Expected impact – patients
Paradoxical situation in patients with haemophilia: there is a budget, but the treatment is not used. According to the first socio-economic analysis of the impact that haemophilia has in our country, only half of the patients use the treatment available through CNAS, even though the budget has increased 2.5 times over the last two years.
The main causes for this situation are: the disproportionate network of treatment centers, the difficult access of patients to medication and the lack of haematologists.
Only half of the patients with haemophilia are treated.
At the end of October 2020, the first analysis on the economic and social impact of haemophilia in Romania was launched. The study was carried out by EY Romania, at Roche Romania’s request, with 2019 as the reference year.
According to the analysis, 1,600 people live in Romania suffering from haemophilia, but only half of these patients receive treatment under the National Haemophilia Program.
More specifically, over the last five years, the average number of patients treated annually was around 850.
Based on the analysis, funding is not a barrier to treatment access. Despite the 2.5 increase in the budget allocated to the National Haemophilia Program (from RON 70 million in 2016 to RON 179 million in 2018), the persons diagnosed with haemophilia encounter a number of difficulties in accessing treatment.
“We were not prepared enough to use this budget, with so many years of underfunding”, said Prof. Dr. Daniel Coriu, director of the Center for Hematology and Spinal Transplant of the Fundeni Clinical Institute and vice president of the Romanian Society of Hematology. “You see, haemophilia is a special disease. We need haematologists who understand the disease. This is about a multidisciplinary team, not just a haematologist. We need a surgeon, an orthopedist, a dentist, and a physiotherapist. Unfortunately, the Romanian medical system was not prepared for this”.
≈ 300,000 km/year, travelled by patients with haemophilia for treatment.
Within this study, the most frequently mentioned barriers to access, from the perspective of physicians, were: the existence of only three treatment centers in the country (one in Bucharest and two in Timiş County), major differences between access to treatment and medical services between people domiciled in large urban centers compared to those from smaller cities or rural areas, as well as the lack of information regarding the treatment possibilities. According to physicians, these barriers lead to poor management of haemophilia in Romania.
As regards the economic and social aspect of the disease, according to the results of the study, the total costs of haemophilia in Romania are RON 160 million annually (EUR 35 million), of which 91% represent direct costs, namely diagnosis, treatment and monitoring of the disease, while 9% are indirect costs (productivity losses, pensions and disability benefits, holidays, transport). Compared to the other EU Member States, the average cost per patient (EUR 21,000) is 8-9 times lower than similar costs in Germany, Italy, France, Spain and the United Kingdom and is in the lower-cost segment compared to other EU states.
The socio-economic impact of haemophilia in Romania
On 24 October 2018, the first analysis was launched on the economic and social impact of haemophillia in Romania, on patients, their families, the state budget and society in general. The study was carried out by Ernst & Young Romania, at Roche Romania’s request, with 2017 as the reference year.
According to the same database, in the countries from the category of those with higher average incomes, of which Romania forms a part, 47.5% of patients suffer from a severe form of haemophilia .Moreover, 10 out of 100,000 people are born with haemophilia A, while 2 out of 100,000 people are born with haemophilia B. Given that the number of patients with haemophilia B in Romania is very low and the specific data are very poor, the analysis focuses on haemophilia A.
The life expectancy of people diagnosed with haemophilia varies according to the severity of the disease, the lifestyle adopted and access to adequate treatment.
The literature states that the average life expectancy of persons with severe forms of haemophilia who have appropriate access to treatment may be up to 15 years lower than in the general population and 3 years lower for moderate/mild forms of haemophilia, these figures varying considerably depending on the country and the medical system in which the person with haemophilia lives.
“The treatment of haemophilia in Romania is a paradox because we have a generous budget, we have a new protocol, but yet physicians fail to treat us with 4 IU per capital, in accordance with the European Union standards.” – said Nicolae Oniga, President of the National Association of Haemophiliacs in Romania.
Barriers to treatment access
In accordance with the analysis, funding is not a barrier to treatment access for patients. Despite the 2.5 increase in the budget allocated to the National Haemophilia Program (from RON 70 million in 2016 to RON 179 million in 2018), the persons diagnosed with haemophilia face a series of difficulties in accessing treatment.
“We have funding, but we are not ready for this funding and the funding does not solve all the problems”, said one of the medical experts consulted during the analysis.
Among the problems leading to poor haemophilia management in Romania, identified from the perspective of medical professionals, are the following:
• existence of only three comprehensive treatment centers in the country, with uneven geographical distribution: one in Bucharest and two in Timis County;
• major differences in treatment access and medical services between people with haemophilia residing in large urban centers and those residing in small urban or rural centers, the latter being severely disadvantaged;
• lack of information on the possibilities of treating people with haemophilia leads to poor management of the disease.
