Orphan Drugs and Rare Diseases
All legal aspects surrounding orphan drugs and rare diseases in the Polish pharma market. Prepared in association with DLA Piper, a leading law firm in Poland, this is an extract from The Pharma Legal Handbook: Poland, available to purchase here for GBP 75.
1. What is the definition of Rare Diseases in your country?
There is no legal definition of Rare Diseases in Poland. On official websites the government uses the definition recommended by the EU, i.e. a rare disease is one that affects not more than 5 out of 10,000 persons. Moreover, rare diseases are defined as diseases which occur very rarely, are most often genetically determined, have a chronic and often severe course, and about half of them manifest themselves in childhood.
2. Does the designation of ‘Orphan Drug’ exist in your country? (Does it correspond with the definition of Rare Diseases?)
Regulation (EC) no. 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (“Orphan Drug Regulation”) applies in Poland. Pursuant to the Orphan Drug Regulation, a medicinal product may be designated as an orphan medicinal product if its sponsor can establish:
- that it is intended for the diagnosis, prevention or treatment of a life-
threatening or chronically debilitating condition affecting not more than 5 in 10,000 persons in the Community when the application is made, or
that it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the Community and that without incentives it is unlikely that the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment;
- that there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Community or, if such method exists, that the medicinal product will be of significant benefit to those affected by that condition.
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 Orphan Drug Regulation establishes the Committee for Orphan Medicinal Products (“COMP”) within the European Medicines Agency (“EMA”), which is responsible for examining applications for orphan medicinal product designations.
Once an orphan medicinal product has been designated as such, it requires, like other medicinal products, a marketing authorisation before it is marketed. Marketing authorisation applications for designated orphan medicines must be submitted to the EMA for assessment through the centralised procedure, and the European Commission oversees final authorisation, taking into account the opinion of the Committee for Medicinal Products for Human Use (“CHMP”).
4. Does your country have provisions for relaxed clinical trial/scientific evidence requirements in respect of Orphan Drugs as compared to other drugs?
There are no specific provisions for relaxed clinical trial or scientific evidence requirements with respect to Orphan Drugs.
5. Is there an expedited pathway for Orphan Drugs?
In general, the maximum timeframe for the evaluation of marketing authorisations under the centralised procedure is 210 days. In exceptional cases, the CHMP may decide that the product is of major interest for public health and therapeutic innovation, and reduce the timeframe to 150 days, if the applicant provides sufficient justification for an accelerated assessment.
The CHMP may also grant a conditional marketing authorisation valid for one year if it finds that all of the following criteria are met:
- the benefit-risk balance of the medicine is positive;
- it is likely that the applicant will be able to provide comprehensive data post-authorisation;
- the medicine fulfils an unmet medical need;
- the benefit of the medicine’s immediate availability to patients is greater than the risk inherent in the fact that additional data are still required.
6. Are foreign marketing authorizations recognized in your jurisdiction for Orphan Drugs? If yes, marketing authorizations from which countries are recognized?
Poland, being a member state of the EU, recognises marketing authorisations granted by the European Commission under the centralised procedure.
7. Can Orphan Drugs be reimbursed? If so, is there a specific reimbursement procedure for Orphan Drugs?
There is no specific reimbursement procedure for Orphan Drugs. See Question 2 on Regulatory, Pricing and Reimbursement Overview.
In November 2020, the Act of 7 October on the Medical Fund (the “Act on the Medical Fund”) came into force. The Medical Fund set up by this law acts as a complementary element of the public system for financing healthcare. It will be funded from the state budget. One of the relevant sub-funds in terms of orphan drugs is the therapeutic-innovation sub-fund, which would finance emergency access to medicinal technologies and unlimited hospital and specialist services for children.
Under the Act on the Medical Fund, the Agency for Health Technology Assessment and Tarification (“AOTMiT”) prepares a list of drug technologies of high clinical value and a list of drug technologies of a high innovation level, including therapies for oncology and rare diseases.
8. How are the prices of Orphan Drugs regulated?
Prices of Orphan Drugs subject to the general regulatory framework for pricing of medicinal products. See Question 2, Regulatory, Pricing and Reimbursement Overview.
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, there have not been any significant legal/judicial developments in relation to Orphan Drugs.
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?
Pursuant to the Council Recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) (OJ C 151, 3.7.2009, pp. 7-10), member states should take national actions in the field of rare diseases. The Ministry of Health announced the National Plan for Rare Diseases in February 2021. It presents a three-year strategy, for the period 2021-2023, defining specific objectives that are to be achieved in that period.
It plans to ensure sustainable implementation of health policies aimed at the needs of patients with rare diseases, introducing systemic solutions to the health and social problems of those patients.
In February 2021, the Medical Research Agency, a governmental body, announced the launch of its PLN 100 million grant dedicated to research and development activities focused on rare diseases. The aim of this programme is to increase access to diagnostics and drug therapies in rare diseases.