Orphan Drugs and Rare Diseases
The legal framework for orphan drugs and rare diseases in the Slovakian Pharma. Prepared in association with PRK Partners, a leading global law firm, this is an extract from The Pharma Legal Handbook: Slovakia, available to purchase here for GBP 75.
1. What is the definition of Rare Diseases in your country?
The Slovak legislation does not have a specific legal definition for Rare Diseases. The Slovak Act on Medicinal Products rather refers to the Regulation (EC) No. 141/2000 with respect to rare diseases.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Yes, this designation exists in Slovakia. However, as described in our answer to question No. 1 above, the Slovak legislation refers to the Regulation (EC) No. 141/2000 with respect to the definition of rare diseases.
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?)
There is no specific regulatory framework for the authorization of Orphan Drugs in Slovakia. According to the Annex to the Regulation (EC) No. 726/2004, medicinal products that are designated as orphan medicinal products developed are authorized through the centralized procedure (the respective marketing authorization is issued by the European Commission and is valid for all EEA member states).
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
As stated above, there is no specific regulatory framework for the authorization of Orphan Drugs in Slovakia and related requirements and all orphan medicinal products are authorized through the centralized procedure.
No relaxed clinical trial or scientific evidence requirements in respect of Orphan Drugs are provided by the EU legislation. However, the EU offers other “incentives” to encourage the development of Orphan Drugs by way of, e.g. scientific advice, “protocol assistance” (a type of scientific advice relating to the criteria for authorization of an orphan drug), market exclusivity, possibility of administrative fees reduction (depending on the status of the sponsor and the type of service required).
5. Is there an expedited pathway for Orphan Drugs?
According to the European Medicines Agency, orphan medicinal products will not automatically qualify for accelerated assessment (150 days, instead of 210 days for the opinion of the Committee for Medicinal Products for Human Use). However, an accelerated evaluation might be initiated by the CHMP in exceptional cases when a medicinal product is intended to meet a major public health need.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
In general, foreign marketing authorizations are not valid in the Slovak Republic and authorization of local authorities is required, except for the authorization issued by the European Commission through the centralized procedure which is then valid for all EEA member states. Please also refer to Answer No. 6 to Chapter 3: Marketing, Manufacturing, Packaging & Labelling, Advertising.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
In Slovakia, there is no specific reimbursement regulation related to the orphan drugs. Reimbursement of orphan drugs is regulated by the same legislation as the Reimbursement of other medicinal products. Please refer to Answers Nos. 12 and 13 to Chapter 1: Regulatory, Pricing, and Reimbursement Overview. Only a part of the orphan drugs is currently fully reimbursable from the Slovak public health insurance system.
8. How are the prices of Orphan Drugs regulated?
In Slovakia, there is no specific pricing regulation related to the orphan drugs. Pricing of orphan drugs is regulated by the same legislation as the pricing of other medicinal products. Please refer to Answer No. 12 to Chapter 1: Regulatory, Pricing, and Reimbursement Overview.
9. In case of reference price based on a basket of countries, what countries are included?
For the calculation of the “European reference price of a medicinal product”, the average price comparison of three lowest prices in other (only) EU countries is used.
10. Have there been any significant legal/judicial developments in relation to Orphan Drugs in your country?
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?