CEE Expert Guide on the reimbursement of orphan drugs in Poland

Polish law does not provide a separate definition of orphan drugs. Instead, Polish law simply refers to Regulation 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products.

Yes, orphan drugs can be reimbursed in Poland. However, Polish law does not set out a separate procedure specifically for obtaining reimbursement for an orphan drug. This means that general reimbursement regulations apply to orphan drugs, mainly the Act of 12 May 2011 on the Reimbursement of Medicinal Products, Food for Special Nutritional Purposes and Medical Devices (“Reimbursement Act”) and the Act of 27 August 2004 on Health Care Services Financed from Public Funds.

Under Polish law, depending on the circumstances of a case there are generally two different paths to obtain reimbursement for a drug (including orphan): (i) standard application; and (ii) reimbursement for off-label characteristics. Below is an overview of each procedure. Please note that there are also other ways to obtain financing in individual cases, e.g. emergency access to drug technologies (Ratunkowy dostęp do technologii lekowych).

To obtain reimbursement for a product, a marketing authorisation holder (“MAH”) or its representative must file an application with the Ministry of Health (“MOH”). The application can only be filed electronically via the System for Reimbursement Lists (“SOLR”). The SOLR also serves as a means of communication between the applicant and MoH at a later stage of the proceedings.

The reimbursement application must contain the following elements and data:

• basic information regarding the applicant, e.g. name of the company, registered seat, e-mail address, name of the representative;
• basic information about the product to be reimbursed, e.g. name of the product, its GTIN number, its form, method of administration and type of packaging;
• proposed conditions of reimbursement including (i) indications of the product that would be subject to reimbursement; (ii) proposed official sale price; (iii) level of payment; (iv) reimbursement category, e.g. outpatient reimbursement for all or part of registered indications, used in chemotherapy, in a therapeutic programme or used for healthcare services rendered from public funds; or (v) risk-sharing schemes.

If the application relates to a drug that does not have a reimbursable equivalent in the given characterisitcs (as is usually in the case of orphan drugs), the applicant must attach to the application a clinical analysis, an economic analysis, and an analysis of the impact on the budget of the entity obliged to finance the services 

from public funds. Polish law provides for how such analyses must be prepared; the requirements are specified in the Regulation of the Minister of Health of 8 January 2021¹. 

The reimbursement application is reviewed by the MoH. Depending on the scope of the reimbursement application, the MoH is required to consult Agency for Health Technology Assessment and Tarification (AOTMiT). At the MoH’s request, the AOTMiT issues an analysis regarding the potential reimbursement of a product and the recommendation of whether the product should be reimbursed, and if so, under what conditions. This information (together with the reimbursement application) is then passed onto the Economic Commission for further review and negotiations.

The Economic Commission is responsible for negotiating specific conditions for reimbursement, including: (i) the official sale price; (ii) the level of payment; and (iii) risk sharing-schemes. The negotiations are confidential and are concluded by a resolution issued by the Economic Commission. The said resolution summarises the outcome of the negotiations and is presented to the MoH for further review.

Based on all the documents gathered in the reimbursement proceedings, the MoH decides to either grant the reimbursement or refuses to do so. To this end, the MoH considers a variety of factors including: (i) the assessment of the AOTMiT and recommendation of its President; (ii) the resolution of the Economic Commission; and (iii) the importance of the clinical conditions that the reimbursement application covers.

When necessary to save health and life, the MoH may issue a decision to reimburse a drug different from those registered (off-label). Before issuing a decision on reimbursement, the MoH must consult the Transparency Council (an advisory council of the AOTMiT) and a national consultant in the relevant field of medicine.

In making the decision, the MoH considers the following criteria:

• clinical and practical efficacy;
• safety of use;
• the relationship of health benefits to the risk of use;
• the impact on the expenditures of the entity obliged to finance benefits from public funds and beneficiaries;
• existence of an alternative medical technology;
• the amount of the threshold for the cost of obtaining an additional quality-adjusted life year,
• the ratio of costs to the effects obtained.

Reimbursement is only temporary. Reimbursement is granted in the form of a decision and as a rule should last up to three years. It is possible to reapply for reimbursement when the decision is due to expire.

Yes, as indicated in question No. 2, there is no specific procedure construed specifically for orphan drugs. Given this, Polish general drug reimbursement regulations would apply, as medicinal products could potentially be subject to reimbursement in Poland.

The list of reimbursed products is publicly available. The Minister's summary for 2021 indicated 29 drugs reimbursed for rare diseases and 37 in 2022. There are no statistics as to how many of the reimbursed products are in fact orphan drugs registered with the EMA. However, as of March 2022, there were 19 EMA-listed orphan drugs reimbursed in Poland.

As a rule, an application for drug reimbursement should be processed within 180 days. As mentioned in question No. 2, the review of a reimbursement application is a multi-step process and includes the evaluation conducted by the AOTMiT. The duration of this part should not exceed 60 days. Please note that the deadline is extended if the authorities request the applicant to provide additional information.

In practice, these deadlines are often missed, and the time application review may take up to two years (three years for orphan drugs).

On 24 August 2021, the Polish government adopted a Plan for Rare Diseases, a comprehensive model of care for patients with rare diseases. The plan aims to improve monitoring the incidence and treatment of rare diseases along with the visibility of the patient in the health care system, by means of the Rare Disease Patient Passport and the Polish Register of Rare Diseases.

The main goals of the Plan are to improve diagnostics and treatment of rare diseases in Poland in accordance with standards adopted in the European Union, but also to improve access to high-quality, innovative healthcare services and to expand knowledge about rare diseases.

The Plan for Rare Diseases contains 37 tasks to be implemented in 2021-2023. As of now, many solutions are waiting to be implemented and we expect and hope for improvements in the field of rare/orphan diseases in Poland.

¹Regulation of the Minister of Health of 8 January 2021 on the minimum requirements for the analyses included in the applications for inclusion in the reimbursement and determination of the official selling price, for inclusion in the reimbursement and determination of the official selling price of a medicinal technology of high clinical value and for an increase in the official selling price of a medicine, a foodstuff for special nutritional use or a medical device which does not have a reimbursed equivalent in a given indication.