I conducted a small survey by contacting MDS experts and people from the pharmaceutical industry, as well as by searching the available information online to understand the current situation in the field of MDS management in these countries.
The general and expected picture appears. The countries that are members of the European Union (EU) and have a stronger economy, like Czech Republic or Poland, have wide availability of all major drugs and are reimbursed by the national health system. In other EU countries, like Slovenia, only lenalidomide is not reimbursed for MDS. In Croatia, Epo as well as lenalidomide are not reimbursed. All these countries have very well-developed haematopoietic stem cell transplant programmes. Even in these EU countries, the reimbursement of MDS-specific drugs has been lagging compared to that in the Western countries.
Although I was unable to acquire exact data for all Eastern European countries, I could say that the situation in the field of MDS is not better; it is even worse in other Eastern European countries than in Balkan countries. For example, in Ukraine, not only are MDS drugs not reimbursed, but there is also no developed allogeneic transplant programme.
In these countries, in situations with restricted health funding, the drugs that are licenced for MDS (azacytidine, lenalidomide, as well as Epo, which has recently been licenced for MDS), are competing with other drugs or, it is better to say, MDS is competing with other diseases. It does not seem to be a fair competition. Breast, lung, and colon cancers; lymphoma; and multiple myeloma easily gain advantages over MDS. This is rather obvious when we consider the use of lenalidomide for MDS. In my country, lenalidomide is reimbursed for multiple myeloma, but not for MDS. A similar situation occurs in other countries. The general pattern is that when a drug is licenced for two indications, ultimately, it is reimbursed for the more frequent indication.
To summarise, the situation in Eastern Europe regarding MDS management with the exclusion of a majority of EU countries is not very favourable. There is a low level of reimbursement, as well as lower transplant activity.
What can be done to improve the situation regarding the management of patients with MDS in these countries?
I will begin with an example from my country. A patient who was 16 years old did not have MDS. He had a very rare disease called paroxysmal nocturnal haemoglobinuria, which in some way is associated with MDS, because some patients with MDS have small PNH clones. The drug for this disease is eculizumab, a monoclonal antibody which is very expensive, and it is not reimbursed in my country. The patient generated a video in which he bravely and with dignity described the disease and the necessity of obtaining the drug. He directly addressed his plea to our government. The video became viral and the result was that the patient achieved access to the drug. I do not think that this example could easily apply to MDS. This was a case of a very young patient with a very rare disease. Patients with MDS are usually elderly.
However, it clearly indicates that the voice of patients is very important. Patients with MDS should find a way to communicate to society, and they should be heard by important people from the government, health funding organisations, as well as medical societies.
Therefore, it seems that it is imperative in Eastern Europe that MDS patient groups should be founded and be proactive.
There is also a huge responsibility of haematologists and its societies in Eastern European countries. They should not always do as the pharmacological industry or government wants. They should speak for themselves and for their patients. MDS, as well other rare haematological diseases, are the cases wherein haematologists should firmly stand up and raise their voices.
In addition, I think the EU should proceed further in promoting the treatment of rare diseases. Since many Eastern countries are candidates for EU membership and, through the process of negotiations, integrate EU laws and recommendation, as well as EU values in their societies, any improvement in the EU regarding rare diseases could have an influence on these countries. If this is the case, we might not encounter the situation wherein lenalidomide is reimbursed for multiple myeloma and not for 5q- syndrome.
Any of the countries in Eastern Europe should be firmly oriented to develop successful transplant programmes. That is what is happening presently in my country and it should be encouraged by every government in Eastern Europe. This transplant treatment is not only of importance for patients with MDS, but also for patients with many other haematological diseases.
In conclusion, the management of MDS in Eastern European countries is faced with many obstacles and financial restrictions. Patients and doctors should be more active in raising public awareness of this rare disease. The final goals are to improve quality of life and survival of patients with MDS.
Acknowledgement: I would like to particularly thank my colleagues Njetocka Gredelj-Šimec, Jaroslav Cermák, and Krzysztof Madry for answering the survey, as well as other colleagues and people from the pharmaceutical industry who provided me information about the management of MDS in the Eastern Europe.