THE ASSOCIATION WHICH REPRESENTS CHILDREN AFFECTED BY DUCHENNE MUSCULAR DYSTROPHY DENOUNCES THIS SITUATION
● Experts in the diagnosis and treatment of Neuromuscular Diseases affirm that this Community is discriminating these children who are affected by a devastating pathology compared to other children from other communities and other European countries.
● The affected family in Salamanca reveals that the politicians of the Ministry of Health of Castilla y León are misleading the truth contradicting the medical indications of Doctor Andrés Nascimento, Neuropediatrician of the Neuromuscular Pathology Unit of the Neurology Service of the Sant Joan de Déu Hospital, in Barcelona, who precisely indicates that their child meets all the requirements for access to treatment due to his genetic mutation and age. They provide all the documents that prove it.
● The family of Salamanca affected by this situation has written to Her Majesty the Queen Letizia.
22/04/2018
On the European Day of Patients’ Rights, the Duchenne Parent Project Spain Association denounces that Castilla y León is the only Autonomous Community where children affected by Duchenne Muscular Dystrophy (DMD), who present the nonsense mutation and who comply the requirements set by the EMA and the NICE, CANNOT access the treatment prescribed by their physicians in several hospitals.
The affected family whose child is affected by this pathology and members of the Board of Duchenne Parent Project Association (Spain)
This Association – which brings together more than 400 families affected by this disease – claims equity for all children to have the ability to access treatment in all the Autonomous Communities in Spain. DMD is a devastating disease which is diagnosed between the ages of 2 and 5, according to experts. “In the beginning these little ones lose the ability to walk and can lose other muscular functions which affect the main vital organs. Subsequently, with the progress of the disease and without treatment, those affected may suffer from respiratory and cardiac insufficiency, which can lead to their death at an early age. That is why an early therapeutic approach is so important” Neuropediatricians experts in neuromuscular pathologies explain.
The President of this Association, who is part of FEDER, claims “We don’t understand how in 2018 discrimination is taking place in Castilla y León. These children meet the requirements stated by the EMA and the NICE (National Institute for Health and Care Excellence) to be able to access the medication Ataluren. Children affected by this disease can’t retrieve their days and functional losses,” says Silvia Ávila.
Outrage with the Ministry of Health of Castilla y León which plays with our lives
Milagros M. O., mother of a 9-year-old child who meets all the requirements set by the health authorities to access this treatment, represents one of the families who are affected by this situation in Castilla y León. “We have presented all the claims, backed by the Association Duchenne Parent Project Spain, in all Public Health Agencies of Castilla y León. Proof showing that the Health authorities of this Community are playing with the life of our son is that the last response we have received from the Health Service of Castilla y León (SACYL), explains that Dr. Andrés Nascimento, Neuropediatrician of the Unit of Neuromuscular Pathology of the Neurology Service of the Hospital Sant Joan de Déu, in Barcelona, which has also evaluated our son, considers our 9-year-old son too old to access treatment. This is a lie. We can show in writing that Doctor Nascimento says the opposite in his clinical report.“ This family wonders Why are they lying to us? “This is our child’s life. We have in our possession the results of clinical trials in more than 400 children in Europe which have been sent to us by different doctors. In addition, we are in contact with other mothers of our Association whose children suffer from the same mutation, have accessed treatment and are much better. With these lies they are making us want to leave Castilla y León” says this family.
A pathology whose clinical approach is a race against time.
Dr. Juan Vilchez Padilla, Head of the Neurology Service of the Teaching Hospital La Fe in Valencia, and Dr. Andrés Nascimento, in whose hospitals the clinical trials have been conducted in phase IIb and in phase III of this treatment with more than 50 children in Spain, comment that this treatment obtains better results as soon as the patient is treated and delays the symptoms of this disease for more than seven years. “It is essential to delay the moment where these children need a wheelchair to as long as possible, to avoid other clinical complications”, these experts emphasize. “These children, being able to end up in a wheelchair at an early age, may later require high financial assistance from different Administrations and other possible hospital expenses such as, for example, surgical interventions, prolonged internments, assisted ventilation, oxygen and special home care, and even personal assistants, among others”, explains Doctor Vilchez. “It is necessary to avoid wheelchairs,” they agree.
A scandalous situation where article 43 of the Constitution in Castilla y León is infringed. Doctor Vilchez comments that this situation is scandalous and entails discrimination for the children of this Community. These children are diagnosed with the same disease and the same genetic mutation as patients in Catalonia, Valencia, Andalucía, Valencia, Castilla La Mancha, Galicia, País Vasco, Canary Islands, Murcia, Extremadura, Aragon, etc. “How is it possible that even by fulfilling the same requirements, some children don’t have access to the same treatment as other affected children in our country, when the Government speaks of a united Spain? “ wonders this expert of European prestige. For now, both Dr. Vilchez and Dr. Nascimento, don’t advise this family to move from their current community. This situation is inadmissible. “It is a scandalous situation socially speaking. The regulations to have access to treatments are already pre-established internationally for all patients who are suffering from low prevalence diseases. And we all have to abide by these rules,” says Dr. Vilchez. Doctor Andrés Nascimento shares the same opinion. “If there is a European consensus described by the EMA, we must take into account the experience in the treatment of this drug by Austria, the United Kingdom, Denmark, Finland, Romania, Norway, Italy, Slovenia and Portugal, among others. In Spain, clinicians must have complete independence when it comes to diagnosing and treating their patients,” says this expert. ”Under no circumstances politicians be politicized in Spain because the time when patients lose confidence in their clinicians may come,” explains this expert. “Politics and medicine are completely different professions. This situation is unthinkable in developed countries in Europe and far less in the United States. Clinicians must have complete independence when diagnosing and prescribing treatment under current international recommendations and consensus. This type of political pressure on clinicians could occur in some underdeveloped countries,” says this Doctor.
