such a country’s market should be in a state of competition.
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
Starting from 2019 the List of high-cost nosologic diseases has been expanded and 5 additional diseases have been included in the list. Some of these additional diseases are rare (orphan) diseases.
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?
It is proposed to purchase all orphan drugs at the expense of the federal budget only. However, this proposal currently is under public discussions and may not be supported at the legislative level.