On the basis of the answers sent by 27 people with haemophilia and close family members, the main barriers to treatment access and medical services for people with haemophilia in Romania were identified, from the perspective of patients:
As for the economic and social aspect of the disease, according to the results of the study, the total costs of haemophilia in Romania are lei 150 million annually (EUR 30 million), of which 90% are direct costs, namely diagnosis, treatment and monitoring of the disease, while 10% are indirect costs (productivity losses, pensions and disability benefits, leave, transport).
The average cost per patient (EUR 21,000) is 8-9 times lower than similar costs in Germany, Italy, France, Spain and the United Kingdom and is in the lower-cost segment compared to other EU countries. Furthermore, even though in Romania the treatment costs represent over 60%, of the overall costs, the percentage is significantly lower than in the rest of the compared European countries, where it is over 90%.
On the basis of the results of this study, it can be said that the proportion of treatment costs in Romania is still below the maximum potential that would lead to proper management of the patient with haemophilia. This aspect is also supported by the inability to consume the entire budget allocated under the National Haemophilia Program in 2017 when the proportion of costs generated by treatment was 60.6% of the total Program budget.
“From a financial point of view, we have nothing to ask the National Health Insurance House. The existing problems are related to us, the professionals, and the patients. There is a need for multidisciplinary teams of dedicated professionals, made up of haematologists who understand the disease, and especially its complications, surgeons, orthopedists, physiotherapists. Unfortunately, the Romanian medical system has not been prepared for this. ” – said Prof. Dr. Daniel Coriu, Director of the Center for Haematology and Bone Marrow Transplant within the Fundeni Clinical Institute.
The socio-economic impact of haemophilia can be reduced if the treatment protocol in force is followed. Therefore, the efficiency of treatment as well as the introduction of new treatments that would reduce the number and severity of bleeding, are recommendations which would increase both the quality of life of patients and make the financial costs of haemophilia more effective for the public health system.
Also, an increase in the number of treatment centers couldlead to increased patient adherence to treatment.
“Unlike other rare diseases, haemophilia is one of the diseases for which cost-economy calculations show that it deserves proper treatment; not treating haemophilia can cause it to become a much more expensive disease through indirect, medical and non-medical costs than if it is treated. ” – said Prof. Dr. Margit Şerban, Head of the Bone Marrow Transplant Center of the Timisoara Children’s Hospital.
The study presented is public and can be accessed here.
Haemophiliais is a haematological condition the main manifestation of which is hemorrhages (uncontrolled bleeding), caused by a genetic mutation leading to coagulation cascade disorders by the absence or reduction of coagulation factors VIII or IX. Depending on the coagulation factor involved, two main types of haemophillia have been described:
• haemophilia A, for which the genetic mutation concerns the coagulation factor VIII;
• haemophilia B, for which the genetic mutation concerns the coagulation factor IX.
Haemophilia can be:
• congenital, inherited and characterized by quantitatively diminished or qualitatively altered synthesis of coagulation factors VIII or IX;
• acquired, characterized by the production by the human organism of inhibiting auto antibodies to its own coagulation factors VIII or IX.
The genes responsible for the normal development of the coagulation cascade are located on the X chromosome, which is the reason why the phenotypic manifestation of haemophilia is much more common in men than in women. Women with one of the X chromosomes affected by the mutation are considered “carriers”, i.e. they have the genotype characteristic of haemophilia but it is not symptomatic.”Carrier” women can transmit the X chromosome on which the specific mutation is found to their children. The disease is therefore genetically transmitted from mothers and is usually diagnosed in the first years of life, with the appearance of the first physical traumas.
The symptomatic manifestation characteristic of the disease is the tendency for spontaneous or induced hemorrhages to occur, depending on the severity of the coagulation factor deficiency. Hemorrhages may be internal, in joints and muscles, or external, caused by minor cuts, dental interventions, or trauma.
Coagulation cascade is a process that takes place through the contribution of coagulation factors, blood proteins that control bleeding. When one of these proteins, for example factor VIII, is absent, the coagulation cascade is interrupted and the coagulation is no longer occurring or the coagulation rate is slower than normal. Without treatment, bleeding can continue for days or sometimes weeks, putting the patient in vital danger. There is no cure for this disease, but modern treatments can successfully control the disease if they are followed.
Haemophillia is also called a “royal disease” because in the 19th and 20th centuries it was prevalent in the royal families from Europe. Queen Victoria of Great Britain was the carrier of a rare form of this disease, which was then passed on to two of her 5 daughters. In the 1900s, life expectancy for those diagnosed was 13 years.
“Haemophilia doesn’t wait until morning !”
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