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The family is not hiding: it is possible to reach an agreement with the disease, but the most difficult thing is to reach an agreement with the bureaucratic system of his country, despite the fact that he considers himself his patriots. The additional quality of a child’s life depends on the decisions of the officials, but for the latter, this child is just another statistical unit for which no money is provided in the budget. And period.
The disease affects all the muscles of the body.
As the boy’s father Rytis Rudzinskas said, until the son’s second year of life there was no suspicion that something was wrong. Parents were sure that the family would grow up to be a healthy child.
“His development was delayed a bit, later he started to turn around, walk, but everything was still normal. Only last summer my mother had the intuitive feeling that something is not right because the child falls very often , and we also noticed that it is not as agile as the other children. When we were concerned, we contacted the Child Development Center.
As the disease progresses, the child loses the ability to walk, breathe independently, and eventually experiences premature death.
Although doctors did not immediately suspect the disease, as it is usually diagnosed in old age, when symptoms become very pronounced, a creatine kinase test was performed ensuring. If the rate of this test is high, it indicates that there are many dying cells in the body. This is usually the case after a serious injury or heart attack. The son’s examination rate was several tens of times higher than in the case of a heart attack, “said the interlocutor.
Therefore, at the end of last year, Bernard was diagnosed with Duchenne muscular dystrophy. It is an extremely ruthless disease, the progression of which makes a child completely disabled: he loses the ability to walk, breathe independently, and eventually die prematurely.
Furthermore, the boy turned out to be in particularly rare shape, accounting for only 10 percent. All cases of Duchenne muscular dystrophy. This disease finally affects absolutely all the muscles of the body, including the internal organs. It progresses individually for each child, but the opportunity to walk is generally lost around 9-13 years.
Photo from personal album / Bernard
3-4 years of life turned out not worth the money
The news caused a shock, but after the genetic testing, the good news also reached us: a genetic mutation was found in the boy, for whose form a drug has been developed. Studies show that when started at a young enough age before the patient can walk on his own, the drug delays disability for at least 3 to 4 years and has a positive effect on lung and heart function, reducing greatly the risk of premature death.
The price of this medicine is 268 thousand. The decision to purchase and reimburse such medicines is made by the Commission for reimbursement of treatment for very rare human health conditions. Unfortunately, the commission’s decision was negative, and the main argument is that the budget of the Mandatory Health Insurance Fund does not provide sufficient funds to treat this rare condition.
It was also mentioned that alternatives to reimbursable treatment with deflazacort have not yet been used, and the pharmacoeconomic benefits of this expensive drug in 2016 was negatively rated in Scotland. Supposedly, the drug adds very few years of life to make it so worthwhile.
The family, in disagreement with the commission’s decision, appealed to the Board of Appeal of the Ministry of Health (SAM), but the latter refused to accept the complaint, arguing that the Pharmacy Law did not entitle the interested person to the Very Rare Human Health Compensation Commission to appeal to the Board of Appeal. The parents only listened to the offer to go to court, which they did, because they were ready to fight for their child’s health and life in every possible way.
It is true that the complaint from the institution treating the child, Kaunas Clinics, was accepted. “As he explained to me on the phone, it was as one of the arguments: we still accept the hospital’s complaint about what else you want. However, I see a major systemic problem here: the impression is that public authorities are doing everything possible to ensure that only the patient is not involved in the process of deciding his fate, so that he cannot provide additional information.Physicians directly exposed to the disease are also not invited to the hearing.
The Vilnius Regional Administrative Court, in examining a similar complaint, has clarified that patients have the right to file a complaint in such cases. The Court also stated that patients should have an opportunity to comment and appeal against the Commission’s decisions before making an unfavorable decision. However, according to the interlocutor, the officials did not interpret this decision as a rule, but as an exception adapted to a specific case.
Photo of D. Ščiuka / Bernardas with his father.
Practice from other countries
Doctors’ request to treat their young patient with innovative medications is based primarily on an identified genetic mutation that caused his illness. This recommendation was also indicated in the extract by the geneticist who carried out the study.
“Doctors said the clinical indications for the drug correspond to the child’s health status and that it is the only drug that fights the cause of the disease.” Recent research gathering data from children already treated with the drug has shown that the treatment eliminates the total risk of disability and premature death in an average of 3.5 years. Neighboring Latvians even reimburse this medicine for two of their children.
Treatment eliminates the total risk of disability and premature death in an average of 3.5 years.
And treatment with a drug prescribed by the commission can be compared to a situation where a person in need of appendicitis is told that the opportunity to administer pain relievers has not yet run out. This medication is prescribed for many diseases as a muscle aid, bodybuilders also use it to artificially increase muscle mass, but it doesn’t work on the cause of the disease itself.
Also, in case the muscles die, they do not grow on the muscles, but on the scars or sebaceous glands. Yes, this medicine is considered the standard of care for patients like our son, but it is not an alternative to treatment. These are completely incomparable drugs with different actions and purposes, ”said R. Rudzinskas.
Incidentally, such a deflazac card is inexpensive, costs only around 20 euros and is sold in other European countries with a simple medical prescription. In Lithuania, the decision on your appointment is made by the commission, so it will not generally take it.
