• Basque Country, Catalonia, Murcia and Canary deny treatment to patients with Paroxysmal Nocturnal Hemoglobinuria (HPN), a rare disease of blood that deteriorates progressively as the patients and is a vital threat.
• Unequal access to the only medicine available for this disease has led to several patients to move their home to the Autonomous Communities that are guaranteed treatment, vital for those involved.
• To report this situation, patients and relatives from all over Spain are mobilized and the foundation of the Association of HPN patients.
The platform claims that HPN patients 13 patients Paroxysmal Nocturnal Hemoglobinuria (HPN) are expecting their Ministries of Health to authorize access to the only drug capable of halting the progress of their disease and reduce the risk of thromboembolic events, the main cause of mortality of those affected.
According to the platform, despite the serious health condition of patients, if a drug approved in March 2007 by the FDA (Food and Drug Administration) and EMEA (European Medicines Agency) and to have funding approved, there governments that deny strictly economic grounds. In fact, he explains, this treatment has a relatively high cost, but given the low incidence of this disease (as "ultra-orphan" to affect nearly 5 out of 1,000,000 Spanish) only creates an impact of around 0 , 01% of the total budget of each Ministry of Health.
"It is unacceptable that the authorities continue contradicting the medical criteria and denying vital treatment solely on economic grounds," says Jordi Cruz, spokesman for the platform of HPN patients. It added that by saying that "of 20 million euros that the state devoted to research for Rare Disorders (EERR) last year, little has been done in support of access to treatment and improving their quality of life .
Inequality between the Autonomous Communities
According to the platform, although Spanish law recognizes the right of health protection and territorial equity in access to medical advances, some governments deny these drugs now due to budgetary issues, not based on medical and scientific criteria. In fact, the difference between regions in this area is alarming. As confirmed by Cruz, as well as patients from Andalucia, Comunidad Valenciana, Castilla León, Madrid, etc. have access to treatment, the affected communities and other Canary Islands, Catalonia, the Basque Country and Murcia are denied access after long struggles and applications, or simply relegated to eternal waiting processes. "Every day that these patients go without treatment is vital," says Jordi Cruz, "35% of patients die within a period of 5 years after diagnosis and without treatment, are at high risk of thrombosis, which is the cause of between 40% and 67% of deaths in patients with HPN.
Specifically, they target members of the platform, in the Community are currently Canaria 3 patients who are being denied access to treatment by a purely economic consideration. A Catalina dark circles, canaria patient of 44 years, was diagnosed with the disease 17 years ago HPN. Since then, "I have applied only transfusions and cortisone to ease the symptoms." "With new orphan drugs markedly improved," adds dark circles, "but are very expensive to me and the administration denies it. They say the reason but I am sure that this is a question of money. "
The hematologist Fernando Fernández, Hospital Insular de Gran Canaria, and the doctor responsible for patient Catalina dark circles, corroborates the "silence" that currently exists in relation to the HPN and criticizing his patient. "Since last May we have asked three times to help pay for the treatment of dark circles and patient we have not received any response. We are in a position of absolute ignorance. Basically, the health authorities do not inform us of the Canary Islands. We know nothing, "said Dr. Fernandez.
In Catalonia, there are 5 patients who were awaiting a form that will allow medical staff to the application of the drug. In any case, it is still uncertain when they will be served. At the moment, must remain on hold for more than six months while the disease runs its course.
In the Basque Country, 4 patients were forced for over 8 months to assume that their disease continues to evolve because of Osakidetza that has not made a decision. In contrast, patients from other communities such as Madrid, are benefiting from the treatment for almost a year.
The blockade also persists in Murcia, where a patient with HPN at the Hospital Virgen de la Arrixaca that is not given response to be treated.
Other CCAA maintain policies more responsive to their patients with NPH, which also require urgent treatment, explained from the platform.
"The postal code of the patient just determining the quality of life and medical treatment of the patient," criticizes Jordi Cruz. "We should follow the example of other countries, like France, have a specific model of care for rare diseases, according to its merit as problems and needs, so different to other pathologies," he adds.
Cruz says in this connection that in some cases, health authorities, regional and / or addresses of the hospitals are contradicting criteria prescribed emergency medical treatment for patients with HPN "," not only because of the risks of life disease, but because this medicine allows perform their daily activities to some normality, which is very important when we consider who are mostly young people of working age. "
Involving patients
Patients with Paroxysmal Nocturnal Hemoglobinuria (HPN) have joined to form a partnership to help patients get needed care and access to treatment when the clinician to consider it.
In addition, the platform seeks to promote scientific research on the causes, development and therapies for disease of the HPN. Another of the future missions of this association is to advise, support and inform patients and families affected, and promoting public events, science scholarships and other initiatives to foster the exchange of knowledge and its dissemination to the medical, scientific and patient .
Paroxysmal Nocturnal Hemoglobinuria
The paroxysmal nocturnal hemoglobinuria (HPN) is an Ultra-Rare disease that affects approximately 240 patients in Spain. It is a genetic disorder in which normal natural proteins do not adhere adequately to red blood cells and, consequently, they are vulnerable to the immune system and are destroyed prematurely and intermittently. Impact of a disease that gradually deteriorating minority of patients and is a true life-threatening.
First described in 1882, adopted the name because it was observed that intravascular hemolysis (destruction of red blood cells) occurred in crisis situations and at night during sleep, after stressful situations, infections, physical exertion, or vaccination ingestion of acetylsalicylic acid.
NPH often occurs without being recognized initially, usually between 30 and 40 years, and the delay in diagnosis may take from 1 to more than 10 years. The value of the median survival of patients with HPN between 10 and 15 years from the time of diagnosis. NPH develops without warning and affects both sexes. Is estimated to affect between 8,000 and 10,000 people in Europe and North America.
Until now, there is no specific therapy for the treatment of NPH. The treatment of this disease was limited to symptom management through periodic blood transfusions, a non-specific immunosuppressive therapy and, rarely, bone marrow transplants, a procedure that carries considerable mortality risk.
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