President Maćkowiak: We are waiting for the new Plan for Rare Diseases

President Maćkowiak: We are waiting for the new Plan for Rare Diseases

The Rare Diseases Plan, adopted by resolution of the Council of Ministers in 2021, expired at the end of 2023. This means we are currently without a plan. We are waiting for the new plan and its implementation, says Stanisław Maćkowiak, president of the Federation of Polish Patients.

A lot has changed in recent years when it comes to rare diseases: above all, their awareness has increased significantly. There are the first centers treating patients, more and more drugs are reimbursed, although it is still only a drop, because most rare diseases have no treatment. The Federation of Polish Patients brings together many organizations helping people with rare diseases. What are patients most looking forward to today?

Stanisław Maćkowiak: From the patients' point of view, the most important thing is that we have the opportunity to obtain comprehensive and coordinated services. It is primarily about creating a Polish network of expert centers, belonging to the European network. Polish branches already meet the conditions for this, but in order to be able to say that they constitute a Polish network, they must be provided with appropriate financing.

Patients suffering from rare diseases require a special amount of work, diagnostic and therapeutic procedures. Therefore, more financial resources are necessary. Today, patients with rare diseases are often referred to reference centers, where their treatment is scarce and generates debt for these centers. Money is available for this purpose, there is money in the Medical Fund, it should be used. This year, in accordance with the Prime Minister's declaration, there is an additional PLN 3 billion, including: for the treatment of rare diseases.

There is also a need to expand the availability and reimbursement of many drugs for patients with rare diseases – so far, the criteria for drug reimbursement are the same as in the case of population diseases. Better access to diagnostics, including genetic diagnostics, is also necessary.

Many of these diseases have no treatment, but patients also need help.

In the Rare Disease Plan, which was valid until the end of 2023, we focused mainly on medical aspects. The Plan ended in December 2023, although after a conversation with the Ministry of Health there was a determination to adopt the new Plan for implementation after the update. However, it is important not only to prepare a plan, but also to implement the proposed changes.

As I mentioned, these are not only medical issues, so in order to provide patients and their caregivers with support, it is necessary to involve the Ministry of Family and Social Policy in the work. It is also worth including the Ministry of Science and Education in the discussion to educate about rare diseases. We should also focus on the education of doctors. Access to information is also very important in this matter – when creating the National Plan for Rare Diseases, an online platform called Rare Diseases was created on the website of the Ministry of Health, but it is awaiting supplements and updates with new information.

Comparing the situation with a dozen or even a few years ago, we can see huge positive changes. However, there is still a lot that needs to be changed, because Poland is no longer a poor country, patients with rare diseases in Poland are not “worse” than patients in Germany or France.

It's just a matter of how much of our GDP we will spend on health care and how we will organize it. I am glad that rare diseases have been recognized as one of the priorities of health policy. When it comes to rare diseases, in some respects we are at the vanguard of Europe: I am thinking here, for example, of the newborn screening system. There is nothing stopping us from being at the vanguard of Europe in other aspects of care for people with rare diseases.

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