New medicines in practice: what, for whom and when in Poland?

In this article, you will learn:

• Why, despite innovative therapies being authorised for marketing, their actual availability to patients in Poland is still sometimes limited
• What is the current status of the registration of selected new treatments in the US and Europe, and what are the reasons for the differences in the time it takes for them to become available?
• Why does the mere registration of a medicine in the European Union not in itself mean that it is actually available to patients in Poland?
• What are early access programmes, and in what circumstances can they enable the use of a treatment before it is authorised or reimbursed?
• When is it possible to use targeted importation, and what are the procedures for importing a medicine for a specific patient?
• in what situations can Emergency Access to Drug Technologies be used, and what role does it play before the treatment becomes eligible for standard reimbursement?
• What exceptional mechanisms could bridge the gap between the authorisation of an innovative therapy and its routine availability in Poland?

Abstract

In recent years, there has been a wave of groundbreaking therapies across various fields of medicine – from neurodegenerative diseases, through oncology, to rare diseases. Many of these medicines are first-in-class or the first effective treatments for previously incurable conditions. Despite these therapies being authorised for marketing in Poland, they often remain unavailable to patients due to a lack of reimbursement by the National Health Fund. However, extraordinary mechanisms may provide a solution to the lack of reimbursement: targeted import, Emergency Access to Drug Technologies (RDTL) and early access programmes.

Keywords

innovative therapies, early access programmes, Emergency Access to Medicinal Products (RDTL), targeted imports

Advances in medicine are entering a phase in which innovations are no longer isolated occurrences but are becoming a systemic phenomenon. The convergence of several trends – from routine genome sequencing, through protein and cell engineering, to gene editing and improved biomarkers – has radically shortened the path from discovery to therapy. At the same time, regulatory bodies (including the EMA and the FDA) have introduced accelerated assessment pathways and advisory tools, which facilitate the market entry of medicines with high clinical value. The common thread running through these therapies is the precise targeting of the disease mechanism, often confirmed by molecular criteria and measurable endpoints. Importantly, this shift is not merely theoretical – it is already translating into specific interventions that are available (or about to be introduced) in clinical practice.

In this context, recent years have seen a wave of groundbreaking therapies emerge across various fields of medicine – from neurodegenerative diseases, through oncology, to rare diseases (Table 1). Many of these medicines are first-in-class or the first effective treatments for previously incurable conditions. For example, donanemab is a monoclonal antibody that clears pathogenic β-amyloid, used to slow the progression of Alzheimer’s disease in patients at an early stage; lifileucel is the first therapy based on tumour-infiltrating lymphocytes (TILs) to be approved in oncology, exagamglogene autotemcel is the first clinically approved gene therapy utilising CRISPR/Cas9, whilst atidarsagene autotemcel is the first gene therapy for children with metachromatic leukodystrophy (MLD) that prevents neurodegeneration. In oncology, therapies targeting specific molecular abnormalities have emerged: zenocutuzumab – the first drug targeting tumours with an NRG1 gene fusion; inavolisib – a PI3Kα inhibitor used for PIK3CA mutations; and tarlatamab – a new immunotherapy (against DLL3) for refractory small-cell lung cancer. Meanwhile, in endocrinology, crinecerfont is the first drug in decades for congenital adrenal hyperplasia (a CRF1 receptor antagonist, enabling a reduction in steroid doses). The list also includes innovative RNA therapies (olezarsen – a new option for familial chylomicronemia, targeting lipid metabolism), new medicines for autoimmune diseases (nemolizumab – an IL-31 receptor-blocking antibody used to treat prurigo) and the long-awaited resmetirom – the first medicine for NASH with fibrosis.

In this article, we explain to what extent these medicines are already available in Europe and in Poland, and what mechanisms can ensure that patients have access to life-saving treatments.

Most of the therapies discussed were approved by the US Food and Drug Administration (FDA) in 2024. In the United States, many of them were granted accelerated approval or breakthrough therapy status due to the high level of unmet clinical needs and the groundbreaking nature of their mechanism of action.

Europe is also gradually approving these therapies, usually with a delay of several months or even a year following the FDA’s decision. By autumn 2025, the following, amongst others, had already been approved in the European Union: exagamglogene autotemcel, atidarsagene autotemcel, seladelpar, nemolizumab, inavolisib, sotatercept, olezarsen, vorasidenib, resmetirom and donanemab.

Some of the other therapies are still under review by the EMA (e.g. arimoclomol, tovorafenib, tarlatamab, nogapendekin alfa and inbakicept), whilst for others, no marketing authorisation application has yet been submitted. It is worth emphasising that obtaining EU authorisation is only the first step – actual access still requires national reimbursement decisions and price negotiations.

In Poland, in accordance with European Union pharmaceutical legislation, medicines authorised through the centralised procedure by the European Commission are automatically authorised for marketing within the territory of the Republic of Poland as well. This means that if a particular treatment has been granted a marketing authorisation by the European Medicines Agency and the European Commission, it may also be legally sold and used in our country. However, registration alone does not guarantee automatic availability to patients. A key condition for effective access to a treatment is its inclusion in the public funding scheme, i.e. reimbursement by the National Health Fund.

Without reimbursement, patients would have to cover the full cost of treatment themselves, which is practically impossible in the case of modern, innovative medicines – prices often run into hundreds of thousands of zlotys, and in the case of one-off gene therapies, even several million zlotys. At present, none of the newly registered innovative medicines has yet been included on the reimbursement list in Poland, even though some of them have already been authorised in the European Union. In many cases, manufacturers have not even submitted reimbursement applications. This is because Poland is rarely treated as a priority market by pharmaceutical companies – priority is usually given to countries such as Germany, France or the United Kingdom. Manufacturers employ a strategy known as ‘sequential market launch’ to minimise the risk of adverse effects from external price references, which naturally delays the submission of applications in Poland and thus postpones the prospect of reimbursement.

