Until recently, the diagnosis of a neurological disease was a suspended sentence. Recently, we have seen huge changes, we are glad that patients in Poland also have access to very effective medicines, experts said during a Wprost debate with Deputy Minister Maciej Milkowski during the Economic Forum. However, organizational and administrative changes are needed.
Neurology is no longer Cinderella, it is becoming a priority, and the treatment of many diseases such as multiple sclerosis, multiple sclerosis, epilepsy and migraine has completely changed in recent years. A real revolution has also taken place in the treatment of spinal muscular atrophy (SMA).
- if a person suffering from multiple sclerosis receives treatment in a timely manner, he will not have to use a wheelchair; will live and work normally. If a child with SMA is diagnosed during neonatal screening and treated, they may never develop symptoms of the disease. Every day I am happy to see that something I started has been completed: I have a personal joy that it has happened, but I also see a lot going on in neuroscience. This deserves to be a priority area in Polish healthcare, said prof. Marcin Czech, President of the Polish Pharmacoeconomic Society, Deputy Minister of Health in 2017-19
Also prof. Agnieszka Slovik, national consultant in the field of neurology, stressed that the treatment of neurological diseases in Poland is developing very dynamically and harmoniously. - The report “The State of Polish Neurology and Directions of its Development until 2030” prepared by the University of Lazarski and the Polish Neurological Society (the so-called White Book of Polish Neurology), systematized knowledge of the most common neurological diseases, set directions for further development, - emphasized prof. Nightingale.
- Neurology is of interest to all health systems, as the direct, indirect and social costs of neurological diseases are many times higher than the costs of oncology, diabetology and rheumatology combined. Neurology is becoming a priority in European politics and should become a priority in Polish health policy. The document establishes a roadmap that allows organizing activities, - said Dr. Malgorzata Galonzka-Sobotka, Director of the Institute of Health Management at Lazarski University.
Deputy Minister Maciej Milkowski, who was present at the debate, stressed that the Ministry of Health is mindful of neurological patients and tries to be very honest in all reimbursement decisions for new medicines.
MS: Leader in neuroscience but changes needed
prof. Konrad Rejdak, President of the Polish Neurological Society, highlighted that of the six most common neurological diseases mentioned in the Polish Scientific Society and Lazarski University report, multiple sclerosis is the frontrunner for change. “It was here that the changes in treatment that are the driving force behind other neurological diseases first appeared. However, the situation is changing, new solutions are emerging. Naturally, we must change the treatment and the method of treatment, - said prof. Reidak.
prof. Monika Adamczyk-Sowa, President of the Multiple Sclerosis and Neuroimmunology Section of the Polish Neurological Society, emphasized that the treatment of multiple sclerosis has changed significantly in recent years thanks to the registration of new drugs. Also in Poland, access to modern methods of treatment has been systematically improved. - New therapies were reimbursed very quickly, and we are very happy about this. However, the progress is huge, so more capabilities are needed. We also badly need administrative changes in terms of access to highly effective treatments: some of these drugs appeared in the drug program in 2013, and since then there have been no changes in access to them. We are asking the minister for these changes. We would like the drugs, which are currently second-line drugs, to become available to patients earlier, according to our knowledge and recommendations of specialists, - emphasized prof. Adamchik-Owl.
It deals, among other things, with the issue of softening the criteria for the transition of patients from program B.29 to program B.46. The Health Technology and Tariff Evaluation Agency also recommended a combination of first-line and second-line drug programs: the idea was that patients could benefit more quickly from effective treatment. - The combination of programs 1 and 2 is one of the most urgent problems of access to therapy in MS; is one of our most important postulates. We had many meetings with the minister; I hope that the new MS treatment program will take into account our postulates, - emphasized prof. Adamchik-Owl. She also emphasized that an important change for patients will also be the possibility of using the subcutaneous form of natalizumab. - This is a drug that we know very well, with high efficacy, it would be important to know how to use it, especially in chronically treated patients, in whom venous access may be more and more difficult. The subcutaneous form is also less costly to the healthcare system, said Prof. Adamchik-Owl.
