BETHESDA CHILDREN’S HOSPITAL
ZOLGENSMA GENE TREATMENT
We treat patients up to the age of 18 from across Hungary, as well as providing care for socially disadvantaged and physically handicapped children and their families.
Although we enjoy an enviable reputation for delivering consistently high standards of paediatric care generally, we place particular importance on the field of neurology. The work of our Neurology Department focuses primarily on epilepsy treatment, where our Central-Eastern European Pre-surgical Epilepsy Centre provides an extensive programme of examinations prior to surgery being carried out.
Founded by the German Filial Community of the Reformed Church more than 150 years ago, Bethesda Children’s Hospital is one of the oldest children’s hospitals in Budapest and the only children’s hospital in Central-Eastern Europe maintained by the Church.
The Hospital was nationalised in 1952. Like other Church institutions, it was subsequently run for 40 years by the state, before again becoming the property of the Reformed Church in Hungary in 1992. The Hospital has since undergone major renovation work.
Our healing community sets great store by spirituality, while combining expert medical consultation skills with modern medical technology in advanced facilities.
2002 saw our range of care services expand further with the establishment of our Specialised National Burns Care Centre. Treating children suffering burn injuries is highly complex and requires close multidisciplinary co-operation and team work. The Centre offers centralised care for severe burn injuries and permanent patient admissions in cases of more than 5% total body surface area (TBSA) burns in infancy, more than 20% TBSA burns in children up to the age of 4 and more than 40% TBSA burns up to the age of 18, and care provision to suit.
Our Rehabilitation Ward treats children following severe burn injuries, car accidents and strokes, children emerging from coma, as well as providing motion rehabilitation. Our Psychosomatic Ward delivers care for children suffering dysthymia, behavioural disorders, generalised anxiety disorder and learning difficulties.
Our Department of Paediatric Surgery provides comprehensive medical services in general paediatric surgery and traumatology, complemented with paediatric urology and orthopaedic surgery. We specialise in laparoscopic or minimally invasive surgery and our consultants have particular expertise in performing orthopaedic- and gastrointestinal surgery on children with neurological damage.
We can gladly provide accommodation for parents to stay together with their children during their time in the Hospital.
Bethesda Children’s Hospital plays a key role in the Hungarian health and social education programme. We welcome students from a number of different medical universities, colleges, and vocational schools for health services who come to undertake their practical medical training with us. Our doctors, nurses and other specialists regularly lecture and teach at different educational institutions, and frequently attend training courses and conferences, both as delegates and speakers, in Hungary and abroad. We ourselves organise national and international conferences exploring different fields of paediatrics and forums for senior- and junior doctors and nurses, who also publish articles in respected medical journals.
The management of Bethesda Children’s Hospital has a strong belief in the vital role staff’s physical and mental wellbeing plays in the quality of the work and treatment they deliver. The Hospital has its own pastors, teachers and catechists, and a mental health team who take care of the patients. The Hospital’s Diaconal Committee also constantly seeks to help those in need, whether they be patients or staff. Bethesda Children’s Hospital has always enjoyed strong links internationally. As these were broken off with the Hospital’s nationalisation, one of the first tasks we faced in 1992 was to set about rebuilding all our international relations. We have since succeeded in restoring strong links with the German, Dutch, Swiss and Scottish Reformed Churches, hospitals and institutions. We enjoy the benefits provided by flourishing professional collaborations in both Great Britain and the United States. We regularly welcome visitors from the American- and Irish Churches and civil society organisations, while members of the Hospital staff frequently take part in study trips to the United States, Germany and Great Britain, all with a view to ensuring we can bring the latest medical knowledge and skills to bear in delivering the very best levels of medical care.
WHAT IS ZOLGENSMA® GENETHERAPY AND TO WHOM IT CAN BE APPLIED?
ZOLGENSMA® (onasemnogene abeparvovec–xioi) is a genetherapy used in the treatment of children diagnosed with muscular dystrophy (spinal muscular atrophy (SMA), which was granted valid marketing authorisation as a registered drug treatment across the entire territory of the European Union on 18 May 2020. (In the U.S. prescription, the use of Zolgensma genetherapy is possible only in children under 2 years of age.)
Based on clinical experience to date, an international recommendation has been issued on the suitability of the medicine, according to which genetherapy is recommended for children who do not exceed the weight limit of 13.5 kg.
Our hospital is continuously studying the relevant international medical literature and we have to emphasize that the results of clinical trials are based on data from infants and asymptomatic infants and children who does not exceed the weight limit of 13.5 kg and there are limited data on efficacy and safety in the advanced stage of the disease. Based on the facts written before, there are very little available international experience and data in terms of the risk and benefit ratio of genetherapy in children over 2 years of age and children over 13.5 kg. Considering this fact our Hospital has taken the decision that until we have a sufficient number of literature recommendations, we will not be able to perform genetherapy for children over 30 months (2,5 years) of age or for children who are heavier than 13.5 kg.
SUMMARIZE: The last date for administering Zolgensma is the 30 month from the birth of the child.
We have to emphasize that the final decision about the genetherapy treatment can be made only if all the relevant medical documents of the child have been reviewed by the Hospital and our medical team takes its decision always individually in each case.