Data from the European Federation of Pharmaceutical Industries and Associations indicate that the average time taken to gain access to an innovative medicine in Europe is around 531 days. In Poland, this period is one of the longest in the entire European Union – on average, over 800 days from the moment of registration to actual access for the patient [1]. However, there are exceptions to this rule. In the case of sotatercept, used to treat pulmonary arterial hypertension, the reimbursement process is ongoing – the Transparency Council has issued a positive opinion on four proposals to include the medicine in the reimbursement scheme, but a final decision is still pending. This raises the real prospect of the treatment being included in the scheme in the near future, which would be of great significance for patients with this severe and rare condition.

To summarise, none of the innovative medicines discussed are currently available in Poland for routine clinical use under the reimbursement scheme. Whilst some of them are registered and authorised for marketing, the lack of public funding means that, in practice, they are out of reach for the vast majority of patients. Other medicines are still awaiting EU registration and, until then, cannot be used as standard. As a result, Polish patients for whom standard treatments have failed face a serious challenge: how to gain access to modern therapies before they become widely available under the reimbursement scheme. The answer to this question lies in early access schemes, which are discussed later in this article.

Although the medicines in question are not currently reimbursed, the Polish system provides mechanisms enabling patients to access treatment even before formal funding is granted or even before registration. These include: the early access programme (compassionate use), Emergency Access to Drug Technologies (RDTL) and targeted importation.

Compassionate use (early access programme) is the organised provision of a medicinal product to multiple patients by the manufacturer prior to the granting of a marketing authorisation and prior to reimbursement. It is used for patients with severe, life-threatening or chronically debilitating conditions, where available treatments have been exhausted and the patient is not eligible for a clinical trial. Following consultation with the regulatory authorities, the manufacturer launches a programme with clearly defined eligibility criteria and a list of centres; the medicine is usually provided free of charge, whilst the centre covers the costs of standard care and safety monitoring. This mechanism operates in the ‘window’ between the completion of pivotal trials and formal registration and reimbursement decisions.

The legal basis at EU level is Article 83 of Regulation (EC) No 726/2004 (referred to in the Polish version as ‘individual use’), which provides for CHMP/EMA opinions on the conditions of use and distribution, whilst implementation remains the responsibility of the Member States and the manufacturer. The EMA publishes selected opinions but does not maintain a comprehensive centralised register of active programmes – in practice, the most reliable source of up-to-date information is the manufacturer itself (the ‘Medical Information’ and ‘Early/Compassionate Access/EAP’ sections). It should be borne in mind that the launch of a programme depends on the company’s discretion, its scope is often geographically limited, and the medical criteria are strictly defined.

In the absence of an early access programme, targeted importation is an alternative for individual patients. This is an individual procedure enabling the import from abroad of a medicinal product not authorised for marketing in Poland and for which there is no equivalent available on the domestic market, provided that a doctor considers it essential for a specific patient and there are no effective therapeutic alternatives. The application is prepared by a doctor (with a medical justification) and submitted – together with the opinion of the relevant consultant (national or regional) – to the Minister of Health. Once authorisation has been granted, the medicine may be imported by an authorised pharmaceutical wholesaler and dispensed to the patient in accordance with the indications specified in the application. As a general rule, targeted import does not guarantee automatic reimbursement: the costs are borne by the patient or the hospital, unless the Minister of Health grants separate authorisation for funding. The procedure can be time-consuming and requires close cooperation between the doctor, the patient and the importer. It should be emphasised that the import depends on the consent of the manufacturer or the responsible entity, which may refuse to grant it without giving a reason.

If a medicine has already been authorised for marketing in the European Union or in Poland but has not yet been included on the reimbursement list, there is an alternative support mechanism for patients – Emergency Access to Drug Technologies (RDTL). This enables a hospital to obtain public funding for treatment for a specific patient whose life or health is at risk, where no effective treatment options are available. The procedure is centralised. The doctor submits an application together with a justification and the opinion of a national or regional consultant in the relevant field, and the final decision is taken by the Minister of Health. Once the decision has been issued, the National Health Fund may cover the costs of the treatment for a period of 3 months, with the possibility of extending funding if the treatment’s effectiveness is confirmed. In practice, the RDTL acts as a ‘bridge’ – it allows patients to gain access to therapies already registered in the EU but not yet reimbursed in Poland. This makes it possible to introduce innovative medicines earlier, before they are included in medicines programmes or standard reimbursement schemes. However, a significant limitation of the mechanism is the budget. High-cost therapies, such as one-off gene therapies worth several million zlotys, may be difficult to fund under the RDTL, as their cost could place a significant strain on available resources. Nevertheless, this procedure offers a realistic opportunity to treat patients in critical situations. In the past, the RDTL has often been used to fund modern cancer therapies for patients who had exhausted other reimbursed treatment options.

To summarise, Poland is at the end of the chain when it comes to access to new treatments – following approval in the US and the EU, patients in the country must wait for reimbursement. Currently, none of the treatments discussed are reimbursed in Poland, which creates what is known as an ‘innovation gap’. Other EU countries make these medicines available more quickly, often through special programmes or funds. In Poland, however, there are exceptional mechanisms in place: targeted import, Emergency Access to Drug Technologies (RDTL) and compassionate use. Although limited in scope, these have enabled patients to benefit from modern therapies.

1. European Federation of Pharmaceutical Industries and Associations (EFPIA). Patients’ W.A.I.T. Indicator Survey 2024. EFPIA; 2024.