Polish government approves Rare Diseases Plan for 2024-2025

Warsaw’s Rare Diseases Plan for 2024-2025, recently approved by the Council of Ministers, envisions comprehensive and coordinated healthcare services, with approximately €22 million allocated to achieve its implementation.

The previous strategy for rare diseases expired at the end of last year.

“This is very good news; otherwise, we would have wasted the tremendous effort put in by experts over the past few years to create the previous document, which was never truly implemented,” Michał Byliniak, General Director of INFARMA, told Euractiv.

“Now we are all waiting for tangible results that both patients and the clinicians treating these diseases, along with their institutions, can see and feel,” he added.

Rare Diseases priorities

Rare diseases encompass a wide range of diverse, chronic conditions, united by their low prevalence—affecting fewer than five in 10,000 people. There are currently around 8,000 known rare diseases, and diagnosis can often take years.

All of these conditions are serious, chronic, and frequently life-threatening. Globally, approximately 350 million people live with a rare disease, with around 30 million in the European Union and 2-3 million in Poland.

Work on a plan for rare diseases has been ongoing in Poland for years, meaning current actions will largely build on existing initiatives. The document outlines several key initiatives aimed at improving the management and care of rare diseases.

One of the primary proposals is the establishment of additional Rare Disease Expert Centres, which will play a critical role in providing specialised healthcare services, mostly for patients with rare conditions.

Currently, only 23 such centres are operational in Poland and are part of the European network. Including their branches, there are a total of 44 locations conducting expert evaluations for rare diseases. The plan aims for the new specialist centres to be established by 31 December 2025.

“We want these centres to be established closer to where patients live,” Urszula Demkow, Deputy Minister of Health, remarked during an August meeting of the Medical Raison d’etat.

The Deputy Minister noted, a significant help in establishing centres will be the new valuation of services. “If something is valued below cost, hospital directors are not particularly keen on setting up or supporting such centres. However, if the centre is at least not making a loss, one can expect more favourable support from the management,” she assessed.

Another key element of building the plan is a platform for rare diseases. “It is essentially ready. Now we just need to upload many documents that have already been prepared,” Demkow said.

She emphasised that the platform will primarily serve patients and their families. “They will be able to learn more about their or their loved one's condition—what the disease is, what the prognosis is, what the natural course of the disease may be, and what possible therapies are available, with links to international resources,” she explained.

Still much room for improvement

In addition, there will be significant efforts to enhance access to diagnostic and treatment tests for rare diseases.

By the end of 2024, analyses will be conducted to update the list of guaranteed healthcare services and establish new funding mechanisms for genetic testing, and there will be a review of funding for high-specialist non-genetic laboratory tests used in rare disease diagnostics and monitoring, with updates expected by March 2025.

A new Rare Diseases System will also be established to gather data for the Polish Rare Diseases Register and the Rare Disease Patient Card, which will be instrumental in monitoring medical care processes and improving patient safety for those with rare diseases.

Byliniak told Euractiv that INFARMA supports the actions outlined in the plan.

“This is a step in the right direction and necessary for patients who are often lost in the system,” he added.

Byliniak further noted that, according to INFARMA, there is a need to enhance access to early diagnostics. This includes expanding the range of screening tests for newborns and introducing funding for genetic diagnostics.

“We must not forget about improving access to orphan drugs, as there are still significant gaps in this area,” he said. While access to orphan drugs has improved in recent years, only 46 out of 147 therapies registered since 2015 are currently reimbursed. “Much remains to be done in this area as well,” he concluded.

[Edited by Vasiliki Angouridi, Brian Maguire | Euractiv’s Advocacy Lab]