Our Hospital can not be held responsible for the efficacy, side effects and complications of Zolgensma therapy, as well as the costs incurred.
COSTS INVOLVED IN DELIVERING THE ENTIRE GENETHERAPY TREATMENT
- cost of the ZOLGENSMA® medicine
- cost of 3 months’ follow-up treatment and care in Budapest, Hungary
Total cost of the entire genetherapy treatment: 2.132.000 EUR
The medicine, the insurance and the shipment by special airplane (Vials has to be transported frozen at or below -60ºC.) are extremely expensive. 95% of the total cost is the price of the medicine. Therefore the total cost cannot be reduced and there is no any possibility to pay in installments.
The medicine can be ordered after the total cost has been transferred to the Hospital’s account.
GENETHERAPY TREATMENT INCLUDES THE FOLLOWING HEALTH SERVICES, AND SUPPLEMENTARY AND RELATED SERVICES:
A 3-MONTH PERIOD OF TREATMENT TAKES PLACE ONCE THE MEDICINE HAS BEEN ADMINISTERED, WHICH WILL REQUIRE YOU MOVING TO BUDAPEST, HUNGARY. DURING THIS 3-MONTH PERIOD WE ARE FOLLOWING THE CHILD PHYSICAL STATUS; CARRYING OUT DIFFERENT CONTROL EXAMINATIONS, LABORATORY TESTS, DIETARY EXAMINATION, CONSULTATION WITH SPECIALISTS ETC.)
- carrying out the preparatory work for the treatment itself
- ensuring the proper hygiene standards of the hospital area
- procuring and properly storing the medicine
- providing, preparing and administering the medicine
- hospitalisation for the time the medicine is administered
- 3 months’ treatment delivered as outpatient care
- conducting on-going physical examinations and laboratory tests
- calibrating medication doses
- delivering supplementary immunosuppressive drug therapy
- dealing with complications, if necessary through hospitalisation
- conducting additional examinations depending on your child’s actual condition (imaging examinations, consultation with specialists etc.)
- calibrating medical settings of the ventilator in case of non-invasive ventilation
- repairing or replacing medical devices in the event of failure (In the case of a machine provided by a hospital)
- physiotherapy after the first month (because infection) regulary
- certain hygiene products, medicines, formula for the care of children
- providing dietary advice,
- optimising nutrition
- tube feeding if necessary and regular tube replacement.
COST OF DELIVERING THE ENTIRE GENETHERAPY TREATMENT DOES NOT INCLUDE:
- travel to and from Hungary, accommodation and living costs during your stay in Hungary
- patient care beyond the specified 3-month treatment period (in your country)
- The price of the medical technology equipment and devices recommended by the medical specialists of the Hospital in the course of the gene therapy treatment for use subsequent to the completion of said 3-month gene therapy treatment and which are customized to the child (you have the opportunity to buy these equipment in our Hospital, but it is not obligatory).
The main points of the authorization procedure
- Inquiry from the Parents
- Bethesda Children’s Hospital sends the following documents:
- General information sheet about the genetherapy
- Data sheet of application to fill and sign
- Parents send back the filled and signed Data sheet of application and the documents listed in the Data sheet of application. After receiving the requested documents our medical team decides — based on the supplied data of the child— whether the Parents should send further documents or not (that means, further examination of the child’s suitability continues or not).
- Based on the information provided in the Data sheet of application our medical team takes a decision whether to accept the application.
- If the child’s application has been accepted, the Hospital will send the Parents a price offer for performing the genetherapy (The price offer does not mean any obligation regarding the genetherapy treatment carried out by the Hospital).
- In case of accepting the application you take the decision as parents to have your child undergo genetherapy at Bethesda Children’s Hospital in Budapest, Hungary.
- You provide documented proof that sufficient funds are available to cover the entire cost of the treatment.
- The Hospital sends a Medical record and information list about all the medical records and information our medical team needs (only English version is accepted).
- We contact your child’s local treating doctor, requesting additional medical documentation if necessary.
- Once our doctors have studied your child’s complete medical records, Bethesda Children’s Hospital will make a final decision on your child’s suitability for genetherapy and, provided there are no contraindications, on undertaking your child’s treatment.
- Bethesda Children’s Hospital plans the genetherapy and draws up a treatment schedule
- Local treating doctor signs an official statement (we send a form to fill and sign) to the effect that they will undertake aftercare of your child at the local SMA Centre in your country of residence upon your return home
- You will need to move to Budapest, Hungary 2 weeks prior to the proposed date of the genetherapy being administered. It is important you have to bring all equipment your child uses (devices related to ventilation and every medical equipment for movement etc.)
- We meet you at Bethesda Children’s Hospital for an examination of your child and for a detailed briefing and to answer any further questions you may have, to sign the necessary legal documents. Because of the examinations you have to ensure your child doesn’t eat or drink before the meeting for at least 2 hours
- The total cost of the entire genetherapy treatment is credited to Bethesda Children’s Hospital bank account upon which the medicine will be ordered
- Bethesda Children’s Hospital orders the medicine and arranges for the medicine to be imported (the medicine will reach the Hospital within 10 days of the date of ordering)
- Medicine is administered
- Your child is under the treatment and care of our doctors for 3 months in Budapest (Hungary)