Children with phenylketonuria (PKU) born in Poland are unlucky. If they were born in another country of the European Union, they would have wider access to treatment – in Poland, the possibilities are limited, especially in the light of recent changes. From September 1, 2020, the rules for reimbursement of the special nutritional preparation for children over 1 year of age Milupa PKU 2 mix have changed, which in most PKU patients with children covers 80% of the protein requirement and is necessary for proper development. Parents now have to pay over PLN 3,20 per packaging for the preparation, which has so far cost PLN 120.
The Transparency Council at the Agency for Health Technology Assessment and Tariffication (AOTMiT) decided that the preparations used in phenylketonuria are similar, can be used interchangeably and do not have to cost the same, i.e. PLN 3,20. This means that from January 1, 2021, adult patients with PKU will probably have to pay more for their preparations. Unfortunately, due to the lack of dialogue with the Ministry of Health on this matter, patients do not yet know what their situation is. What’s worse, the recently published draft list of reimbursed drugs deprives us of hope – since January, Milupa is still with a high subsidy.
One package is enough for about 7 days … Many parents simply cannot afford to pay extra a few hundred zlotys for the preparation a month. Diet is the basis for the correct treatment of phenylketonuria, because the intellectual development of a child depends on the proper supply of ingredients. What the body adopts today will affect its functioning in the future. Lack of access to appropriate nutritional preparations, and thus failure to follow the diet in PKU, inevitably leads to mental retardation in a child.
People with PKU do not have to be different from the rest of society, they can lead a normal, full life. The condition for this is access to the preparation and low-protein food. Do parents of children with phenylketonuria have an alternative? Yes, but only on paper, because the cheaper preparation, which has set the reimbursement limit, is practically unavailable, and at the same time unknown to doctors and parents. The Transparency Council in its opinion of October 26, 2020 emphasizes that since there are difficulties in accessing the preparation setting the limit, it is possible to repeal the administrative decision, because it is necessary to ensure continuity in access to the products covered by the reimbursement. Is it possible, then, to reverse this decision so that sick children have access to effective nutritional management?
The changes on the reimbursement list that hit the parents of sick children in such a way result from the creation of a limit group in which Milupa PKU 2 mix was found. The limit was set by a new product that is hardly available in pharmacies (only one in Poland). For comparison, in most European Union countries, subsequent preparations are entered on the reimbursement list at the same cost, so that the patient can choose the best preparation for himself. Moreover, patients with phenylketonuria pay attention to the plans of the Ministry of Health regarding limit groups for other products for phenylketonuria, which may aggravate the problem as other preparations will no longer be available to patients. The ministry wants to create a single limit group for all products for phenylketonuria.
– Creating a limit group for the Milupa preparation in September was not a good decision. It hit the sick and their families the most. The more disturbing are the premises for the Ministry’s plans to create limit groups for the remaining preparations. If it is undertaken, adult patients will also have to pay more for their preparations from January 1, 2021. This is very disadvantageous for us, the more that each product is different and should not be treated as substitutes. Unfortunately, no one consulted this decision with patient organizations, says Maria Słupska from the Gdańsk Society of Friends of Children with Phenylketonuria.
Changes in the payment for Milupa PKU 2 mix may lead to children stopping taking any other product. The medical community speaks with patients in one voice – depriving children of phenylketonuria from access to the preparation, which is the basis of treatment, will have a negative impact on their health as well as intellectual and psychomotor development. Phenylketonuria is an inherited metabolic disease in which the metabolism of phenylalanine – an amino acid whose excess can lead to many complications, mainly in the central nervous system is disturbed. Phenylalanine is part of the protein ingested in foods.
– Patients with phenylketonuria must follow a strict elimination diet that will limit the supply of phenylalanine-containing proteins. The mainstay of treatment is the administration of special phenylalanine-free mixtures, such as Milupa PKU 2 mix. There are no two identical preparations for patients with phenylketonuria. They differ in composition, as well as taste, smell, consistency, which is very important for sick children. The composition of the product, its quality and consistency in its use guarantee that young patients will develop properly. This is especially important in children whose neurological system develops in the first years of life – then the level of phenylalanine, as well as ensuring the proper level of micro and macro elements is crucial. Introducing changes in treatment can have a very negative impact on the development of children – it is unethical and inhumane – says Agnieszka Chrobot, MD, PhD from the Metabolic Diseases Clinic of the Provincial Children’s Hospital in Bydgoszcz.