“Going back to the medicine we need, Lithuania has already considered prescribing this medicine to a child whose condition was worse, but the answer was that the effect of the medicine in the pharmacoeconomic sense is too small. There was no such argument in our response to us, but it was mentioned that in Scotland in 2016. The pharmacoeconomic benefits of the drug were negatively evaluated. However, today in Scotland, this medicine is reimbursed without restriction to all patients for whom it is suitable.
I also have similar commission decisions in the UK, France, Germany, and Italy, showing that the situation is much broader when evaluating the prescription of medications to a patient, taking into account not only the length of life, but also what effect will have the treatment in the family: both parents will be able to work more time, therefore, the state will have to offer less support to the family, reason why they will save in a wider context “, explained the interlocutor.
In other countries, not only length of life is taken into account, but also the impact of treatment on the family.
The man said he had been in contact with the drug makers themselves after the commission’s decision, who were surprised that he had not been invited to negotiate a price. Because they have multiple programs, for example, half a year can give the drug for free, so the state would have to pay only half the price.
It is also a fairly popular practice for the state to enter into a conditional reimbursement agreement with drug manufacturers, i. and. The drug is free immediately, but it is claimed that if the health indicators are not positive within five years, the drug manufacturer will receive only 40%. negotiated prices Different countries buy the drug at very different prices, it can even cut the quantity in half. Of course, it depends on the number of patients, but in any case, it is the subject of negotiations.
Photo by D. Ščiuka / Bernardas
Race against the clock
“This medicine is only for children who are still walking, since he cannot walk again.” It is very important for us to preserve the function of walking for as long as possible, also because much research related to genetic engineering is currently being carried out both in the world and in Lithuania. Many scientists hope that a solution to the disease will be found within a decade. And that is our only hope.
But if our child has already missed the opportunity to walk, no one will lift him or her out of a wheelchair or any other bed into which the disease may sink. The wheelchair is only the first stage of the consequences. This is followed by the loss of respiratory and other organ functions. Probably the most terrifying thing is not a short life expectancy, but a terrible quality of life, “said R. Rudzinskas.
The father revealed that the son’s illness was undoubtedly a major blow, but both spouses struggled to stay healthy, socially active, and provide as much care as possible.
“The most painful thing I would have ever expected would be more stressful bureaucratic factors than dealing with the disease itself. The disease is like a lottery, no one is immune to it. Family genetic research shows that neither I nor my wife nor our parents have genetic abnormalities. This is a case that came out of nowhere. Usually, we all think it could happen to someone else, but not to me. Our example shows that this can happen to anyone, even young and healthy people.
But the most painful thing in this whole story is that as SAM bureaucrats get involved in the bureaucracy and send patients to court, and our son, who needs treatment here and now, years go by, his health deteriorates and the consequences become irreversible, it is impossible to repair it.
We have also interacted with other patients who have similar illnesses. Parents, especially those raising older children, are very tired of the constant encounter with the system, when they have to demand even basic help, which seems to be readily available in such conditions, “said the interlocutor.
Photo by Saulius Chadasevičius / 15min / Tememis, goddess of justice.
The SAM response contains the same arguments.
Questions raised during the conversation with the child’s father. 15 minutes SAM representatives asked. We ask why patients and doctors treating a disease cannot attend Commission meetings for reimbursement of costs for the treatment of very rare human health conditions. Are they accepting the main argument of not receiving treatment because they are not budgeted? Why were opportunities not sought to reduce the cost of the drug, negotiate a partial payment, or include the need for patient treatment in future budget planning? Finally, is there really a way for a patient who does not agree with the Commission’s arguments: to sue?
The response from the chairman of the meeting of the commission for reimbursement of treatment costs for very rare human health conditions Anželika Balčiūnienė states that the commission, which also includes a representative of the Kaunas clinics, makes decisions according to the procedure settled down. The decisions and minutes made by the Commission are published on the SAM website.
For a patient who disagrees with the Commission’s arguments, there is only one way: to sue?
“Under the current procedure, there is no possibility that the patient or his representatives participate in the examination of the applications. For its part, the commission has the right to receive all the necessary information from SAM units and treatment institutions for the decision-making as well as the right to request additional information from patient or physician organizations and other institutions.
Regarding reimbursement, it is stipulated that only the costs of purchasing a medicine that meets all the established criteria can be reimbursed. The criteria are set out in the description of the procedure for accepting reimbursement of the costs of treating very rare human health conditions. This procedure applies in all cases. We note that the commission, after receiving additional or modified information, for example, if new data on the therapeutic benefits of the drug is published, reconsiders the request, “said A. Balčiūnienė.
The Seimas Ombudsman, who launched an investigation into this incident, asked similar questions to SAM and the Commission for reimbursement of expenses for the treatment of very rare human health conditions and to the Appeals Commission. The study will also try to find out what is the scheme to calculate the cost of treatment, according to which it is decided that there are no funds for Bernard’s medicine, why the commission bases its decision on the therapeutic benefits of medicine in 2016. information but does not take into account the latest data, etc.
However, as the institutions question each other and wait for a court hearing, the days that are worth the gold for three years pass. Therefore, parents are looking for a variety of ways to get the much-needed medicine at least initially. To this end, the Bernard Support Foundation was established, which describes the child’s history and provides details for those who, during this difficult time, however, have the desire and opportunity to contribute to the child’s treatment with at least a small sacrifice. .
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