Dr. Małgorzata Galonzka-Sobotka emphasized that SM is a success story, but new technologies and new solutions are emerging. “We still have a lot to do: we need to better organize the path from suspected disease to treatment in order to reduce time and start treatment as soon as possible,” said Dr. Galonzka-Sobotka. prof. Konrad Reidak stressed that the organizational changes postulated in the white paper on Polish neuroscience are also important. - We want to improve the system of neurological care, hence the postulate to create a clinic with a higher level of specialization. We would like the patient to go to the target clinic, where he can count on faster diagnosis, qualification for treatment and its management, he said.
SMA: comprehensive diagnosis and treatment
As of September 1, 2022, two additional drugs for patients with spinal muscular atrophy (SMA) were included in the reimbursement program: a gene therapy (zolgensma) and a drug administered in syrup (risdiplam) to the nusinersen drug program. - Important changes started earlier: since April of this year, we have been screening newborns for SMA throughout Poland. Thanks to this, there is a chance that the child will be covered very quickly by one of the currently reimbursed therapies, and this will bring the greatest efficiency, - said prof. Katarzyna Kotulska-Juzwiak, Head of the Department of Neurology and Epileptology, Memorial Institute of the Children’s Health Center.
Thanks to the introduction of screening tests, a child can be diagnosed with SMA as early as 11-12 years old. day of life, when there are no symptoms of the disease. - This creates a chance to stop the progression of the disease at a stage when it has not yet caused the development of disability, has not deprived of motor functions. A child who received treatment at such an early age has a chance for proper development, - emphasized prof. Kotulska-Yuzwiak.
Conducted by prof. The Kotulska-Jóźwiak study of a population of Polish patients treated with nusinersen showed the effectiveness of its administration: none of the patients stopped treatment due to treatment failure. - From September 1, we have all types of therapy in the panel of compensated drugs. In the case of children identified during screening, there is a chance to receive gene therapy once. This is a chance to live without symptoms of the disease. Perhaps due to this, a group of patients will appear in whom SMA will be written in the genes, but will not have symptoms of the disease and will not require chronic treatment,” said Prof. Kotulska-Yuzwiak.
prof. Marcin Cech, who made the decision to reimburse nusinersen when he was Deputy Minister of Health, stressed that he is pleased that SMA can now be effectively treated in Poland with all the drugs available in the world. - I realized that turning to innovative therapy is the first step to clarify the fate of patients suffering from this disease, their families and those who care for them. Then I realized that I had to take the next step: screening. He is already here, and now I’m glad that min. Milkovsky led to further reimbursement decisions. This is an undeniable success; we are one of the four countries in the world where the treatment of SMA is best organized, - said prof. Marcin Czech.
The revolution in treatment was also emphasized by Kacper Ruczynski, member of the Strategic Council of the SMA Foundation: - Next to me are the heroes of the Polish SMA community: Minister prof. Marcin Cech and Minister Maciej Milkowski: the people who made the most important decisions for patients: the reimbursement of all three drugs and the introduction of newborn screening. Thanks to this, we have one of the most complete diagnostic and therapeutic programs in the world,” said Kacper Ruczynski. - Previously, it was an inclined plane: the baby was healthy at birth, and then symptoms appeared. This has changed: today patients think what to do, how to develop. Neurology has made great strides, and the Ministry of Health has made great strides in making treatment affordable, said Kacper Ruczynski.
Decisions of the Ministry of Health: weighted
Deputy Minister Maciej Milkowski stressed that when making decisions on reimbursement of expenses, the ministry tries to be very honest in this matter, and also analyzes the situation over several years. - As for gene therapy, we have no evidence that it will be effective in 70-80 years. We will monitor the effectiveness of treatment for 4 years, this is a key period in a child’s life, we will publish this data. We analyzed in detail who this therapy is most useful for, said Deputy Minister Milkowski.
He emphasized that there is a lot going on in neurology: an example of a very effective treatment is mechanical thrombectomy after a stroke. - We have reimbursed the cost of new medicines for chronic migraine. Changes to MS regimens for September 1 have not yet been made, so this should be done by November 1. We are starting to work on closing the program completely by the end of September, which will be completely revolutionary,” Deputy Minister Milkowski assured.
Source: Wprost