In Poland, neonatal screening is financed from the state budget, which leads to the early detection of phenylketonuria. This only confirms that the Ministry of Health recognizes the importance of implementing appropriate treatment as soon as possible in the event of a diagnosis of PKU. Recent decisions contradict this logic and deprive patients of an opportunity for effective dietary management. Spending money on neonatal screening and then blocking access to modern nutritional treatment for people diagnosed with the disease through screening is a waste of that money.
What really, in a very practical dimension, happened in the lives of families with children with phenylketonuria, is illustrated by the story described by Ms Aneta from Krakow:
– I have two children on the preparation Milupa PKU 2 Mix, which from 01.09.2020/129,00/3,20. costs PLN 3 for the patient (previously PLN 4). One child is 12 years old and needs 7 packages of the preparation per month, and the other is 11 years old and uses 1 packages per month, so in total I buy 419,00 packages of the preparation each month, which in the current situation amounts to PLN 3 per month! Phenylketonuria is a lifelong disease in which you must strictly follow a diet, which involves the use of the preparation 2012 times a day for the rest of your life. Each change of it is associated with difficulties. The child refuses to drink the substitute for the preparation, cries, vomits – unfortunately, all this results in an immediate increase in the level of phenylalanine in the blood, which in the long term may cause mental retardation in the child. I am terrified of the current situation because I remember what happened in XNUMX, when we were denied reimbursement overnight for PKU. And now the same thing happened. I would like to add that the cost of the preparation is not the only expense we incur in connection with food for children with PKU. In addition, there are also low-protein foods, which are very expensive. In many countries in Europe, low-protein food is reimbursed. In our country, not only do we have to pay for it, but we also have to fight for the reimbursement of the basic preparation that enables our children to live a normal life. Therefore, I appeal to the Ministry of Health once again for a thorough analysis and a look at the problem related to the reimbursement of the preparation for patients with phenylketonuria in Poland. Do not deprive our children of the preparation that allows them to develop properly.
In turn, Ms. Monika from Ruda Śląska, mother of a 3.5-year-old boy, is very worried about where to get the funds to buy Milupa PKU 2 mix – We use 6 packages per month, which means that we have to pay PLN 779 per month for the preparation, not as before September 19,20 ,3.20 zł. I tried to switch my son to other preparations, but the little one does not accept them, spits them out, says that they are not good. This is a really huge problem for us, because ensuring a proper diet is essential, but where should we, parents, have the funds for it? Our children were used to the preparation for XNUMX, how can the Ministry of Health suddenly raise the price in such a drastic way?
Ms Anna from Lublin has two daughters aged 8 and 11 – From September I have to pay almost PLN 2 for Milupa PKU 1000 mix, previously I paid PLN 25, where should I get the funds? And yet we still have to buy low-protein foods, which are also very expensive. I am asking the Minister of Health to restore the state from before September. Diet is very important for the development of our children, and we really cannot pay a thousand zlotys a month for just one preparation.
Representatives of organizations working for patients with phenylketonuria also point to this problem, emphasizing that many parents will not bear the costs associated with the purchase of Milupa PKU 2 mix at the new price. This means that children will stop taking the product that is necessary for their proper functioning and development, because they will not want to take anything else. Discontinuation of treatment for a few weeks may seriously deteriorate your health and lead to irreversible changes that may not be visible immediately, but will certainly affect the proper development of children in the future.
– You cannot deprive patients of something that has been available for years and was the basis of their treatment. After all, these decisions will affect not only the health of patients, but also the indirect costs. A child who develops badly, due to the fact that he is not properly treated, will have to be looked after by a parent – and these are the costs related to absenteeism from work, costs of social assistance or benefits. Do we really have to condemn children to disability and lack of social acceptance? – adds Dr. Agnieszka Chrobot.
Patients and the medical community agree and have been appealing to the Ministry of Health for several months to restore the lump-sum payment for Milupa PKU 2 mix, unfortunately with no response from Miodowa. They also strive not to change the limit groups on the reimbursement lists, which will ensure access to the most appropriate preparations for all patients. Let all preparations be reimbursed at the same price, then doctors and patients will have the best choice. Let’s hope policymakers understand the concerns and plight of patients, and that Christmas will be a time of peace for the sick. There is still time to change the decision, so far the Ministry of Health has published a draft list. Is it on the final reimbursement list for January 2021? Will there be Milupa PKU 2 mix for 3,20?