Orphan Drugs & Rare Diseases
An intro to the legal situation for orphan drugs and 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 GBP 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 dd 21.11.2011 No. 323-FZ “On fundamental healthcare principles in the Russian Federation” (the “Law on fundamental healthcare principles”). Rare (orphan) diseases are diseases that have a prevalence of no more than 10 cases per 100 thousand populations. A list of rare (orphan) diseases is maintained by the Russian Ministry of Healthcare based on statistical data and is placed on its official website on the Internet.
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 Art. 4 of the Federal Law dd 12.04.2010 No. 61-FZ “On drugs circulation” (the “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 is the same as the regulatory framework for authorization of drugs in general.
Russia, being the member state of the Eurasian Economic Union (“EAEU”), complies with the EAEU provisions regarding the formation of the Common Market of medicines and medical devices under Art. 30 and Art. 31 of the Eurasian Economic Union Treaty dd 29.05.2014. Starting from 2021, authorization (i.e. registration) of new medical drugs in Russia is performed under the regulations of the EAEU that include the Rules of registration and examination of medical drugs approved by the decision of the Council of the Eurasian Economic Commission dd 03.11.2016 No. 78 (the “EAEU Rules”) and other applicable regulations of the EAEU.
4. Does your country have pro- visions for relaxed clinical trial/ scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
Yes. According to the EAEU Rules, to orphan drugs, the main provisions of Part II (registration on conditions) may be applied. The applicant must justify in the preclinical and clinical summaries the reasons why it is not possible to provide complete information and provide a justification for the balance of benefits and risks for the orphan drugs in question.
Besides, the submission of samples, specific reagents, and other materials is not required if it is impossible to conduct tests in an expert organization due to the inaccessibility of samples of medicinal products (if they are classified as an orphan), the impossibility of observing the conditions for transporting these samples to the territory of the member state and (or) their storage, lack of special equipment and consumables in an expert organization.
5. Is there an expedited pathway for Orphan Drugs?
The EAEU Rules do not establish any specifics regarding time period of orphan drug registration.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Yes, but only those that are issued within the EAEU and recognized under the EAEU Rules.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
Reimbursement procedure for orphan drugs in Russian Federation implies a special procedure for providing citizens with orphan drugs and their purchasing to:
- Some orphan drugs are included in the List of high-cost nosologic diseases and thus are purchased at the expense of the federal budget pursuant to the Law on fundamental healthcare principles;
- Other orphan drugs are purchased at the expense of the relevant regional budget.
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.
Besides, special rules for purchasing drugs (including those that are not registered in Russia) for children with rare diseases are introduced starting from April 20, 2021. The need for drugs for a specific child (or group of children) will be determined by the expert council of the Fund for Support of Children with Severe Life-threatening and Chronic Diseases, including rare (orphan) diseases called “Krug Dobra”. These drugs will be purchased by the Russian Ministry of Healthcare or its subordinate institutions, as well as the Fund itself.
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 List of vital essential and necessary drugs (“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 based on 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. Definitions of monopolistically high and low prices are provided in Art. 6-7 of the Federal law dd 26.07.2006 No. 135-FZ “On protection of competition”. Besides, in its official Statement dd 10.02.2016 (“Statement”) FAS clarifies the definitions of monopolistically high and low prices as follows:
- Monopolistically low price of goods is the price that 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 high price of goods is the price that 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 the 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).
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 2020 the List of high cost nosologic diseases has been expanded and 2 additional diseases have been included in the list. 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.
Starting from 2021 Fund for Support of Children with Severe Life-threatening and Chronic Diseases, including rare (orphan) diseases called “Krug Dobra” is established. The fund’s founder is the Russian Ministry of Healthcare. The Fund is funded by deductions from the increased personal income tax rate (15%), which is applied for income over RUB 5 million per year. These funds go to the federal budget and then go to the fund in the form of subsidies. Krug Dobra is created to provide medical assistance to seriously ill children who need expensive treatment. This fund will participate in purchasing drugs (including those that are not registered in Russia) for children with rare (orphan) diseases, the rules for the purchasing are in force starting from April 20, 2021. The list of diseases and the list of drugs to be covered by these rules are expected to be approved soon.