Your Majesty the Queen
Together with the Parent Project Spain Association, this family has filed claims with the Counselor of this Community, Antonio María Sáez Aguado, who has not even answered back to them. Desperate, they have written to Her Majesty The Queen. Representatives of the Royal House have replied that, unfortunately, in this matter between Communities they don’t have competences. However, the Royal House has derived this appeal to the Ministry of Health. This family has contacted the Ministry of Health, but again, they haven’t answered back, (See minute 5 of the video). This family comments that “In the Ministry of Health they have answered us that they don’t know anything. We feel as if they were playing with the life of our son.”
United against an injustice
The president of the Association of Rare Diseases of Castilla y León (AERSCYL), Cristina Díaz, who is also part of the Board of Directors of FEDER, believes that this is not the first time that this has happened in this Community, “to avoid social differences between some patients and others, the budgetary heading of Health should be centralized. But it’s a very “greedy” game and they are not interested in centralizing the political parties themselves, as no one is sure that the funds destined for health are used for that purpose and could – presumably – be diverted to other destinations“, Diaz explains. “What is the purpose of organizing Congresses and investigating if we are not going to finance these treatments later?” Diaz asks. FEDER considers it essential to speed up the processes of access to orphan medicines and point out, based on EURORDIS’s data, that “only 5% of rare diseases are treated. Thus, there are more than 7,000 rare diseases and in Europe there are 102 authorized drugs with an orphan designation”, explains Juan Carrión, President of FEDER. From this entity they demand a centralized process in the budgets to finance orphan drugs that is clear and transparent and doesn’t lead to second interpretations. “We believe in a centralized process that guarantees access to the patient’s treatment regardless of their place of residence.” explains Alba Ancochea, Director of FEDER, affirms that “we seek to cover the lack of representativeness that organizations have in procedures of vital importance such as the commercialization or not of a treatment”. Some political parties support these families. Where are the health funds? These doubts are precisely what Francisco Igea, Spokesperson of Health of Ciudadanos Party wants to resolve by proposing a single centralized heading for all patients suffering from a disease of low prevalence. The deputy of this Party is very concerned about the lack of equity in health matters “all Spaniards should have the same right to the same benefits. “We ask for the General Health Law and the Cohesion Law to be complied with”, says Igea. María Luisa Carcedo, Secretary of Health and Consumer Affairs of the Federal Executive Commission of PSOE Party, says that her party will demand, again, through an amendment to the General State Budget, which is precisely being processed right now in the Cortes Generales, to create a Health Innovation Fund to face these therapeutic innovations, analyze their effectiveness and recover the universality of health. Amparo Botejara, Health spokesperson for the Podemos Party in the Congress of Deputies, comments that this case is very serious because, in effect, article 43 of the Spanish Constitution is being violated. “Our fundamental mission is to recover the accessibility and equity that should govern our Health System. We will not stop until we achieve this goal. It is unthinkable that such a situation could occur in Spain,” says Botejara. What is happening now with these children in Castilla y León is an absolute discrimination for those affected by this devastating pathology who meet all the criteria set by the EMA of access to treatment compared to the rest of other European countries and the rest of the Communities from Spain. These families have the unconditional support of the Association Duchenne Parent Project Spain, FEDER, the Association (AERSCYL) and most of the political parties that have joined to endorse this official statement.
María Huerta
Ce Consultores®
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We support Research Association against pain (www.pacientesatm.com), Infancia Solidaria (www.infanciasolidaria.org), Fundaicyl –a Foundation which helps children and Association of Patients which suffer from Genetic and Autoimmune Diseases.
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About Duchenne Muscular Dystrophy (DMD)
1.- DMD is a severe condition, progressive and recessive, linked to X chromosome, that affects mainly males.
Carrier mothers are also part of the population and doctors advice that medical tests must be performed.
2.- Nonsense mutation DMD is caused by a DNA change that affects to the production of a protein, called dystrophin, that is in the muscle.
3.- Normally, dystrophin production is affected since birth and DMD symptoms usually begin around the age of two to five. The main symptom is movement disorder. Children affected start having difficulties when they walk and, eventually main vital organs would be seriously damaged. Most will lose the ability to walk and assisted ventilation may be required. Development of heart failure is also one of the symptoms that might lead to death at the age of 30.
4.- DMD treatment includes corticosteroids, cardiac and respiratory monitoring and support, occasional orthopedic surgery. Physical therapy is also helpful. Nutritional advice might also be needed and gastric feeding when the disease has progressed.
5.- There are many Sanitary professionals involved on the treatment of this condition: neurologists, neuropediatricians, neuromuscular specialists, rehabilitation specialists, genetists, pediatricians and family doctors.
